UCB bolsters gene therapy efforts with Handl buy, Lacerta deal

UCB on Thursday announced that it acquired Handl Therapeutics in a move it says will strengthen its existing early gene therapy pipeline with the addition of two research programmes, as well as adeno-associated virus (AAV) capsid technology platform and capabilities. The company also unveiled a separate research collaboration and licensing agreement with Lacerta Therapeutics that provides access to a gene therapy programme, as well as proprietary AAV capsids. Financial details were not disclosed for either deal.

Dhavalkumar Patel, chief scientific officer at UCB, suggested the two transactions "[offer] us the potential to drive a fundamental change in how diseases are treated, by moving us from treating symptoms to disease modification and eventually towards a cure." UCB noted that "a vast array of diseases are amenable to gene therapy and [we are] embracing this modality."

Focus on neurodegenerative diseases

According to UCB, Handl is working to harness disease-modifying in vivo gene therapy to treat complex neurodegenerative diseases through AAV capsid technology. UCB said the company, which was founded last year, has built a "strong international network to access global capabilities and expertise," with technology platforms and scientific advances licenced from various universities and research organisations "to address unmet medical needs." The Handl team will remain based in Leuven, Belgium, and will work closely with UCB's international research teams.

Meanwhile, UCB's R&D partnership with Lacerta will focus on an unspecified central nervous system (CNS) disease with a high unmet need, with Lacerta leading research, preclinical activities and early manufacturing process development, while the Belgian drugmaker will handle investigational new drug-enabling studies, manufacturing and clinical development. UCB said the tie-up will give it access to Lacerta's expertise in AAV-based CNS-targeted gene therapies that it hopes will shore up its own abilities to produce treatments for neurodegenerative diseases.

Research at Lacerta, a spin-off from the University of Florida founded in 2017, is currently focused on developing AAV-based therapies for Sanfilippo Syndrome Type B, Friedreich's ataxia, spinocerebellar ataxia, Pompe disease, and Alzheimer's disease. The company has a licensing deal with Sarepta Therapeutics for up to three CNS-targeted gene therapy programmes, including exclusive rights to the former's candidate for Pompe disease.

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