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    Home > Active Ingredient News > Blood System > The AAV gene therapy company founded by the Chinese uses a lower dose to treat hemophilia A doctor Mai Meng broke the news

    The AAV gene therapy company founded by the Chinese uses a lower dose to treat hemophilia A doctor Mai Meng broke the news

    • Last Update: 2022-04-27
    • Source: Internet
    • Author: User
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    April 5, 2022/eMedClub News/--Recently, ASC Therapeutics announced that its investigational hemophilia A gene therapy ASC618 has been granted Fast Track designation by the U.
    S.
    Food and Drug Administration (EMA) and an orphan drug designation by the European Medicines Agency (EMA).
    affirmed positive opinion
    .

    ASC618 is an AAV gene therapy that carries a codon-optimized B-domain-deleted factor VIII gene and a minimal-length, liver-specific promoter capable of endogenously producing factor VIII for the treatment of hemophilia
    A.

    In preclinical studies, ASC618 was able to produce sufficient factor VIII levels at doses significantly lower than other hemophilia A gene therapies
    .

    This strategy greatly reduces the dose level of AAV gene therapy, directly reduces the hepatotoxic side effects caused by gene therapy, and improves the safety of gene therapy
    .

    Previously, ASC618 has received FDA IND clearance and FDA orphan drug designation
    .

    In July 2021, ASC Therapeutics announced that the FDA has approved the IND application for ASC618 for the treatment of patients with severe or moderate hemophilia
    A.

    This also makes ASC Therapeutics the first Chinese-founded company to submit an IND application to the FDA in the field of gene therapy
    .

    Recommended reading: Lower doses! U.
    S.
    FDA approves "high-secretion" protein optimization strategy AAV gene therapy for the treatment of hemophilia AASC Therapeutics was established in 2008 and is headquartered in the San Francisco Bay Area, California, and has strategies in the United States and China Deploy
    .

    The company has unique allogeneic cell therapy and precision gene modification technologies, and has established an international leading position in the fields of cell therapy, precision gene editing and gene therapy
    .

    At the same time, the company is also the first high-tech enterprise with gene editing therapy, gene therapy and cell therapy technologies authorized by exclusive global and Chinese patents
    .

    In April 2014 and September 2016, the company was twice rated as "Global TOP10 Companies Leading Precision Gene Editing Application Technology" by Nature Biotechnology
    .

    ▲ The development and status of AAV gene therapy for hemophilia A, Dr.
    Jiang Ruhong, founder, chairman, president and CEO of ASC Therapeutics In the field of AAV gene therapy, hemophilia is one of the hottest tracks
    .

    In recent years, due to advances in gene therapy and protein engineering, gene therapy has achieved significant breakthroughs in the clinical application of hemophilia, and the drug market for hemophilia is undergoing major changes
    .

    The original mutant gene of hemophilia A is very large, exceeding the packaging upper limit of the AAV vector.
    Therefore, after more than ten years of efforts, researchers successfully cut out the unnecessary part of the gene, which has since brought about blood type A blood.
    The pomp of gene therapy development for palliative disease
    .

    According to a report published by Coherent Market Insights, the hemophilia gene therapy market is estimated to exceed USD 780 million in 2026, with a compound annual growth rate (CAGR) from 2018 to 2026 or as high as 56.
    0%
    .

    Compared with hemophilia B, the incidence of hemophilia A is about 4 times that of hemophilia B, and the market prospect is broad
    .

    At present, in addition to ASC Therapeutics, many international companies are deploying gene therapy products for hemophilia A.
    Here are some examples of candidate products that are progressing faster: ➤ BioMarin's valoctocogene roxaparvovec (valrox) In January 2022, the company announced Complete data for valrox in Phase 3 clinical study GENEr8-1, 134 subjects met all primary and secondary efficacy endpoints in the two-year analysis
    .

    Phase 3 clinical study GENEr8-1 performed the designated primary analysis on participants from a previous non-interventional study, all of whom received a single 6e13 vg/kg dose
    .

