GENE THERAPY RARE DISEASES – Page 2 – REGENHEALTHSOLUTIONS (RHS)

PIER MARIA FORNASARI
2024-05-11

Fred Hutch scientist Dr. Adair partners with colleagues around the globe to make gene therapies more effective and more widely available

Fred Hutch Cancer Center scientist Jennifer E. Adair, PhD, is on a mission to foster worldwide collaboration on potentially curative gene therapies. Holder of the Fleischauer Family Endowed Chair in Gene Therapy Translation, Adair just co-authored two articles published today in Science Translational Medicine as part of a special series on global access to these therapies that she

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PIER MARIA FORNASARI
2024-05-07

Gene Therapy Treatment Increasing Body’s Signal for New Blood Vessel Growth Shows Promise

EXACT Trial Demonstrated Improvements in Exercise Duration, Ischemia, and Decreased Symptoms for Patients with Advanced CAD Final 12-month data from the EXACT trial demonstrates safety and efficacy results for a vascular endothelial growth factor (VEGF) gene therapy treatment for patients who have advanced coronary artery disease (CAD). The late-breaking results were presented today at

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PIER MARIA FORNASARI
2024-03-19

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

The U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Metachromatic leukodystrophy is a debilitating, rare genetic disease affecting the brain and nervous system. It is caused by a

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PIER MARIA FORNASARI
2024-01-24

Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at the Association for Research in Otolaryngology 2024 MidWinter Meeting

Hearing restoration was observed within 30 days of a single administration of AK-OTOF in the initial AK-OTOF-101 study participant, the first to receive gene therapy in the United States for a genetic form of hearing loss AK-OTOF is a gene therapy being developed for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF)

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PIER MARIA FORNASARI
2023-12-10

FDA Approves First CRISPR-based Gene Therapies to Treat Patients with Sickle Cell Disease

The U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an

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PIER MARIA FORNASARI
2023-10-24

FDA frees Verve CRISP-R base editing clinical trial for Heterozygous Familial Hypercholesterolemia

The trial, which is ongoing in the U.K. and New Zealand, has been on hold in the U.S. since late last year as the FDA sought more details from Verve. Verve Therapeutics plans to soon start testing a gene editing therapy for heart disease in the U.S. after federal regulators lifted an order that had

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PIER MARIA FORNASARI
2023-10-21

New Gene Therapy Trials to fight Children Deafness

Researchers are testing an approach that involves replacing a mutated gene in the inner ears of children with severe hearing loss. Two companies have launched clinical trials to see if they can restore hearing to children with a rare type of genetic deafness. Akouos and Decibel Therapeutics, both based in Boston, are testing experimental therapies in

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PIER MARIA FORNASARI
2023-10-19

Researchers Design Gene Therapy That Can Effectively Target Glioblastoma

Glioblastoma (GBM), an aggressive brain cancer, is notoriously resistant to treatment, with recurrent GBM associated with survival of less than 10 months. Immunotherapies, which mobilize the body’s immune defenses against a cancer, have not been effective for GBM, in part because the tumor’s surrounding environment is largely impenetrable to assaults from the body’s immune system.

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PIER MARIA FORNASARI
2023-01-23

Results on 5 gene therapy trials expected in the first 2023 semester

Can gene therapy trials for vision loss, hemophilia, and melanoma break through in 2023? fter another turbulent year in gene therapy development, all eyes are on five major trial readouts set for H1 2023. The clinical studies target rare blood disorders, inherited retinal diseases, and metastatic melanoma—each of which poses distinct trial design challenges. First, two small

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PIER MARIA FORNASARI
2022-12-29

Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate

Pfizer announced positive top-line results from the Phase 3 BENEGENE-2 study (NCT03861273) evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B. The BENEGENE-2 study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion

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REGENERATIVE AND PERSONALISED MEDICINE

DIGITAL HEALTHCARE

Category GENE THERAPY RARE DISEASES

Fred Hutch scientist Dr. Adair partners with colleagues around the globe to make gene therapies more effective and more widely available

Gene Therapy Treatment Increasing Body’s Signal for New Blood Vessel Growth Shows Promise

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at the Association for Research in Otolaryngology 2024 MidWinter Meeting

FDA Approves First CRISPR-based Gene Therapies to Treat Patients with Sickle Cell Disease

FDA frees Verve CRISP-R base editing clinical trial for Heterozygous Familial Hypercholesterolemia

New Gene Therapy Trials to fight Children Deafness

Researchers Design Gene Therapy That Can Effectively Target Glioblastoma

Results on 5 gene therapy trials expected in the first 2023 semester

Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate