Kate Therapeutics – a next-generation gene therapy company – recently emerged from stealth mode with a $51 million Series A financing co-led by founding investor Westlake Village BioPartners and Versant Ventures with participation from Osage University Partners and UF Innovate | Ventures. And the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM), the details of which are described in a separate press release issued today.
There are many genetically defined and complex muscle and heart diseases that cannot currently be addressed due to a lack of specific and effective delivery to these tissues. And adeno-associated virus (AAV)-based capsids have shown promise to deliver therapeutic cargoes in other organs, but have been hampered for use in muscle and heart by limited potency, lack of tissue selectivity, and minimal regulation of cargoes.
The proceeds from the financing and license agreement will support the advancement of KateTx’s initial internal portfolio of muscle and heart disease programs, including myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD), which are 2 of the leading causes of adult-onset muscular dystrophy.
For capsids, KateTx’s DELIVER platform utilizes directed evolution, stringent RNA-based selection of functional capsid variants and machine learning in various in vivo models. And the platform already has yielded the MyoAAV class of capsids, which were developed by KateTx scientific co-founder and Chief Scientific Officer Sharif Tabebordbar, Ph.D., and colleagues at the Broad Institute of MIT and Harvard while he was a supervisor, and research scientist in the lab of Pardis Sabeti, M.D., Ph.D. Results were first published in Cell in 2021.
The technologies related to Dr. Tabebordbar’s work are licensed to KateTx. And internally, the company is also developing next-generation MyoAAV capsids with further targeting enhancements.
MyoAAV capsids target skeletal and cardiac muscle with dramatically higher efficiency across species compared with naturally occurring adeno-associated virus (AAV) capsids including AAV8, AAV9, and AAVrh74. And these breakthroughs have the potential to improve efficacy and safety of gene therapy and enable the pursuit of a broader set of targets that are otherwise difficult to treat with current technologies.
KateTx’s cargo platform includes internally generated proprietary capabilities and technologies licensed from the University of Florida developed in the laboratory of KateTx scientific co-founder Eric Wang, Ph.D. And the overarching goal of the cargo platform is to ensure the company’s therapies are produced only in tissues of interest and not elsewhere in the body.
Beyond capsid and cargo platform efforts, KateTx built a team with gene therapy discovery, development, manufacturing, and disease area expertise. KateTx’s current focus is identifying and advancing clinical candidates for DM1 and FSHD, as well as for other genetic muscle and heart diseases.
KEY QUOTES:
“We are excited to announce KateTx’s launch and what this means for patients suffering from muscle and heart diseases. KateTx is applying novel capsid and cargo technology platforms to enable skeletal and cardiac muscle targeting and liver de-targeting. We believe our technologies can provide safer and more effective medicines for patients.”
— Kevin Forrest, Ph.D., president, CEO and a director of KateTx
“My father lives with FSHD, so I saw firsthand the consequences of this devastating disease on patients and families. It is the reason I entered this field in the first place. I am beyond excited that KateTx’s unique technologies are being used to develop first- and best-in-class gene therapies for patients living with serious muscle and heart diseases.”
— Dr. Tabebordbar
“KateTx is leading the way to develop capsid and cargo combinations to address muscle and heart diseases in ways that have not been previously possible. With Kevin at the helm and a world-class team, we look forward to the exciting future for the company and the medicines it will advance for patients.”
— Beth Seidenberg, M.D., co-founding managing director at Westlake and chair of KateTx’s board
“KateTx’s platform has the potential to bring life-changing new therapies to patients suffering from muscle and heart disease who have limited or no treatment options. With this Series A financing and licensing agreement, KateTx will be able to progress its deep pipeline of internal programs.”
— Clare Ozawa, Ph.D., managing director at Versant and a KateTx board member
