Sometimes, hard science is personal.
Sharif Tabebordbar’s father suffers from facioscapulohumeral muscular dystrophy, a genetic disease that systematically robs the body of muscular strength starting in the face and descending to the legs until they have difficulty walking. There are no approved treatments.
After earning a doctorate from Harvard and working at a biotech company with a different approach to treating muscular disorders, Tabebordbar joined colleagues at The Broad Institute of MIT and Harvard, a renowned life sciences research house, to find a better solution.
The result is Kate Therapeutics, where Tabebordbar is co-founder and chief science officer. The San Diego startup on Thursday announced $51 million in venture capital funding as well as a licensing agreement with pharmaceutical firm Astellas Pharma of Japan.
The venture money will be used to advance Kate Therapeutics’ novel medicine delivery platform, as well as a few genetic therapy candidates targeting muscle and heart disorders.
“The reason we are focused on this is because (Tabebordbar) made it his life’s mission to develop and improve delivery technology so he can address the disease that his dad has,” said Kevin Forrest, chief executive of Kate Therapeutics. “That is why we exist.”
The company is named after a young girl in Florida who has a pediatric version of myotonic dystrophy Type 1 — another muscular dystrophy-like disorder. Symptoms include muscle weakness and an inability to relax muscles.
“It is definitely emotional work,” said Forrest. “It is important work, and we finally have the tools to potentially give these patients some options.”
It’s early days for Kate Therapeutics. The 45-employee firm, founded in 2020, is pre-clinical and has significant work to do before advancing its technology to safety and efficacy trials.
But investors see promise in Kate Therapeutics’ platform to deliver and treat complex muscle and heart diseases — which are difficult to target precisely with genetic therapies without significant side effects.
The funding round was led by Westlake Village BioPartners and Versant Ventures with participation from Osage University Partners and University of Florida Innovate/Ventures.
“One particular aspect of the platform that caught our attention was the cardiac component,” said Clare Ozawa, managing partner of Versant Ventures in San Diego. “The platform is special because they basically re-engineered these viral vectors into very special ones that much more specifically get (treatments) to the muscles and heart, and also reduce the ability to get into other tissues like the liver, which you don’t want to get into.”
In addition, Kate Therapeutics entered into a licensing agreement with Astellas Pharma for its pre-clinical KT430 gene therapy for young children with X-linked myotubular myopathy — a life-threatening neuromuscular disease with symptoms including extreme muscle weakness and difficulty breathing.
Astellas will make an upfront payment to Kate Therapeutics, which is also eligible to receive development, regulatory and milestone payments, plus royalties on worldwide sales. Other details were not disclosed.
“The combination of Kate’s unique scientific approach and Astellas’ depth of experience in developing gene therapies for X-linked myotubular myopathy provides a solid foundation for the advancement of KT430 as it progresses towards the clinic,” said Adam Pearson, chief strategy officer at Astellas.
