Jaguar snags a CMO after banking $139M to propel its preclinical gene therapies

Jaguar Gene Therapy emerged in February with about $40 million from Deerfield Management and a plan to get its leading assets into the clinic in the next couple of years. Now, having raised another $139 million to fuel that mission, the company is adding a chief medical officer to its team. 

Joe McIntosh, M.D. arrives from Aruvant, a Roivant subsidiary where he led development of a gene therapy for sickle cell disease as the company’s chief medical officer. Before joining Aruvant in May 2020, McIntosh was head of clinical development at PTC Therapeutics, where he led clinical studies in areas such as genetic diseases and oncology. 

He joins a team led by CEO Joe Nolan, formerly general manager of U.S. operations at AveXis, the company that developed Zolgensma, the first gene therapy for children with spinal muscular atrophy. Novartis bought AveXis for $8.7 billion in 2019. 

RELATED: AveXis name recognition and Zolgensma legacy hook Lilly in Jaguar's $139M series B 

The team’s experience at AveXis drew its series B investors, including Goldman Sachs and Eli Lilly, as well as its new CMO. 

“When I saw that former AveXis leadership had formed Jaguar, I was eager to join the team and contribute to their efforts to accelerate the development of breakthrough gene therapies,” McIntosh said in a statement on Tuesday. “I believe Jaguar’s initial pipeline is really exciting and holds great promise for patients and families suffering from severe genetic diseases.” 

That pipeline includes gene therapies for galactosemia, an inborn error of metabolism; autism spectrum disorder with a genetic cause; and Type 1 diabetes. Its U.K.-based subsidiary, Axovia Therapeutics, is working on treatments for disorders of the cilia, starting with a subtype of Bardet-Biedel syndrome. 

Axovia is developing a gene therapy for subtype of Bardet-Biedl called BBS1, a life-threatening neurometabolic condition with no effective treatment. This program is the furthest along, with an IND planned for the second half of 2022, Nolan said in a previous interview. 

RELATED: Deerfield unveils Jaguar Gene Therapy with 4 preclinical programs and access to more 

INDs for the programs in galactosemia—which affects the body’s ability to process and produce energy from galactose, a sugar found in milk—and autism spectrum disorder will follow in 2023. 

“With his expertise and insights in genetic diseases, he is perfectly suited to be our Chief Medical Officer, responsible for advancing our robust pipeline of AAV9-based gene therapies targeting severe genetic diseases in patient populations with large unmet need.” said Sukumar “Suku” Nagendran, M.D., Jaguar’s R&D chief, who served as chief medical officer at AveXis. 

Like Taysha Gene Therapies, another company founded and run by members of the former AveXis team, Jaguar is focusing on treatments delivered using adeno-associated viruses, or AAVs. 

Other companies are working on other delivery vehicles for gene therapies, such as lentiviral and nonviral vectors, but Jaguar is sticking to a proven platform that they know well—at least, until the other methods get proven out, Nolan said in a previous interview. 

For now, Jaguar will be busy working on its preclinical programs, but down the line, it may license more prospects from Deerfield’s network of 18 academic institutions.