- Sarepta Therapeutics is paying privately-held StrideBio $48 million to develop gene therapies for at least four central nervous system conditions, potentially adding to a beefy pipeline of candidates for genetically driven rare diseases.
- Stride will develop the first four identified therapies through preclinical research and also share the cost of early clinical-stage development. Sarepta will have an option to license up to four more using Stride's capsid engineering platform.
- The deal is the third for StrideBio, following collaborations with CRISPR Therapeutics and Takeda. Between industry collaborations and venture funding, the North Carolina-based group has raised more than $93 million.
The companies identified Rett, Angelman and Dravet syndromes and Niemann-Pick disorder as the first four conditions that the new collaboration will try to treat. Four more could emerge, which would trigger an additional $42.5 million in additional payments.
Sarepta is paying the initial $48 million upfront fee in the form of combined cash and shares. StrideBio will be eligible for additional undisclosed development, regulatory and sales milestones, as well as royalties. The privately-held partner also will have an option to co-commercialization rights to one of the gene therapies, if successful.
The four StrideBio agents will join a Sarepta pipeline that already has 23 identified projects in clinical or pre-clinical development.
Cambridge, Massaschusetts-based Sarepta stated the collaboration will utilize StrideBio's "unique approach" to engineering capsids, the shells surrounding the adeno-associated virus (AAV) used by many researchers to deliver genes to cells.
StrideBio's technology tries to better target which cells their AAV-based therapies reach, as well as avoid triggering neutralizing antibodies, which can reduce the effectiveness of a gene therapy.
Immune responses to some AAV-based therapies have raised safety concerns. On Tuesday, Solid Biosciences announced the Food and Drug Administration had put a hold on its AAV9-based gene therapy for Duchenne muscular dystrophy (DMD) because of immune responses, although Sarepta's own DMD gene therapy has not seen anything similar.
As part of the agreement, Sarepta and StrideBio "plan to focus on strategies intended to address re-dosing challenges in patients who have received AAV-delivered gene therapy."
Re-treatment of patients who don't respond or have unlimited response to gene therapies is an unanswered question in this quickly evolving field, and drug developers and payers alike will closely watch any developments that emerge from the Sarepta-StrideBio re-dosing work.