Roche Commits Over $1 Billion to Tackle Gene Therapy Delivery Challenge with Dyno Therapeutics

Swiss pharmaceutical company Roche (RHHBY.US) said on Thursday that it has reached a new agreement with Dyno Therapeutics, Inc., and is willing to pay more than 1 billion US dollars to address one of the biggest challenges in gene therapy: drug delivery. Dyno Therapeutics is a biotechnology company that focuses on helping companies ensure their gene therapies reach the intended targets.

Gene therapy is a one-time treatment that addresses the underlying cause of genetic diseases by repairing spelling errors in DNA. Biotech companies typically deliver it by inserting a working copy of the gene into the shell of an inactive virus, known as a capsid. This shell carries the gene to the correct cells in the body to replace the mutated gene.

But gene therapy is difficult to reach certain parts of the body, thus limiting its potential. In addition, this therapy often fails to achieve the desired therapeutic effect, forcing pharmaceutical companies to use higher doses, which may bring toxicity issues and lead to immune system rejection. This also increases costs, causing the price of some treatments to reach millions of dollars.

This collaboration builds on a previous agreement between the two companies, allowing Roche to utilize Dyno Therapeutics' technology more extensively. Under the new agreement, Roche will make an upfront payment of $50 million to Dyno, which will leverage artificial intelligence to design improved vectors for gene therapy, thereby enhancing the potential to cure neurological diseases. If these therapies pass clinical trials and achieve commercial success, Roche will pay over $1 billion in milestone payments and royalties.

Eric Kelsic, CEO of Dyno Therapeutics, said that Dyno's technology can ensure gene therapies reach the correct cells while avoiding other organs in the body. The company designs capsids that are better able to cross the blood-brain barrier, which is crucial for treating neurological diseases. Due to previous exposure to viruses, many patients develop antibodies against the capsids used in gene therapy, often rendering them unable to benefit from the treatment. Dyno is designing methods to avoid this issue.

While some pharmaceutical companies have scaled back their ambitions in the gene therapy field, Roche continues to pursue this cutting-edge science. However, it also faces challenges. In July this year, the company halted the development of a gene therapy for Pompe disease, a rare condition that causes weakness in the heart and skeletal muscles.

In 2019, Roche agreed to spend $4.8 billion to acquire Spark Therapeutics, a manufacturer of treatments for hereditary vision loss and other diseases. However, by 2022, Roche had written down more than $1 billion of the acquisition's value, citing reduced sales expectations for Spark’s approved and experimental products.

Boris L. Zaïtra, head of business development at Roche, stated that Roche hopes to develop a gene therapy through Dyno Therapeutics to address "historically difficult-to-treat neurological diseases." Kelsic said this could open the door to potential treatments for diseases such as Alzheimer's.

Dyno Therapeutics doesn’t have its own gene therapy. Instead, the company has decided to focus on helping other companies improve their treatment options. In addition to Roche, Dyno also collaborates with Sarepta Therapeutics, which sells gene therapies for Duchenne muscular dystrophy, and Astellas Pharma.

Kelsic said, "Solving the drug delivery problem is a huge challenge in this field." "When we solve the delivery issue, it will provide better treatment options for more diseases and more patients."

Editor: Guo Mingyu