AskBio Reels In $235M to Advance Gene Therapy Clinical Trials

Asklepios BioPharmaceutical (AskBio) has raised $235 million in financing to support its research and development of gene therapies for rare, genetic disorders.

The funding came from TPG Capital and Vida Ventures, which together invested $225 million and gained a minority stake in the Research Triangle Park, NC, company. Concurrent with that investment, AskBio’s founders and board members invested $10 million. The AskBio funding is the latest in a flurry of gene therapy investments that have swept across the sector in recent months.

Gene therapy uses an engineered virus to deliver a functioning gene to replace a mutated or dysfunctional one. The goal is to provide a one-time treatment with a long-lasting, perhaps even permanent therapeutic effect.

AskBio has successfully placed gene therapy programs in early development in the hands of larger pharmaceutical companies. In 2014, Baxter (NYSE: [[ticker:BAX]]) paid $70 million up front for Chatham Therapeutics, an AskBio spinout developing hemophilia gene therapies. Those programs are now part of Takeda Pharmaceutical (NYSE: [[ticker:TAK]]). In 2016, Pfizer paid $150 million to acquire Bamboo Therapeutics, an AskBio spinout with programs for rare neuromuscular disorders.

AskBio’s current pipeline has experimental gene therapies for neuromuscular diseases, central nervous system disorders, and cardiovascular conditions. Earlier this year, the company began a clinical trial of its gene therapy for Pompe disease, an inherited enzyme deficiency that leads to a buildup of a sugar in muscle tissues that leads to muscle weakness. Another AskBio gene therapy for Parkinson’s disease is in Phase 1/2 testing.

AskBio was founded in 2001 by Jude Samulski, the company’s president chief scientific officer, and CEO Sheila Mikhail, a veteran life sciences executive. The company’s technology is based on research from the Gene Therapy Center at the University of North Carolina at Chapel Hill. The company says it will apply the new capital toward clinical trials and manufacturing of its experimental therapies.

The past year has seen a number of pharmaceutical companies strike open their wallets to boost their presence in gene therapy. In January, Johnson & Johnson (NYSE: [[ticker:JNJ]]) paid $100 million for rights to gene therapies developed by MeiraGTx (NASDAQ: [[ticker:MGTX]]) for vision loss. The next month, Roche agreed to pay $4.8 billion for Spark Therapeutics (NASDAQ: [[ticker:ONCE]]), which has a treatment for a rare form of blindness that became the first FDA-approved gene therapy in 2017. Biotech startups are also jumping into gene therapy. New York-based Neurogene emerged in February with $68 million in Series A financing to support development of its preclinical treatments for rare, genetic brain disorders.

March continued the gene therapy dealmaking. Biogen (NASDAQ: [[ticker:BIIB]]) agreed to pay $800 million to acquire Nightstar Therapeutics and its portfolio of gene therapies for rare eye diseases. That deal was followed by Pfizer, which paid $51 million for a minority stake in French gene therapy developer Vivet Therapeutics. And lab equipment maker Thermo Fisher Scientific (NYSE: [[ticker:TMO]]) reached a $1.7 billion deal for Brammer Bio, a contract manufacturer of gene therapy tools called “viral vectors.”