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Gene Therapy

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May 15, 2024
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Researchers hope one-time gene therapy ‘amounts to a cure’ for hemophilia B

Researchers hope one-time gene therapy ‘amounts to a cure’ for hemophilia B

The FDA’s decision to approve a one-time gene therapy for adults with moderate to severe hemophilia B could have a considerable impact on the treatment landscape, according to a researcher involved in the pivotal trial for the agent.

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May 06, 2024
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FDA grants regenerative medicine advanced therapy designation to Rett syndrome treatment

FDA grants regenerative medicine advanced therapy designation to Rett syndrome treatment

The FDA has granted regenerative medicine advanced therapy designation to an intrathecally delivered AAV9 gene transfer therapy to treat younger individuals with Rett syndrome, according to the manufacturer.

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May 02, 2024
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Gene therapy improves symptoms for patients with ‘no-option’ treatment-resistant angina

Gene therapy improves symptoms for patients with ‘no-option’ treatment-resistant angina

Gene therapy improved exercise capacity and blood flow and reduced chest pain occurrence and nitroglycerine use for patients with advanced ischemic heart disease, a speaker reported.

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April 26, 2024
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The American Association for the Advancement of Science elects Lieberman as 2024 fellow

The American Association for the Advancement of Science elects Lieberman as 2024 fellow

The American Association for the Advancement of Science elected orthopedic surgeon Jay R. Lieberman, MD, as a 2024 fellow for his research in gene therapy and joint replacement, according to a press release.

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April 26, 2024
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Ocugen doses second cohort in trial of OCU410 for geographic atrophy

Ocugen doses second cohort in trial of OCU410 for geographic atrophy

Ocugen has announced that dosing is complete in the second cohort of its phase 1/2 ArMaDa clinical trial to assess the safety of OCU410, a modifier gene therapy candidate for geographic atrophy.

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April 26, 2024
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FDA approves one-time gene therapy Beqvez for hemophilia B

FDA approves one-time gene therapy Beqvez for hemophilia B

The FDA approved fidanacogene elaparvovec-dzkt for treatment of adults with moderate to severe hemophilia B.

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April 25, 2024
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Exa-cel a ‘first step’ in gene therapy for sickle cell disease, beta-thalassemia

Exa-cel a ‘first step’ in gene therapy for sickle cell disease, beta-thalassemia

Exagamglogene autotemcel gave most patients with beta-thalassemia transfusion independence and drastically reduced the number of vaso-occlusive crises in those with sickle cell disease in a pair of phase 3 trials.

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April 18, 2024
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Gene therapy to treat congestive heart failure gains FDA fast track designation

Gene therapy to treat congestive heart failure gains FDA fast track designation

Bayer AG and its subsidiary Asklepios BioPharmaceutical announced the FDA granted fast track designation for its investigational gene therapy for the treatment of congestive heart failure.

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April 18, 2024
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VIDEO: AMD research should focus on regenerative medicine to restore vision

VIDEO: AMD research should focus on regenerative medicine to restore vision

In a video interview, Majda Hadziahmetovic, MD, retinal specialist and assistant professor of ophthalmology at Duke University discusses improving early detection and risk prediction for AMD patients.

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April 18, 2024
1 min watch
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VIDEO: Collaboration needed to care for patients with AMD

VIDEO: Collaboration needed to care for patients with AMD

In a video interview, Majda Hadziahmetovic, MD, retinal specialist and assistant professor of ophthalmology at Duke University discusses the necessary collaboration between ophthalmologists and optometrists on AMD care and management.

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