Decanoic acid and its compositions for use in the prevention and / or treatment of peroxisomal disorders.

Description

1 / 39 Decanoic acid and its compositions for use in the prevention and / or treatment of peroxisomal disorders. FIELD OF THE INVENTION

[001] The present invention relates to decanoic acid and compositions comprising the same for treating and / or preventing a peroxisomal disorder. BACKGROUND OF THE INVENTION

[002] Peroxisomes are small membrane-bound organelles that are indispensable for human health and development. Peroxisomes contain enzymes involved in a variety of metabolic reactions, such as β-oxidation of fatty acids, ether phospholipid biosynthesis, and metabolism of reactive oxygen species (Islinger, M., et al., 2012. Histochemistry and cell biology, 137(5), pp.547-574).

[003] Peroxisomal disorders are a group of medical conditions characterized by peroxisome dysfunction. Defects in genes encoding peroxisomal proteins can lead to a variety of diseases that can be grouped into two main classes of peroxisomal disorders based on the cause and consequence of the defects: single peroxisomal enzyme deficiencies (PEDs) and peroxisome biogenesis disorders (PBDs), including Zellweger Spectrum Disorders (ZSDs). Taken as a group, peroxisomal disorders constitute a substantial fraction of inborn errors of metabolism and have an estimated combined incidence of 1 in 5000 (Waterham, HR, et al., 2016. BBA-Molecular Cell Research, 1863(5), pp.922-933).

[004] For many peroxisomal disorders, there is no standard treatment, with treatment strategies focusing on symptomatic or supportive therapies. Currently, there is no single diet. Petition 870250088218, dated 09 / 29 / 2025, page 9 / 81 2 / 39 specific recommended for patients with PBD-ZSD (Braverman, NE, et al., 2016. Molecular genetics and metabolism, 117(3), pp.313-321).

[005] Thus, there is a demand for new strategies for the treatment and / or prevention of peroxisomal disorders. SUMMARY OF THE INVENTION

[006] The inventors have surprisingly discovered that decanoic acid can exert positive effects on peroxisomal content and function. These positive effects support the application of decanoic acid or compositions comprising it to treat and / or prevent a peroxisomal disorder.

[007] In one aspect, the present invention provides decanoic acid for use in the treatment and / or prevention of a peroxisomal disorder.

[008] In another aspect, the present invention provides a method of treating and / or preventing a peroxisomal disorder, wherein the method comprises administering an effective amount of decanoic acid to an individual who needs it.

[009] In another aspect, the present invention provides the use of decanoic acid for the manufacture of a medicament for the treatment and / or prevention of a peroxisomal disorder.

[0010] In another aspect, the present invention provides the use of decanoic acid as a peroxisome proliferator, optionally in vitro.

[0011] Peroxisomal disorder can be any medical condition characterized by peroxisome dysfunction. Properly, peroxisomal disorder is a peroxisome biogenesis disorder (PBD) or a peroxisomal enzyme deficiency (PED). Properly, peroxisomal disorder is a Zellwe spectrum disorder. Petition 870250088218, dated 09 / 29 / 2025, page 10 / 81 3 / 39 ger (ZDL); rhizomelic chondriodysplasia punctata (RCDP) type 1; RCDP type 5; X-linked adrenoleukodystrophy (X-ALD); acyl-CoA oxidase deficiency; bifunctional protein D deficiency; SCPx deficiency; 2-methylacyl-CoA racemace deficiency (AMACR); Refsum disease primary hyperoxaluria type 1; RCDP type 2; RCDP type 3; RCDP type 4; ABCD3 deficiency; BAAT deficiency; or acatalasemia. In some modalities, the peroxisomal disorder is a peroxisome biogenesis disorder (PBD). In some modalities, the peroxisomal disorder is a Zellweger spectrum disorder (ZSD), rhizomelic chondriodysplasia punctata (RCDP) type 1, or RCDP type 5. In some modalities, the peroxisomal disorder is ZSD. In some forms, the peroxisomal disorder is Heimler syndrome (HS), infantile Refsum disease (IRD), neonatal adrenoleukodystrophy (NALD), or Zellweger syndrome (ZS). In some forms, the peroxisomal disorder is HS, IRD, or NALD.

[0012] Decanoic acid can be used to treat and / or prevent a peroxisomal disorder in any individual who needs it. Appropriately, the individual is a mammal. Appropriately, the individual is a human being. Appropriately, the individual is a child or an adult. Appropriately, the individual has a defect in a PEX gene or a gene encoding a peroxisomal protein. Appropriately, the individual has a defect in one or more genes selected from: PEX1, PEX2, PEX3, PEX5, PEX6, PEX7, PEX10, PEX12, PEX13, PEX14, PEX16, PEX19, PEX26, ABCD1, ACOX1, HSD17B4, SCP2, AMACR, PHYH, AGXT, GNPAT, AGPS, FAR1, ABCD3, BAAT, and CAT. In some forms, the individual has a defect in a PEX gene. In some forms, the individual has a defect in one or more genes selected from: PEX1, PEX2, PEX3, PEX5, PEX6, PEX7, PEX10, PEX11β, PEX12, PEX13, PEX14, PEX16, PEX19, PEX26. Petition 870250088218, dated 09 / 29 / 2025, page 11 / 81 4 / 39

[0013] Decanoic acid can be supplied in any suitable form. Suitablely, decanoic acid is in the form of triglycerides, diacylglycerides, monoacylglycerides, a pharmaceutically acceptable salt and / or an ester. In some embodiments, decanoic acid is in the form of triglycerides, diacylglycerides and / or monoacylglycerides. In some embodiments, decanoic acid is in the form of triglycerides. In some embodiments, decanoic acid is in the form of medium-chain triglycerides. In some embodiments, decanoic acid is in the form of a C10 homotriglyceride.

[0014] Decanoic acid may be comprised in a composition. The composition may be any suitable composition. Suitably, the composition is a pharmaceutical composition, a nutritional composition, or a nutritional supplement. Suitably, the composition is in the form of an oil-in-water emulsion, a powder, or a food. In some embodiments, the composition is in the form of an oil-in-water emulsion. In other preferred embodiments, the composition is in a ready-to-use form.

[0015] Decanoic acid may be present in the composition in any suitable amount and in combination with other suitable components. Suitably, at least about 50% by weight of the fat is decanoic acid. In some embodiments, at least about 55% by weight, at least about 60% by weight, at least about 65% by weight, or at least about 70% by weight of the fat is decanoic acid.Appropriately, decanoic acid is present in the composition in an amount of at least about 100 g / L. In some embodiments, decanoic acid is present in the composition in an amount of at least about 110 g / L, at least about 120 g / L, at least about 130 g / L, at least about 140 g / L, at least about 150 g / L, or at least about 150 g / L. Petition 870250088218, dated 09 / 29 / 2025, p. 12 / 81 5 / 39 in approximately 160 g / L, at least approximately 180 g / L, at least approximately 200 g / L, at least approximately 220 g / L, or at least approximately 240 g / L. In some embodiments, the composition is free from, or substantially free from, fatty acid moieties other than octanoic acid or decanoic acid. In some embodiments, the composition is free from, or substantially free from, fatty acid moieties other than decanoic acid. In some embodiments, the composition is free from, or substantially free from, fatty acid moieties other than octanoic acid or decanoic acid. In some embodiments, the composition is free from, or substantially free from, MCTs comprising fatty acid moieties other than decanoic acid. Suitably, the composition comprises proteins, fats, and carbohydrates.

[0016] Decanoic acid can be administered by any suitable route of administration and in any suitable administration regimen. Suitablely, decanoic acid is administered enterally or parenterally. In some embodiments, decanoic acid is administered enterally. In some embodiments, decanoic acid is administered orally or via a feeding tube. Suitablely, decanoic acid is administered once daily, twice daily, three times daily, or four times daily. In some embodiments, decanoic acid is administered as part of a meal, snack, or enteral ration. Suitablely, decanoic acid is administered in an amount of about 2 ga (about 200 g) per day, about 5 ga (about 150 g) per day, or about 10 ga (about 100 g) per day.

[0017] Decanoic acid can improve peroxisomal function and / or peroxisomal count. Decanoic acid can improve peroxisomal function. For example, decanoic acid can increase acyl CoA oxidase activity, increase Pex11a expression and / or Petition 870250088218, dated 09 / 29 / 2025, page 13 / 81 6 / 39 increase peroxisomal beta-oxidation capacity. DESCRIPTION OF THE DRAWINGS

[0018] Figure 1 - Effect of decanoic acid on peroxisomal content and function

[0019] SH-SY5Y cells were exposed to 250 μM of C10 or C8 for 6 days. The following parameters were studied: (A) acyl CoA oxidase activity; (B) PEX11a expression; and (C) assessment of beta-oxidation of peroxisomal fatty acids. DETAILED DESCRIPTION

[0020] Several preferred features and embodiments of the present invention will now be described as non-limiting examples. This disclosure is not limited to the exemplary methods and materials disclosed herein, and any methods and materials similar or equivalent to those described herein may be used in the practice or testing of embodiments of this disclosure. Those skilled in the art will understand that they can combine all the features of the invention disclosed herein without departing from the scope of the invention as disclosed.

