Gene Therapy

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Genetic Hearing Loss Improves With DB-OTO Gene Therapy: CHORD Trial

By Rose McNulty

February 27th 2025

Ten children who received DB-OTO for profound genetic hearing loss due to variants of the otoferlin gene showed notable improvements in hearing, according to initial results of the CHORD trial.

In the INSPIRE DUCHENNE trial, interim 90-day biopsy data showed an average microdystrophin expression of 110% among the first 3 participants in the study. | Image credit: RFBSIP - stock.adobe.com

SGT-003 Gene Therapy Shows Potential in DMD

By Rose McNulty

February 26th 2025

The authors emphasized the importance of early diagnosis and treatment with CGTs, especially for patients with genetic conditions. | Image credit: RFBSIP - stock.adobe.com

Mitigating Barriers to Cell and Gene Therapy Access

By Rose McNulty

February 14th 2025

Duchenne muscular dystrophy | Image credit: hafakot - stock.adobe.com

EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD

By Rose McNulty

January 28th 2025

Intrathecal onasemnogene abeparvovec is an investigational SMA gene therapy that has been evaluated in the phase 1/2 STRONG study, the phase 3B STRENGTH study, and the STEER study. | Image credit: wladimir1804 - stock.adobe.com

Phase 3 STEER Study of SMA Gene Therapy Meets Primary End Point

By Rose McNulty

January 15th 2025

More News

Duchenne Muscular Dystrophy | Image credit: luchschenF - stock.adobe.com

February 5th 2024

Pre-, Postinfusion Protocol Recommendations for Delandistrogene Moxeparvovec Use in DMD

The gene therapy delandistrogene moxeparvovec-rokl, also known as Elevidys (Sarepta Therapeutics) is indicated to treat Duchenne muscular dystrophy (DMD) in ambulatory patients aged 4 to 5 years, and Sarepta recently filed supplemental data with the FDA seeking to expand the labeled indication.

January 31st 2024

What We’re Reading: Sickle Cell Treatment Access; Patent Thicket Attacks; US Syphilis Cases Rise

Sickle cell disease (SCD) will be the first focus of the Cell and Gene Therapy (CGT) Access Model; the FTC has challenged the validity of over 100 drug product patents to help increase competition and potentially lower prices; the rate of infectious syphilis cases in the US rose 9% in 2022.

January 24th 2024

What We’re Reading: Phase 3 Gene Therapy Trial; COVID Infection Surge; Patients Maintain Anti-Obesity Drug Weight Loss

Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial to test its gene therapy candidate SRP-9003 in children with limb-girdle muscular dystrophy type 2E; wastewater testing points to a new COVID-19 infection wave fueled by the JN.1 variant; most patients on anti-obesity medications kept at least some weight off up to a year after they stopped taking them.

DNA molecule and dollar sign | Image Credit: Sergey Nivens-stock.adobe.com

January 22nd 2024

Analysis Explores Gene Therapy’s Potential to Be Cost-Effective in SCD

This new economic valuation of the societal and health care benefits of gene therapy to treat sickle cell disease (SCD) concludes the highly personalized treatment has potential to be cost effective if priced below $2 million.

Card saying hemophilia B

January 16th 2024

Pfizer’s Hemophilia B Gene Therapy Approved as Beqvez in Canada

Victoria Johnson

The FDA and the European Medicines Agency have accepted marketing authorization applications and decisions are pending.

Updated Data for Odronextamab Show Durable Responses in R/R DLBCL, 73% Complete Response in R/R Follicular Lymphoma

January 8th 2024

Updated Data for Odronextamab Show Durable Responses in R/R DLBCL, 73% Complete Response in R/R Follicular Lymphoma

Sarepta Therapeutics Inc | Image Credit: Timon - stock.adobe.com

January 5th 2024

Sarepta Therapeutics Seeks to Expand Delandistrogene Moxeparvovec Indications

Delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) is currently approved to treat Duchenne muscular dystrophy in 4- and 5-year-old patients who have a confirmed DMD gene mutation.

child in wheelchair and parent | Image credit: Art_Photo - stock.adobe.com

December 29th 2023

Descriptions of Physical Limitations, Symptoms of DMD From the Patient, Caregiver Perspective

Skylar Jeremias

A recent study encapsulates how patients and caregivers characterize the impact of Duchenne muscular dystrophy (DMD) on patients’ physical limitations and symptom burden, potentially helping to inform patient-centered strategies for assessing clinical outcomes in DMD research.

3d rendered HIV Virus in Blood Stream in color background | Image Credit: RAJCREATIONZS - stock.adobe.com

December 23rd 2023

Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy

Victoria Johnson

Excision BioTherapeutics has so far released positive safety data from the first 3 participants living with HIV-1, with no evidence of vector shedding in sexual organ tissue.

ICYMI: AMCP Nexus Conference Highlights

December 19th 2023

ICYMI: AMCP Nexus Conference Highlights

From the 2023 Academy of Managed Care Pharmacy (AMCP) Nexus meeting, held in October, expert interviews and insightful updates on the Inflation Reduction Act, gene therapy affordability and access, and the 340B Drug Pricing Program comprise the conference highlights.

Medical team meeting to discuss research | Image credit: mrmohock - stock.adobe.com

December 15th 2023

Gene Therapy for Mucopolysaccharidoses Type IIIB Requires More Testing, Research

Also known as Sanfilippo syndrome, this rare genetic syndrome has no known cures and what current treatment options are available are expensive and limited in supply.

FDA approval stamp in red

December 10th 2023

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

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

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

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

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

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

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

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

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

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).

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

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

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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