San Diego-based Kate Therapeutics (KateTx) has emerged from stealth mode with a $51 million Series A co-led by founding investor Westlake Village BioPartners and Versant Ventures, with participation from Osage University Partners and UF Innovate | Ventures.
In addition to completion of the funding round, KateTx, which bills itself as “a next-generation gene therapy company,” announced that the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KateTx’s KT430 to treat X-linked myotubular myopathy.
KateTx was founded during the pandemic in 2020 by Kevin Forrest, Ph.D., Eric Wang, Ph.D., Sharif Tabebordbar, Mark Fielden, Ph. D. and Alan Beggs, Ph.D. The pre-clinical startup focuses on difficult to treat genetically defined muscle and heart diseases.
President, CEO and Director
KateTx
Forrest, the company’s president and CEO, said KateTx’s technologies can “provide safer and more effective medicines for patients.”
“We are excited to announce KateTx’s launch and what this means for patients suffering from muscle and heart diseases,” said Forrest, adding that proceeds from the Series A 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 and facioscapulohumeral muscular dystrophy (FSHD), which are two of the leading causes of adult-onset muscular dystrophy.
According to the company, KateTx’s DELIVER platform for capsids leverages “directed evolution, stringent RNA-based selection of functional capsid variants and machine learning in various in vivo models.”
The platform already has yielded the MyoAAV class of capsids, which were developed by Dr. Tabebordbar, KateTx scientific co-founder and chief scientific officer, together with colleagues at the Broad Institute of MIT and Harvard.
Scientific Co-founder and Chief Scientific Officer
KateTx
The development happened when Dr. Tabebordbar was a supervisor and research scientist in the lab of Pardis Sabeti, M.D., Ph.D. Results were first published in Cell in 2021. Technologies related to Dr. Tabebordbar’s work are licensed to 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,” said Dr. Tabebordbar. “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.”
KateTx’s cargo platform includes both internally generated proprietary capabilities and technologies licensed from the University of Florida that were developed in the laboratory of KateTx scientific Co-founder Dr. Wang, who said 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.
Near-term Focus
Beyond capsid and cargo platform efforts, KateTx has 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.
Managing Director
Westlake Village BioPartners
“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,” said Beth Seidenberg, M.D., co-founding managing director at Westlake and chair of KateTx’s board of directors. “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.”
Myotonic dystrophy type 1 is a progressive multisystem disorder affecting approximately 40,000 individuals in the U.S. Common symptoms include weakness and myotonia (inability to relax muscles) of the lower legs, hands, neck and face.
Onset of FSHD, a muscle disorder affecting approximately 40,000 Americans, typically occurs between ages 15 to 30. Weakness usually progresses in a descending pattern, affecting the face, shoulders and upper arms, lower legs and hips. Currently, there are no approved medicines for FSHD.
Kate Therapeutics
FOUNDED: 2020
CEO: Kevin Forrest, Ph.D.
HEADQUARTERS: San Diego
EMPLOYEES:
BUSINESS: Gene therapy
WEBSITE: katetherapeutics.com
NOTABLE: KateTx develops AAV-based gene therapies to treat genetically defined muscle and heart diseases. AAV vectors are bioengineered tools that use a non-enveloped virus to transport modified genetic material safely into tissues and cells impacted by otherwise difficult-to-treat conditions.
