Gene therapy may be 'one-shot stop' for rare bone disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
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Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
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Gene Therapy Shows Promise for Rare Bone Disease in Mice
Researchers have developed a gene therapy that may provide a long-term treatment for hypophosphatasia. Unlike current ...
Sangamo reports positive Fabry disease gene therapy results
RICHMOND, Calif. - Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company with a market capitalization of $263 million, has announced promising updated results from its Phase 1/2 STAAR ...
Sangamo reports updated data for gene therapy candidate ST-920
Sangamo Therapeutics (NASDAQ:SGMO) reported positive updated data from a Phase 1/2 study of its gene therapy candidate isaralgagene civaparvovec, or ST-920, in the treatment of Fabry disease. The ...
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New Base Editing Gene Therapy Cures its First Patient: A Sickle-Cell Sufferer Now in the ‘Gym Every Day’
Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.
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New gene therapy shows promise for lifelong treatment of hypophosphatasia
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.
Tenaya Therapeutics Receives $8 Million Clinical Grant from California Institute for Regenerative Medicine
Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of ...
Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease
FDA aligned on single-arm Phase 3 study to support potential accelerated and full approvalOn track to dose first patient in Phase 3 trial in ...
NHS England8d
Revolutionary gene-editing therapy for sickle cell ‘offers hope of a cure’ for NHS patients
NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as ...
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Gene Therapy: One-Shot Cure for Rare Bone Disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by ...