Cell & Gene Therapy Commercial Insight – February 2019

CELL THERAPY:

We saw Gilead drop its anti-BCMA CAR-T therapy this past month amidst a crowded competitive landscape and clinical data that didn’t support further development work. The BCMA space has picked up rapidly the last few years and some companies that are further along in the clinic than Gilead, including bluebird, have generated impressive data. Certainly, with the plethora of CAR-T technologies that are in the clinic for hematological malignancy Gilead won’t be the only company pulling candidates as the pool of clinical data grows and the leading products are identified.

GENE THERAPY:

This month’s news sees good progress for several companies, with the announcement of positive data for AVROBIO’s treatment for Fabry disease and uniQure’s treatment for haemophilia B, and advances in clinical trials for Solid Biosciences in Duchenne muscular dystrophy and Lysogene’s treatment for Sanfilippo A. It’s not all good news though, with disappointment coming from Gensight Biologic’s treatment for Leber’s hereditary optic neuropathy (LHON); this was always going to be a tough nut to crack, and thus it proved. There’s only one treatment available for LHON – Santhera’s idebenone (Raxone), approved under exceptional circumstances in Europe – and the negative interim results from Gensight’s RESCUE study suggest there will be no new therapies for this devastating condition in the near future.

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