[0021] It should be mentioned that, as used here and in the attached claims, the singular forms a, an, oea include plural references, unless the context clearly dictates otherwise.

[0022] The terms comprising, includes and comprised of, as used herein, are synonymous with including, includes, containing or contains and are inclusive or not limited and do not exclude additional members, elements or steps not mentioned. The terms comprising, includes and comprised of also include the term consisting of.

[0023] The number ranges are inclusive of the numbers that define the range. As used herein, the term about means approximately, in the region of, approximately, or around. When the term Petition 870250088218, dated 09 / 29 / 2025, page 14 / 81 7 / 39 mo about is used in conjunction with a numerical value or range; it modifies that value or range by extending the thresholds above and below the stated numerical value(s). In general, the terms about and approximately are used in the present invention to modify numerical value(s) above and below the stated value(s) by 10%.

[0024] The publications discussed in the present invention are provided for disclosure only prior to the filing date of this application. Nothing in this invention should be construed as an admission that such publications constitute the prior art to the claims appended hereto.

[0025] Unless otherwise indicated, the practice of the present invention will employ conventional techniques that are within the capabilities of a person skilled in the art. All publications mentioned in the descriptive report are incorporated herein by reference. Decanoic acid and compositions comprising the same.

[0026] The present invention relates to decanoic acid and compositions comprising the same for treating and / or preventing a peroxisomal disorder. Decanoic acid

[0027] Decanoic acid (also known as capric acid) is a saturated fatty acid with the formula CH3(CH2)8COOH. Decanoic acid can be in free form (or as a salt or ester thereof) or in the form of, for example, triglycerides, diacylglycerides, or monoacylglycerides.

[0028] In some embodiments, decanoic acid is in the form of triglycerides, diacylglycerides and / or monoacylglycerides. In preferred embodiments, decanoic acid is in the form of triglycerides. Petition 870250088218, dated 09 / 29 / 2025, page 15 / 81 8 / 39

[0029] In some embodiments, decanoic acid is in the form of medium-chain triglycerides. A medium-chain triglyceride (MCT) may refer to a triglyceride in which all three fatty acid moieties are medium-chain fatty acid moieties. The term fatty acid moiety may refer to the portion of the MCT that originates from a fatty acid in an esterification reaction with glycerol. For example, an esterification reaction between glycerol and only decanoic acid would result in an MCT with decanoic acid moieties. Appropriately, as defined herein, medium-chain fatty acids (MCFAs) are fatty acids having 6 to 12 carbon atoms, although fatty acids with 8 and 10 carbon atoms (i.e., octanoic acid and decanoic acid) are preferred and are referred to in the present invention as C8 or C8 fatty acids, and C10 or C10 fatty acids.

[0030] Homotriglycerides (i.e., all fatty acid moieties of the triglyceride are of the same identity, for example a C10 homotriglyceride may comprise 3 decanoic acid moieties) and / or heterotriglycerides (i.e., the fatty acid moieties of the MCT do not all have the same identity) may be used in the present invention. Preferred heterotriglycerides are heterotriglycerides consisting of octanoic acid and decanoic acid moieties. In some embodiments, the decanoic acid is in the form of a C10 homotriglyceride. In some embodiments, the decanoic acid is in the form of a C8 / C10 homotriglyceride.

[0031] Decanoic acid can be obtained by any suitable method known in the art. Examples of natural sources of MCTs include plant sources such as coconuts, coconut oil, palm kernel oil, and animal sources such as milk. Decanoic acid makes up about 5 to 8% by weight of the fatty acid composition of coconut oil. MCTs can also be synthesized Petition 870250088218, dated 09 / 29 / 2025, p. 16 / 81 9 / 39 obtained by the esterification of glycerol with one or more medium-chain fatty acids (MCFAs). For example, MCT-C10 can be synthesized by esterification of glycerol with decanoic acid.

[0032] Alternatively, the decanoic acid referred to in this invention may be in the form of a pharmaceutically acceptable salt or an ester. Salts and esters of decanoic acid are also known in the art as decanoates or caprates.

[0033] Decanoic acid or a composition comprising it may be supplied in any suitable form, such as in the form of an oil-in-water emulsion, a powder or a food.

[0034] In one embodiment, decanoic acid or a composition comprising it is in the form of an oil-in-water emulsion. The emulsion may not substantially comprise any protein or carbohydrate. Suitably, the total fat content of the oil-in-water emulsion is about 5 to about 40 g / 100 ml. In one embodiment, the total fat content of the oil-in-water emulsion is about 10 to about 40 g / 100 ml, about 15 to about 40 g / 100 ml, about 20 to about 40 g / 100 ml, about 25 to about 40 g / 100 ml, about 30 to about 40 g / 100 ml, or about 34 g / 100 ml. In other embodiments, the total fat content of the oil-in-water emulsion is approximately 5 to approximately 30 g / 100 ml, approximately 5 to approximately 25 g / 100 ml, approximately 10 to approximately 25 g / 100 ml, approximately 10 to approximately 20 g / 100 ml, or approximately 15 to approximately 25 g / 100 ml. Appropriately, the energy value of the emulsion is approximately 50 to approximately 500 kcal per 100 ml.In one embodiment, the energy value of the emulsion is approximately 100 to approximately 500 kcal per 100 ml, approximately 200 to approximately 500 kcal per 100 ml, approximately 200 to approximately 400 kcal per 100 ml, approximately 250 to approximately 350 kcal per 100 ml, or approximately 308 kcal per 100 ml. In other embodiments, the energy value of the emulsion is between approximately 50 and approximately 300 kcal per 100 ml. Petition 870250088218, dated 09 / 29 / 2025, p. 17 / 81 10 / 39 ml, for example, about 100 to about 300 kcal per 100 ml, about 50 to about 200 kcal per 100 ml, about 150 to about 250 kcal per 100 ml or about 160 to about 200 kcal per 100 ml.

[0035] In one embodiment, decanoic acid or a composition comprising it is in the form of a tablet, dragee, capsule, gel capsule, powder, granule, solution, emulsion, suspension, coated particle, spray-dried particle or pill.

[0036] In one embodiment, decanoic acid or a composition comprising it may be in powder form. The powder may, for example, be a spray-dried powder or a freeze-dried powder. In another embodiment, decanoic acid or a composition comprising it is in a spray-dried form.

[0037] Decanoic acid or a composition comprising it may be supplied in a ready-to-use form or in a form suitable for reconstitution in water. In preferred embodiments, the decanoic acid or composition comprising it is in a ready-to-use form. Suitably, the decanoic acid or composition comprising it is supplied in packages of about 50 mL to about 200 mL, about 80 mL to about 150 mL, or about 120 mL in volume. The decanoic acid, or the composition comprising it, may be incorporated into or mixed into a food substance. The decanoic acid or the composition comprising it may be in the form of a food product or a food. In one embodiment, the food product is a food product for humans. In one embodiment, the decanoic acid is comprised within a fortifying beverage or food. Compositions

[0038] Decanoic acid (e.g. in the form of triglycerides) Petition 870250088218, dated 09 / 29 / 2025, page 18 / 81 11 / 39 may be in the form of a composition.

[0039] The composition may be any type of composition in which decanoic acid can be incorporated, such as a composition in the form of a food product, a feeding tube, a nutritional supplement, a complete nutritional product or a pharmaceutical composition.

[0040] Decanoic acid can be formulated for human consumption. Decanoic acid or the composition comprising it can be formulated for enteral or parenteral administration. In a preferred embodiment, the composition is formulated for oral administration.

[0041] Decanoic acid or a composition comprising it may be in the form of a medical food. The term medical food, as used in the present invention, may refer to a food product specifically formulated for the dietary management of a disease or medical condition. For example, the disease or medical condition may have distinct nutritional needs that cannot be met by normal diet alone. The medical food may be administered under medical supervision. The medical food may be for oral intake or enteral nutrition.

