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

Fierce Pharma

MARCH 15, 2024

Takeda will shut down a gene therapy manufacturing and R&D facility in Austria. The FDA questioned whether Carvykti should be approved in an earlier line of multiple myeloma after noticing an early detriment to patient survival in a clinical trial. . | WuXi AppTec left BIO amid alleged biosecurity concerns and as U.S.

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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

XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. The global incidence of DMD is approximately one in every 3,500 to 6,000 male births.

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FDA Approval Genetics Gene Therapy Gene 105

Drug Discovery World

MARCH 6, 2024

UK-based biotech SynaptixBio has secured a second Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA). This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination.

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Drugs Protein Gene Clinical Trials 52

XTalks

MARCH 28, 2024

Therefore, there is a need to provide better treatment options and increase public awareness of the possible therapies available for CRC. Targeted Therapies Targeted therapies have been developed to specifically attack cancer cells demonstrating certain characteristics and reduce side effects and not affect normal cells.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

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Gene Therapy Gene Drugs FDA Approval 111

XTalks

MARCH 1, 2024

While there is no cure for most types of tinnitus, treatments can help manage the condition, including sound therapy, counseling and hearing aids. Related: Interview with Neuromod COO Diarmuid Flavin About the FDA Approval of Tinnitus Treatment Lenire – Xtalks Life Science Podcast Ep.

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Gene Therapy Gene Life Science Genetics 52

Drug Discovery World

MARCH 19, 2024

Lenmeldy (atidarsagene autotemcel) has become the first Food and Drug Administration (FDA)-approved gene therapy for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

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Gene Therapy Gene FDA Approval Genetic Disease 52

FDA Law Blog

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). The primary basis for approval, like other drugs for DMD, was based on a single placebo-controlled randomized trial. By Charles G.

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FDA Approval Trials Genetics Drugs 59

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

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