Gene Therapy

Latest News

The first gene therapies for sickle cell disease were FDA approved in 2023, but high upfront costs have been barriers to patient access to these life-changing treatments. | Image credit: RFBSIP - stock.adobe.com
CGT Access Model Aims to Expand Access to Sickle Cell Treatments

July 18th 2025

States participating in the Cell and Gene Therapy (CGT) Access Model will be testing outcomes-based payments for sickle cell disease treatments.

SRP-9003 is currently being evaluated in a phase 1/2 clinical trial and the phase 3 EMERGENE clinical trial. | Image credit: vladimircaribb - stock.adobe.com
FDA Grants Platform Technology Designation to Limb-Girdle Muscular Dystrophy Gene Therapy Vector

July 1st 2025

An analysis of outcomes from part 2 of the EMBARK trial included 14 patients who had received a placebo in part 1 of the study and were aged 8 to 9 years at crossover. | Image Credit: RFBSIP - stock.adobe.com
DMD Gene Therapy Shows Clinical Benefits in 8- and 9-Year-Old Patients

June 13th 2025

Participants who received RGX-202 demonstrated functional improvement and better outcomes than controls for all measures. | Image Credit: Treecha- stock.adobe.com
RGX-202 Gene Therapy Improves DMD Trajectory, Biomarkers

June 12th 2025

While significant progress has been made, innovations in vector design, immune modulation, and delivery strategies are needed to further improve gene therapy for DMD. | Image credit: RFBSIP - stock.adobe.com
Hurdles Remain Despite Progress in Gene Therapy for DMD

May 30th 2025

More News

3D rendering of a CAR T cell | Image Credit: Naeblys-stock.adobe.com
May 9th 2024

New Strategy Shows Promise for CD7-Positive Leukemia/Lymphoma

Kimberly Rath, PharmD
This study introduces a novel "all-in-one" strategy combining CD7 chimeric antigen receptor T-cell therapy with haploidentical hematopoietic stem-cell transplantation, showing significant potential to improve survival and reduce toxicities in patients with relapsed or refractory CD7-positive hematologic cancers.
CRISPR rendering | Image credit: © Jacqueline Weber - stock.adobe.com
May 2nd 2024

Phase 3 Data Show Exa-Cel Prevents Vaso-Occlusive Crises for At Least 1 Year in SCD

AJMC Contributor
Data from the study showed that exa-cel prevented vaso-occlusive crises in all but 1 of the 30 evaluable patients for at least 12 months.
CAR T | Image Credit: DesignCells - stock.adobe.com
April 22nd 2024

AE Analysis Finds Low Rate of T-Cell Malignant Neoplasms in CAR T Recipients

Jared Kaltwasser
Only 11 cases of adverse events (AEs) were reported over a 6-year time frame, the investigators found, following administration of chimeric antigen receptor T-cell therapy (CAR T).
CAR T cell AI-generated image - AIGen - stock.adobe.com.jpg
April 22nd 2024

Review Finds Anti-BCMA CAR T-Cell Therapy Effective in Patients With MM

Jared Kaltwasser
The analysis also found the therapy has a manageable safety profile.
Genetic and gene manipulation concept | Image Credit: © ipopba - stock.adobe.com
April 18th 2024

Gene, Light Therapy Combo Shows Promise Against Prostate Cancer Cells in Proof-of-Concept Study

AJMC Contributor
In their preclinical model, the researchers found efficacy both in vitro and in vivo by using CRISPR-Cas9 to mimic porphyria and combining the technology with light therapy.
Older woman's eye | Image credit: Syda Productions - stock.adobe.com
April 13th 2024

Gene Therapy Well Tolerated in Wet AMD, Shows Promise in Visual Acuity

Laura Joszt, MA
A single injection of a gene therapy was well tolerated and showed the potential to control exudation in patients with neovascular age-related macular degeneration (wet AMD).
FDA Approved Products or Drugs | Image Credit:Olivier Le Moal - stock.adobe.com
March 19th 2024

