Sarepta, Codiak BioSciences Team Up For Neuromuscular Disease Targeting

Sarepta Therapeutics (SRPT) has announced that it has teamed up with Codiak BioSciences to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing and RNA technologies for neuromuscular diseases.

According to Sarepta, the engineered exosome approach offers the potential to effectively deliver genetic therapeutics without triggering the adaptive immune response.

The two-year agreement includes up to five neuromuscular targets. Codiak is eligible to receive up to $72.5 million in upfront and near-term license payments plus research funding, as well as milestone payments and tiered royalties on future sales. Sarepta is granted an option to any of the candidates developed as part of the research alliance.

Exosomes are natural nanoparticles that serve as the body’s intercellular communication system, facilitating the transfer of a wide variety molecular payloads between cells. Through its proprietary engEx Platform, Codiak can design exosomes with precisely engineered properties, incorporate various types of biologically active molecules and direct them to specific cell types.

“Codiak’s engEx technology could potentially address some of the limitations of current treatment approaches and offers broad utility across Sarepta’s therapeutic modalities” commented Sarepta CEO Doug Ingram. “Codiak’s exosomes are engineered for precise tissue targeting and offer a non-viral delivery approach with non-immunogenic potential, thus opening up avenues for more efficient delivery and potential re-dosing,” he added.

If Sarepta elects to exercise its option on a target, Codiak will be responsible for research and preclinical development through IND preparation, and Sarepta will take charge of clinical development and commercial activities.

Shares in Sarepta have spiked 33% year-to-date, and analysts have a bullish Strong Buy consensus on the stock’s outlook. That’s with a $197 average analyst price target (14% upside potential). (See Sarepta stock analysis on TipRanks)

However, Oppenheimer’s Hartaj Singh is taking a more cautious approach. He recently reiterated his Hold rating on the stock without a price target, writing “While Sarepta continues to enhance its gene therapy manufacturing scale-up across its platform of programs, we await clarity on several key aspects for the DMD (Duchenne Muscular Dystrophy) program (SRP-9001).”

For Singh, success for the name hinges on several key SRP-9001 steps: scale-up (GMP material by July), study 301’s initiation (2H20), study 102’s readout in 1Q21 and what size/scope of 301 data will support a registrational filing.

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