Nearly 300 gene and cell therapies in development - report


Pharma is becoming increasingly focused on gene and cell therapies, with nearly 300 in clinical development or awaiting review by the FDA, according to a new report from US trade body PhRMA.

The first ever Medicines in Development report on cell and gene therapies found that there are 289 novel gene and cell therapies in development or awaiting review, and 111 of these are aimed at cancer.

More than 100 diseases are being studied for potential treatment with cell and gene therapies, including Alzheimer’s disease, cardiovascular disease, neurological disorders, eye disorders, and infectious diseases.

The report follows several landmark developments in the field over the last year and a half, starting with the FDA’s first approval of Novartis’ cancer cell therapy Kymriah last August.

Gilead/Kite then got its rival CAR-T, Yescarta approved by the FDA shortly afterwards.

This was followed by the news about a year ago that Spark Therapeutics’ Luxturna, a gene therapy for a form of inherited blindness, had got the nod from the FDA too.

This year also saw the first ever RNA interference (RNAi) drug approved by the FDA, after Alnylam’s Onpattro got a licence for nerve damage caused by hereditary transthyretin-mediated amyloidosis in adults.

The report shows that cancer is by far the most common target for the cell therapies in development, although the majority of the 111 therapies in the clinic are in early stage development.

There are 28 cell or gene therapies in development for eye disorders, 24 for cardiovascular disease, 22 for neurologic diseases, and 21 for blood disorders.

Other diseases being targeted include haemophilia A, haemophilia B, and the report noted efforts by biotechs such as bluebird bio to develop a second generation of CAR-T therapies that target B-cell maturation antigen and kill malignant myeloma cells.

There is also hope that a gene therapy for the vision loss disorder Stargardt disease can provide long-term or potentially permanent correction by correcting a mutation in photoreceptors in the retina.