Sarepta Therapeutics Signs Long-term Strategic Investment and License Agreements with Lacerta Therapeutics, Gaining Rights to Multiple CNS-targeted Gene Therapy programs and Access to Important Gene Therapy Talent and Tools
-- Lacerta is an AAV-based gene therapy company founded on technologies licensed from the
“Today’s investment with Lacerta bolsters Sarepta’s position as a leader in precision genetic medicine and moves us forward on our mission is to deliver life-enhancing therapies to those living with underserved diseases and in so doing to become one of the most meaningful global genetic medicine companies in the coming few years,” said
Specifically, the Lacerta relationship provides
- Expansion of Sarepta’s gene therapy pipeline to up to 11 unique candidates: With this transaction,
Sareptahas added up to three CNS gene therapy targets to its already significant pipeline of 20 programs in various stages of development, expanding the Company’s presence in gene therapy and broadening its therapeutic focus into CNS-targeted therapies.
- Access to world class talent: Lacerta’s founders, nine in all, whom are widely published (over 500 papers among them) in leading peer-reviewed journals, are highly regarded in gene therapy clinical research and have worked at leading centers across
the United States, including University of Florida, Nationwide Children’s Hospital, CHOP/ University of Pennsylvaniaand Weill Medical College of Cornell.
Regarding Sarepta’s license and option to three CNS gene therapy assets, Lacerta will manage the majority of pre-clinical development while
“Our co-founders have dedicated their careers to the development of AAV gene therapy platforms for the treatment of multiple diseases. Lacerta’s mission is to advance these technologies to develop novel treatments for patients with CNS disorders,” said Dr.
About Sarepta Therapeutics
About Lacerta Therapeutics
Lacerta Therapeutics is a clinical-stage gene therapy company using a constellation of proprietary adeno-associated virus (AAV) vector technologies to develop treatments for central nervous system and lysosomal storage diseases. We are advancing our clinical programs using proprietary capsid variants and a scalable vector manufacturing platform. Currently, Lacerta is focused on gene therapy solutions for Pompe Disease, Sanfilippo Syndrome Type B, Aromatic L-amino acid decarboxylase deficiency, Neurodegenerative Proteinopathies, Spinocerebellar Ataxias and Glioblastoma. For more information, visit www.lacertatherapeutics.com
This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Sarepta’s plan to invest
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the expected benefits and opportunities related to the transactions with Lacerta may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development; the collaboration with Lacerta may not result in any viable treatments suitable for clinical research or commercialization due to a variety of reasons including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates or may never become commercialized products due to other various reasons including any potential future inability of the parties to fulfill their commitments and obligations under the agreements, including any inability by
Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties
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Source: Sarepta Therapeutics, Inc.
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