CAMBRIDGE, Mass. and RESEARCH TRIANGLE PARK, N.C., Nov. 14, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc., (NASDAQ: SRPT), the chief in precision genetic drugs for uncommon illnesses, and StrideBio, Inc., a number one developer of novel adeno-associated viral (AAV) based mostly gene therapies, at the moment introduced the signing of a collaboration and license settlement to develop in vivo AAV-based therapies for as much as eight central nervous system (CNS) and neuromuscular targets. Pursuant to the settlement, Sarepta is granted an unique license on chosen targets to leverage StrideBio’s novel, structure-driven capsid know-how, supposed to boost particular tropism to tissues of curiosity and evade neutralizing antibodies. The events additionally plan to concentrate on methods supposed to handle re-dosing challenges in sufferers who’ve acquired AAV-delivered gene remedy. StrideBio will conduct all investigational new drug (IND) enabling analysis, improvement and manufacturing for the primary 4 CNS targets, that are MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome), and NPC1 (Niemann-Choose). Moreover, Sarepta could have an unique choice to 4 extra targets based mostly on StrideBio’s capsid know-how.
StrideBio possesses an modern and proprietary platform that’s enabled by a deep information of AAV construction and a singular strategy to engineering capsids with novel features, and was co-founded by Mavis Agbandje-McKenna, Ph.D., and Aravind Asokan, Ph.D., main scientists within the area of AAV biology and gene remedy. StrideBio has created a rising portfolio of novel AAV capsids advanced in non-human primates that present diminished seroprevalence and potential for improved tropism to focused tissues.
“With our partnership with StrideBio, Sarepta continues to construct on its management place in gene therapies to deal with uncommon illnesses. We’re excited to work with StrideBio and entry its modern AAV platform for next-generation capsids,” mentioned Doug Ingram, Sarepta’s President and Chief Govt Officer. “Our partnership with StrideBio expands our analysis portfolio by as much as eight new targets and, by way of our strategic partnering strategy that has our collaborator lead all IND-enabling analysis and improvement, ensures that we achieve entry to new know-how and targets whereas not distracting Sarepta from its near-term priorities.”
“We’re very excited to provoke this multi-target collaboration with Sarepta, a longtime chief within the improvement and commercialization of genetic medicines,” said Sapan Shah, Ph.D., Chief Govt Officer, StrideBio. “This partnership will present vital assets and experience to allow StrideBio’s continued speedy growth of our analysis and manufacturing platform, in addition to speed up the event of AAV gene therapies for a number of uncommon illness targets. We’re trying ahead to working along with Sarepta to deliver novel therapies to sufferers as rapidly as attainable.”
Phrases of Settlement
Beneath the phrases of the settlement, StrideBio might be liable for AAV capsid improvement, non-clinical improvement and manufacturing of preclinical candidates to be chosen for development into scientific research. The events may even share early scientific improvement actions for chosen targets, with Sarepta liable for late stage improvement and commercialization. A complete of 4 preliminary targets are specified underneath the collaboration. StrideBio will obtain a $48 million upfront fee within the type of money and Sarepta inventory, along with vital future improvement, regulatory and business milestones for the 4 applications. StrideBio may even obtain royalties on worldwide internet gross sales of any business merchandise developed by way of the collaboration. Sarepta has additionally obtained an unique choice to increase the collaboration to incorporate as much as a further 4 targets with an upfront fee of as much as $42.5 million together with future downstream milestone funds, whereas StrideBio has an choice to acquire co-development and co-commercial rights to one of many collaboration targets. As well as, Sarepta has made a dedication to put money into StrideBio’s subsequent financing spherical. Additional monetary phrases weren’t disclosed.
About Sarepta Therapeutics
Sarepta is on the forefront of precision genetic drugs, having constructed a formidable and aggressive place in Duchenne muscular dystrophy (DMD) and extra lately in gene therapies for Limb-girdle muscular dystrophy illnesses (LGMD), MPS IIIA and different CNS-related issues, totaling over 20 therapies in numerous levels of improvement. The Firm’s applications and analysis focus span a number of therapeutic modalities, together with RNA, gene remedy and gene modifying. Sarepta is fueled by an audacious however essential mission: to profoundly enhance and prolong the lives of sufferers with uncommon genetic-based illnesses. For extra data, please go to www.sarepta.com.
