Spark's Luxturna Approval Ushers In A New Gene Therapy Era
Spark's gene therapy for patients with an inherited form of blindness will be available in the US late in the first quarter, but on the big question – whether it will cost $1m for the one-time treatment – Spark would not say.
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A bluebird executive spoke during ARM’s Cell and Gene Meeting On The Mesa about why the firm pulled Zynteglo out of the EU, but while progress has been made in the US, long-term challenges remain.
AGTC hopes to gain the first approval in X-linked retinitis pigmentosa for its gene therapy AGTC-501, now in a Phase II/III study. It also is advancing development in achromatopsia, another unmet medical need.
BIO panel with Roche’s James Sabry, Spark’s Federico Mingozzi and Adaptive’s Harlan Robins imagines a world where cell and gene therapies are preferred over small molecules and antibodies.