By Denny Jacob
American depositary receipts of Freeline Therapeutics Holdings rose 14% to $4.29 following positive data from an ongoing trial evaluating FLT201, its adeno-associated virus gene therapy candidate.
The stock is down 40% on the year.
The clinical-stage biotechnology company said data from the first two patients in its trial showed that a single infusion of the gene therapy candidate led to increases in GCase activity in plasma and normalization of GCase activity in leukocytes in addition to the favorable safety and tolerability profile.
Freeline Chief Medical Officer Pamela Foulds said the company’s belief in FLT201’s therapeutic potential was strengthened following the data. She also disclosed that the company has decided to treat a third patient at the same levels rather than a higher dose as initially planned given the response and clean safety and tolerability to date.
FLT201 is a gene therapy candidate for Gaucher disease, a genetic disorder in which a deficiency of the GCase enzyme leads to a buildup of harmful substrates causing symptoms including an enlarged spleen and liver.
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