[0042] Decanoic acid or a composition comprising it may be in the form of a feeding tube. The term feeding tube refers to a product intended to introduce nutrients directly into an individual's gastrointestinal tract via a feeding tube. Enteral feeding may be administered, for example, via a feeding tube placed through an individual's nose (such as nasogastric, nasoduodenal, and nasojejunal tubes) or a feeding tube placed directly into an individual's abdomen (such as a Petition 870250088218, dated 09 / 29 / 2025, p. 19 / 81 12 / 39 feeding tube for gastrostomy, gastrojejunostomy or jejunostomy).

[0043] Decanoic acid or a composition comprising it may be in the form of a nutritional composition or a nutritional supplement. The term nutritional composition may refer to a composition that nourishes an individual. The term nutritional supplement may refer to a product intended to supplement an individual's overall diet. Decanoic acid or a composition comprising it may be in the form of a complete nutritional product. The term complete nutritional product may refer to a product that is capable of being the individual's sole source of nutrition.

[0044] Decanoic acid or a composition comprising it may be in the form of a beverage, mayonnaise, salad dressing, margarine, low-fat spread, dairy products, cheese spread, processed cheese, dairy dessert, flavored milk, cream, fermented dairy product, cheese, butter, condensed milk product, ice cream mix, soy product, pasteurized liquid egg, bakery product, confectionery product, confectionery bar, chocolate bar, high-fat bar, liquid emulsion, spray-dried powder, freeze-dried powder, UHT pudding, pasteurized pudding, gel, jelly, yogurt, or a food with a fat-based or water-containing filling. Decanoic acid or a composition comprising it may be used to coat a food.

[0045] Decanoic acid or a composition comprising it may be in the form of a pharmaceutical composition. A pharmaceutical composition may refer to a composition comprising or consisting of a therapeutically effective amount of a pharmaceutically active agent, i.e., decanoic acid. A pharmaceutical composition preferably includes a vehicle, Petition 870250088218, dated 09 / 29 / 2025, page 20 / 81 13 / 39 pharmaceutically acceptable diluent or excipient (including combinations thereof).

[0046] Acceptable vehicles, diluents, and excipients for therapeutic use are well known in the pharmaceutical art. The choice of pharmaceutical vehicle, excipient, or diluent may be selected with respect to the intended route of administration and standard pharmaceutical practice. Pharmaceutical compositions may comprise, as, or in addition to, the vehicle, excipient, or diluent, any suitable binders, lubricant(s), suspending agent(s), coating agent(s), or solubilizing agent(s). Nutritionally acceptable vehicles, diluents, and excipients include those suitable for human or animal consumption and which are used as a standard in the food industry. Typical nutritionally acceptable vehicles, diluents, and excipients will be familiar to those skilled in the art.

[0047] Examples of suitable vehicles include lactose, starch, glucose, methylcellulose, magnesium stearate, mannitol, sorbitol and the like. Examples of suitable diluents include ethanol, glycerol and water. Examples of suitable binders include starch, gelatin, natural sugars such as glucose, anhydrous lactose, free-flowing lactose, beta-lactose, corn sweeteners, natural and synthetic gums such as acacia, tragacanth or sodium alginate, carboxymethylcellulose and poly(ethylene glycol). Examples of suitable lubricants include sodium oleate, sodium stearate, magnesium stearate, sodium benzoate, sodium acetate, sodium chloride and the like.

[0048] In addition to decanoic acid, the composition may comprise any additional components in any suitable quantity. The composition may comprise proteins, fat and / or carbohydrates.

[0049] In one embodiment, at least about 50% by weight of the fat in the composition is decanoic acid. In one embodiment, Petition 870250088218, dated 09 / 29 / 2025, page 21 / 81 14 / 39 at least about 51% by weight, at least about 52% by weight, at least about 53% by weight, at least about 54% by weight, at least about 55% by weight, at least about 56% by weight, at least about 57% by weight, at least about 58% by weight, at least about 59% by weight, at least about 60% by weight, at least about 65% by weight, at least about 70% by weight, at least about 75% by weight, at least about 80% by weight, at least about 85% by weight, at least about 90% by weight, at least about 95% by weight, at least about 98% by weight, at least about 99% by weight or at least about 100% by weight of the fat in the composition is decanoic acid. In one form, at least about 70% by weight of the fat in the composition is decanoic acid.

[0050] In one embodiment, decanoic acid is in the form of medium-chain triglycerides wherein said triglycerides comprise at least about 50% by weight, at least about 51% by weight, at least about 52% by weight, at least about 53% by weight, at least about 54% by weight, at least about 55% by weight, at least about 56% by weight, at least about 57% by weight, at least about 58% by weight, at least about 59% by weight, at least about 60% by weight, at least about 70% by weight, at least about 80% by weight, at least about 85% by weight, at least about 90% by weight, at least about 95% by weight, at least about 99% by weight or 100% by weight of the total fat content of the composition. In one embodiment, decanoic acid is in the form of medium-chain triglycerides, with said triglycerides constituting at least about 70% by weight of the total fat content of the composition.

[0051] In one embodiment, decanoic acid is present in the composition in an amount of at least about 5 g / L, by Petition 870250088218, dated 09 / 29 / 2025, p. 22 / 81 15 / 39 at least about 10 g / L, at least about 15 g / L, at least about 20 g / L, at least about 30 g / L, at least about 40 g / L, at least about 50 g / L, at least about 60 g / L, at least about 70 g / L, at least about 80 g / L, at least about 90 g / L, at least about 100 g / L, at least about 110 g / L, at least about 120 g / L, at least about 130 g / L, at least about 140 g / L, at least about 150 g / L, at least about 160 g / L, at least about 180 g / L, at least about 200 g / L, at least about 220 g / L, at least about 240 g / L, at least about 260 g / L, at least about 280 g / L, at least about 300 g / L, at least about 400 g / L, or at least about 500 g / L. In one embodiment, decanoic acid is present in the composition in an amount of at least about 240 g / L.In one embodiment, the composition comprises decanoic acid in an amount of about 100 g / L to about 500 g / L, about 150 g / L to about 400 g / L, or about 200 g / L to about 300 g / L. In another embodiment, the composition comprises decanoic acid in an amount of about 245 g / L.

[0052] The composition may comprise total saturated fatty acids in an amount of about 23 g about 50 g, about 25 g about 45 g, or about 33 to about 44 g per 100 g of lipids, on a fatty acid basis. The composition may comprise total saturated fatty acids in an amount of about 27 g about 35 g, about 28 g about 34 g, about 29 g about 33 g, or about 30 g about 32 g per 100 mL. In one embodiment, the composition comprises total saturated fatty acids in an amount of about 31 g per 100 mL. It is preferred that the majority of the saturated fatty acids be decanoic acid (C10:0). In one embodiment, the composition comprises decanoic acid in an amount of about 15 g, about 50 g, about 18 g, about Petition 870250088218, dated 09 / 29 / 2025, p. 23 / 81 16 / 39 g, or about 23 g, about 44 g per 100 g of lipids, or about 30 g, about 37 g per 100 g of lipids.

[0053] In one embodiment, the composition is free or substantially free of fatty acid moieties other than octanoic acid or decanoic acid. In one embodiment, the composition is free or substantially free of fatty acid moieties other than decanoic acid. However, there may be traces of such fatty acid fractions in the composition (e.g., less than about 5% by weight, less than about 4% by weight, less than about 3% by weight, less than about 2% by weight, less than about 1% by weight, less than about 0.5% by weight, or less than about 0.1% by weight).

[0054] In one embodiment, the composition is free or substantially free of medium-chain triglyceride moieties other than octanoic acid and decanoic acid. In another embodiment, the composition is free of or substantially free of medium-chain triglyceride moieties other than decanoic acid. However, there may be traces of such MCTs in the composition (e.g., less than about 5% by weight, less than about 4% by weight, less than about 3% by weight, less than about 2% by weight, less than about 1% by weight, less than about 0.5% by weight, or less than about 0.1% by weight).

[0055] The composition may also comprise long-chain triglycerides (LCTs). Preferably, the LCTs are present in the composition in an amount less than about 5% by weight, less than about 4% by weight, less than about 3% by weight, less than about 2% by weight, less than about 1% by weight, less than about 0.5% by weight or less than about 0.1% by weight). In one embodiment, no LCTs are present in the composition.

[0056] In one modality, the composition is substantially Petition 870250088218, dated 09 / 29 / 2025, p. 24 / 81 17 / 39 free of any other saturated fatty acid. However, there may be traces of other saturated fatty acids in the composition (for example, less than about 5% by weight, less than about 4% by weight, less than about 3% by weight, less than about 2% by weight, less than about 1% by weight, less than about 0.5% by weight or less than about 0.1% by weight).

