gene therapy

Solid Biosciences Provides Update on IGNITE DMD Status and New Clinical Sites

Solid Biosciences shared an update on the progress of their IGNITE DMD Phase I/II clinical trial for investigational microdystrophin gene transfer, SGT-001. The update includes news about initiating dosing for their higher dose cohort of…

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PPMD Awards $100,000 Grant to Nationwide Children’s Hospital to Further Explore GALGT2 Gene Therapy

PPMD is excited to announce that Drs. Paul Martin and Kevin Flanigan from the Research Institute at Nationwide Children’s Hospital have completed work under a $100,000 grant exploring GALGT2 gene therapy technology as a potential treatment…

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Audentes Therapeutics Announces Expansion of AAV Technology Platform and Pipeline for Duchenne

Audentes Therapeutics announced that it is expanding its scientific platform and beginning development of new AAV-based genetic medicines for Duchenne. Audentes is a leading AAV-based genetic medicines company based in San Francisco. The company is…

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Sarepta Provides Micro-dystrophin Gene Therapy Update

Sarepta Therapeutics hosted a webcast earlier this morning, providing an update on encouraging 9-month functional and creatine kinase (CK) data from baseline for the original four patients in the Phase 1 open-label study of Sarepta’s…

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February 7, 2019

Solid Biosciences Announces Intention to Dose Escalate in IGNITE DMD Clinical Trial

Solid Biosciences announced today plans to move to a higher dose of SGT-001 in IGNITE DMD, their Phase 1/2 clinical trial evaluating SGT-001 microdystrophin gene therapy as a potential treatment for Duchenne. This decision is…

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January 24, 2019

PPMD Awards $105,000 Grant to Explore A Mini-Dystrophin Gene Aimed at Protecting Cardiac Function

PPMD is excited to award the University of Missouri School of Medicine a grant for $105,000 to continue development of a minimized dystrophin gene aimed at protecting cardiac function in people with Duchenne. The project is led by Dongsheng…

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December 19, 2018

PPMD Awards $329,000 Grant to Binghamton University to Examine Role of Immune System on Dystrophin Restoration

PPMD is excited to award Dr. Kanneboyina Nagaraju and his team at Binghamton University’s School of Pharmacy and Pharmaceutical Sciences a $329,000 grant to continue his work examining the immune response to the production of…

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November 13, 2018

Solid Provides Update on IGNITE DMD Clinical Trial

Solid Biosciences Inc. today shared on a updated on the IGNITE DMD clinical trial, noting the dosing of two additional patients with SGT-001 gene transfer and the company’s plan to report preliminary results from IGNITE…

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October 4, 2018

Jerry Mendell, M.D. Shares Positive Updated Results from Gene Therapy Micro-dystrophin Trial

PPMD is excited to learn that Dr. Jerry Mendell of Nationwide Children’s Hospital today shared positive results for the fourth patient in Sarepta’s micro-dystrophin trial, indicating positive functional signals for all of the patients….

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September 24, 2018

Sarepta Therapeutics Announces Clinical Hold Lifted for its Micro-dystrophin Gene Therapy Program

We are thrilled to learn that the FDA has lifted the clinical hold for Sarepta’s Duchenne micro-dystrophin gene therapy program. Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical…

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