Sarepta Therapeutics, Inc. (SRPT - Free Report) and privately-held Codiak BioSciences announced that the companies have entered into a global research and option agreement to develop potent genetic therapeutics using engineered exosomes developed with Codiak’s engEx platform.
While Sarepta is a leader in the field of precision genetic medicine for rare diseases, Codiak BioSciences is one of the leading companies advancing engineered exosomes.
Please note that exosomes are natural nanoparticles that serve as the body’s intercellular communication system, facilitating the transfer of a wide variety of molecular payloads between cells. Exosomes provide a unique advantage as a targeted delivery system for genetic medicines because they are inherently non-immunogenic since they are derived from human cells. The companies will design exosomes that can deliver and functionally release select payloads, such as nucleic acids and gene therapy and gene editing constructs, with precise tissue targeting in neuromuscular indications. This approach is believed to effectively deliver genetic therapeutics without triggering the adaptive immune response.
The two-year agreement between the two companies includes up to five neuromuscular targets. Per the terms of the agreement, Sarepta will pay Codiak BioSciences up to $72.5 million in upfront payment and near-term license payments as well as research funding. Codiak BioSciences will also be eligible to receive significant development and regulatory milestone payments and tiered royalties on future sales from Sarepta.
Sarepta will receive exclusive option to license Codiak BioSciences’ proprietary engEx Platform to develop and commercialize engineered exosome therapeutics. Codiak BioSciences will be responsible for research and preclinical development through IND preparation following exercise of option by Sarepta. While Sarepta will lead clinical development and commercial activities.
Shares of Sarepta have risen 33% so far this year compared with the industry’s increase of 12.8%.
The company is developing several gene therapies targeting muscular dystrophy indications.Its lead gene therapy candidate, SRP-9001, is being evaluated as a treatment for Duchenne muscular dystrophy (“DMD”).Encouraging results from the company’s gene therapy studies boost its leading position in this field as competitors continue to face trouble during development. Pfizer (PFE - Free Report) is also developing a gene therapy for treating DMD but reported underwhelming data last month, which boosted investors’ confidence in Sarepta. Solid Biosciences (SLDB - Free Report) is another company developing gene therapy for treating DMD. However, the company’s early-stage study in under clinical hold. Sarepta has also signed a licensing agreement with Roche (RHHBY - Free Report) , granting exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets.
The company is also developing another gene therapy candidate, SRP-9003, in patients with limb-girdle muscular dystrophy Type 2E. The company reported encouraging preliminary data from an early-stage study earlier this month.
Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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