gene therapy

February 13, 2024

PPMD Provides $500,000 in Funding to Kinea Bio Through PPMD Venture Pathways Program to Support Next-Gen Midi-Dystrophin Gene Therapy Development

PPMD is excited to announce that the organization has provided $500,000 in funding to Kinea Bio, Inc. (Kinea) through PPMD Venture Pathways, the organization’s venture philanthropy program which provides industry funding to accelerate therapeutic development….

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January 11, 2024

Introducing PPMD’s Gene Therapy Hub

We are excited to announce the launch of PPMD’s Gene Therapy Hub! Gene therapy has made significant strides in Duchenne, with one therapy approved and other potential therapies in various stages of development. As our…

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FDA grants approval via accelerated approval pathway to ELEVIDYS: First-ever gene therapy for individuals with Duchenne ages 4-5

Today is an incredible milestone for the entire Duchenne community. It is a first step. But a huge step forward. Today, the FDA has granted accelerated approval to ELEVIDYS (delandistrogene moxeparvovec-rokl), a micro-dystrophin gene therapy…

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Sarepta Announces PDUFA Extension and Update for the FDA’s Review of SRP-9001

Sarepta Therapeutics announced today that they received an update from the FDA on the Biologics License Application (BLA) for SRP-9001. The PDUFA date reportedly has been extended to June 22, 2023. …

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SRP-9001: Submit Your Community Perspectives to FDA

On May 12, the FDA held an Advisory Committee meeting for SRP-9001, a gene therapy intended to treat Duchenne. The Advisory Committee meeting was used by FDA to obtain independent expert advice on scientific, technical,…

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A Community United: Thank You for Raising Your Voice During the SRP-9001 Advisory Committee

Today was a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy intended to treat Duchenne. This meeting was held as part of the regulatory review process for…

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May 12: Advisory Committee Meeting for SRP-9001 – Live Stream Information & Meeting Materials Released

The FDA has shared live stream access information for the May 12th Advisory Committee for SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics’s investigational gene therapy, which has applied for FDA approval through the Accelerated Approval pathway with…

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What You Need To Know Ahead of the SRP-9001 Advisory Committee on Friday, May 12

This Friday, May 12th, will be a historic day for the Duchenne community. It will be the day of the first ever FDA Advisory Committee meeting for a gene therapy specifically to treat Duchenne. PPMD…

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PPMD Submits Findings from New Gene Therapy Preference Study to FDA for SRP-9001 Advisory Committee

May 12 will be a historic day for the Duchenne community: the first FDA Advisory Committee meeting for a gene therapy specifically intended to treat Duchenne. PPMD, our community partners, and many others have long…

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PPMD & Community Partners Submit Letter to FDA Encouraging Appropriate Regulatory Flexibility in SRP-9001 Review

PPMD along with community partners and key opinion leaders and physicians in the Duchenne space have submitted a letter to the US Food and Drug Administration (FDA) and FDA’s Center for Biologics Evaluation and Research…

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