gene therapy

Sarepta Therapeutics Signs Agreement with Hansa Biopharma for Imlifidase

PPMD is encouraged to learn that Sarepta Therapeutics announced an agreement with Hansa Biopharma, a company that has developed an IgG antibody-cleaving enzyme called imlifidase. Imlifidase is an investigational product initially developed to aid in…

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Applications Open for PPMD and Duchenne UK $1 Million Joint Research Grant Call for Gene Therapy

PPMD is thrilled to announce our partnership with Duchenne UK to launch a joint 2020 Research Grant Call, inviting research proposals for submission and review. Up to $1 million (US) is committed to a project…

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Sarepta Announces Positive Safety and Efficacy Data from SRP-9001 Gene Therapy Trial Published

Sarepta Therapeutics today announced safety and tolerability data at one year from four Duchenne clinical trial participants who received SRP-9001 micro-dystrophin (AAVrh74.MHCK7.micro-dystrophin) have been published in JAMA Neurology. PPMD is proud to have been an…

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Duchenne UK and PPMD Announce $1 Million Joint Research Grant Call for Gene Therapy

PPMD is thrilled to announce our partnership with Duchenne UK to launch a joint 2020 Research Grant Call, inviting research proposals for submission and review. Up to $1 million (US) is committed to a project…

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Pfizer Shares New Phase 1b Results of Gene Therapy in Ambulatory Boys With Duchenne

PROGRESS! Pfizer announced updated Phase 1b clinical data on PF-06939926, their investigational gene therapy being developed to treat Duchenne. The preliminary data from 9 ambulatory boys with Duchenne, aged 6 to 12 indicate that the…

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Sarepta and Dyno Therapeutics Announce Agreement to Develop Next-Generation Gene Therapy Vectors

Sarepta Therapeutics and Dyno Therapeutics announced an agreement to develop next-generation gene therapy vectors for muscle diseases. PPMD is pleased to see Sarepta’s continued investment and contribution in enhancing the future of gene therapy technologies…

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Solid Biosciences Shares Update on SGT-001 Phase I/II Clinical Hold for IGNITE DMD Program

Solid Biosciences shared today that their IGNITE DMD Phase I/II clinical trial will remain on clinical hold. Solid will continue to work with the FDA to provide further data and analyses relating to their manufacturing…

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Sarepta Therapeutics Shares COVID-19 Resources

Sarepta Therapeutics has shared a new COVID-19 resource page on its website, along with a new video providing contact phone numbers for those whom may have questions about continuity of access to Sarepta sponsored clinical…

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Audentes Therapeutics Shares Community Update on Duchenne Program & COVID-19

Audentes Therapeutics has shared an update with the community regarding their Duchenne program and COVID-19. Read the Update from Audentes Dear Duchenne Patient Leaders, On behalf of the team at Audentes, we hope that you…

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Solid Biosciences Shares Letter to the Duchenne Community: COVID-19 Update

Solid Biosciences Inc. has shared an update with the community regarding the IGNITE DMD clinical hold and COVID-19. Solid’s Letter to the Duchenne Community: Letter to the Duchenne Community: COVID-19 Update Dear Duchenne Community, During…

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