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

-- Sarepta expands its presence in gene therapy and broadens its reach with license to up to three new CNS-targeted gene therapy programs, including exclusive rights to Lacerta’s  gene therapy candidate for Pompe Disease and options to two additional candidates --

-- Sarepta will make an equity investment of $30 million --

-- Lacerta is an AAV-based gene therapy company founded on technologies licensed from the University of Florida, ranked a top center of excellence in gene therapy research; its founders have led numerous clinical-stage gene therapy programs and made significant advances in and contributions to the gene therapy field --

CAMBRIDGE, MA, USA I August 08, 2018 I Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced today that it has made a strategic investment and entered into a license and option agreement with Lacerta Therapeutics, a gene therapy company using a constellation of proprietary adeno-associated virus (AAV) vector technologies to develop CNS-targeted treatments and lysosomal storage diseases. Sarepta expects to benefit from Lacerta’s expertise in AAV-based CNS-targeted gene therapies, will gain access to Lacerta’s capsid screening and proprietary OneBac manufacturing platform and process for the licensed products, and bolsters its pipeline to 11 gene therapy programs, with three CNS-focused programs from Lacerta.

“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 Doug Ingram, Sarepta’s president and chief executive officer.

Specifically, the Lacerta relationship provides Sarepta with the following:

  • Expansion of Sarepta’s gene therapy pipeline to up to 11 unique candidates: With this transaction, Sarepta has 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 Pennsylvania and 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 Sarepta will lead clinical development and commercialization. Sarepta will owe development and sales-based milestones to Lacerta and pay single-digit royalties on net sales. 

“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. Joseph Reddy, president and chief executive officer of Lacerta Therapeutics. "We are pleased to begin our collaboration with Sarepta Therapeutics, a gene therapy leader, as it represents a significant step in advancing Lacerta’s treatments to the clinic,” added Dr. Reddy.

About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates. For more information, please visit www.sarepta.com.

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

SOURCE: Sarepta Therapeutics

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