[0057] In one embodiment, the composition is substantially free of mono- or polyunsaturated fatty acids. However, there may be traces of mono- or polyunsaturated fatty acids in the composition (for example, less than about 5% by weight, less than about 4% by weight, less than about 3% by weight, less than about 2% by weight, less than about 1% by weight, less than about 0.5% by weight, or less than about 0.1% by weight).

[0058] In one embodiment, the composition comprises pure or substantially pure decanoic acid.

[0059] The composition may comprise lipids in an amount of about 60 g about 80 g, about 63 g about 75 g, or about 65 g about 72 g per 100 g of dry mass. In one embodiment, the composition comprises lipids in an amount of about 30 g about 40 g, about 31 g about 38 g, about 32 g about 36 g, or about 33 g about 35 g per 100 mL. In one embodiment, the composition comprises lipids in an amount of about 34 g per 100 mL. The composition may comprise MCT in an amount of about 30 g about 40 g, about 31 g about 38 g, about 32 g about 36 g, or about 33 g about 34 g per 100 mL. In one embodiment, the composition comprises MCT in an amount of approximately 33.5 g per 100 mL.

[0060] Coconut oil or palm oil may be a preferred source for at least 50% by weight, or from about 70% by weight to about 90% by weight of the lipids. The remainder of the lipids may be Petition 870250088218, dated 09 / 29 / 2025, p. 25 / 81 18 / 39 selected from, for example, sources of medium-chain triglycerides, such as fractionated coconut oil, macadamia oil, palm oil or palm kernel oil, or sources of long-chain triglycerides, such as safflower oil, sesame seed oil, soybean oil (which may be obtained from soybeans), sunflower oil, high oleic sunflower oil, corn oil, canola oil, walnut oil, evening primrose oil, peanut oil, cottonseed oil, rapeseed oil, olive oil, fish oil, palm olein or algae oil, or mixtures thereof, preferably soybean oil (suitably between 2% by weight and 30% by weight), medium-chain triglycerides (suitably between 0% by weight and 14% by weight), marine oils (suitably between 0% by weight and 14% by weight, or between 2% by weight and 12% by weight) and phospholipids, mono- and diglycerides.

[0061] The composition may comprise carbohydrate in an amount of about 0 g about 9 g, about 3.2 g about 9 g, about 4 g about 8.6 g, or about 5 g about 8.2 g per 100 g dry. In one embodiment, the composition comprises carbohydrate in an amount of about 0 g about 1 g per 100 mL, about 0.1 g about 0.8 g per 100 mL, about 0.2 g about 0.6 g per 100 mL, or about 0.3 g about 0.5 g per 100 mL. In one embodiment, the composition comprises carbohydrate in an amount of about 0.42 g per 100 mL.

[0062] The composition may comprise protein in an amount of about 5 g about 20 g, about 13 g about 20 g, about 13 g about 18 g, about 13.8 g about 17 g, or about 14.2 g about 16.2 g per 100 g of dry mass. In one embodiment, the composition comprises protein in an amount of about 0 g about 1 g per 100 mL, about 0 g about 0.5 g per 100 mL, about 0 g about 0.2 g per 100 mL, or about 0 g about 0.1 g per 100 mL. In one embodiment, the composition Petition 870250088218, dated 09 / 29 / 2025, p. 26 / 81 19 / 39 comprises protein in an amount of approximately 0 g per 100 mL. The inclusion of alpha-lactalbumin or ingredients comprising large amounts of protein is particularly suitable. The presence of more than 20% by weight of alpha-lactalbumin in the protein fraction of the product results in easy compliance with the requirements for leucine, lysine, methionine, and cysteine, excellent palatability, and digestibility properties. In one embodiment, more than approximately 20% by weight or approximately 40 to approximately 80% by weight of the protein fraction is alpha-lactalbumin.

[0063] In another embodiment, the composition is free, or substantially free, of carbohydrates and proteins; for example, the composition has less than 2% by weight, 1% by weight, 0.5% by weight, or 0.1% by weight of carbohydrates and proteins. In one embodiment, the weight amounts of lipids to the sum of proteins and carbohydrates in the composition is about 1-5 to 1 or more. For example, the weight amounts of lipid to the sum of proteins and carbohydrates may be about 1:1, about 2:1, about 3:1, about 4:1, about 5:1, about 2.0-5.0 to 1, about 2.4-4.0 to 1, or about 2.6-3.8 to 1, or more.

[0064] The composition may also include substances such as minerals, vitamins, salts, functional additives including, for example, palatability enhancers, colorants, emulsifiers, antimicrobial agents or other preservatives. In one embodiment, the composition further comprises at least one substance selected from the group consisting of minerals, vitamins, salts, preservatives, and combinations thereof. Minerals that may be useful in such compositions include, for example, calcium, phosphorus, potassium, sodium, iron, chlorine, boron, copper, zinc, magnesium, manganese, iodine, selenium, chromium, molybdenum, fluorine and the like. Examples of vitamins that may be useful in the compositions described herein include vitamins H Petition 870250088218, dated 09 / 29 / 2025, page 27 / 81 20 / 39 fat-soluble vitamins (such as thiamine (vitamin B1)), riboflavin (vitamin B2), niacin (vitamin B3), pantothenic acid (vitamin B5), pyridoxine (vitamin B6), biotin (vitamin B7), myo-inositol (vitamin B8), folic acid (vitamin B9), cobalamin (vitamin B12 and vitamin C) and fat-soluble vitamins (such as vitamin A, vitamin D, vitamin E and vitamin K), including salts, esters or derivatives thereof. Inulin, taurine, carnitine, amino acids, enzymes, coenzymes and the like may be useful in various embodiments. In one embodiment, the composition comprises salt in an amount of about 0.0 to about 0.2 g per 100 mL. In another embodiment, the composition comprises salt in an amount of about 0.11 g per 100 mL. Preservatives, stabilizers, colorants, and even flavoring agents may be included in the composition. Examples of preservatives include sodium benzoate, sorbic acid, and p-hydroxybenzoic acid esters. Antioxidants and suspending agents may also be used.

[0065] The composition may be relatively energy dense. In one embodiment, the composition comprises about 2520 to about 3780, about 2520 to about 3080, or about 2800 to about 3040 kJ per 100 grams of dry matter. The composition may comprise about 2500 to about 3100 kJ per 100 grams of dry matter, as well as about 2505, about 2510, about 2515, about 2520, about 2525, about 2530, about 2535, or about 2540 to about 3100, about 3095, about 3090, about 3085, or about 3080 kJ per 100 g. In one embodiment, the composition comprises approximately 2984, approximately 2985, approximately 2986, approximately 2987, approximately 2988, approximately 2989, or approximately 2990 kJ per 100 g. In one embodiment, the composition comprises approximately 2500 to approximately 3100 kJ per 100 grams of dry matter. In one embodiment, the composition comprises approximately 2540 to approximately 3080 kJ per 100 grams of dry matter. In one embodiment, the composition comprises Petition 870250088218, dated 09 / 29 / 2025, p. 28 / 81 21 / 39 contains approximately 1000 to approximately 1500, approximately 1100 to approximately 1400, or approximately 1200 to approximately 1300 kJ per 100 mL. In one embodiment, the composition comprises approximately 1265 kJ per 100 mL.

[0066] The composition may have a ketogenic ratio of about 0.2 to about 0.3:1. Other embodiments include, but are not limited to, ratios of about 0.5:1, about 1:1 to about 5:1, up to about 4:1, optionally where most of the fat is decanoic acid. In one embodiment, the composition has a ketogenic ratio of about 2:1 to about 4:1. In one embodiment, the composition has a ketogenic ratio of about 2.7:1 to about 3.4:1. Exemplary composition

[0067] An exemplary composition is shown in the table below. In one embodiment, the composition is a ready-to-drink oil-in-water emulsion and comprises the following: Table 1 Per 100 mL per 120 mL package Energy kJ 1265 1518 Fat g 34 41 of which saturated g 31 37 MCT g 33.5 40 Decanoic acid g 24.5 29 Carbohydrates g 0.42 0.50 Protein g 0 0 Salt g 0.11 0.13 Peroxisomal disorders

[0068] In one aspect, the present invention provides decanoic acid, or a composition comprising the same, for use in the treatment and / or prevention of a peroxisomal disorder.

[0069] In one aspect, the present invention provides a method of treating and / or preventing a peroxisomal disorder, the method comprising administering a therapeutically effective amount. Petition 870250088218, dated 09 / 29 / 2025, page 29 / 81 22 / 39 of decanoic acid, or a composition comprising the same, to an individual who needs it.

[0070] In one aspect, the present invention provides the use of decanoic acid, or a composition comprising the same, for the manufacture of a medicament for the treatment and / or prevention of a peroxisomal disorder.

