Tag - ORTX

Markets

Stifel starts coverage on 10 rare disease-focused companies

Stifel began coverage on 10 rare disease-focused companies, with seven “buy” and three “hold” ratings, noting that with an estimated 7,000-plus rare diseases – 95% of which lack approved therapy – these are early...

March 1, 2021

Developments

FDA clears Orchard Therapeutics IND for OTL-200

The FDA cleared Orchard Therapeutics’ (NASDAQ:ORTX) IND for OTL-200, a stem cell-based gene therapy for the treatment of metachromatic leukodystrophy (MLD). MLD is caused by a mutation in the arylsulfatase-A gene, which...

November 23, 2020

Developments

Orchard Therapeutics gets EMA accelerated assessment for OTL-200

The EMA granted Orchard Therapeutics’ (NASDAQ:ORTX) OTL-200 accelerated assessment for the treatment of metachromatic leukodystrophy (MLD). MLD is caused by a mutation in the arylsulfatase-A gene, which results in the...

November 18, 2019

Developments

Orchard Therapeutics gets FDA RMAT designation for Wiskott-Aldrich Syndrome drug

Orchard Therapeutics’ (NASDAQ:ORTX) OTL-103 received FDA regenerative medicine advanced therapy (RMAT) designation for the treatment of Wiskott-Aldrich Syndrome (WAS). WAS is an inherited immune disorder where...

July 31, 2019

Developments

Orchard Therapeutics doses first patient in cryopreserved gene therapy trial

Orchard Therapeutics (NASDAQ:ORTX) has dosed the first patient in a study evaluating a cryopreserved formulation of OTL-103, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of Wiskott...

April 25, 2019

Tag - ORTX

Stifel starts coverage on 10 rare disease-focused companies

FDA clears Orchard Therapeutics IND for OTL-200

Orchard Therapeutics gets EMA accelerated assessment for OTL-200

Orchard Therapeutics gets FDA RMAT designation for Wiskott-Aldrich Syndrome drug

Orchard Therapeutics doses first patient in cryopreserved gene therapy trial

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