Regenerative medicine is beginning to add real value to healthcare not just by treating disease, but by curing disease, according to Edward Lanphier, President and CEO of Sangamo BioScience, and Chair of the Alliance for Regenerative Medicine. “That is the area of great differentiation, value creation and enthusiasm,” he said, speaking at Biotech Showcase™ 2015, January 12 in San Francisco.
Most people—even those in the industry—don’t realize that more than 60 regenerative products have been approved, he said. Some 500 companies are operating in this space globally, with 375 ongoing clinical trials. Of those, 340 are in Phase I or Phase II. Therefore, a great many products can be expected to be commercialized in the next few years.
Edward Lanphier, President and CEO of Sangamo BioScience, and Chair of the Alliance for Regenerative Medicine
Despite the opening of the IPO window and notable partnering last year, significant challenges remain. “Despite the value of these regenerative products, there can be resistance to their pricing,” Lanphier said. Because their merits aren’t always recognized, he calls for increased education throughout the healthcare sphere to ensure that effective therapeutics can be reimbursed. That means engaging the patient community and foundations, as well as physicians, regulators and payers.
The development of new tools is vital, too. For example, CRISPR genome editing technology is catalyzing the field. “There’s tremendous excitement around it because it is robust. Scientists’ first experiments using CRISPR/Cas9—with many different targets, cell types and species—work,” said Katrine Bosley, CEO, Editas Medicine. More tools like this are needed “to enable a broad set of possibilities across genetic-driven diseases,” thus helping gene therapy and genome editing to be more exact and, therefore, more effective.
Today’s breakthroughs have all evolved from decades of earlier work, the panelists emphasize. “You have to be prepared for a marathon,” Bosley said.
Voyager Therapeutics “has been working in stealth mode for two years,” selecting products that can be developed quickly, said Steven Paul, CEO. Its vectors can deliver therapeutics to the brain and the central nervous system. Voyager’s focus includes Parkinson’s disease, Friedreich’s ataxia and monogenic ALS. In December, it entered a collaborate agreement with MassBiologics to develop adeno-associated viral (rAAV) vectors for scalable production.
Avalanche Biotechnologies is focused on ophthalmic diseases using an AAV delivery platform. “There’s a tremendous opportunity, including wet and dry AMD and orphan indications,” said Thomas Chalberg, Founder and CEO. Animal studies show that after only one treatment 12 years ago, a retina is still delivering therapeutic proteins. “Our lead product is in Phase IIa for wet macular degeneration and in preclinical work for ALS. This is transformative, and an area of continuing innovation,” he said.
Panelist Larry Bullock, CFO of AGTC, also is focusing on orphan ocular diseases. Programs for XLRS and ACHM using AAVs as the delivery mechanism are preparing for INDs, with initial clinical data expected mid-year. A Phase IIb program also is underway for lung disease.
At bluebird bio, lentiviral vectors are used ex-vivo to target replication cells, according to Jeffrey Walsh, COO. “We remove cells from the body and introduce viral vectors carrying specific DNA to the cells in which it was missing.” The program uses hemopoietinc stem cells and T cells, and has three programs in clinical development, including one for sickle cell anemia.
“Celladon is targeting enzymes important in calcium regulation that are deemed undruggable,” said CEO Kristina Zsebo. The lead program is for heart failure, “the enormous unmet need that causes USD 60 billion in healthcare costs annually. We’re opening a 250 patient trial in April.”
ARM State of the Industry Briefing Panel, Biotech Showcase 2015
But, the panelists agreed, innovative science isn’t enough to build a successful product. Because this is cutting edge work, “education is very important. Stay close to the patient community and articulate what you’re trying to accomplish,” Walsh advised.
As companies think about commercialization, they also must “engage with regulators early and often” so they each understand what’s coming, he continued. In a later panel on advanced therapy drug development, Paul Wotton, President and CEO at Ocata Therapeutics, reiterated that sentiment. Despite having GMP manufacturing facilities ready, he said, “you need to continue to have productive dialog with regulators. Double-check everything with the agency.”
Take the conversation forward, beyond immediate science and manufacturing concerns, Bosley suggested. “Begin the right conversations with regulatory authorities and learn to anticipate ethical issues. The ethics are different for regenerative medicine (than for anything that’s come before). You’re talking about changing a person’s genomics. This issue is far greater than any company can deal with alone.”