[0071] As used in this document, the term treat may refer to preventing, diminishing, reducing or improving at least one symptom associated with the condition and / or slowing, reducing or blocking the progression of the condition in an individual with one or more symptoms of the condition.

[0072] As used in this document, the term prevention may refer to the reduction or prevention of the development of at least one symptom associated with the condition in an individual who does not have any symptoms of the condition.

[0073] Peroxisomes are dynamic organelles that play an essential role in a variety of catabolic and anabolic cellular metabolic pathways, including alpha and beta-oxidation of fatty acids and synthesis of plasmalogen and bile acids. Defects in genes encoding peroxisomal proteins can result in a wide variety of peroxisomal disorders affecting specific metabolic pathways, i.e., single peroxisomal enzyme deficiencies, or causing a generalized defect in peroxisome function and assembly, i.e., peroxisome biogenesis disorders (Waterham, HR, et al., 2016. Biochimica et Biophysica Acta (BBA)-Molecular Cell Research, 1863(5), pp.922-933).

[0074] Decanoic acid can improve peroxisomal function and / or peroxisomal count. Any suitable assay can be used to determine the effect on peroxisomal function and / or peroxisomal count. For example, peroxisomal function and / or count Petition 870250088218, dated 09 / 29 / 2025, p. 30 / 81 23 / 39 can be determined by gene expression analysis of genes encoding peroxisomal proteins or by direct measurements of peroxisomal proteins (see, for example, Huang, TY, et al., 2019. Metabolism, 98, pp. 53-61). Peroxisomal function can also be determined by measuring the levels of substrates normally handled by peroxisomes, such as VLCFAs, pristanic acid, phytanic acid, DHCA, THCA, and pipecolic acid, and / or the levels of end products of peroxisomal metabolism, such as plasmalogens, cholic and chenodeoxycholic acid, and docosahexaenoic acid (see, for example, Ferdinandusse, S., et al., 2016. Journal of the Society for the Study of Inborn Errors of Metabolism, 39(4), pp. 531-543). The peroxisomal number can also be determined by immunofluorescence and counting. peroxisomes, as described for example, Colton, HM, et al., 2004. Toxicological Sciences, 80(1), pp.183-192.Peroxisomes can be stained, for example, with a polyclonal antibody against a peroxisomal membrane protein, and the number of peroxisomes per cell can be determined manually or using automated software.

[0075] Decanoic acid can improve peroxisomal function. In some embodiments, decanoic acid increases acyl CoA oxidase activity, increases PEX11a expression, and / or increases peroxisomal beta-oxidation capacity. In some embodiments, decanoic acid increases acyl CoA oxidase activity by at least 50% or at least 100%. In some embodiments, decanoic acid increases PEX11a expression by at least 10%, at least 20%, or at least 30%. In some embodiments, decanoic acid increases peroxisomal beta-oxidation capacity by at least 10%, at least 20%, or at least 30%.

[0076] The peroxisomal disorder treated or prevented in the present invention may be a peroxisome biogenesis disorder (PBD). Petition 870250088218, dated 09 / 29 / 2025, page 31 / 81 24 / 39 or a peroxisomal enzyme deficiency (PED). Peroxisome biogenesis disorders (PBDs)

[0077] In some forms, the peroxisomal disorder is a peroxisome biogenesis (PBD) disorder.

[0078] Peroxisome biogenesis disorders (PBDs) are a group of autosomal recessive disorders comprised of three distinct subtypes recognized among PBDs: Zellweger spectrum disorders (ZSDs), RCDP type 1 and type 5, and peroxisomal fission defects. PBDs can be caused by defects in any of at least 14 different PEX genes, which encode proteins involved in peroxisome assembly and proliferation. Genetic testing for all PEX genes is available as a diagnostic service (Waterham, HR and Ebberink, MS, 2012. Biochimica et Biophysica Acta (BBA)-Molecular Basis of Disease, 1822(9), pp.1430-1441; and Waterham, HR, et al., 2016. Biochimica et Biophysica Acta (BBA)-Molecular Cell Research, 1863(5), pp.922-933).

[0079] In some forms, the peroxisomal disorder is a Zellweger spectrum disorder (ZSD), RCDP type 1, RCDP type 5, or a peroxisomal fission defect. Zellweger spectrum disorders (ZSDs)

[0080] In some modalities, the peroxisomal disorder is a Zellweger spectrum disorder (ZSD).

[0081] ZSDs include the three historically defined clinical entities cerebrohepatorenal syndrome or Zellweger syndrome (ZS), neonatal adrenoleukodystrophy (NALD), and infantile Refsum disease (IRD), which are currently considered different presentations within the same clinical and biochemical spectrum, with ZS being the most severe and IRD a less severe presentation (Waterham, HR, et al., 2016. Biochimica et Biophysica Acta (BBA)-Molecular Cell Research, 1863(5), pp.922-933). ZSDs may also include the syndrome of Petition 870250088218, dated 09 / 29 / 2025, p. 32 / 81 25 / 39 Heimler syndrome (HS) is caused by hypomorphic mutations in PEX1 and PEX6 (Ratbi, I., et al., 2015. The American Journal of Human Genetics, 97(4), pp. 535-545).

[0082] In some forms, the peroxisomal disorder is Heimler syndrome (HS), infantile Refsum disease (IRD), neonatal adrenoleukodystrophy (NALD), or Zellweger syndrome (ZS). In some forms, the peroxisomal disorder is HS, IRD, or NALD. RCDP type 1 or type 5

[0083] In some modalities, the peroxisomal disorder is RCDP type 1 or RCDP type 5.

[0084] RCDP type 1 is caused by mutations in the PEX7 gene, which encodes the PTS2 protein receptor. RCDP type 5 is caused by a frameshift mutation located in exon 9 specific to PEX5L, which results in the loss of only the PEX5L isoform, while PEX5S is still expressed (Waterham, HR, et al., 2016. Biochimica et Biophysica Acta (BBA)-Molecular Cell Research, 1863(5), pp.922-933). Peroxisomal fission defects

[0085] In some modalities, the peroxisomal disorder is a peroxisomal fission defect.

[0086] Patients have been reported with mutations in DLP1, Mff, GDAP1, and Pex11β (Waterham, HR, et al., 2007. New England Journal of Medicine, 356(17), pp. 1736-1741; Nasca, A., et al., 2018. Frontiers in Genetics, 9, p. 625; Huber, N., et al., 2013. EMBO Reports, 14 (6), pp. 545-552; and Ebberink, MS, et al. 2012. Journal of Medical Genetics, 49 (5), pp. 307-313). Peroxisomal enzyme deficiencies (PEDs)

[0087] In some forms, the peroxisomal disorder is a deficiency of peroxisomal enzyme (PED).

[0088] Peroxisomal enzyme deficiencies (PEDs) are a Petition 870250088218, dated 09 / 29 / 2025, p. 33 / 81 26 / 39 group of disorders caused by a defect in a single peroxisomal protein involved in peroxisomal function, such as (1) phospholipid ether biosynthesis (plasmalogen); (2) beta-oxidation of fatty acids; (3) peroxisomal alpha-oxidation; (4) glyoxylate detoxification and (5) H2O2 metabolism (Wanders, RJ and Waterham, HR, 2006. Biochimica et Biophysica Acta (BBA)-Molecular Cell Research, 1763(12), pp.1707-1720; and Waterham, HR, et al., 2016. Biochimica et Biophysica Acta (BBA)-Molecular Cell Research, 1863(5), pp.922-933)

[0089] In some embodiments, the peroxisomal disorder is a disorder of beta-oxidation of peroxisomal fatty acids, a disorder of alpha-oxidation of peroxisomal fatty acids, a disorder of peroxisomal glyoxylate metabolism, a disorder of peroxisomal ether phospholipid biosynthesis, a disorder of peroxisomal bile acid synthesis, or a disorder of peroxisomal H2O2 metabolism.

[0090] In some forms, the peroxisomal disorder is X-linked adrenoleukodystrophy (X-ALD); acyl-CoA oxidase deficiency; bifunctional protein D deficiency; SCPx deficiency; 2-methylacyl-CoA racemace (AMACR) deficiency; Refsum disease primary hyperoxaluria type 1; RCDP type 2; RCDP type 3; RCDP type 4; ABCD3 deficiency; BAAT deficiency; or Acatalasemia. Peroxisomal fatty acid beta-oxidation disorders

[0091] In some forms, the peroxisomal disorder is a disorder of beta-oxidation of peroxisomal fatty acids, such as X-linked adrenoleukodystrophy (X-ALD); acyl-CoA oxidase deficiency; bifunctional protein D deficiency; SCPx deficiency; or 2-methylacyl-CoA racemace (AMACR) deficiency.

