Deerfield Management-backed Jaguar Gene Therapy, and its ciliopathy-focused subsidiary Axovia Therapeutics, launched today with an undisclosed Series A.
Deerfield Management-backed Jaguar Gene Therapy, and its ciliopathy-focused subsidiary Axovia Therapeutics, launched today with an undisclosed Series A to develop adeno-associated virus serotype 9 (AAV9)-driven gene therapies. Jaguar’s C-suite cut their teeth at another Deerfield company, AveXis, which developed Zolgensma gene therapy for spinal muscular atrophy, and was subsequently purchased by Novartis in 2018 for $8.7 billion. AveXis was renamed Novartis Gene Therapy last year.
The companies have been in stealth mode for the past year. Jaguar is developing gene replacement therapies for severe genetic diseases, using a platform based on the AAV9 capsid, the same vector used for Zolgensma. The first three preclinical programs in Jaguar’s pipelines are JAG101, in development for galactosemia; JAG201, targeted at a genetically driven form of autism spectrum disorder; and JAG301, to be targeted at a subpopulation of newly diagnosed Type 1 diabetes patients.
Jaguar’s President and CEO is Joe Nolan, who was previously AveXis’s General Manager for the U.S. He is joined by Sukumar Nagendran, President of R&D, who led development of AVXS-101, which is now being groomed for regulatory submission in older patients with spinal muscular atrophy by Novartis Gene Therapy. Jaguar’s Executive Chairman Sean Nolan was previously President and CEO at AveXis, and the company’s Chief Operating Officer Andrew Knudten, Chief Operating Officer Phil Donenberg, and Chief Legal Officer Michael Johannesen all came from AveXis as well.
“We believe that the combination of our proven management team and gene therapy expertise coupled with the financial resources and access to academic institutions provided by Deerfield Management will facilitate our ability to identify critical clinical needs,” said Sean Nolan, “and address them by expediting the development of gene therapies from bench to bedside as safely and quickly as possible.”
Meanwhile, Axovia – a majority-owned subsidiary of Jaguar that has also received funding from the UCL Technology Fund – is working on an AAV9-delivered gene replacement therapy for Bardet-Biedl Syndrome-1 (BBS1), a subset of patients with the rare ciliopathy BBS, with symptoms including early onset retinal blindness. The subsidiary was cofounded by BBS research pioneer Philip Beales, who developing diagnostics for the disease and helping identify the genetic causes of cilial dysfunction while head of genetics and genomic medicine at University College London (UCL). Another gene therapy, Luxturna, has been approved for genetic retinal diseases Leber congenital amaurosis and retinitis pigmentosa.
Deerfield’s strategy in recent years has been to ink research deals with academic institutions in order to access early-stage opportunities, most recently signing a new $130 million collaboration with Dana-Farber Cancer Institute. Axovia’s program is based on a program licensed from UCL and Apollo Therapeutics last year. Deerfield will help Jaguar access academic labs that could supply the company with future gene therapy candidates.
Zolgensma, a one-time therapy, costs $2.125 million per dose, making it among the most expensive medicines in the world.
