An AAV therapy alignment

Sarepta and StrideBio announce multi-target strategic collaboration

Mel J. Yeates
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CAMBRIDGE, Mass. and RESEARCH TRIANGLE PARK, N.C.—Sarepta Therapeutics, Inc., a company in precision genetic medicine for rare diseases, and StrideBio, Inc., a developer of novel adeno-associated viral (AAV)-based gene therapies, have announced the signing of a collaboration and license agreement to develop in vivo AAV-based therapies for up to eight central nervous system (CNS) and neuromuscular targets.
“With our partnership with StrideBio, Sarepta continues to build on its leadership position in gene therapies to treat rare diseases. We are excited to work with StrideBio and access its innovative AAV platform for next-generation capsids,” said Doug Ingram, Sarepta’s president and chief executive officer. “Our partnership with StrideBio expands our research portfolio by up to eight new targets and, through our strategic partnering approach that has our collaborator lead all IND-enabling research and development, ensures that we gain access to new technology and targets while not distracting Sarepta from its near-term priorities.” 
Under the terms of the agreement, StrideBio will be responsible for AAV capsid development, non-clinical development and manufacturing of preclinical candidates to be selected for advancement into clinical studies. The companies will also share early clinical development activities for selected targets, with Sarepta responsible for late stage development and commercialization. Four initial targets are specified under the collaboration, which are MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome) and NPC1 (Niemann-Pick).
Sarepta will be granted an exclusive license on selected targets to leverage StrideBio’s novel, structure-driven capsid technology, intended to enhance specific tropism to tissues of interest and evade neutralizing antibodies. The companies also plan to focus on strategies intended to address re-dosing challenges in patients who have received AAV-delivered gene therapy. StrideBio will conduct all investigational new drug-enabling research, development and manufacturing for the first four CNS targets.
StrideBio will receive a $48 million upfront payment in the form of cash and Sarepta stock, in addition to significant future development, regulatory and commercial milestones for the four programs. StrideBio will also receive royalties on worldwide net sales of any commercial products developed through the collaboration. Sarepta has obtained an exclusive option to expand the collaboration to include up to an additional four targets, with an upfront payment of up to $42.5 million along with future downstream milestone payments. StrideBio has an option to obtain co-development and co-commercial rights to one of the collaboration targets. Sarepta has also made a commitment to invest in StrideBio’s next financing round. Further financial terms were not disclosed.
“We are very excited to initiate this multi-target collaboration with Sarepta, an established leader in the development and commercialization of genetic medicines. This partnership will provide significant resources and expertise to enable StrideBio’s continued rapid expansion of our research and manufacturing platform, as well as accelerate the development of AAV gene therapies for multiple rare disease targets,” added Sapan Shah, Ph.D., chief executive officer, StrideBio. “We are looking forward to working together with Sarepta to bring novel treatments to patients as quickly as possible.”
This isn’t the only recent AAV collaboration StrideBio has made. Earlier this year in March, StrideBio signed a collaboration and license agreement with Takeda Pharmaceutical Company Ltd. to develop in vivo AAV-based therapies for Friedreich’s Ataxia and two additional undisclosed targets. These programs aim to utilize novel AAV capsids developed by StrideBio to improve potency, evade neutralizing antibodies and enhance specific tropism to tissues, including the central nervous system.
“We are very excited to partner with Takeda given their expertise and commitment to developing treatments for patients with neurological diseases,” noted Shah in a press release.
According to the agreement, StrideBio is responsible for AAV capsid development, non-clinical development and manufacturing of preclinical candidates to be selected for advancement into clinical studies. Takeda is responsible for clinical development and commercialization of selected candidates arising from the collaboration. 
StrideBio is eligible to receive approximately $30 million in upfront and near term preclinical milestones, as well as an additional $680 million in future development and commercial milestones from Takeda. StrideBio will also receive royalties on worldwide net sales of any commercial products developed through the collaboration. No further financial terms were disclosed.
“StrideBio’s expertise and unique gene therapy technology holds great potential for significantly advancing the field of neurological disease research,” stated Emiliangelo Ratti, head of the Neuroscience Therapeutic Area unit at Takeda. “Our collaboration is a natural extension of Takeda’s neuroscience research and development strategy, including modality diversification, identifying targets with a high degree of association with disease, and a focus on developing innovative medicines for neurologic diseases that have a high unmet medical need.”

Mel J. Yeates

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