[0092] In some forms, the peroxisomal disorder is X-linked adrenoleukodystrophy (X-ALD). X-ALD is the most common peroxisomal disorder and is caused by mutations in the gene Petition 870250088218, dated 09 / 29 / 2025, page 34 / 81 27 / 39 ne ABCD1, which encodes one of the three known peroxisomal ABC transporters located in the peroxisomal membrane, which are involved in the ATP-driven transport of fatty acids across the peroxisomal membrane.

[0093] In some forms, the peroxisomal disorder is acyl-CoA oxidase deficiency. Acyl-CoA oxidase deficiency is caused by mutations in the ACOX1 gene that encodes one of three different peroxisomal acyl-CoA oxidases.

[0094] In some forms, the peroxisomal disorder is a deficiency of bifunctional D-protein. Bifunctional D-protein deficiency is caused by mutations in the HSD17B4 gene that encodes 17-beta-estradiol dehydrogenase, an enzyme that catalyzes the second and third steps of peroxisomal beta-oxidation.

[0095] In some modalities, the peroxisomal disorder is SCPx deficiency. SCPx deficiency is caused by mutations in the SCP2 gene, which encodes the 58 kDa SCPx protein composed of a 46 kDa thiolase domain and a 13 kDa SCP2 domain.

[0096] In some forms, the peroxisomal disorder is 2-methylacyl-CoA racemace (AMACR) deficiency. AMACR deficiency is caused by mutations in the AMACR gene that encodes the 2-methylacyl-CoA racemace (AMACR) enzyme. Peroxisomal fatty acid alpha-oxidation disorders

[0097] In some forms, the peroxisomal disorder is a disorder of alpha-oxidation of peroxisomal fatty acids, such as Refsum disease.

[0098] In some forms, the peroxisomal disorder is Refsum disease. Refsum disease is caused by mutations in the PHYH gene that encodes the peroxisomal enzyme phytanoyl-CoA 2-hydroxylase, which catalyzes the first step in the peroxisomal alpha-oxidation pathway. Petition 870250088218, dated 09 / 29 / 2025, page 35 / 81 28 / 39 Disorders of peroxisomal glyoxylate metabolism

[0099] In some forms, the peroxisomal disorder is a disorder of peroxisomal glyoxylate metabolism, such as primary hyperoxaluria type 1.

[00100] In some forms, the peroxisomal disorder is primary hyperoxaluria type 1. Primary hyperoxaluria type 1 is caused by mutations in the AGXT gene that encodes the liver-specific peroxisomal enzyme alanine-gluoxylate aminotransferase (AGT), a pyridoxal 5'-phosphate-dependent enzyme that catalyzes the transamination of alanine and glyoxylate to pyruvate and glycine. Disorders of peroxisomal ether phospholipid biosynthesis

[00101] In some forms, the peroxisomal disorder is a disorder of peroxisomal ether phospholipid biosynthesis, such as RCDP type 2, RCDP type 3, or RCDP type 4.

[00102] In some forms, the peroxisomal disorder is RCDP type 2. RCDP type 2 is also known as GNPAT deficiency and is caused by mutations in the GNPAT gene that encodes GNPAT (DHAPAT), the enzyme that catalyzes the first of two subsequent peroxisomal conversions in ether phospholipid synthesis.

[00103] In some forms, the peroxisomal disorder is RCDP type 3. RCDP type 3 is also known as APGS deficiency and is caused by mutations in the APGS gene that encodes APGS, the enzyme that catalyzes the second peroxisomal conversion in ether phospholipid synthesis.

[00104] In some forms, the peroxisomal disorder is RCDP type 4. RCDP type 4 is also known as FAR1 deficiency and can be caused by mutations in the FAR1 gene that encodes fatty acyl-CoA reductase 1, which converts fatty acyl-CoA into its respective fatty alcohol. Disorders of peroxisomal bile acid synthesis Petition 870250088218, dated 09 / 29 / 2025, page 36 / 81 29 / 39

[00105] In some forms, peroxisomal disorder is a disorder of the synthesis of peroxisomal bile acids, such as ABCD3 deficiency or BAAT deficiency.

[00106] In some forms, the peroxisomal disorder is ABCD3 deficiency. ABCD3 deficiency can be caused by a truncated mutation in the ABCD3 gene. The ABCD3 peroxisomal transporter half is involved in the transport of branched-chain fatty acids and C27 bile acids to the peroxisome.

[00107] In some forms, the peroxisomal disorder is BAAT deficiency. BAAT deficiency is caused by mutations in the BAAT gene that encodes the liver-specific peroxisomal enzyme bile acid-CoA:amino acid N-acyltransferase, which converts bile acid CoA esters into taurine or glycine conjugates. Disorders of peroxisomal H2O2 metabolism

[00108] In some forms, peroxisomal disorder is a disorder of peroxisomal H2O2 metabolism, such as Acatalasemia.

[00109] In some forms, the peroxisomal disorder is Acatalasemia. Acatalasemia is caused by mutations in the CAT gene that encodes the prototypical peroxisomal catalase enzyme, which breaks down H2O2 into H2O and O2. Individual

[00110] The agents and methods can be used to treat or prevent a peroxisomal disorder in any individual who needs it.

[00111] The individual can be a human being or an animal. The individual can be a mammal, such as a human being, canine, feline, equine, caprine, bovine, ovine, porcine, cervine, and primates. In preferred modalities, the individual is a human being. In other modalities, the individual is an animal, preferably, where the animal is Petition 870250088218, dated 09 / 29 / 2025, page 37 / 81 30 / 39 a pet. A pet can be an animal selected from dogs, cats, birds, fish, rodents such as mice, rats and guinea pigs, rabbits, etc.

[00112] The individual can be of any age. Properly, the individual can be a child or an adult. In some embodiments, the individual is a young person, an adolescent, a child, or an infant. The term young person can refer to an individual who has not yet reached adulthood. The term adolescent can refer to an individual during the period from the onset of puberty to adulthood. The term child means a human being in the stages between birth and puberty. The term infant can refer to a human individual aged approximately 0 to approximately 1 year. In other embodiments, the individual is an adolescent or an adult. In one embodiment, the individual is a human being aged approximately 3 months or more.

[00113] The individual may have any peroxisomal disorder described in this document. Any appropriate diagnostic method may be used to determine if the individual has a peroxisomal disorder (see, for example, Ferdinandusse, S., et al., 2016. Journal of the Society for the Study of Inborn Errors of Metabolism, 39(4), pp.531-543; and Klouwer, FC, et al., 2016. Neuropediatrics, 47(04), pp.205-220). For example, a peroxisomal disorder may be confirmed by biochemical tests and / or genetic testing.

[00114] The individual may have increased plasma levels of substrates normally handled by peroxisomes, such as VLCFAs, pristanic acid, phytanic acid, DHCA, THCA, and pipecolic acid. The individual may have decreased levels of end products of peroxisomal metabolism, such as plasmalogens, cholic and chenodeoxycholic acid, and docosahexaenoic acid.

[00115] The individual may have a defect in a PEX gene, a gene Petition 870250088218, dated 09 / 29 / 2025, page 38 / 81 31 / 39 associated with a peroxisomal fission defect and / or a gene encoding a peroxisomal protein. As described above, PBDs can be caused by defects in any one of at least 14 different PEX genes, which encode proteins involved in peroxisome assembly and proliferation, and PEDs caused by defects in any peroxisomal protein involved in peroxisomal function. The development of next-generation sequencing technology allows parallel sequencing of multiple genes involved in peroxisomal disorders. PBDs can be diagnosed by PEX cDNA transfection complementation assays followed by sequencing of the implicated PEX gene (see, for example, Krause, C., et al., 2009. European journal of human genetics, 17(6), pp.741-748).

[00116] In some forms, the individual has a defect in one or more genes selected from among: PEX1, PEX2, PEX3, PEX5, PEX6, PEX7, PEX10, PEXIla, PEXIie, PEX11y, PEX12, PEX13, PEX14, PEX16, PEX19, PEX26, DLP1, Mff, GDAP1, Fis1, ABCD1, ACOX1, HSD17B4, SCP2, AMACR, PHYH, AGXT, GNPAT, AGPS, FAR1, ABCD3, BAAT, and CAT.

[00117] In some modalities, the individual has a defect in one or more PEX genes. In some modalities, the individual has a defect in one or more genes selected from: PEX1, PEX2, PEX3, PEX5, PEX6, PEX7, PEX10, PEX11a, PEX11ie, PEX11y, PEX12, PEX13, PEX14, PEX16, PEX19, and PEX26.

[00118] In some forms, the individual has a defect in one or more genes associated with a PBD.

