More than 525 clinical trials are underway worldwide to test new ways of growing human tissue, implanting cells, and modifying genes to treat patients with a range of diseases, drawing nearly $5 billion in financial backing in the second quarter, according to new data.
But the emerging field of regenerative medicine, which could bring its first commercial products to the US market over the next three to five years, faces formidable hurdles. Companies must identify disease targets, develop drug-delivery technologies and manufacturing processes, and figure out how to bill health insurers for procedures that could cure diseases.
“We’re all chasing this Holy Grail of one-shot lifelong therapies,” said Frank Borriello, vice president for global business development at Baxalta Inc., which is working on a gene therapy for hemophilia patients.
He spoke at a panel on gene and cell therapies Thursday at the annual BioPharma America conference at the Boston Marriott Copley Place.
Because some of the new therapies will treat rare diseases affecting only hundreds of patients, a giant one-time price tag — or even an annuity where payments would be made over the patient’s lifetime — could radically change the economics of prescription drug reimbursements as insurers already are being strained by costly new medicines.
“These are going to be eye-popping numbers,” warned Edward Lanphier, chief executive of Sangamo Bioscience Inc., a Richmond, Calif., gene therapy company working on ways to modify a cell’s DNA at specific locations to treat AIDS and other diseases. “It’s going to take a shift away from traditional business models toward a different kind of model.”
While gene therapy research has been around for decades, the field is still recovering from a setback in 1999 when 18-year-old Jesse Gelsinger, who suffered from a rare metabolic disorder, died in a clinical trial run by the University of Pennsylvania after a technique to inject a gene into his cells led to massive organ failure. Scientists have since improved the technique that allows such injections, enabling a new generation of clinical studies to move forward.
There are currently 55 programs in late-stage trials, according to the Alliance for Regenerative Medicine, a global advocacy group promoting advanced therapies in the emerging field.
In the United States, some leaders in the field include Bluebird Bio, a Cambridge company developing treatments for children with a rare blood disorder called beta-thalassemia, and Spark Therapeutics Inc., a Philadelphia company working on cures for inherited retinal diseases.
With such programs moving through the research-and-development pipeline, money is flowing into the field. Regenerative medicine companies raised $4.9 billion through venture financing, corporate partnerships, acquisitions, and public offerings in the three months ended June 30, up 129 percent from the same period a year ago, according to the regenerative medicine alliance.
One of the most promising niches taking root in the Boston area is genome editing, which seeks to repair defective mutations responsible for thousands of genetic disorders. But speakers at the BioPharm America conference acknowledged there is still much work to do.
“It’s beautiful biology, but it’s still basic science,” said Alexandra Glucksmann, chief operating officer for Editas Medicine, a gene-editing startup in Cambridge. “We’re not there yet with the technology or the delivery.”Robert Weisman can be reached at firstname.lastname@example.org. Follow him on Twitter @GlobeRobW.
Correction: An earlier version of this story incorrectly stated the percentage increase of investments in regenerative medicine companies. It increased 129 percent in the second quarter from the same period a year ago.