Gene therapy company Kate Therapeutics Inc. (KateTx), has emerged from stealth mode with a $51 million Series A financing.
In addition, the company has awarded Astellas Pharma Inc. exclusive worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM).
There are a large number of complex and genetically determined heart and muscle diseases that are currently intractable due to the lack of specific and effective delivery to these tissues. Adeno-associated virus (AAV)-based capsids have shown promise for delivering therapeutic payloads in other organs, but have been hampered for use in muscle and heart due to limited potency, lack of tissue selectivity and minimal payload regulation.
“We are excited to announce the launch of KateTx and what it means for patients with musculoskeletal and cardiac disease,” said Kevin Forrest, president, CEO and director of KateTx.
“KateTx implemented a new capsid and cargo technology platform to enable skeletal and cardiac muscle targeting as well as liver targeting. We believe our technology can provide patients with safer and more effective drugs.”
The proceeds from the financing and licensing agreement will support the advancement of KateTx’s early internal portfolio of cardiac and muscular disease programs, including myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD), which are two of the leading causes of adult onset. muscular dystrophy.
About DM1 and FSHD
Type 1 myotonic dystrophy is a progressive multisystem disorder that affects approximately 40,000 people in the US. Common symptoms include weakness and myotonia (inability to relax muscles) of the lower legs, hands, neck, and face, leading to upper limb disability and difficulty walking, as well as extramuscular symptoms including cognitive problems, daytime sleepiness and sleep disturbances, and arrhythmias. The adult-onset form is the most common; Child-onset and congenital forms are more severe and sometimes life-threatening. There are no approved drugs for DM1.
Facioscapulohumeral muscular dystrophy is an inherited muscle disorder that also affects about 40,000 people in the US. FSHD usually starts at the age of 15 to 30 years. Weakness usually develops in a descending pattern, affecting the face, shoulders and upper arms, lower legs and hips. Upper limb weakness is often most prominent, but many patients will have significant difficulty walking, and about 20% eventually require a wheelchair. There are no approved drugs for FSHD.
Different delivery approaches
For capsids, the KateTx DELIVER platform leverages directed evolution, RNA-based selection of functional capsid variants, and machine learning in multiple in vivo models. The platform has produced the MyoAAV-class capsid, which was developed by KateTx scientific co-founder and chief scientific scientist Sharif Tabebordbar and colleagues at the Broad Institute of MIT and Harvard while he was supervisor, research scientist in Sabeti’s Pardis lab.
Technology related to Tabebordbar’s work is licensed to KateTx. Internally, the company is also developing the next generation of MyoAAV capsids with further targeting enhancements.
MyoAAV capsids target skeletal and cardiac muscles with significantly higher efficiency across species compared to naturally occurring adeno-associated virus (AAV) capsids including AAV8, AAV9, and AAVrh74. This breakthrough has the potential to increase the efficacy and safety of gene therapy and enable the pursuit of a broader set of targets that are difficult to treat with current technology.
“My dad lives with FSHD, so I see firsthand the toll this devastating disease has on patients and families. That is the reason I entered this field in the first place,” said Tabebordbar.
“I am thrilled that KateTx’s unique technology is being used to develop a first and best-in-class gene therapy for patients living with serious heart and muscle diseases.”
The KateTx cargo platform includes in-house proprietary capabilities and technology licensed from the University of Florida developed in the lab of scientific co-founder KateTx, Eric Wang. The overarching goal of the cargo platform is to ensure enterprise therapies are produced only in the tissues of interest and nowhere else in the body.
Short term focus on continuing to the clinic
Beyond the capsid and cargo platform efforts, KateTx has built a team with discovery, development, manufacturing, and gene therapy expertise. KateTx’s current focus is on identifying and advancing clinical candidates for DM1 and FSHD, as well as other genetic heart and muscle diseases.
The funding was co-led by founding investors Westlake Village BioPartners and Versant Ventures, with participation from Osage University Partners and UF Innovate | Business.