Cell and Gene Therapy Development Increased 20%, PhRMA Finds

There are 362 investigational cell and gene therapies currently in clinical development, a 20 percent increase from 2018, according to a recent report from Pharmaceutical Research and Manufacturers of America (PhRMA).

The first cell and gene therapy was approved in 2017 and the increasing rate of research and development is enhancing the reality of personalized gene therapy treatments for patients in the near future. 

The report mentioned five gene therapies in development for potential treatments: 

• Gene therapy using adeno-associated virus (AAV)- factor VIII- designed to stimulate the production of factor VIII for the treatment of hemophilia A
• Gene therapy using AAV vectors to deliver a high-activity Factor IX gene to the liver for the treatment of hemophilia B
• A second-generation CAR-T cell therapy comprising genetically-modified T cells designed to target B-cell maturation antigen and redirect the T-cells to recognize and kill malignant myeloma cells
• Gene therapy for the treatment of Stargardt disease, which delivers a corrected version of the ABCR gene directly in the photoreceptors in the retina
• Gene therapy used to recombinant AAV9 capsid to deliver a shortened version of human dystrophin to treat Duchenne muscular dystrophy 

A handful of innovative medicines have previously been approved by the FDA and are currently helping patients. Thus far, the gene or cell therapies have centered on treating cancer, eye diseases, and rare hereditary diseases.

But increases in cell and gene therapy development could help patients with other conditions and diseases.

Last week, Oregon Health & Science University (OHSU) held the first-ever gene therapy clinical trial, BRILLIANCE, to address blindness-causing gene mutation, LCA10. 

Using new gene-editing tool, CRISPR, the clinical trial performed in vivo gene editing to treat Leber congenital amaurosis 10, a rare form of inherited blindness. During this clinical trial, one patient living with LCA10 was treated. This is a promising outcome, as patients currently affected by the disease have had no prior treatment options.

“This dosing is a truly historic event – for science, for medicine, and most importantly for people living with this eye disease,” said Cynthia Collins, president and CEO of Editas Medicine. “The first patient dosed in the BRILLIANCE clinical trial marks a significant milestone toward delivering on the promise and potential of CRISPR medicines to durably treat devastating diseases such as LCA10. We look forward to sharing future updates from this clinical trial and our ocular program.”

Despite positive outcomes, scientists and manufacturers still face technical and logistical challenges to bring the treatments to market in sufficient quantities. A recent Oxford Economics paper explored the challenges and suggested that large capital investments are “likely needed to bring future breakthroughs that will standardize processes, simplify delivery logistics, boost efficiency, and increase competition.” 

Researchers believe that they need to explore next-generation therapies and ensure patients receive affordable access to cell and gene therapies in the future. Biopharmaceutical companies are currently partnering with insurers to develop innovative contracting arrangements. 

The arrangements include result-based or value-based contracts that focus on real-world results for patients and may improve outcomes and alternative financing arrangements to help limit or spread costs over time for a payer, the report highlighted. But older federal rules and policies can provide uncertainty for manufacturers. This may limit the growth of innovative contracting arrangements. 

“Novel cell and gene therapies in the development pipeline today are the results of pioneering research by America’s biopharmaceutical research companies. It is crucial we foster an environment that encourages continued innovation in this space,” researchers concluded.