[00119] In some modalities, the individual has a defect in one or more genes associated with a peroxisomal fission defect. In some modalities, the individual has a defect in one or more genes selected from: DLP1, Mff, GDAP1, Fis1, PEXIla, PEXIie, and PEXIIy. Petition 870250088218, dated 09 / 29 / 2025, p. 39 / 81 32 / 39

[00120] In some modalities, the individual has a defect in one or more genes that encode a peroxisomal protein. In some modalities, the individual has a defect in one or more genes selected from: ABCD1, ACOX1, HSD17B4, SCP2, AMACR, PHYH, AGXT, GNPAT, AGPS, FAR1, ABCD3, BAAT, and CAT. Administration

[00121] Decanoic acid can be administered to the individual by any suitable route. Decanoic acid or the composition comprising it can be administered enterally or parenterally.

[00122] In some embodiments, decanoic acid or a composition comprising the same is administered enterally. Enteral administration may be oral, gastric and / or rectal. In general terms, the administration of the combination or composition, as described in the present invention, may be, for example, orally or by another route that enters the gastrointestinal tract, for example, administration may be via enteral feeding. In some embodiments, decanoic acid or a composition comprising the same is administered orally or via a feeding tube.

[00123] Decanoic acid or a composition comprising the same may be administered to the individual via any suitable administration regimen. Suitablely, decanoic acid or a composition comprising the same is administered once daily, twice daily, three times daily, or four times daily. Suitablely, decanoic acid or a composition comprising the same is administered as part of a meal, snack, or enteral feeding. Suitablely, decanoic acid or a composition comprising the same is administered daily for at least six days.

[00124] Decanoic acid or a composition comprising the same Petition 870250088218, dated 09 / 29 / 2025, pp. 40 / 81 33 / 39 mo can be administered to the individual in any appropriate dosage. Appropriately, decanoic acid is administered to the individual in an amount of at least about 2 g, at least about 5 g, or at least about 10 g per day. Appropriately, decanoic acid is administered in an amount of about 2 g to about 200 g per day, about 5 g to about 150 g per day, or about 10 g to about 100 g per day. In one embodiment, decanoic acid is administered in an amount of about 14.5 to about 58 g per day. In one embodiment, decanoic acid is administered in an amount of about 14.5 g per day, about 29 g per day, or about 58 g per day.

[00125] In one embodiment, decanoic acid or a composition comprising the same is administered as part of a ketogenic diet. If decanoic acid or a composition comprising the same is administered as part of a ketogenic diet, the ratio of total fat to protein / carbohydrate content may be altered during therapy to achieve nutritional goals and optimize clinical benefit.

[00126] The classic version of the ketogenic diet uses ratios to determine and describe fat content. The ketogenic ratio represents the relationship between grams of fat and the combined grams of protein and carbohydrate. At a ratio of 4:1, there are four times more grams of fat for every 1 g of protein and carbohydrate combined. The ratio is traditionally intended to regulate the degree of ketosis, with higher ratios theoretically stimulating greater ketosis. The ketogenic ratio can range from, for example, about 1:1 to about 7:1, about 1:1 to about 5:1, for example, about 1:1, about 1.5:1, about 2:1, about 2.5:1, about 3:1, about 3.5:1, about 4:1, about 4.5:1 or about 5:1. In one embodiment, the ketogenic ratio is about 2.25:1 to about 3.9:1. In another embodiment, the ketogenic ratio is about 2.25:1 to about 3.9:1. Petition 870250088218, dated 09 / 29 / 2025, p. 41 / 81 In the 34 / 39 modality, the ketogenic ratio is approximately 2.26 to approximately 3.8:1 or approximately 2.7 to approximately 3.4:1. In other modalities, the ketogenic ratio is approximately 3.21:1, approximately 3.23:1, approximately 3.24:1, approximately 3.25:1, approximately 3.26:1, approximately 3.27:1, approximately 3.28:1, or approximately 3.29:1. Peroxisome proliferator

[00127] In one aspect, the present invention provides the use of decanoic acid as a peroxisome proliferator. The use can be in vitro. As used herein, a peroxisome proliferator can refer to an agent that induces peroxisome proliferation. Examples

[00128] The invention will now be further described by means of examples, which are intended to assist those skilled in the art in carrying out the invention and are not intended to limit the scope of the invention. Example 1 - Decanoic acid and peroxisomal function

[00129] SH-SY5Y cells were stored frozen, and cells with passage numbers between 20 and 24 were used. When needed, SH-SY5Y cells were thawed at 20-25°C and rapidly seeded at a density of 13.3 χ 103 cells / cm2 in 75 cm2 flasks. SH-SY5Y cells were then cultured in media containing 1:1 DMEM / F-12 + 100 mL / L FBS + 10 mL / L l-glutamine at +37°C and 5% CO2. SH-SY5Y cells were exposed to 250 uM decanoic acid (C10) or octanoic acid (C8) for 6 days.

[00130] To evaluate the effects on peroxisomal content and function, the following parameters were studied: (a) activity of the peroxisomal marker enzyme, acyl CoA oxidase, (b) expression of Pex11a, a gene associated with peroxisomal biogenesis, and (c) evaluation of beta-oxidation of peroxisomal fatty acids by monitoring the en Petition 870250088218, dated 09 / 29 / 2025, page 42 / 81 35 / 39 chain shortening of a very long-chain fatty acid, C22, to C18, C16 and C14.

[00131] Exposure to C10 was associated with an increase in acyl CoA oxidase activity (Figure 1A), an increase in Pex11a (Figure 1B), and an increase in the ability to perform peroxisomal beta-oxidation (Figure 1C). These results show that C10 may exert additional positive effects on peroxisomal function. MODALITIES

[00132] Several preferred features and embodiments of the present invention will now be described with reference to the following numbered paragraphs (paragraphs).

[00133] 1. Decanoic acid for use in the treatment and / or prevention of a peroxisomal disorder in an individual.

[00134] 2. Decanoic acid for use in accordance with paragraph 1, wherein the peroxisomal disorder is a peroxisome biogenesis disorder (PBD) or a peroxisomal enzyme deficiency (PED).

[00135] 3. Decanoic acid for use in accordance with paragraph 1 or 2, wherein the peroxisomal disorder is a Zellweger spectrum disorder (ZSD); rhizomelic chondriodysplasia punctata (RCDP) type 1; RCDP type 5; X-linked adrenoleukodystrophy (X-ALD); acyl-CoA oxidase deficiency; bifunctional protein D deficiency; SCPx deficiency; 2-methylacyl-CoA racemace (AMACR) deficiency; Refsum disease primary hyperoxaluria type 1; RCDP type 2; RCDP type 3; RCDP type 4; ABCD3 deficiency; BAAT deficiency; or Acatalasemia.

[00136] 4. Decanoic acid for use in accordance with any preceding paragraph, wherein the peroxisomal disorder is a peroxisome biogenesis disorder (PBD).

[00137] 5. Decanoic acid for use according to any para Petition 870250088218, dated 09 / 29 / 2025, page 43 / 81 36 / 39 previous graph, where the peroxisomal disorder is a Zellweger spectrum disorder (ZSD), rhizomelic chondriodysplasia punctata (RCDP) type 1 or RCDP type 5, optionally where the peroxisomal disorder is a ZSD.

[00138] 6. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the peroxisomal disorder is Heimler syndrome (HS), infantile Refsum disease (IRD), neonatal adrenoleukodystrophy (NALD) or Zellweger syndrome (ZS), optionally wherein the peroxisomal disorder is HS, IRD or NALD.

[00139] 7. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the individual has a defect in a PEX gene or a gene encoding a peroxisomal protein.

[00140] 8. Decanoic acid for use in accordance with any preceding paragraph, wherein the individual has a defect in one or more selected genes from: PEX1, PEX2, PEX3, PEX5, PEX6, PEX7, PEX10, PEX12, PEX13, PEX14, PEX16, PEX19, PEX26, ABCD1, ACOX1, HSD17B4, SCP2, AMACR, PHYH, AGXT, GNPAT, AGPS, FAR1, ABCD3, BAAT, and CAT.

[00141] 9. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the individual has a defect in a PEX gene.

[00142] 10. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the individual has a defect in one or more of the following selected genes: PEX1, PEX2, PEX3, PEX5, PEX6, PEX7, PEX10, PEX12, PEX13, PEX14, PEX16, PEX19, PEX26.

[00143] 11. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the individual is a mammal, preferably wherein the individual is a human.

[00144] 12. Decanoic acid for use in accordance with any of the preceding paragraphs, where the individual is a child or an adult.