FDA Approves Arsa-Cel for Metachromatic Leukodystrophy

Maggie L. Shaw
The approval makes atidarsagene autotemcel (arsa-cel [Lenmeldy]; Orchard Therapeutics) the first approved treatment for metachromatic leukodystrophy, a disease caused by a mutation in the ARSA gene and marked by progressive declines in both the central and peripheral nervous systems.
Benjamin N. Rome, MD, MPH | Image Credit: Brigham and Women's Hospital
March 15th 2024

Dr Ben Rome Scrutinizes the FDA’s Accelerated Approval Pathway for DMD Treatments, Calls for Policy Reform

Maggie L. Shaw
In a recent issue of JAMA, a team led by Benjamin N. Rome, MD, MPH, examined how the FDA’s accelerated approval process has moved 5 genetically targeted treatments for Duchenne muscular dystrophy (DMD) through its pipeline despite limited evidence on their efficacy.
DNA molecule and dollar sign | Image credit: Sergey Nivens - stock.adobe.com
March 14th 2024

Experts Issue Call for Reform to DMD Drug Pricing, Trial Approval Process

Maggie L. Shaw
Since 2016, 5 targeted treatments have received accelerated approval from the FDA for use in Duchenne muscular dystrophy (DMD), and the total spend for just 3 of them is $3.1 billion.
CAR T-cell therapy | Image Credit: Deisgn Cells - stock.adobe.com
March 7th 2024

Addressing CAR T Challenges: Enhancing Collaboration for Patient Care

Maggie L. Shaw
Knowledge gaps persist in awareness of and education on the use of chimeric antigen receptor (CAR) T-cell therapies for hematologic malignancies, especially multiple myeloma and lymphoma.
genetically engineered CAR T | Image credit: christoph burgstedt - stock.adobe.com
February 29th 2024

Optimizing CAR T for Patients With BCL and CNS Involvement

Maggie L. Shaw
With little data available on outcomes among patients who have B-cell lymphoma (BCL) and secondary central nervous system (CNS) involvement who were administered CD19-targeting chimeric antigen receptor (CAR) T-cell therapy, these investigators enrolled 4 male patients in their study.
Molecular engineering of DNA for Gene therapy | Image credit: immimagery-stock.adobe.com
February 22nd 2024

Identifying Patients at High Risk of Myeloid Neoplasms After CAR T: Implications for Care, Surveillance

Maggie L. Shaw
For chimeric antigen receptor T-cell therapies (CAR T) approved to treat B-cell lymphoproliferative disorders and multiple myeloma, more data are needed on the effects of posttreatment myeloid neoplasms to optimize patient counseling, risk stratification, and surveillance.
When Choosing Between Bispecifics and CAR T, the Best Therapy May Be What Is Accessible
February 15th 2024

When Choosing Between Bispecifics and CAR T, the Best Therapy May Be What Is Accessible

Mary Caffrey
Coverage from the session, "Which Comes First? Bispecifics, CAR T-Cell Therapy, and Cost Consideration in Sequencing,” at Patient-Centered Oncology Care 2023.
Scientist analyzing gene transfer | Image credit: Bojan-stock.adobe.com
February 15th 2024

In Adolescents With Obesity, FMT Shows No Negative Impact on Horizontal Gene Transfer

Maggie L. Shaw
For this analysis, investigators used metagenomic sequencing files from the Gut Bugs trial, a placebo-controlled multidonor fecal microbiota transplantation (FMT) trial conducted among adolescents classified as obese.
Duchenne Muscular Dystrophy | Image credit: luchschenF - stock.adobe.com
February 5th 2024

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

Maggie L. Shaw
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.
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January 31st 2024

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

AJMC Staff
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.
what we're reading logo
January 24th 2024

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

AJMC Staff
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

Maggie L. Shaw
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

Mary Caffrey
Sarepta Therapeutics Inc | Image Credit: Timon - stock.adobe.com
January 5th 2024

Sarepta Therapeutics Seeks to Expand Delandistrogene Moxeparvovec Indications

Maggie L. Shaw
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

Maggie L. Shaw
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

Maggie L. Shaw
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

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.
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