About StrideBio, Inc.
StrideBio, Inc is a gene remedy firm centered on creating and creating novel adeno-associated viral (AAV) therapies for uncommon illnesses. Our STRucture Inspired DEsignal strategy holds the potential to generate distinctive AAV capsids with improved traits together with efficiency, tissue tropism, and skill to evade pre-existing antibodies. This highly effective new strategy has broad utility, enabling gene addition, gene silencing and gene modifying modalities for a variety of illnesses, together with uncommon genetic illnesses. StrideBio is headquartered in Analysis Triangle Park, NC. Present buyers embrace Hatteras Enterprise Companions, Takeda Ventures, UCB Ventures and Alexandria Actual Property Equities, Inc. For extra data, please go to www.stridebio.com.
This press launch incorporates “forward-looking statements.” Any statements contained on this press launch that aren’t statements of historic truth could also be deemed to be forward-looking statements. Phrases resembling “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “attainable” and comparable expressions are supposed to establish forward-looking statements. These forward-looking statements embrace statements concerning the potential advantages of the collaboration between Sarepta and StrideBio, together with enhancing particular tropism to tissues of curiosity, evading neutralizing antibodies, granting Sarepta entry to new know-how and targets, offering vital assets and experience to allow StrideBio’s continued speedy growth of its analysis and manufacturing platform, and accelerating the event of AAV gene therapies for a number of uncommon illness targets; the potential of StrideBio’s portfolio to enhance tropism to focused tissues; the events’ plan to concentrate on methods supposed to handle re-dosing challenges in sufferers who’ve acquired AAV-delivered gene remedy; the phrases of the settlement between Sarepta and StrideBio, together with anticipated and future funds, future improvement, regulatory and business milestones, future royalties, choices and Sarepta’s dedication to put money into StrideBio’s subsequent financing spherical; and Sarepta’s mission to profoundly enhance and prolong the lives of sufferers with uncommon genetic-based illnesses.
These forward-looking statements contain dangers and uncertainties, lots of that are past Sarepta’s management. Identified danger components embrace, amongst others: the anticipated advantages and alternatives associated to the collaboration between Sarepta and StrideBio might not be realized or might take longer to appreciate than anticipated as a result of challenges and uncertainties inherent in product analysis and improvement; specifically, the collaboration might not end in any viable therapies appropriate for commercialization as a result of a wide range of causes, together with any incapacity of the events to carry out their commitments and obligations underneath the settlement, the outcomes of analysis might not be in line with previous outcomes or might not be constructive or might in any other case fail to fulfill regulatory approval necessities for the security and efficacy of product candidates, attainable limitations of firm monetary and different assets, manufacturing limitations that might not be anticipated or resolved for in a well timed method, and regulatory, courtroom or company selections, resembling selections by the US Patent and Trademark Workplace with respect to patents that cowl Sarepta’s product candidates; and people dangers recognized underneath the heading “Threat Components” in Sarepta’s most up-to-date Annual Report on Kind 10-Ok for the yr ended December 31, 2018 and most up-to-date Quarterly Report on Kind 10-Q filed with the Securities and Alternate Fee (SEC) in addition to different SEC filings made by the Firm which you’re inspired to evaluate.
Any of the foregoing dangers may materially and adversely have an effect on the Firm’s enterprise, outcomes of operations and the buying and selling worth of Sarepta’s widespread inventory. For an in depth description of dangers and uncertainties Sarepta faces, you’re inspired to evaluate Sarepta’s 2018 Annual Report on Kind 10-Ok and most up-to-date Quarterly Report on Kind 10-Q filed with the SEC in addition to different SEC filings made by Sarepta. We warning buyers to not place appreciable reliance on the forward-looking statements contained on this press launch. Sarepta doesn’t undertake any obligation to publicly replace its forward-looking statements based mostly on occasions or circumstances after the date hereof.
Sarepta Therapeutics, Inc.
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