Gene Therapy

Latest News

Researcher working with microplate in the laboratory | Image credit: angellodeco – stock.adobe.com
Sarepta Halts Development, Testing of DMD Therapy SRP-5051

November 14th 2024

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with the developer citing safety concerns, FDA feedback, and an evolving therapeutic landscape.

FDA Approved
FDA Approves Gene Therapy to Treat AADC Deficiency

November 14th 2024

Fidanacogene elaparvovec is a promising gene therapy for patients with hemophilia B | image credit: ipopba - stock.adobe.com
Fidanacogene Elaparvovec Shows Strong Results in Open-Label Phase 3 Study

November 6th 2024

Screenshot of an interview with Adam Colborn, JD
Medicaid Aiming to Improve Patient Access to High-Cost Therapies

November 2nd 2024

Gene therapy research | Image credit: RFBSIP – stock.adobe.com
A Year of DMD Gene Therapy Trial Failures

October 29th 2024

More News

Colored Genetic Code DNA Molecule Structure | Image credit: ktsdesign - stock.adobe.com
November 2nd 2023

Despite Risk, Patients With SCD Hopeful About Gene Therapies

Maggie L. Shaw
Sickle cell disease (SCD) has known complications that include organ damage and failure, rhabdomyolysis, glaucoma, splenic sequestration, and vaso-occlusive crises. At present, the only potential cure is a bone marrow transplant, but it can be difficult to find a donor and there is a high rejection risk.
DNA concept art | Image credit: nobeastsofierce - stock.adobe.com
October 25th 2023

Success of Gene Therapy Relies on Integration Location in DNA

Laura Joszt, MA
The location of integration of a gene therapy has been crucial for the safety and efficacy of the treatment to cure infants with X-linked severe combined immunodeficiency.
Duchenne medicial concept | Image Credit: hafakot - stock.adobe.com
October 19th 2023

Case Report of Patient Death Following High-Dose Gene Therapy for DMD

Maggie L. Shaw
In this case study, a patient who had Duchenne muscular dystrophy (DMD) and received high-dose transgene therapy to upregulate cortical dystrophin subsequently developed acute respiratory distress syndrome and died.
Genetic engineering and gene manipulation concept | Image Credit: andrianocz - stock.adobe.com
October 13th 2023

Taysha Doses Second Patient With Rett Syndrome With TSHA-102, per IDMC Recommendation

Noah Stansfield
Both the second and first patients dosed in the study received the lower of the 2 doses of the gene therapy that Taysha Gene Therapies is evaluating in the phase 1/2 REVEAL trial.
Sarah Boyce, president and CEO, Avidity Biosciences | Image Credit: Avidity Biosciences
October 9th 2023

Avidity Biosciences Gains FDA Orphan Drug Designation for Patients With DMD

Pearl Steinzor
Sarah Boyce, president and CEO at Avidity Biosciences, discusses her leading role at the company, as well as antibody oligonucleotide conjugate drug, AOC 1044, currently in development for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping (DMD44).
FDA logo | Image credit: Araki Illustrations - stock.adobe.com
October 7th 2023

New FDA Pilot Program Will Provide Hands-On Regulatory Guidance for Cell and Gene Therapy Trials in Rare Disease

Noah Stansfield
The program will initially allow 6 sponsors of clinical trials to participate and will provide sponsors with the opportunity for frequent advice and regular communication regarding clinical trial design and other development issues.
Male hands showing protection and protection for family security, finances and health ,Insurance management planning ,property and family risk reduction ,Caring and caring for loved ones: © chaylek - stock.adobe.com
June 28th 2023

Researchers Highlight Restrictive Coverage of Gene, Cell Therapies

Jaime Rosenberg
The researchers focused on coverage trends across an array of disease states, including lymphoma, vision loss, and spinal muscular atrophy, finding discrepancies in coverage across 16 states and Medicaid Care Organizations.
FDA Approves Delandistrogene Moxeparvovec, First Gene Therapy to Treat Duchenne Muscular Dystrophy
June 22nd 2023

FDA Approves Delandistrogene Moxeparvovec, First Gene Therapy to Treat Duchenne Muscular Dystrophy

Mary Caffrey
The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.
DNA strand
May 12th 2022

A Look at the Current, Future State of Cell and Gene Therapies in the United States

Gianna Melillo
Experts evaluated the future of cell and gene therapies in the United States and offered insights on potential value-based payment models for these treatments.
Gene Therapies Present Reimbursement Challenges That Have Yet to Be Answered
May 19th 2021

Gene Therapies Present Reimbursement Challenges That Have Yet to Be Answered

Laura Joszt, MA
With more than 3000 gene therapies in development, payers will have to grapple with the challenges of paying for these innovative but expensive therapies.
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