Sarepta picks up a slate of preclinical gene therapy programs, seeding new StrideBio deal with $48M upfront
Having tasted early success in treating a number of muscular dystrophies with gene therapy, Sarepta is setting itself up to tackle more rare diseases.
The Boston-based biotech has picked StrideBio for a partnership evidently designed to run years, kicking things off with $48 million upfront and four central nervous system targets. The smaller partner is tasked with all R&D work up to the IND stage, coming up with the AAV-based therapies to be tested in Rett syndrome (MECP2), Dravet syndrome (SCN1A), Angelman syndrome (UBE3A) and Niemann-Pick (NPC1).
Co-founded by Mavis Agbandje-McKenna and Aravind Asokan, StrideBio’s pitch centers around its structure-driven capsid technology drawn from non-human primates, which it says can better direct these shells to tissues of interest and evade neutralizing antibodies. For Sarepta, the lure also lies in the potential to redose patients who have received AAV-delivered gene therapies before.
Sarepta’s first foray into the field was a microdystrophin approach to Duchenne muscular dystrophy, an extension of its efforts with its marketed exon-skipping drug Exondys-51. In a recent bout, it appeared to score higher than Pfizer in a Phase Ib, buoying its prospects. Preliminary functional data on a limb girdle muscular dystrophy acquired from Myonexus also appeared positive.
Its new deal also provides an exclusive option to four additional genetic targets for CNS or neuromuscular ailments, which could cost up to $42.5 million upfront. StrideBio may co-develop and co-commercialize one of the eight total products — just the right number for the small biotech, which is also juggling a preclinical pact with Takeda featuring three targets including Friedreich’s Ataxia.
CEO Sapan Shah talked up the resources and expertise they will gain for expanding their research and manufacturing platform rapidly.
“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,” Sarepta chief Doug Ingram added in a statement.