Gene therapy could be a game changer for various diseases, but it can be hobbled by its packaging, most often a virus to which some people are naturally immune. Sarepta Therapeutics is on the hunt for alternatives, and its latest target is a system the body’s cells use to communicate with each other: exosomes.
It is teaming up with Codiak Biosciences to develop engineered exosomes that can deliver gene therapy, gene editing or RNA-based treatments—all of which Sarepta is already working on. Under the two-year deal, Codiak will reel in up to $72.5 million in upfront and near-term license fees along with research funding, the pair said in a statement Monday. As usual, Codiak stands to pick up “significant” development and regulatory milestones, but the partners kept those numbers under wraps.
Sarepta has the option to take forward candidates for up to five targets in the neuromuscular space. If Sarepta pulls the trigger on any of those targets, Codiak will take care of research and preclinical development through IND preparation, at which point Sarepta will pick up clinical development and commercial activities.
Sarepta has more than 20 programs in its gene therapy pipeline tackling diseases like Duchenne muscular dystrophy and Limb-girdle muscular dystrophy, which rely on adeno-associated virus (AAV) vectors. Many gene therapies, including approved ones like Novartis’ Zolgensma, are delivered using AAVs, but these natural viruses come with challenges that can limit the use of gene therapies. For starters, some people may have developed a natural immunity to this type of virus, making the treatment ineffective. Multiple players are hunting down nonviral alternatives to get gene therapies into patients.
Enter exosomes. Long thought of as the cell’s garbage removal system, exosomes carry payloads like proteins or RNAs from one cell to another. Codiak’s engEx Platform engineers drug molecules into exosomes to get drugs where they need to go. Because they are derived from human cells and released and received by nearly every cell in the body, exosomes can bypass the immune response that repels invaders.
“As Sarepta expands its leadership position in precision genetic medicine, this alliance with Codiak furthers our goal to deliver the most advanced therapies to patients … 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,” said Doug Ingram, CEO of Sarepta, in the statement.
The Codiak deal comes on the heels of a collaboration with Dyno Therapeutics, a company using artificial intelligence to generate and evaluate millions of new AAV variants with the goal of creating synthetic AAV capsids—the protein shells of a virus—that work better than current options.