Gene Therapy

Latest News

Real-world data suggest delandistrogene moxeparvovec is well-tolerated and effective in patients with DMD. | Image credit: natali_mis - stock.adobe.com
Real-World Study Suggests DMD Gene Therapy Is Safe, Effective

March 26th 2025

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort.

Delandistrogene moxeparvovec is an adeno-associated virus vector-based gene therapy approved for the treatment of DMD. Image credit: natali_mis - stock.adobe.com
Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD

March 21st 2025

Just 4 of the 20 cell and gene therapy trials in the study submitted the same trial evidence to both the FDA and EMA. | Image credit: Treecha - stock.adobe.com
Uniform Global Standards, Processes Could Accelerate CGT Approvals, Access

March 13th 2025

human-dna-strand-double-helix-macro-generative-ai_634358-316.jpg
Genetic Hearing Loss Improves With DB-OTO Gene Therapy: CHORD Trial

February 27th 2025

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

February 26th 2025

More News

Gene therapy trials | Image Credit: ArtemisDiana-stock.adobe.com
August 23rd 2024

An Updated Snapshot of Gene Therapy Clinical Trials Worldwide

Gayle Turim Dickstein
The gene therapy landscape has changed dramatically in the past 5 years, and investigators wanted to capture and analyze the details of current and upcoming trials, along with their potential impact on patients.
CAR T interaction | Image Credit: Alpha Tauri 3D-stock.adobe.com
August 16th 2024

Novel CAR-E Platform Enhances CAR T-Cell Functionality in Cancer Treatment

Kimberly Rath, PharmD
Researchers introduce a chimeric antigen receptor–enhancer (CAR-E) therapeutic platform that significantly extends the effectiveness and memory of CAR T cells, potentially preventing relapse in patients who have cancer.
expensive medicine | Image Credit: jadamprostore-stock.adobe.com
August 9th 2024

High Costs of Sickle Cell Therapies Highlight Paradox of Drug Development

Jared Kaltwasser
Experts argue for new mechanisms to fund research in oft-ignored rare diseases.
epidermolysis bullosa | Image Credit: Yurii Kibalnik-stock.adobe.com
August 2nd 2024

CRISPR/Cas9 Gene Therapy Holds Promise to Treat Epidermolysis Bullosa

Karen Jacobson-Sive
An exon-skipping technique using dual single-guide RNA/Cas9 ribonucleoproteins targeted at 3 novel COL7A1 exons with pathogenic heterozygous mutations achieved exon deletion rates of up to 95%.
Artist rendering of a gene being edited | Image credit: Degimages - stock.adobe.com
July 27th 2024

Progress in Precise Gene Expression Control With CRISPR-Cas12f

Kimberly Rath, PharmD
An innovative CRISPR-Cas12f-based activator system may hold significant potential in the future of gene therapy and biological research, suggest researchers of a new study.
Red blood drop with double helix | Image credit: Елена Бутусова-stock.adobe.com
July 26th 2024

Hemophilia A Gene Therapy Superior to Standard of Care in Phase 3 Trial

Laura Joszt, MA
The gene therapy giroctocogene fitelparvovec demonstrated superiority compared with the standard of care, routine prophylaxis, in hemophilia A treatment.
symptoms of epidermolysis bullosa | Image Credit: ангелина ковальчук-stock.adobe.com
July 22nd 2024

Key Considerations for Optimizing B-VEC in Dystrophic Epidermolysis Bullosa

AJMC Contributor
Beremagene geperpavec-svdt (B-VEC) entered the market in 2023 as the first approved corrective treatment for dystrophic epidermolysis bullosa (DEB), a rare genetic disease affecting the skin and nails that is caused by mutations in the COL7A1 gene.
Medicine doctor hold icon health and electronic medical record on interface. Digital healthcare and network connection on hologram virtual screen, insurance. medical technology and network concept - ipopba - stock.adobe.com.jpeg
July 21st 2024

Understanding Coverage, Financing, and Future Trends of Gene Therapy in Employer Health Plans

