Skip to main content
Premium Trial:

Request an Annual Quote

Gene Therapy Hope

Gene therapies have been getting a lot of notice lately, leading CNBC to ask a biotech investor what all the fuss is about.

Novartis' CAR-T cell therapy, Kymriah, became the first approved gene therapy in the US in August. The US Food and Drug Administration approved it treat a type of acute lymphoblastic leukemia. A second gene therapy, Kite Pharmaceuticals' Yescarta, was approved earlier this month to treat certain kinds of large B-cell lymphoma. An FDA panel has also voted to recommend the approval of Spark Therapeutics' Luxturna for a type of childhood blindness and a final decision about which is expected in January.

Canaan Venture Partners' biotech investor Nina Kjellson tells CNBC that gene therapies are promising as they could target a range of diseases, especially ones caused by single-gene mutations. "It's still early days, but the hope is that gene therapy might someday replace drugs or surgery by fixing the problem at its root," CNBC says.

Kjellson notes that one issue for gene therapies could be their price, as costs could reach $1 million, which might lead insurance companies to balk at covering them.

Filed under