2017: The dawning of cell and gene therapies

By Dan Stanton

- Last updated on GMT


Related tags Gene therapy Medicine Food and drug administration Cell therapy manufacturing & gene therapy congress

Product approvals and a regulatory focus on regenerative medicines has made 2017 a breakthrough year for cell and gene therapies, say consultants.

“This is a year where in the field of cell and gene therapy it is easier to talk about what has not changed in regulatory affairs,”​ Anthony Davies, chairman of Dark Horse Consulting Inc., said earlier this month.

Speaking to delegates at the Cell Therapy Manufacturing & Gene Therapy Congress in Amsterdam he highlighted the efforts being made by regulators to clarify and drive development of such therapies.

The year began with US Food and Drug Administration (FDA) guidance for the freshly introduced Regenerative Advanced Therapy designation, later renamed regenerative medicine advanced therapy (RMAT) – possibly to avoid the Murinae connotations and pun headlines, ahem​, associated with its abbreviation.

Humacyte’s Humacyl (human acellular vessel) became the first product to receive RMAT designation (at least publically) back in March

But Davies said regulatory momentum never slowed down, driven in part by the new FDA Commissioner Scott Gottlieb who “has a significant agenda of change and has a significant level of interest in cell and gene therapy.”

Gottlieb described cell and gene therapies as “no longer the stuff of science fiction” in November​ while publishing a series of guidelines for industry on regenerative medicines. He has also led efforts to clampdown on rogue stem cell clinics​ to help fully regulate the sector.

Approvals offsetting safety concerns

However, it is the approvals of two chimeric antigen receptor (CAR) T cell therapies and – as of this week – a gene therapy product which have cemented 2017 as the year cell and gene therapies broke through.

In September​, Novartis received FDA approval for Kymriah (tisagenlecleucel) as a single treatment for patients with acute lymphoblastic leukemia (ALL). Weeks later​ Kite Pharma – fresh from its acquisition by Gilead Sciences – saw its CAR-T therapy Yescarta (axicabtagene ciloleucel) approved for patients with relapsed or refractory large B-cell lymphoma.

“We have all witnessed this year the great news of first successful authorisations of CAR T products – two of them approved in the US and currently under evaluation in the EU,”​ said Paula Salmikangas, director of Biopharmaceuticals and ATMP (Advanced Therapy Medicinal Products) at NDA Group AB, and a former Finnish regulator.

She told delegates the approvals largely offset some of the “worrying news about the safety concerns”​ of cell therapies after deaths in clinical trials led to the FDA temporarily halting trials for Juno Therapeutics’ JCAR015 candidate in 2016​ and Cellectis’ allogeneic candidate UCART123 in September​ this year.

“When some of the clinical trials were on hold people were even thinking whether successful translation of such complex products would be even possible.”

Meanwhile, Spark Therapeutics was given an early Christmas present this week​ with the approval of Luxturna (voretigene neparvovec-rzyl), its one-time gene therapy treatment for an inherited form of vision loss.

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