Annapurna Therapeutics will collaborate with Weill Cornell Medicine to build a gene therapy portfolio that will include three of the institution’s programs, the partners said today. The value of the collaboration was not disclosed. Annapurna will pay Weill Cornell Medicine milestone payments and royalties on sales for products developed.

Through licensing agreements, Annapurna plans to advance gene therapy programs launched at Weill Cornell Medicine’s Department of Genetic Medicine to treat Alpha1-antitrypsin (A1AT) deficiency, hereditary angioedema, and severe allergies.

Annapurna holds rights through an agreement with Weill Cornell Medicine to an IND application to initiate clinical studies of a gene therapy for A1AT. Weill Cornell Medicine has achieved encouraging results in a mouse model created with grafts of immune cells from human donors suffering from severe allergies, the partners said.

Annapurna, formerly AAVLife, will continue to use adeno-associated viral (AAV) vectors. The company will also partner with Weill Cornell Medicine to scale production of gene therapies by manufacturing processes that the institution has already used to produce GMP material for gene-therapy trials.

Separately, Annapurna said it will continue development of a gene therapy for cardiomyopathy associated with Friedreich’s ataxia, for which it holds an exclusive worldwide license to a recently issued U.S. patent covering the use of an AAV vector. Annapurna is doing observational studies of Friedreich’s ataxia patients to determine appropriate measures for future safety and efficacy trials.

Annapurna has named Carlo Russo, M.D., as CMO and head of development. Dr. Russo served as a senior vp in various R&D capacities at GlaxoSmithKline, including head of development of the Biopharm Unit and head of R&D of the Rare Diseases Unit. He has also held academic appointments at Weill Cornell Medicine.

Annapurna also named three researchers to its Scientific Advisory Board:

  • Hélenè Puccio, Ph.D., head of a research team at the Institute of Genetics and Molecular and Cellular Biology, University of Strasbourg;
  • Fulvio Mavilio, Ph.D., scientific director of the French biotechnology institute Genethon and professor of molecular biology at the University of Modena and Reggio Emilia, Italy;
  • and Ronald Crystal, M.D., chairman of genetic medicine and the Bruce Webster professor of internal medicine at Weill Cornell Medicine, as chief scientific adviser to Annapurna.

In 2014 the journal Human Gene Therapy honored Dr. Crystal, a professor of genetic medicine and of medicine at Weill Cornell Medicine, with its Pioneer Award in recognition of his seminal work on viral vectors to deliver therapeutic genes to patients. Human Gene Therapy is published by GEN publisher Mary Ann Liebert Inc.

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