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Adeno-associated virus vector pharmaceutical compositions and methods

A composition and drug technology, applied in the direction of drug combination, botany equipment and method, biochemical equipment and method, etc., can solve the problems of excipient concentration, affecting supply chain and distribution, affecting the stability of biological preparations, etc.

Pending Publication Date: 2022-07-08
再生生物股份有限公司
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0007] Maintaining various buffer properties within target specifications is critical to ensure product stability, but storage at -80°C impacts supply chain and distribution
Crystallization of water during slow freezing can lead to excipient concentration, which can affect the stability of biologics
Phase separation or pH changes may also occur, which can affect the stability of biologics

Method used

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  • Adeno-associated virus vector pharmaceutical compositions and methods
  • Adeno-associated virus vector pharmaceutical compositions and methods
  • Adeno-associated virus vector pharmaceutical compositions and methods

Examples

Experimental program
Comparison scheme
Effect test

example 3

[0747] 5.3 Example 3: Comparison of Formulation A and Formulation B in Stability

[0748] This example shows a comparison of Formulation A and Formulation B in terms of their stability.

[0749] The new Formulation B prevented capsid rupture and release of small amounts of free DNA under freeze / thaw cycles and temperature stress. The current 12-month long-term stability study demonstrated that the in vitro relative potency and other quality attributes in FDP Formulation B were maintained at -80°C (≤-60°C) and -20°C (-25°C to -15°C).

[0750] Available freeze / thaw data, temperature stress data, and long-term stability data suggest that the new formulations have similar or improved stability.

[0751] The FDP batches in the new FDP formulation B can be set for long-term stability at -80°C (≤-60°C) and -20°C (-25°C to -15°C) and stability trend data can be used as stability planning Part of the monitoring to ensure that the expiration date of the new FDP complies with regulatio...

example 7

[0889] 5.7 Example 7: Comparison of Formulation A and Formulation B in Efficacy after 30 Freeze and Thaw Cycles

[0890] This example shows a comparison of Formulation A and Formulation B in efficacy after 30 freeze and thaw cycles.

[0891] The studies were performed by procedures similar to those shown in Section 4.5.1, Section 5.4.5(c), and / or other relevant sections provided herein.

[0892] The example was performed for AAV8 with the green fluorescent protein gene. Freeze-thaw cycles are used to simulate temperature changes in shipping and storage logistics, and can also act as an "accelerating" stress to force AAV degradation for formulation optimization work. like Figure 36 As shown, "modified dPBS with 4% sucrose" Formulation B (dark grey bars) maintained potency after 30 freeze-thaw cycles. In contrast, the reference formulation (dPBS, light grey bars) efficacy decreased to between 66% and 72% after 15 to 30 freeze-thaw cycles.

[0893]The shift to an acidic pH r...

example 10

[0900] 5.10 Example 10: Comparison of Physicochemical Properties of Formulations

[0901] This example shows the characterization of the physicochemical properties of different Construct II formulation buffer candidates, including pH, glass transition temperature (Tg'), osmolarity and density. Construct II is currently designed to be stored in a frozen formulation. The effect of freezing and freezer temperature fluctuations on buffer pH was investigated by tracking pH and temperature in real time using a cryogenic pH probe in a -20°C automatic defrost (-20°C AD) freezer. The phase transition of the formulations upon freezing and thawing was assessed by calorimetry. The results of this study indicate that pH changes of varying magnitudes depend on the formulation buffer composition, which is a key factor considering the importance of stable pH for long-term storage of pharmaceutical products. Additionally, the glass transition temperatures of the different formulation buffers...

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Abstract

Provided herein are pharmaceutical compositions comprising a recombinant adeno-associated virus (AAV), a salt excipient or buffer, a sugar, and a surfactant. Also provided herein are methods for treating or preventing a disease in a subject by administering to the subject in need thereof a therapeutically effective amount of the pharmaceutical composition.

Description

[0001] Cross-reference to related applications [0002] This application claims the benefit of US Provisional Application No. 62 / 911,968, filed October 7, 2019, the contents of which are incorporated herein by reference in their entirety. [0003] References to Sequence Listings Submitted Electronically [0004] This application incorporates by reference the Sequence Listing entitled "Sequence_Listing_12656-124-228.TXT", filed with this application, created on September 28, 2020 and having a size of 97,652 bytes, as a text file. Background technique [0005] A member of the Parvoviridae family designated as virus-dependent, adeno-associated viruses (AAVs) are small non-enveloped icosahedral viruses with single-stranded linear DNA genomes ranging from approximately 4.7 kilobases (kb) to 6 kb. Properties of non-pathogenicity, infectivity over a broad host and cell type tropism range, including dividing and non-dividing cells, and the ability to establish long-term transgene ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K39/00A61P27/02C12N15/63C12N15/86C12N15/864C07K16/22A61K48/00
CPCC12N15/86A61P27/02A61K39/00C07K16/22C12N2750/14143A61K9/08A61K9/19A61K47/26A61K47/183A61K47/02A61K47/10A61K9/0019A61K9/0048A61K35/76A61K2039/505A61K2039/5256A61K35/761
Inventor T·马歇尔J·比K·欧贝里Y·张R·德帕斯
Owner 再生生物股份有限公司
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