    The data showed that the annualized bleeding rate (ABR) was reduced by 85% from baseline, and the cumulative mean annualized bleeding rate (ABR) was less than 1
    .

    The EMA has previously accepted a Marketing Application (MAA) for this product candidate for adults with severe hemophilia
    A.

    In March 2021, the FDA granted valrox Regenerative Medicine Advanced Therapy Designation (RMAT)
    .

    Based on these results, BioMarin plans to resubmit its marketing application (BLA) to the FDA in the second quarter of 2022, followed by a six-month review by the FDA
    .

    Recommended reading: The first hemophilia gene therapy is about to hit the market? BioMarin Announces Phase 3 Data to Complete the Regulatory PuzzleRoche/Spark: SPK-8011, SPK-8016 Expression after AAV Gene Transfer for Hemophilia A" Phase 1/2 clinical trial results, and the latest follow-up data for this product candidate will be announced at the ISTH2021 meeting
    .

    Of the 18 patients treated with SPK-8011, 16 maintained persistent factor VIII expression; the patients' annual bleeding rate (ABR) decreased by 91.
    2% and the annual factor VIII infusion rate decreased by 97%
    .

    The company has another candidate gene therapy, SPK-8016, also targeting hemophilia A, which updated preliminary data from a Phase 1/2 clinical trial in February 2021
    .

    Recommended reading: Roche/Spark announces the latest clinical data on gene therapy for hemophilia AYimai Meng broke the news ➤ Pfizer/Sangamo: giroctocogene fitelparvovec (SB-525) giroctocogene fitelparvovec (SB-525, PF-07055480) is a Recombinant AAV2/6 vector-based gene therapy co-developed by Pfizer and Sangamo
    .

    Its design improves liver-specific promoters, factor VIII-encoding transgenes, and polyA and viral vector sequences, which not only optimize the efficiency of vector production, but also enhance liver-specific factor VIII protein expression
    .

    On October 7, 2020, Pfizer/Sangamo announced the initiation of AFFINE, a Phase 3 clinical trial of its hemophilia A candidate gene therapy SB-525, which will evaluate the efficacy and safety of SB-525 in patients with moderate to severe hemophilia A.
    security
    .

    The study has an observation period of 5 years to further assess the durability and efficacy of this gene therapy
    .

    ➤ Bayer/Ultragenyx Pharmaceuticals: BAY2599023 (DTX201, AAVhu37 FVIII) In August 2018, Ultragenyx announced a collaboration with Bayer to develop a gene therapy BAY2599023 (DTX201, AAVhu37 FVIII) for the treatment of hemophilia
    A.

    DTX201 uses REGENXBIO's proprietary NAV AAVhu37 vector to deliver the coagulation factor VIII gene to hepatocytes and promote its sustained expression to potentially overcome the deficiency in hemophilia A patients
    .

    Preliminary data from Phase 1/2 clinical trial results of DTX201 showed that DTX201 was able to safely and effectively increase levels of coagulation factor VIII (FVIII) and prevent or reduce bleeding in the first two patients with severe hemophilia A in the clinical trial
    .

    Recommended reading: ASH2019: Bayer AAV gene therapy in hemophilia A Phase 1/2 clinical trial results are positiveYimai Meng broke the news that other clinical candidates include Freeline's FLT210, Shire/Baxalta's SHP654,
    etc.

    Not long ago, Suzhou Huayi Lejian Biotechnology Co.
    , Ltd.
    , founded by Professor Rao Yi, also applied for an IIT study of AAV gene therapy for hemophilia A, which was approved by the Genetics Office
    .

    The clinical trial will be carried out at the Hematology Hospital of the Chinese Academy of Medical Sciences
    .

    Recommended reading: The company founded by Professor Rao Yi chose hemophilia A, and another AAV gene therapy passed the approvalYimai Meng broke the news Reference: 1.
    https:// therapeutics-receives-key-regulatory-designations-in-us-and-europe-to-advance-its-second-generation-gene-therapy-for-hemophilia-a/——List of recent popular events——▼April 12 July 1st-3rd, Shanghai, 2022 Shanghai International Bio-Innovative Drug Industry Expo
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