Giuliana Grossi
The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as the challenges brought by manufacturing complexities and the need to address accessibility to treatment.
cardiac genetics | Image Credit: bluebay2014-stock.adobe.com
July 16th 2024

Lexeo Shares Positive Interim Data for FA Cardiomyopathy Gene Therapy

Jared Kaltwasser
The therapy led to increased frataxin levels and decreased left ventricular mass in patients with Friedreich ataxia (FA) cardiomyopathy, the authors said.
Female reproductive system | Image Credit: mi_viri-stock.adobe.com
July 15th 2024

GPR176 May Be Potential Gene Therapy Target in Ovarian Cancer

AJMC Contributor
The biomarker, G-protein-coupled receptor 176 (GPR176), may be a driver in the progression of ovarian cancer and potential target for gene therapy, suggested researchers of a new study.
Hearing test for children | Image Credit: Microgen-stock.adobe.com
July 8th 2024

Five Children, 2 Hearing-Improved Ears Each: Successful DFNB9 Gene Therapy

Gayle Turim Dickstein
The interim results of a single-arm trial in children with autosomal recessive deafness 9 (DFNB9) show the benefits of binaural administration of gene therapy: better hearing with no serious adverse effects.
DNA research | Image Credit: vladimircaribb-stock.adobe.com
July 1st 2024

AAV Therapy: A Successful 1-Patient SPG50 Gene Therapy Trial

Gayle Turim Dickstein
An adeno-associated virus (AAV) gene therapy for hereditary spastic paraplegia type 50 (SPG50)—funded by a young patient’s family—gives hope not only to a 4-year-old, but to researchers working on similar projects for other rare diseases.
Gene editing shows promise in Duchenne muscular dystrophy. | Image credit: Degimages - stock.adobe.com
June 29th 2024

Stem Cell–Based Gene Editing Strategy Shows Promise in DMD

Jared Kaltwasser
Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein.
Blue stamp of FDA approval | Image Credit: argus-stock.adobe.com
June 20th 2024

FDA Grants 2 Approvals to Delandistrogene Moxeparvovec for DMD

Maggie L. Shaw
Accelerated approval was originally granted in June 2023 for patients aged 4 to 5 years, indicating there was an unmet clinical need for a potentially life-saving treatment for the rare genetic muscle disorder.
Molecular engineering of DNA for Gene therapy | Image Credit: © immimagery - stock.adobe.com
June 12th 2024

Rett Syndrome Gene Therapy Granted RMAT Designation

Victoria Johnson
The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation after reviewing the phase 1 safety and efficacy data.
mRNA | Image Credit: ArtemisDiana-stock.adobe.com
June 11th 2024

Advances in Colon Cancer: Delivering Dual-mRNA With Lysate-Based Nanoparticles

Gayle Turim Dickstein
Building on prior research, a team tackled mouse-model colon cancer with a novel nonviral nanovector to carry dual–messenger RNA (mRNA) immunogene therapy.
Genetic and gene manipulation concept | Image Credit: © ipopba - stock.adobe.com
June 2nd 2024

Proof-of-Concept Study Highlights Potential of Gene-Based Therapy for Timothy Disease

AJMC Contributor
The proof-of-concept study showed that antisense oligonucleotides developed by the researchers successfully restored cellular development and brain cell function in patients with the disease characterized by multiorgan dysfunction.
CRISPR DNA Editor | Image Credit: NathanDeverycom-stock.adobe.com
May 20th 2024

Gene Therapy Offers Promise in Treating Inborn Errors of Immunity

Jared Kaltwasser
A new report says early evidence of using gene therapy to treat monogenic diseases offers reason for optimism.
Hemophilia definition | Image Credit: VitezslavVylicil-stock.adobe.com
May 15th 2024

Valoctocogene Roxaparvovec Dramatically Reduces Bleeding in Hemophilia A

Kimberly Rath, PharmD
New study findings demonstrate that valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, significantly reduces bleeding episodes and dependency on factor VIII infusions over a 3-year period, while maintaining a favorable safety profile.
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.
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