UCB strengthens its gene therapy activities with additional pipeline programs, capabilities and platforms

- Acquisition of Handl Therapeutics BV augments UCB's existing early gene therapy pipeline with two research programs, a proprietary adeno-associated virus (AAV) capsid technology platform and capabilities

- New R&D collaboration with Lacerta Therapeutics provides access to a novel gene therapy program and proprietary AAV capsids

- Gene therapy investment strengthens UCB's capabilities to discover and develop differentiated solutions with unique outcomes for patients

BRUSSELS, Belgium I November 12, 2020 I UCB today announced the acquisition of Handl Therapeutics BV, a rapidly growing and transformative gene therapy company based in Leuven, Belgium and a new collaboration with Lacerta Therapeutics, a Florida based clinical stage gene therapy company. The new acquisition and collaboration will together serve to rapidly accelerate UCB's ambition in gene therapy.

UCB has a vision to move from symptomatic treatments to disease modification and eventually towards a cure. AAV-mediated gene therapy offers to deliver that potential and drive a fundamental change in how diseases are treated with the ability to remove or add disease-related proteins with a single treatment. A vast array of diseases are amenable to gene therapy and UCB is embracing this modality to expand its capabilities and ultimately transform the lives of patients with severe diseases.

"Handl Therapeutics BV was formed with the goal of harnessing today's new frontiers in science and technology, and delivering transformative and advanced medicines to patients," said Professor Michael Linden, Founder and Chief Scientific Officer at Handl Therapeutics BV. "Knowing that UCB shares this goal too gives me great confidence that our combined resources and expertise will provide the best possible chance of making this a reality and I am happy that we are joining the UCB family."

Founded in 2019, Handl Therapeutics BV has a vision to deploy the power of disease modifying in vivo gene therapy to treat complex neurodegenerative diseases through AAV capsid technology. Operating in a highly collaborative manner, Handl Therapeutics BV has built a strong international network to access global capabilities and expertise. To this end, it combines state of the art technology platforms and scientific advances licenced from KU Leuven (Belgium), Centre for Applied Medical Research (CIMA Universidad de Navarra, Spain), University of Chile (Chile) and King's College London (UK) to address unmet medical needs.

Florent Gros, Handl Therapeutics BV Founder and Chief Executive Officer said, "UCB's global footprint and scientific expertise in neurodegenerative diseases, coupled with our shared cultures of scientific advancement and commitment to patients, creates an exceptional environment in which we can accelerate the development of gene therapies and change patients' lives."

The Handl Therapeutics team will continue to be based in Leuven, Belgium, and will work very closely with UCB's international research teams.

In addition to the Handl Therapeutics BV acquisition, today's announcement of a new collaboration with Lacerta Therapeutics underlines UCB's strategic focus in gene therapy to fulfil its Patient Value Ambition. These transactions build upon the strategic acquisition of Element Genomics, Inc. (acquired in 2018) that strengthened UCB's genomics and epigenomics research platforms aiding the identification of novel drug targets.

Founded in 2017, and a spin-off from the University of Florida, Lacerta Therapeutics' mission is to make AAV-based therapies available for all patients with rare and serious neurological disorders. The research collaboration and licensing agreement with UCB will focus on a central nervous system (CNS) disease with a high unmet need. Lacerta Therapeutics will lead research, preclinical activities and the early manufacturing process development, while UCB will complete IND-enabling studies, manufacturing and clinical development. This new collaboration will allow UCB to access Lacerta Therapeutics' expertise in AAV-based CNS targeted gene therapies, fortifying UCB's ability to produce effective treatments for neurodegenerative diseases.

"At Lacerta Therapeutics, we have dedicated our careers to the development of AAV gene therapy platforms and it remains our mission to advance these platforms and develop novel therapeutics for patients with neurodegenerative disorders," said Dr. Edgardo Rodrὡguez-Lebròn, President and Chairman of the Board at Lacerta. "We are looking forward to entering a new era, working in partnership with UCB. Our hope is that our combined expertise will lead to significant advances in identifying treatments for orphan CNS diseases."

Dhavalkumar Patel, UCB's Chief Scientific Officer said, "UCB's ambition for patients relies on our ability to innovate and deliver highly differentiated medicines. The acquisition of Handl Therapeutics BV and the new partnership with Lacerta Therapeutics offers 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." He added, "We are delighted to be able to welcome a rich diversity of talent and expertise from both Handl Therapeutics BV and Lacerta Therapeutics. With their deep and wide-ranging knowledge, novel gene therapy platforms and drive for innovation, I am confident that together we will transform the lives of people living with severe neurodegenerative diseases."

This acquisition and new collaboration do not impact UCB's financial outlook for 2020.

About UCB

UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 7,600 people in approximately 40 countries, the company generated revenue of € 4.9 billion in 2019. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.

About Handl Therapeutics BV

The vision of Handl Therapeutics is to deploy the power of disease modifying in vivo gene therapy to treat complex neurodegenerative diseases to begin addressing significant unmet medical needs in healthcare. The innovative business model is based on a highly integrative approach that is built on tight links with an international network of academic partners. Handl Therapeutics goal is to build a streamlined business that is designed to deliver transformative advanced medicines to the market.

About Lacerta Therapeutics

Lacerta Therapeutics Inc (www.lacertatx.com) is a clinical-stage gene therapy company that is leveraging its proprietary adeno-associated virus (AAV) vector technology and manufacturing platforms to develop treatments for central nervous system and lysosomal storage diseases. Currently, Lacerta is focused on gene therapy solutions for Sanfilippo Syndrome Type B, Friedreich's ataxia, Spinocerebellar ataxia, Pompe disease, and Alzheimer's.

SOURCE: UCB

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