Pharmaceutical Technology

MARCH 19, 2024

The US FDA has granted approval to Orchard Therapeutics' Lenmeldy for specific forms of metachromatic leukodystrophy (MLD).

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Gene Therapy FDA Approval Gene 246

Fierce Pharma

MARCH 18, 2024

Kyowa Kirin’s bet on Orchard Therapeutics has paid off, furnishing the Japanese drugmaker with a newly approved gene therapy in the U.S. | The FDA on Monday gave a thumbs up to Lenmeldy as the first gene therapy in the U.S. for kids with certain types of metachromatic leukodystrophy (MLD).

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Gene Therapy Gene FDA Approval 111

Bio Pharma Dive

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

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Gene Therapy Gene FDA Approval Development 169

BioSpace

MARCH 18, 2024

Orchard Therapeutics on Monday secured the FDA’s first approval for an autologous gene therapy to treat the rare metabolic disease metachromatic leukodystrophy in children.

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Gene Therapy Gene FDA Approval 118

BioSpace

MARCH 19, 2024

Two days after winning FDA approval, Orchard Therapeutics on Wednesday provided its U.S. launch plans for metachromatic leukodystrophy gene therapy Lenmeldy, which has a wholesale acquisition cost of $4.25 million for the one-time treatment.

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Gene Therapy Gene FDA Approval 130

BioPharma Reporter

MARCH 19, 2024

Food and Drug Administration has cleared AGC Biologicsâ Milan site to begin manufacturing of Orchard Therapeuticsâ Lenmeldy (atidarsagene autotemcel), a gene therapy for early-onset metachromatic leukodystrophy (MLD).

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Gene Therapy Manufacturing Gene FDA Approval 59

Drug Discovery World

MARCH 22, 2024

This has been an interesting week for cell and gene therapies, with two landmark FDA approvals, two significant fundraising efforts and potentially ground-breaking study results in glioblastoma. The post This week in drug discovery (18-22 March) appeared first on Drug Discovery World (DDW).

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Gene Therapy In-Vivo FDA Approval Drugs 52