AAVLife, registered in Paris, is a privately held company dedicated to advancing gene therapy for rare diseases. AAVLife's lead indication is for Friedreich's Ataxia Cardiomyopathy. Its program is based on the successful use of a gene-therapy approach in a mouse model developed to mimic the gene defect and cardiac symptoms of Friedreich's Ataxia. The approach made use of an adeno-associated virus (AAV) to introduce a normal gene into cardiac tissue. The therapy restored cardiac function and reversed pathological heart enlargement in mice that had already progressed to heart failure.
Latest AAVLife News
Jan 5, 2016
Reprint When GlaxoSmithKline veteran Amber Salzman started up Paris-based AAVLife and ramped up its first gene therapy program for Friedrich's Ataxia, she started working early on with adenovirus vector pioneer Ron Crystal at Weill Cornell Medicine. And now, less than two years after rounding up the $12 million Series A for the biotech, Crystal is delivering three new gene therapy programs that the company--now renamed Annapurna--plans to put into the clinic as Salzman hunts up a Series B. First up is an IND-level program for Alpha1-antitrypsin (A1AT) deficiency, a genetic condition that destroys lung tissue. Hereditary angioedema is next in line, with some intriguing animal data setting the stage for an attempt at a new program for a one-time cure for severe allergies. "These targets are tractable from a development perspective," says Salzman, who was happy to "cherry pick" the three programs that offered the best, most straightforward shot at a successful clinical development program. Salzman has also signed up another longtime GSK exec for the company's operations in Philadelphia. Carlo Russo, a former top R&D exec at Glaxo, has signed on as chief medical officer and head of development. While still in its early days, Annapurna is following a well thumbed playbook in the gene therapy field. Like other startups, it allied itself closely with one of the big names that made an early rep for themselves in a field that has gone through multiple advances and some huge setbacks. But Salzman notes that gene therapy companies have been posting some positive results, which has helped lift the prospects for all the biotechs operating in the field. And Crystal takes the long view, noting that breaking ground in any new therapeutic arena often takes a considerable amount of time after the initial hype fades away. "It's not dissimilar from monoclonal antibodies," says Crystal, the chairman of genetic medicine at Weill Cornell. "Gene therapy needed a big learning curve. " It's been quite a trek for a field which is now headed to a new peak. To help highlight the quest it's on, the biotech's new name was drawn from a set of mountains in the Himalayas that Crystal has climbed. And Salzman believes that this is a good year to hunt up a new venture round to fuel the next big climb at Annapurna. Filed Under
AAVLife Frequently Asked Questions (FAQ)
What is AAVLife's latest funding round?
AAVLife's latest funding round is Series A.
How much did AAVLife raise?
AAVLife raised a total of $12M.
Who are the investors of AAVLife?
Investors of AAVLife include Versant Ventures and Inserm Transfert.
Who are AAVLife's competitors?
Competitors of AAVLife include Audentes Therapeutics.
Compare AAVLife to Competitors
Renova Therapeutics is a San Diego-based biopharmaceutical company and an industry leader in gene therapy, pursuing the development of treatments for congestive heart failure (CHF) and other chronic diseases. CHF is the most rapidly growing cardiovascular disorder in the United States, with an estimated six million people afflicted and 550,000 new cases diagnosed every year. Approximately 26 million patients have CHF worldwide. Renova Therapeutics, in a Public-Private Partnership with the National Institutes of Health, is currently supporting a Phase I/II trial of its novel lead therapy for patients with severe CHF (Class II, III and IV). This initial product is a single-dose gene therapy that, in animal studies, has shown substantially improved, sustained outcomes in heart failure models by safely and significantly improving myocardial function. The product is being evaluated for its safety and initial efficacy in a 3:1 randomized, double-blinded, placebo-controlled dose-ranging study involving 56 patients across seven US medical centers.
StrideBio is a company focused on gene therapy in the healthcare industry. It develops gene therapies for people with life-threatening diseases, leveraging its proprietary adeno-associated virus (AAV) vector engineering platform to create unique and differentiated vectors that improve upon naturally occurring AAV serotypes. The company primarily serves the healthcare sector. It was founded in 2015 and is based in Research Triangle Park, North Carolina.
Evive Biotech is a biopharmaceutical company with a focus on developing novel therapies. The company's main offerings include innovative drug candidates for oncology, inflammatory, and metabolic diseases. The company primarily serves the healthcare and pharmaceutical industries. It was founded in 2004 and is based in Singapore, Singapore.
Applied Genetic Technologies (NASDAQ: AGTC) is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. The company was founded in 1999 and is based in Alachua, Florida.
Calimmune is a clinical-stage biotechnology company focused on developing novel gene therapies that have the potential to improve and protect the lives of patients by enhancing their immune systems. Calimmune's lead product candidate for HIV, now in Phase I/II studies, is being evaluated as a one-time treatment to prevent HIV progression to AIDS. Calimmune is developing a full product pipeline of therapeutic candidates to address the unique needs of individuals at different stages of HIV infection and with various treatment histories.
Bluebird Bio runs a company focusing on pioneering gene therapies. The company's main services include the development of one-time treatments designed to deliver a functional copy of a gene to a patient’s own cells, with a particular emphasis on severe genetic diseases such as sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy. It was formerly known as Genetix Pharmaceuticals. It was founded in 1993 and is based in Cambridge, Massachusetts.