Kate Therapeutics is pursuing FSHD treatment

The company’s MyoAAV promises a less toxic way to deliver gene therapies

by June Kinoshita, Senior Director of Research and Education

Kate Therapeutics, Inc. (KateTx), a San Diego-based biopharma company, announced on June 8 that it has “emerged from stealth mode with a $51 million Series A financing” to fuel its program to develop novel therapies targeting muscle and cardiac tissue. Among the disease KateTx plans to tackle are FSHD and myotonic dystrophy Type 1.

Efforts to deliver gene therapies into cells typically rely on adeno-associated virus (AAV) to shuttle therapeutic molecules into cells. AAV is ordinarily harmless, but to treat a human patient, the therapeutic molecule would have to be delivered in such huge quantities that the amount of AAV needed can damage the liver or even kill a patient. This has stymied efforts to develop gene therapies for muscle diseases such as FSHD, but a few years ago, Sharif Tabebordbar, Ph.D., and colleagues at the Broad Institute of MIT and Harvard found an ingenious way to solve the problem. The surface of the AAV particle is covered with “capsids”, proteins that recognize specific types of cells, allowing the virus to target those cells. Tabebordbar and his colleagues found an efficient way to design variants of AAV, called MyoAAV, that produce capsids that prefer muscle cells over other tissues. You can read more about it in our 2021 blog post:  His father’s FSHD inspired scientist to make a breakthrough

Tabebordbar went on to help co-found KateTx, where he now serves as Chief Scientific Officer. Our team at the FSHD Society has been in regular contact with KateTx since 2021, excited by the potential for applying the company’s platform to FSHD. We have no idea whether our quiet advocacy and encouragement helped, but it is great news that yet another leading-edge company has joined the battle!

Read the full press release here.