Pathway Development Consortium Releases Draft White Paper to Facilitate AAV Gene Therapy Development

The Pathway Development Consortium (PDC), a new public-private collaboration founded by REGENXBIO Inc. (Nasdaq: RGNX) and Solid Biosciences Inc. (Nasdaq: SLDB), today announced the publication of a draft white paper, Draft Framework for AAV Gene Therapy Development, which proposes a framework that can be applied to AAV gene therapies to facilitate the use of the accelerated approval pathway at the US Food and Drug Administration (FDA). The white paper identifies different categories of AAV gene therapies that target the underlying monogenetic changes that cause disease and proposes generalized approaches that would clarify the evidence needed to support FDA approval. The PDC is actively seeking feedback from key stakeholders on this framework at The publication of the white paper coincides with the formal launch of the PDC, a multistakeholder initiative which aims to identify, develop, expand and maintain pathways to effective therapies for patients diagnosed early in life with rare diseases. The PDC seeks to achieve these goals by bringing together a broad and diverse group of stakeholders from the rare disease and AAV gene therapy communities, including patients, industry, regulators, academia and payers, among others, for meaningful scientific and policy discussions.

For further information, see SolidBio (

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