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Horae Gene Therapy Center announces collaboration with Spark Therapeutics

Agreement focuses on selecting adeno-associated virus vectors to target genetic diseases

  Guangping Gao, PhD

Guangping Gao, PhD

Spark Therapeutics has announced a multiyear research agreement with Guangping Gao, PhD, the Penelope Booth Rockwell Chair in Biomedical Research, professor of microbiology & physiological systems and director of the Horae Gene Therapy Center. Dr. Gao will collaborate with Spark Therapeutics to identify adeno-associated virus (AAV) vectors from a proprietary library of AAV capsids and evaluate their efficacy, with the goal of enhancing the efficiency of gene delivery to cells in the retina, liver and central nervous system.

“We’re excited to have selected Spark Therapeutics as a collaborator to develop next-generation AAV vectors that deliver genetic information to certain cell types more efficiently and to cell types that are otherwise hard to target,” said Gao.

Under the agreement, Spark Therapeutics has the option to an exclusive worldwide license for the intellectual property developed under the research agreement.

“We are delighted to be working with Professor Gao, a longtime leader in gene therapy and capsid development,” said Katherine A. High, MD, president and chief scientific officer at Spark Therapeutics. “Strategically, these types of relationships and agreements help us build on our research in gene therapy and continue to advance the utility of our proven and proprietary gene therapy platform.”

Spark Therapeutics is a gene therapy company developing investigational, potentially one-time, life-altering treatments, with a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal diseases, liver-mediated diseases such as hemophilia, and neurodegenerative diseases.

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