Gene Therapy

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While patients should be closely monitored in the first 90 days post-treatment, delandistrogene moxeparvovec showed a manageable, consistent safety profile in a broad population of patients. | Image Credit: Hypnosis - stock.adobe.com
Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data

May 16th 2025

Delandistrogene moxeparvovec demonstrated a manageable safety profile across clinical trials for Duchenne muscular dystrophy (DMD), with most adverse events emerging within 90 days of infusion.

The new report highlights several barriers to CGT use, including cumbersome referral processes, high price tags, complex logistics, and uncertainties around reimbursement. | Image Credit: Treecha - stock.adobe.com
CGTs Have Potential to Transform Care, but Challenges Persist

May 14th 2025

Quantitative magnetic resonance imaging seemed to show that delandistrogene moxeparvovec protects muscle from progressive damage in DMD. | Image credit: OlegKachura - stock.adobe.com
Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression

May 14th 2025

gene therapy | Image Credit: © RFBSIP - stock.adobe.com
Expanding Broader Patient Access to CGTs

April 30th 2025

At 6 months, 81% of wounds showed 50% or more healing vs 16% of 43 matched control wounds treated with standard of care in the VIITAL study. | Image credit: wladimir1804 - stock.adobe.com
FDA Approves Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa

April 29th 2025

More News

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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