Pharma Focus Asia

Sarepta Therapeutics and Selecta Biosciences Enter Into Research License and Option Agreement for Selecta’s ImmTOR Immune Tolerance Platform in Neuromuscular Diseases

Friday, June 19, 2020

Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases, and Selecta Biosciences, Inc. announced that they have entered into a Research License and Option agreement granting Sarepta an option to license the rights to develop and commercialize Selecta’s immune tolerance platform, ImmTOR™, for use in Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs).  In advance of exercising its option, Sarepta will conduct research and evaluate the utility of ImmTOR to minimize or prevent the formation of neutralizing antibodies (NAb) to adeno-associated virus (AAV) in connection with the administration of Sarepta’s DMD and LGMD gene therapy candidates.

Sarepta’s late-stage gene therapy candidates are delivered using AAV – in particular, AAVrh74.  AAVrh74 was selected because of its safety profile, superior muscle tropism, empirical demonstration of high expression, and low screen-out rate for pre-existing antibodies. Currently, however, all systemic AAV-delivered constructs are one-time therapies that cannot be re-dosed due to the robust post-administration development of NAbs specific to the AAV vector. Selecta is a leader in immune tolerance and has generated strong preclinical evidence to support the potential for re-dosing patients receiving gene therapy. Selecta has reported that in preclinical studies, when used in combination with AAV gene therapy vectors, Selecta’s ImmTOR immune tolerance platform inhibits the development of NAbs to the vector, permitting re-dosing of the gene therapy. i

“As we build our enduring gene therapy engine, we intend not only to rapidly advance treatments for rare, life-ending diseases, but at the same time, to advance the state of genetic medicine science by continually improving the utility of gene therapy.  If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing. We are encouraged by the data generated on the ImmTOR platform and excited to join with Selecta to explore the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed,” said Doug Ingram, President and Chief Executive Officer, Sarepta Therapeutics.

“We are pleased to build on our already strong foundation of strategic partnerships and expand the clinical application of the ImmTOR platform into neuromuscular diseases. The ability to re-dose gene therapy addresses one of the major challenges of one-time therapies today,” said Carsten Brunn Ph.D., President and Chief Executive Officer of Selecta Biosciences. “We are excited to collaborate with a leader in genetic medicine like Sarepta, and are confident that their expertise in rare diseases combined with our immune tolerance platform has the potential to enhance the long-term therapeutic benefit to patients with these debilitating conditions.”

DMD is a rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. LGMDs are a group of over 30 distinct diseases that cause weakness and wasting of the muscles around the hips and shoulders, eventually progressing to the arms and legs. LGMD can be caused by a single gene defect that affects specific proteins within the muscle cell, including those responsible for keeping the muscle membrane intact.

Under the terms of the research license and option agreement, Sarepta will make an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone fees. If Sarepta exercises its options to enter any commercial license agreements, Selecta will be eligible for additional development, regulatory, and commercial milestone payments, as well as tiered royalties on net product sales. Additional financial details are not being disclosed.

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