Date: 2018-08-08
Type of information: Licensing agreement
Compound: AAV-based CNS-targeted gene therapies
Company: Sarepta Therapeutics (USA - MA) Lacerta Therapeutics (USA - Fl)
Therapeutic area: CNS diseases
Type agreement: licensing
Action mechanism: gene therapy
Disease: CNS diseases
Details:
- • On August 8, 2018, Sarepta Therapeutics announced 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. 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.
Financial terms:
- 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.
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Is general: Yes
