Gene Therapy

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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

The platform technology designation is a new designation established by the FDA.

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

While patients should be closely monitored in the first 90 days post-treatment, delandistrogene moxeparvovec showed a manageable, consistent safety profile in a broad population of patients. | Image Credit: Hypnosis - stock.adobe.com
Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data

May 16th 2025

More News

Gene therapy research | Image credit: RFBSIP – stock.adobe.com
Published: October 29th 2024 | Updated: October 30th 2024

A Year of DMD Gene Therapy Trial Failures

Hayden E. Klein
The outcomes of the EMBARK and CIFFREO trials in boys aged 4 to 7 have raised concerns in the Duchenne muscular dystrophy (DMD) community and prompted layoffs at Pfizer.
Varying models for CAR-T therapy are similarly cost effective, report finds | image credit: inthasone - stock.adobe.com
October 22nd 2024

Different Cost-Effectiveness Models of CAR T Yield Similar Results

Jared Kaltwasser
Investigators said the unique features of chimeric antigen receptor (CAR) T-cell therapy may warrant novel approaches to cost-effectiveness analysis.
Illustration of gene therapy | Image Credit: © RFBSIP - stock.adobe.com
October 17th 2024

CNS-Directed AAV Gene Therapy: Immune Dynamics and Clinical Implications

Kimberly Rath, PharmD
A new study reviews how various routes of adeno-associated virus (AAV) gene therapy administration influence local and peripheral immune responses, with a focus on those observed in clinical trials targeting the central nervous system (CNS).
eye computer | Image Credit: © eevi - stock.adobe.com
October 9th 2024

Voretigene Neparvovec Helps Patients With RPE65-Related Retinopathy Regain Bilateral Function

Karen Jacobson-Sive
Three patients with RPE65-related retinopathy showed improved function in both eyes following gene augmentation therapy treatment with voretigene neparvovec.
Molecular model of insulin molecule | Image Credit: © Dr_Microbe - stock.adobe.com
October 4th 2024

Protein's Role in Insulin Signaling Could Have Implications for Gene Therapy

Jared Kaltwasser
Insulin resistance plays a role in many diseases. The new research provides insights into why insulin resistance appears to be associated with neurodegenerative disorders.
Gene editing | Image Credit: © Degimages - stock.adobe.com
September 18th 2024

Meta-Study Analyzes Immunosuppressive Protocols for AAV Gene Therapy for Monogenic Disorders

Karen Jacobson-Sive
Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect profile that is consistent with high-dose steroid use in other conditions, according to a meta-study that analyzed immunosuppressive protocols.
Gene editing | image credit: 4K_Heaven - stock.adobe.com
September 11th 2024

Emerging Technology Engineers Oncogenic Mutation Into Cell Lines

Gwen Mergian
A new proof-of-concept method reliably engineered a hotspot mutation of SF3B1, a gene-splicing gene, into diverse cancer cell lines, outperforming other contemporary editing approaches.
Genetic analysis | Image Credit: © angellodeco - stock.adobe.com
September 4th 2024

Review: CAR T Cells Could Transform Pediatric SLE Therapy

Jared Kaltwasser
Early data suggest chimeric antigen receptor (CAR) T-cell therapy can achieve meaningful long-term results.
CAR T-cell therapy has revolutionized cancer treatment | image credit: Serhii - stock.adobe.com
September 4th 2024

Neurological Complications in CAR T-Cell Therapy: A Case of Parkinsonism

Kimberly Rath, PharmD
A case report highlights a rare instance of parkinsonism following CAR T-cell therapy for multiple myeloma.
The safety profile of CAR T for patients with SLE is more favorable compared with that for the oncology population, likely due to a smaller target amount of T cells and thus a lower underlying risk of cytokine release syndrome. | Image credit: AIGen - stock.adobe.com
August 28th 2024

Looking to CAR T-Cell Therapy in Refractory Pediatric SLE

Gayle Turim Dickstein
Several years of preliminary research results suggest chimeric antigen receptor (CAR) T-cell therapy could benefit pediatric patients with refractory systemic lupus erythematosus (SLE).
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.
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