Gene Therapy

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Adults and adolescents with sickle cell disease experienced early, sustained, and clinically meaningful improvements in HRQOL measures following exa-cel infusion. | Image credit: Ezume Images - stock.adobe.com
Gene Therapy Meaningfully Improves Quality of Life in Severe Sickle Cell Disease

August 29th 2025

Patients with severe sickle cell disease experienced significant quality of life improvements after receiving exagamglogene autotemcel gene therapy.

While the findings suggest that switching to onasemnogene abeparvovec after non–gene therapy treatments could be beneficial to patients with spinal muscular atrophy, it is not clear which patients may benefit the most from switching therapies. | Image credit: Rochu_2008 - stock.adobe.com
Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

August 15th 2025

Gene therapy appears to be a promising approach for a subset of genetic deafness. | Image credit: NathanDeverycom - stock.adobe.com
Progress and Challenges With Gene Therapy in Hereditary Hearing Loss

July 31st 2025

The combination of novel vectors with tissue- and cell-specific regulatory elements to construct a toolbox for targeted delivery is a promising strategy. | Image credit: Dr_Microbe - stock.adobe.com
The State and Future of Viral, Nonviral Vectors for Gene Therapy

July 25th 2025

OneOncology Appoints Michael Byrne, DO, as Medical Director for Cellular Therapy
OneOncology Appoints Michael Byrne, DO, as Medical Director for Cellular Therapy

July 22nd 2025

More News

FDA approval stamp in red
December 10th 2023

FDA Approves bluebird bio's Lovo-Cel Gene Therapy for Sickle Cell Disease

Matt Hoffman
The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185). It was the second approval from FDA for a gene therapy for sickle cell disease.
Female hands show the brain | Image Credit: natali_mis - stock.adobe.com
December 3rd 2023

NK Cell Therapy SNK01 Improves Cognitive Function in Alzheimer Disease for up to 11 Weeks

Noah Stansfield
Among 10 patients evaluated, compared with their baseline at 1 week after receiving their final dose in the trial, 30% demonstrated clinical improvement on the Alzheimer’s disease composite score.
Genetic engineering and gene manipulation concept | Image Credit: andrianocz - stock.adobe.com
November 27th 2023

Gene Therapy Success in UK Likely Depends on Overcoming Education, Psychological Support Challenges

Maggie L. Shaw
Education, psychological support, and implementation guidance are the top unmet needs identified by investigators from the United Kingdom concerning gene therapy use for hemophilia.
Duchenne medicial concept | Image Credit: hafakot - stock.adobe.com
November 22nd 2023

EMBARK Data Support Delandistrogene Moxeparvovec for DMD Across All Study End Points

Marco Meglio
Over a 52-week treatment period, the gene therapy demonstrated robust evidence for a clinically meaningful benefit, including across several prespecified functional secondary end points vs placebo.
DNA molecules | Image Credit: vitstudio - stock.adobe.com
November 17th 2023

Data on Delandistrogene Moxeparvovec Can Inform Future Gene Therapies

Maggie L. Shaw
The FDA approved delandistrogene moxeparvovec-rokl (Elevidys, Sarepta Therapeutics) in June to treat Duchenne muscular dystrophy in ambulatory pediatric patients aged 4 through 5 who have a confirmed DMD gene mutation.
Insurance management planning and risk reduction | Image Credit: chaylek - stock.adobe.com
November 9th 2023

High-Cost Gene Therapies Present Reimbursement, Access Challenges

Maggie L. Shaw
Investigators propose potential payment models for gene therapies that consider equitable patient access and payer reimbursement.
Gene therapy improves auditory response for child with profound genetic hearing loss | Image Credit: Marija - stock.adobe.com.
November 4th 2023

Gene Therapy Improves Auditory Response for Child With Profound Genetic Hearing Loss

Joshua Fitch
A child with profound genetic hearing loss was dosed with the investigational otoferlin gene therapy (DB-OTO), becoming the first patient dosed in a phase 1/2 trial evaluating the treatment, and experienced improved auditory response at week 6 with no concerning safety signals.
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.
Screenshot of Michelle Booth, PharmD, and Andy Killpack, PharmD, side by side
April 10th 2023

Payers, Providers Still Navigating Uncharted Waters for Gene Therapies

Laura Joszt, MA Peter Wehrwein
Many gene therapies promise life-changing effects, but without long-term data it remains to be seen how long the benefits last.
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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