Novartis has acquired Kate Therapeutics to further enhance and strengthen our ongoing efforts to advance gene therapies for patients.

Nov 21, 2024

• Acquisition of Kate Therapeutics enhances and strengthens Novartis strategic priorities in gene therapy and neuroscience innovation
• Transaction includes enabling technology platforms and therapeutic candidates for DMD, FSHD and DM1, currently in preclinical development
• Transaction value of up to USD 1.1bn in total, comprising an upfront payment and potential additional milestone payments

Neuromuscular diseases are a group of disorders that impair the functioning of muscles and nerves, disrupting nerve signal transmission and muscle contraction and movement. Many of these disorders are genetic in nature and present significant challenges to patients and their families. 

At Novartis, we are committed to addressing unmet needs for patients living with inherited neuromuscular diseases. This includes innovating novel gene therapies, which aim to address these diseases by either replacing faulty genes or blocking the effects of toxic RNA or proteins. To further enhance and strengthen our ongoing efforts to advance gene therapies for patients, Novartis has acquired Kate Therapeutics, a San Diego-based, preclinical stage biotechnology company, focused on developing adeno-associated virus (AAV)-based gene therapies to treat genetically defined neuromuscular diseases. 

“We are delighted to bring Kate Therapeutics’ platform technologies and programs together with Novartis scientific expertise and leadership in gene therapy and neuroscience innovation,” said Fiona Marshall, President of Biomedical Research at Novartis. “We have been highly impressed with the rigor and potential of Kate’s science, and we are confident this acquisition will further enhance our ability to bring forward new therapeutic options for patients living with neuromuscular diseases.”

Kate Therapeutics’ primary programs include preclinical candidates for Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1). Kate’s Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA (DELIVER) platform combines diverse capsid library generation with transcript-based in vivo selection and machine learning to evolve functional capsid variants. DELIVER has been used to evolve a novel class of liver de-targeted muscle-tropic capsids, which transduce both skeletal muscle and cardiac tissue with potency and selectivity in vivo, while avoiding the liver. 

“This acquisition builds on our expertise and leadership in neuroscience drug discovery and brings to Novartis talent, expertise and capabilities that are highly complementary to our ongoing internal efforts,” said Robert Baloh, Global Head of Neuroscience Research at Novartis. “It reflects our commitment to addressing unmet medical needs in neuroscience and tackling the limitations of existing gene therapies for patients with inherited neuromuscular conditions.” 

Kate Therapeutics’ technology platforms integrate capsid and cargo technologies to deliver payloads to desired tissues, while potentially mitigating off-target effects to tissues such as the liver. This approach aims to improve both the efficacy and safety of gene therapies, opening potential possibilities for treating complex diseases previously difficult to address with current technologies, including inherited neuromuscular diseases. 

Under the terms of the agreement, Kate Therapeutics’ shareholders are entitled to receive up to USD 1.1bn, comprising a cash payment that was paid at closing of the transaction and additional amounts payable on achievement of specified milestones. 

Learn more about Neuroscience research at Novartis.

Learn more about Novartis Gene Therapies.

Disclaimer:

This media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for any investigational or approved products resulting from the acquisition of Kate Therapeutics and its platform technologies and programs, or regarding potential future revenues from such platforms and programs. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that any investigational or approved products resulting from the platform technologies and programs acquired from Kate Therapeutics will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Neither can there be any guarantee expected benefits or synergies from this transaction will be achieved in the expected timeframe, or at all, nor can there be any guarantee that the platforms and programs acquired from Kate Therapeutics will be commercially successful in the future. In particular, our expectations regarding platforms and programs acquired from Kate Therapeutics or the transaction described in this media update could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise.