Bamboo Therapeutics Gearing Up to Treat Deadly Childhood Neurological Diseases

By Barry Teater, NCBiotech Writer

Pope Francis kisses Italian children with Canavan disease at private Vatican meeting as researcher Jude Samulski (far left background) and the father of one of the children look on. -- Bamboo Therapeutics photos

Bamboo Therapeutics, aiming to treat devastating childhood neurological diseases with next-generation gene therapy, is the latest biotechnology company co-founded by entrepreneurs Sheila Mikhail and R. Jude Samulski, Ph.D., director of the Gene Therapy Center at the University of North Carolina at Chapel Hill.

“These are exciting times for gene therapy,” says Mikhail, Bamboo’s chief executive officer. “We are building Bamboo to be a fully integrated gene therapy company that can deliver potentially life-changing treatments to patients.”

Bamboo has been operating as a virtual company in Chapel Hill with seed money from angel investors, foundations and the motivated parents of children suffering from central nervous system and neuromuscular diseases. It hopes to raise further funds in 2016 to advance its gene therapies.

“We have been fortunate to have substantial support from families and foundations,” Mikhail says. “It’s really powerful when you meet these parents who have children with the diseases that we are pursuing. They are so motivated, and it helps to energize our own employees.”

Bamboo is a spinout of Asklepios Biopharmaceutical (“AskBio” for short), a gene-delivery technology company founded in 2003 by Mikhail, Samulski and UNC Professor Xiao Xiao, Ph.D., also a co-founder of Bamboo and an expert in gene therapy for muscular dystrophies.

Bamboo is one of several companies spun out of AskBio, which has received more than $700,000 in grants and loans from the North Carolina Biotechnology Center to support its research and commercial development.

Next-generation viral vectors

Bamboo’s technology uses a benign type of virus called adeno-associated virus (AAV) as a “vector” to ferry normal genes into the cells of patients who have faulty genes that cause disease. AAV technology has existed for several decades, but recent improvements have made it more precise, with potentially fewer detrimental “off-target” side effects.

Earlier viral vectors tested in clinical trials had to be delivered into the brain through holes surgically bored in the skull, and they didn’t spread broadly throughout the brain, limiting their potential effect.

Bamboo Therapeutics CEO Sheila Mikhail

Nonetheless, “even with that older technology, they (doctors and researchers) got evidence that it worked,” Mikhail says. “There was improved neurological function. It just looked like the patients got better.”

Newer viral vectors can be injected into the spinal canal and can target specific brain cell types without affecting other cells. “Having these vectors where we can put zip codes on them and deliver them to target tissues is a powerful technology,” Mikhail says.

In January 2016, Bamboo purchased UNC’s Vector Core, an 11,000-square-foot facility that manufactures viral vectors for research and clinical use by pharmaceutical and biotechnology companies, as well as universities and foundations.

“We believe that having a leading manufacturing facility fully integrated into our business provides flexibility and a competitive advantage,” says Samulski, scientific founder and chairman of Bamboo. “We anticipate rapidly moving our programs forward, including our DMD (Duchenne muscular dystrophy) program, which is expected to enter the clinic in early 2017.”

Bamboo has hired the Vector Core’s 20 employees, including Director Josh Grieger, Ph.D., who is another co-founder of Bamboo. He is the company’s new vice president of manufacturing and process development.

Focus on rare diseases

Bamboo will focus initially on the clinical development of gene therapies for four neurological diseases:

• Giant axonal neuropathy, a disorder of neurofilaments, the structural framework that helps define the shape and size of nerve cells. As nerve cells die, patients become increasingly paralyzed and typically die in their 20s or 30s.
• Friedreich’s ataxia, a progressive degeneration of nervous tissue in the spinal cord. After 10 to 15 years with the disease, patients are usually wheelchair bound and require assistance with daily living.
• Canavan’s disease, a progressive deterioration of nerve cells in the brains of infants. Patients typically do not live past the age of 10.
• Duchenne muscular dystrophy, a progressive degeneration of muscles. The disease mainly affects boys, and the average life expectancy for patients is about 25 years.

All of these rare diseases are caused by absent or faulty proteins due to mutations in single genes, making gene therapy with AAV vectors a viable treatment strategy.

Samulski is a pioneer in AAV vectors and was the first to clone the virus some 30 years ago. He has more than 20 patents filed or issued relating to AAV biology.

His gene therapy platform for Canavan disease is the first of its kind developed by a U.S. academic institution and allowed to be investigated in a clinical trial by the Food and Drug Administration. It’s also the first vector produced by UNC’s Vector Core to be tested in patients.

Samulski was recruited to UNC from the University of Pittsburgh with the help of a $430,000 Faculty Recruitment Grant from the Biotechnology Center in 1993.

Inspiration from the Pope

In November 2015, Samulski and a scientific collaborator, Paola Leone, Ph.D., a gene therapy researcher at Rowan University in Glassboro, N.J., were invited to join a group of Canavan disease patients and families for a private meeting with Pope Francis at the Vatican in Rome.

“It was very special experience,” says Samulski. “The families and I were touched that the Pope would take the time to meet with us.”

The Pope blessed the audience and wished the researchers success.