AskBio Spins Out New Gene Therapy Company, Actus Therapeutics

Actus logoAsklepios BioPharmaceutical (AskBio), a gene therapy platform company based in Chapel Hill, has spun out its fourth company, Actus Therapeutics, to develop gene therapies for rare genetic diseases including Pompe disease and epilepsy.

Actus will focus initially on treating Pompe disease, an inherited disorder that is typically fatal in infants if untreated but can be less severe in its adult form. 

Actus is preparing for a Phase 1 clinical trial of its therapy in six adults with late-onset Pompe disease. The company’s investigational new drug application and trial protocol have been approved by the Food and Drug Administration, and the trial is expected to begin in the fourth quarter of this year, said Sheila Mikhail, co-founder and chief executive officer of Actus.

The company’s therapy is based on technology developed in the lab of Dwight Koeberl, M.D., Ph.D., professor of pediatrics and a medical genetics specialist at Duke University. Koeberl’s research focuses on using adeno-associated virus (AAV) vectors to insert DNA or RNA into targeted cells where it can repair faulty genes that cause disease.

Pompe disease is triggered by genetic mutations that cause a deficiency of an enzyme that converts glycogen – the body’s reserve of stored energy – into glucose, or blood sugar. The deficiency results in a massive accumulation of glycogen in muscles, organs and tissues, leading to muscle weakness and wasting.

The disease is estimated to affect about one in every 40,000 births, and there is no known cure.

While enzyme-replacement therapy has shown promise for treating Pompe disease in infants and adults, it is expensive, requires frequent infusions and can cause unwanted antibody responses and side effects.  

“We have shown in mice that AAV gene therapies have the potential to overcome the limitations of enzyme-replacement therapy by delivering low doses and restricting expression to the liver,” Koeberl said. “We look forward to working with Actus and bringing these therapies into human clinical trials.”


Actus said its Pompe therapy would benefit from AskBio’s AAV platform technologies, manufacturing process, and experience in progressing AAV gene therapy programs into the clinic. AskBio’s AAVs have the potential to provide treatment at a lower effective dose and with more cell type specificity and less potential systemic effects than other AAVs, Actus said.

Deep expertise in AAV biology

Actus is managed through AskBio, which has about 12 employees and several more coming on board as Actus ramps up operations.

AskBio’s gene therapy platform is based on the work of Jude Samulski, Ph.D., a pioneering scientist in gene therapy who was recruited to the University of North Carolina School of Medicine from the University of Pittsburgh in 1993 with the help of about $250,000 in grant funding from the North Carolina Biotechnology Center. Samulski directed UNC’s Gene Therapy Center for many years.

Several grants and loans from the Biotech Center have supported the development of Samulski’s academic research and commercial technologies respectively.

Samulski and Mikhail founded AskBio in 2003 to commercialize AAV gene therapies. AskBio subsequently spun out three gene therapy companies prior to Actus:

  • NanoCor Therapeutics, developing treatments for cardiovascular disease.
  • Chatham Therapeutics, developing treatments for hemophilia. Chatham was sold to Baxter International (now Shire) in 2014 for $70 million.
  • Bamboo Therapeutics, developing treatments for rare neuromuscular diseases. Bamboo was sold to Pfizer in 2016 for $150 million in a deal that could be worth as much as $645 million if certain milestones are met. 

Samulski joined Pfizer in Sanford, N.C., as vice president of gene therapy after its purchase of Bamboo Therapeutics but is transitioning back to UNC and to AskBio as the company’s scientific founder and chief science officer, Mikhail said.

Barry Teater, NCBiotech Writer
Thu, 04/26/2018 - 11:41