Swiss pharmaceutical giant Roche (RHHBY, Financial) has announced a new agreement with Dyno Therapeutics Inc., committing over $1 billion to tackle one of the biggest challenges in gene therapy: drug delivery. Dyno Therapeutics specializes in ensuring gene therapies achieve their intended targets, potentially transforming treatments for genetic diseases that focus on correcting DNA errors.
Gene therapy often involves inserting a functional copy of a gene into an inactivated virus shell, known as a capsid, to deliver it to the right cells in the body. However, reaching certain parts of the body has been difficult, limiting the therapy's potential. Often, higher doses are needed, leading to toxicity issues and immune system rejection, ultimately increasing costs, with some treatments reaching millions of dollars.
The collaboration builds on a previous agreement between the two companies. Under the new terms, Roche will make an initial payment of $50 million to Dyno, which will use artificial intelligence to design improved delivery vehicles for gene therapies, with a focus on treating neurological disorders. Success in clinical trials and commercial outcomes could see Roche paying over $1 billion in milestones and royalties.
Dyno's CEO, Eric Kelsic, states that their technology ensures that gene therapies reach the correct cells while avoiding other organs. Dyno is developing capsids capable of effectively crossing the blood-brain barrier, crucial for treating neurological conditions. This approach addresses a significant challenge, as many patients form antibodies against capsids due to previous viral exposures, making therapies ineffective.
Despite a reduction in ambition within the gene therapy sector by some pharmaceutical companies, Roche continues to pursue this cutting-edge science, although it faces hurdles. In July, Roche halted the development of a gene therapy for Pompe disease, a rare condition leading to muscle weakness. Previously, in 2019, Roche acquired Spark Therapeutics for $4.8 billion, a company known for its treatments for inherited vision loss. However, by 2022, Roche wrote down over $1 billion from the acquisition value due to lower sales expectations for Spark's marketed and experimental products.
Boris L. Zaïtra, Roche's business development head, emphasizes the company's goal to develop a gene therapy addressing historically challenging neurological diseases through their partnership with Dyno. Kelsic believes this could pave the way for potential treatments for diseases like Alzheimer's.
Dyno does not develop its gene therapies; instead, it focuses on helping other companies enhance their treatment strategies. Besides Roche, Dyno collaborates with Sarepta Therapeutics, which sells gene therapy for Duchenne muscular dystrophy, and Astellas Pharma.
