Annapurna Therapeutics to Collaborate with Weill Cornell Medicine on Gene-Therapy Portfolio

Collaboration To Yield Market-Leading R&D Pipeline

PARIS & NEW YORK--()--Annapurna Therapeutics today announced a collaboration with Weill Cornell Medicine to build a market-leading gene-therapy company.

“This collaboration with Weill Cornell Medicine amounts to a step change in our ambitions at Annapurna”

Under a series of licensing agreements, Annapurna will advance several gene-therapy programs already initiated at Weill Cornell Medicine’s pioneering Department of Genetic Medicine. Annapurna, formerly AAVLife SAS, will continue to make use of adeno-associated viral (AAV) vectors and will 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.

Ronald Crystal, M.D., Chairman of Genetic Medicine, the Bruce Webster Professor of Internal Medicine and a Professor of Genetic Medicine and of Medicine at Weill Cornell Medicine, will serve as Chief Scientific Adviser to Annapurna. In 2014 the peer-reviewed journal Human Gene Therapy presented Dr. Crystal with its Pioneer Award in recognition of his seminal work on viral vectors to deliver therapeutic genes to patients.

“This collaboration with Weill Cornell Medicine amounts to a step change in our ambitions at Annapurna,” said Amber Salzman, Ph.D., President and Chief Executive Officer of Annapurna. “We are bringing together a promising pipeline, a proven manufacturing technology, and a team of managers and advisers with deep experience in gene-therapy development.”

“With Annapurna, we intend to advance promising discoveries made in our laboratories at Weill Cornell Medicine into clinical studies,” said Dr. Crystal, who is a co-founder, shareholder and paid consultant to Annapurna Therapeutics. “Both sides understand the urgent need to make the promise of gene therapy a reality for patients needing better treatment options.”

Thomas Woiwode, Ph.D., a Managing Director at Versant Ventures, which has led the financing of Annapurna, commented: “We’re thrilled to be working with Amber and Ron and the deeply experienced management team at Annapurna to develop our robust portfolio of gene-therapy products.”

In addition, Annapurna has appointed Carlo Russo, M.D., as Chief Medical Officer and Head of Development. Dr. Russo served as a Senior Vice President in various R&D capacities at GlaxoSmithKline, including as head of Development of the Biopharm Unit and of R&D of the Rare Diseases Unit. He led development of the first GlaxoSmithKline gene-therapy product for severe combined immunodeficiency syndrome, for which GlaxoSmithKline has filed a marketing application in Europe.

Previously, Dr. Russo was President and Chief Executive Officer of Vaxinnate Corporation and the head of the Department of Global Strategic Regulatory Development, Vaccines and Biologics, including human papilloma virus and rotavirus vaccines, at Merck Research Laboratories. He has also held academic appointments at Weill Cornell Medicine.

The agreements with Weill Cornell Medicine add these programs to the Annapurna pipeline:

Alpha1-antitrypsin (A1AT) deficiency. Through an agreement with Weill Cornell Medicine, Annapurna holds rights to an Investigational New Drug application to initiate clinical studies of a gene therapy for A1AT, a genetic disorder that results in the destruction of lung tissue. In the United States alone, A1AT affects some 90,000 people, most of them currently untreated. Current treatment requires weekly protein infusions.

Hereditary angioedema. Hereditary angioedema manifests itself in severe swelling of various parts of the body including the hands, face, and throat. Swelling of the airway can lead to death by asphyxiation. The prevalence of the disorder is estimated between one in 10,000 people to one in 50,000. The currently approved prophylactic therapy can require frequent protein infusions and in clinical studies reduced the overall attack rate by about half.

Severe allergies. Weill Cornell Medicine has achieved encouraging results for gene therapy in a mouse model created with grafts of immune cells from human donors suffering from severe allergies. Severe allergies cause extreme discomfort and can be fatal. Peanut allergy, for example, is a common cause of anaphylaxis in children arriving in emergency rooms. There are no definitive treatments for peanut allergy apart from epinephrine during emergencies.

Under the agreements between Annapurna and Weill Cornell Medicine, the institution will receive milestone payments and royalties on sales. Terms were not disclosed.

Annapurna will also continue to fully resource its earlier development of a gene therapy for cardiomyopathy associated with Friedreich’s ataxia. Annapurna is doing observational studies in Friedreich’s ataxia patients to determine appropriate measures for safety and efficacy trials. Friedreich’s ataxia affects 10,000 to 20,000 people in the United States and Europe. Although the disorder is characterized by the loss of full control of bodily movements, most deaths result from progressive degradation of cardiac function. There are no approved treatments.

Annapurna holds an exclusive worldwide license to a recently issued United States patent covering the use of an AAV vector in gene therapy for cardiomyopathy associated with Friedreich’s ataxia.

On Annapurna’s Scientific Advisory Board, Dr. Crystal of Weill Cornell Medicine is joined by two other internationally recognized researchers in fields of interest to the company:

  • Hélenè Puccio, Ph.D., Head of a research team at the Institute of Genetics and Molecular and Cellular Biology, University of Strasbourg. Dr. Puccio was the lead author of a paper in Nature Medicine which in 2014 reported the promise of gene therapy for cardiomyopathy associated with Friedreich’s ataxia in an animal model.
  • 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. Dr. Mavilio has researched gene therapy for rare diseases for more than 20 years.

About Annapurna Therapeutics

Annapurna Therapeutics, headquartered in Paris, is a privately held company dedicated to advancing gene therapy for significant unmet medical needs. It is funded by Versant Ventures, an international life-sciences venture-capital firm; Inserm Transfert Initiative, a seed fund linked to the technology-transfer arm of Institut National de la Santé et de la Recherche Médicale (Inserm); and Bpifrance, a public investment bank. Further information is available at


For Annapurna:
Amber Salzman, +1 610-659-1098
President & Chief Executive Officer
Biosyntax LLC
Richard Koenig, +1 610-213-0725
For Weill Cornell Medicine:
Jen Gundersen, +1 646-317-7402

Release Summary

Annapurna Therapeutics and Weill Cornell Medicine will collaborate to develop novel gene therapies.

Annapurna Therapeutics and Weill Cornell Medicine