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

By Hayden E. Klein

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

By Julia Bonavitacola

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

By Jared Kaltwasser

November 6th 2024

Screenshot of an interview with Adam Colborn, JD

Medicaid Aiming to Improve Patient Access to High-Cost Therapies

By Laura Joszt, MA

November 2nd 2024

Gene therapy research | Image credit: RFBSIP – stock.adobe.com

A Year of DMD Gene Therapy Trial Failures

By Hayden E. Klein

October 29th 2024

More News

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

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

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

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

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

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

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

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

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

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.

2 Commerce Drive
Suite 100
Cranbury, NJ 08512

© 2024 MJH Life Sciences^® and AJMC®.
All rights reserved.

Editorial Boards

Do Not Sell My Information

Terms & Conditions