When GlaxoSmithKline ($GSK) veteran Amber Salzman started up Paris-based AAVLife and ramped up its first gene therapy program for Friedreich'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.