uniQure has outlined plans to move forward with a Biologics License Application for AMT-130, its investigational gene therapy for Huntington’s disease, under the FDA’s accelerated approval pathway. According to reports, the FDA has agreed that three-year Phase 1/2 data from AMT-130 can serve as the primary basis for the application. uniQure plans to submit the […]
Two new treatments across very different therapeutic areas and modalities have received regulatory approvals in the U.S. and UK. AstraZeneca’s Baxfendy (baxdrostat) has been approved by the U.S. FDA as a new treatment option for adults with uncontrolled hypertension, in combination with other antihypertensive medicines. Meanwhile, Krystal Biotech’s VYJUVEK (beremagene geperpavec-svdt) has been approved by […]
The U.S. FDA has approved Otarmeni (lunsotogene parvec-cwha), Regeneron Pharmaceuticals’ gene therapy for severe-to-profound sensorineural hearing loss caused by biallelic OTOF gene variants. Otarmeni is the first approved gene therapy for genetic hearing loss and the first dual-AAV vector-based gene therapy to reach approval. The decision is notable not only for rare disease innovation, but […]
On March 27, the U.S. FDA approved Rocket Pharmaceuticals’ gene therapy Kresladi for severe Leukocyte Adhesion Deficiency-I (LAD-I). Kresladi is the first approved gene therapy for LAD-I. The decision marks an important step forward for gene therapy in ultra-rare diseases, where both clinical value and execution capability remain critical. Key points First approved gene therapy […]
Monash University / Monash Health have announced that a patient with systemic lupus erythematosus (SLE) has achieved remission following treatment with CAR-T cell therapy.This represents the first clinical application of CAR-T therapy for an autoimmune disease in Australia, extending a modality long established in haematological oncology into a new therapeutic area. CAR-T cell therapy involves […]
The U.S. FDA has formally approved Novartis’ Itvisma (onasemnogene abeparvovec-brve) for use in children aged two and older, adolescents, and adults with spinal muscular atrophy (SMA).This represents the first gene-replacement therapy expanded to all eligible age groups, marking a significant advancement in the field of genetic medicine. Significance of the Approval in SMA Treatment SMA […]
The basic premise of search engine reputation management is to use the following three strategies to accomplish the goal of creating a completely positive first page of search engine results for a specific term…
The basic premise of search engine reputation management is to use the following three strategies to accomplish the goal of creating a completely positive first page of search engine results for a specific term…
The basic premise of search engine reputation management is to use the following three strategies to accomplish the goal of creating a completely positive first page of search engine results for a specific term…
The basic premise of search engine reputation management is to use the following three strategies to accomplish the goal of creating a completely positive first page of search engine results for a specific term…
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