[00145] 13. Decanoic acid for use according to any pa Petition 870250088218, dated 09 / 29 / 2025, pp. 44 / 81 37 / 39 previous paragraph, whereby decanoic acid is in the form of triglycerides, diacylglycerides, monoacylglycerides, a pharmaceutically acceptable salt and / or an ester.

[00146] 14. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the decanoic acid is in the form of triglycerides, diacylglycerides and / or monoacylglycerides.

[00147] 16. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the decanoic acid is in the form of triglycerides, preferably wherein the decanoic acid is in the form of medium-chain triglycerides.

[00148] 17. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the decanoic acid is in the form of a C10 homotriglyceride.

[00149] 18. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the decanoic acid is comprised in a composition.

[00150] 19. Decanoic acid for use in accordance with the paragraph, wherein the composition is a pharmaceutical composition, a nutritional composition or a nutritional supplement.

[00151] 20. Decanoic acid for use in accordance with paragraph or 19, wherein the composition is in the form of an oil-in-water emulsion, a powder or a foodstuff, preferably wherein the composition is in the form of an oil-in-water emulsion.

[00152] 21. Decanoic acid for use in accordance with any of paragraphs 18 to 20, the composition being in a ready-to-use form.

[00153] 22. Decanoic acid for use in accordance with any of paragraphs 18 to 21, and the composition is free of, or substantially free of, non-decanoic fatty acid fractions. Petition 870250088218, dated 09 / 29 / 2025, p. 45 / 81 38 / 39 decanoic or octanoic acid, optionally, the composition being free of, or substantially free of, fatty acid fractions other than decanoic acid.

[00154] 23. Decanoic acid for use in accordance with any of paragraphs 18 to 22, the composition being free of MCTs comprising fatty acid fractions other than decanoic acid or octanoic acid, optionally the composition being free of, or substantially free of, MCTs comprising fatty acid fractions other than decanoic acid.

[00155] 24. Decanoic acid for use in accordance with any of paragraphs 18 to 23, wherein the composition comprises proteins, fat and carbohydrates, and wherein at least about 50% by weight of the fat is decanoic acid, optionally at least about 55% by weight, at least about 60% by weight, at least about 65% by weight, at least about 70% by weight of the fat is decanoic acid.

[00156] 25. Decanoic acid for use in accordance with any of paragraphs 18 to 24, wherein the decanoic acid is present in the composition in an amount of at least about 100 g / L, optionally wherein the decanoic acid is present in the composition in an amount of at least about 110 g / L, at least about 120 g / L, at least about 130 g / L, at least about 140 g / L, at least about 150 g / L, at least about 160 g / L, at least about 180 g / L, at least about 200 g / L, at least about 220 g / L, or at least about 240 g / L.

[00157] 26. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the decanoic acid is administered enterally or parenterally, preferably wherein the decanoic acid is administered enterally.

[00158] 27. Decanoic acid for use according to any pa Petition 870250088218, dated 09 / 29 / 2025, pp. 46 / 81 39 / 39 previous paragraph, whereby decanoic acid is administered orally or via a feeding tube.

[00159] 28. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the decanoic acid is administered once daily, twice daily, three times daily or four times daily.

[00160] 29. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein the decanoic acid is administered as part of a meal, snack or enteral feeding.

[00161] 30. Decanoic acid for use in accordance with any preceding paragraph, wherein the decanoic acid is administered in an amount of about 2 ga about 200 g per day, about 5 ga about 150 g per day, or about 10 ga about 100 g per day.

[00162] 31. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein decanoic acid improves peroxisomal function and / or peroxisomal count.

[00163] 32. Decanoic acid for use in accordance with any preceding paragraph, wherein decanoic acid improves peroxisomal function.

[00164] 33. Decanoic acid for use in accordance with any of the preceding paragraphs, wherein decanoic acid increases acyl CoA oxidase activity, increases Pex11a expression and / or increases peroxisomal beta-oxidation capacity.

[00165] 34. Method of treatment and / or prevention of a peroxisomal disorder, the method comprising the administration of decanoic acid to an individual who needs it.

[00166] 35. Use of decanoic acid for the manufacture of a medicament for the treatment and / or prevention of a peroxisomal disorder.

[00167] 36. Use of decanoic acid as a peroxisome proliferator in vitro. Petition 870250088218, dated 09 / 29 / 2025, page 47 / 81

Claims

1 / 3 CLAIMS 1. Use of decanoic acid, characterized in that it is for the preparation of a medicament and / or composition for the treatment and / or prevention of a peroxisomal disorder.

2. Use, according to claim 1, characterized in that the peroxisomal disorder is a peroxisome biogenesis disorder (PBD) or a peroxisomal enzyme deficiency (PED), optionally the peroxisomal disorder being a Zellweger spectrum disorder (ZSD); rhizomelic chondriodysplasia punctata (RCDP) type 1; RCDP type 5; X-linked adrenoleukodystrophy (XALD); acyl-CoA oxidase deficiency; bifunctional protein deficiency; SCPx deficiency; 2-methylacyl-CoA racemace (AMACR) deficiency; Refsum disease primary hyperoxaluria type 1; RCDP type 2; RCDP type 3; RCDP type 4; ABCD3 deficiency; BAAT deficiency; or Acatalasemia.

3. Use, according to claim 1 or 2, characterized in that the individual has a defect in a PEX gene or a gene encoding a peroxisomal protein, optionally the individual has a defect in one or more genes selected from: PEX1, PEX2, PEX3, PEX5, PEX6, PEX7, PEX10, PEX12, PEX13, PEX14, PEX16, PEX19, PEX26, ABCD1, ACOX1, HSD17B4, SCP2, AMACR, PHYH, AGXT, GNPAT, AGPS, FAR1, ABCD3, BAAT, and CAT.

4. Use, according to any one of claims 1 to 3, characterized in that the decanoic acid is in the form of triglycerides, diacylglycerides, monoacylglycerides, a pharmaceutically acceptable salt and / or an ester.

5. Use, according to any one of claims 1 to 4, characterized in that the decanoic acid is in the form of a C10 homotriglyceride. Petition 870250089316, dated 10 / 01 / 2025, page 7 / 12 2 / 3 6. Use, according to any one of claims 1 to 5, characterized in that decanoic acid is comprised in a composition.

7. Use according to claim 6, characterized in that the composition is a pharmaceutical composition, a nutritional composition or a nutritional supplement.

8. Use according to claim 6 or 7, characterized in that the composition is in the form of an oil-in-water emulsion, a powder or a foodstuff, or the composition is in the form of an oil-in-water emulsion.

9. Use, according to any one of claims 6 to 8, characterized in that the composition is in a ready-to-use form.

10. Use, according to any one of claims 6 to 9, characterized in that the composition comprises proteins, fat and carbohydrates, and wherein at least 50% by weight of the fat is decanoic acid, or wherein at least 55% by weight, at least 60% by weight, at least 65% by weight, or at least 70% by weight of the fat is decanoic acid.

11. Use, according to any one of claims 6 to 10, characterized in that decanoic acid is present in the composition in an amount of at least 100 g / L, or in that decanoic acid is present in the composition in an amount of at least 110 g / L, at least 120 g / L, at least 130 g / L, at least 140 g / L, at least 150 g / L, at least 160 g / L, at least 180 g / L, at least 200 g / L, at least 220 g / L, or at least 240 g / L.

12. Use, according to any of claims 1 to 11, characterized in that said medicine and / or composition is formulated and / or prepared to provide decanoic acid via enteral or parenteral route, or that said medicine and / or composition is formulated and / or prepared to provide decanoic acid orally or via a feeding tube.

13. Use, according to any one of claims 1 to 12, characterized in that said medicine and / or composition is formulated and / or prepared to provide decanoic acid once daily, twice daily, three times daily or four times daily.

14. Use, according to any one of claims 1 to 13, characterized in that said medicine and / or composition is formulated and / or prepared to provide decanoic acid as part of a meal, snack or enteral feeding.

15. Use, according to any one of claims 1 to 14, characterized in that decanoic acid improves peroxisomal function and / or peroxisomal number, or in that decanoic acid increases acyl CoA oxidase activity, increases Pex11a expression and / or increases peroxisomal beta-oxidation capacity.

16. Use of decanoic acid, characterized by the fact that it is a peroxisome proliferator in vitro.

17. Invention, characterized by any form of its embodiment or any applicable class or category of claim, for example, product, process or use covered by the object initially described, disclosed or illustrated in this patent application, for example: Decanoic acid for use in the treatment and / or prevention of a peroxisomal disorder; and / or Method for treatment and / or prevention of a peroxisomal disorder, comprising the administration of decanoic acid to an individual who needs it. Petition 870250089316, dated 01 / 10 / 2025, p. 9 / 12

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