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Kate Therapeutics (KateTx) today emerged from stealth mode with a $51 million Series A round and a deal with Astellas Pharma to develop a gene therapy for X-linked myotubular myopathy (XLMTM).

“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,” said Kevin Forrest, PhD, president, CEO and a director of KateTx.

The financing was co-led by founding investor Westlake Village BioPartners and Versant Ventures, with participation from Osage University Partners and UF Innovate Ventures. Proceeds will support the advancement of KateTx’s muscle and heart disease programs, including ones for myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD), which are two of the leading causes of adult-onset muscular dystrophy.

The deal with Astellas is an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat XLMTM, a serious, life-threatening, rare neuromuscular disease characterized by extreme muscle weakness, respiratory failure and early death. Astellas will make an undisclosed upfront payment to KateTx, which is also eligible to receive development, regulatory and commercial milestone payments, plus royalties on worldwide sales.

KateTx’s sweet spot is  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. 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 ability to regulate cargoes.

KateTx’s DELIVER platform leverages directed evolution, stringent RNA-based selection of functional capsid variants, and machine learning. The platform already has yielded the MyoAAV class of capsids, which were developed by KateTx scientific co-founder and Chief Scientific Officer Sharif Tabebordbar, PhD, and colleagues at the Broad Institute of MIT and Harvard while he was a supervisor, research scientist in the lab of Pardis Sabeti, MD, PhD. Results were first published in Cell in 2021.

MyoAAV capsids target skeletal and cardiac muscle with higher efficiency compared with naturally occurring adeno-associated virus (AAV) capsids including AAV8, AAV9, and AAVrh74. They thus 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.

“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 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 Eric Wang, PhD. 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.

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