Gene Therapy

Latest News
April 30th 2025

Expanding Broader Patient Access to CGTs

April 29th 2025

FDA Approves Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa

April 18th 2025

Consensus Guidelines Aim to Optimize DMD Gene Therapy Delivery

March 26th 2025

Real-World Study Suggests DMD Gene Therapy Is Safe, Effective

March 21st 2025

Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD

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April 29th 2025

Real-World Data Crucial to Prove the Value of Expensive Cell and Gene Therapies: Fran Gregory, PharmD

April 7th 2025

Hurdles to Bringing Gene Therapies to Market: Giulio Cossu, MD

March 27th 2025

Future Research Directions for Revakinagene Taroretcel in MacTel

February 28th 2025

Gene Therapy Holds Potential in Otoferlin-Related Hearing Loss

February 5th 2025

Potential Impacts of Halting the CGT Access Model: AMCP's Adam Colborn

May 26th 2024

Dr Migvis Monduy on the Most Promising Areas of DMD Research

October 23rd 2023

Dr Landon Marshall Discusses Gene Therapy Forecasting Benefits, Future Advancements

April 10th 2023

Payers, Providers Still Navigating Uncharted Waters for Gene Therapies

More News

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
March 13th 2025

Uniform Global Standards, Processes Could Accelerate CGT Approvals, Access

Rose McNulty
The study found that just 20% of trials submitted to both the FDA and European Medicines Agency had matching evidence.
human-dna-strand-double-helix-macro-generative-ai_634358-316.jpg
February 27th 2025

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

Rose McNulty
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
February 26th 2025

SGT-003 Gene Therapy Shows Potential in DMD

Rose McNulty
In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants with Duchenne muscular dystrophy (DMD) at 90 days post treatment.
The authors emphasized the importance of early diagnosis and treatment with CGTs, especially for patients with genetic conditions. | Image credit: RFBSIP - stock.adobe.com
February 14th 2025

Mitigating Barriers to Cell and Gene Therapy Access

Rose McNulty
Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, according to a white paper from the National Pharmaceutical Council.
Duchenne muscular dystrophy | Image credit: hafakot - stock.adobe.com
January 28th 2025

EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD

Rose McNulty
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD).
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
January 15th 2025

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

Rose McNulty
Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene abeparvovec, an investigational gene therapy.
The findings highlight a need for improved standardization of measurements for AAV in the context of viral vector–based gene therapies. | Image credit: RFBSIP - stock.adobe.com
January 12th 2025

Standardized Measurements Needed for AAVs in Gene Therapy, Study Finds

Rose McNulty
Common measurements for modified adeno-associated viruses (AAVs), a gene therapy delivery method, showed substantial variation in accuracy and precision.
Among patients younger than 18 years with β-thalassemia, 92% reached transfusion independence with beti-cel, while 80% of adults achieved this outcome. | Image credit: Eleni - stock.adobe.com
January 3rd 2025

Gene Therapy Demonstrates High Efficacy in Severe β-Thalassemia

Kimberly Rath, PharmD
A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β-thalassemia.
Highlights from AMCP Nexus 2024
December 26th 2024

ICYMI: Highlights From AMCP Nexus 2024

Kyle Munz
Check out this year's top coverage from The Academy of Managed Care Pharmacy (AMCP) Nexus 2024 meeting, which included relevant topics in health care policy, novel pharmaceutical developments, financial considerations across multiple conditions, and more.
Gene therapy | Image credit: vchalup - stock.adobe.com
December 19th 2024

New Evidence Highlights Stability of Lentiviral Gene Therapies

Kimberly Rath, PharmD
Researchers study long-term safety of lentiviral gene therapy in primates, finding no evidence of somatic mutations or malignancy.
Blue graphic with the words "Highlights From AMCP Annual 2024" and health care icons
December 19th 2024

ICYMI: Highlights From AMCP Annual 2024

Laura Joszt, MA
Among the most-read coverage for the annual meeting were presentations on the potential and costs of cell and gene therapies, the latest trends in the industry, and pharmacogenomic testing in mental health.
Gene therapy | Image credit: Treecha – stock.adobe.com
December 19th 2024

FDA Pauses PepGen’s DMD Drug Trial in the US

Hayden E. Klein
The CONNECT2-EDO51 phase 2 trial looks at PGN-EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.
Here are the top 5 most-read gene therapy articles in 2024.
December 13th 2024

Top 5 Most-Read Gene Therapy Articles of 2024

Cameron Santoro
The most-read gene therapy articles included topics on FDA approvals, chimeric antigen receptor T-cell therapies, the cost-effectiveness of gene therapies, as well as other findings.
CAR T-cell cancer treatment concept | image credit: catalin -stock.adobe.com
December 12th 2024

Comparing CAR T-Cell Therapies for LBCL: Insights From CART-SIE Study

Kimberly Rath, PharmD
Real-world study compares the efficacy and safety of axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel), finding that axi-cel provides superior progression-free survival but with higher toxicity.
Novel, ultra-high-cost gene and cell therapies are set to have a major influence on pharmaceutical systems | image credit: suyujung - stock.adobe.come
December 9th 2024

Ultra-High-Cost Gene Therapies Set to Influence Major Pharmacy Shifts

Jared Kaltwasser
Leaders of the American Society of Health-System Pharmacists (ASHP) say the time is now to prepare for providing innovative gene and cell therapies.
Imaging of the eye's retina | Image Credit: © Richman Photo - stock.adobe.com
December 3rd 2024

Gene Therapy Enhances Visual Processing for Inherited Retinal Disease

Kimberly Rath, PharmD
Gene therapy partially restores visual processing in the geniculostriate pathway of patients with Leber congenital amaurosis type 2 while maintaining compensatory activity in the retinotectal pathway.
Screenshot of an interview with Kevin Niehoff, PharmD
December 3rd 2024

Expanding Access to Gene Therapies: Addressing Patient Burden, High Costs

Laura Joszt, MA Peter Wehrwein
Gene therapies can be life-changing for people, but the high cost plus the burden of treatment remain barriers to access and utilization, explained Kevin Niehoff, PharmD, BCMAS, of IPD Analytics.
gene therapy | Image Credit: © Anar Mammadov-stock.adobe.com
November 28th 2024

Real-World Data Demonstrate Efficacy of Beti-Cel Gene Therapy

Jared Kaltwasser
Investigators said patients experienced adverse effects in line with those expected for myeloablative conditioning with busulfan following treatment with betibeglogene autotemcel (beti-cel; Zynteglo; bluebird bio).
Boy with DMD using walker | Image credit: Svitlana – stock.adobe.com
November 23rd 2024

Viltolarsen Slows DMD Progression in Small Study

Hayden E. Klein
Further research is needed to determine how once-weekly viltolarsen can slow disease progression and preserve motor function in boys and young men with Duchenne muscular dystrophy (DMD).
Researcher working with microplate in the laboratory | Image credit: angellodeco – stock.adobe.com
November 14th 2024

Sarepta Halts Development, Testing of DMD Therapy SRP-5051

Hayden E. Klein
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
November 14th 2024

FDA Approves Gene Therapy to Treat AADC Deficiency

Julia Bonavitacola
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase (AADC) deficiency.
Fidanacogene elaparvovec is a promising gene therapy for patients with hemophilia B | image credit: ipopba - stock.adobe.com
November 6th 2024

Fidanacogene Elaparvovec Shows Strong Results in Open-Label Phase 3 Study

Jared Kaltwasser
The gene therapy improved annualized bleed rates, though a handful of study participants resumed factor IX prophylaxis.
Screenshot of an interview with Adam Colborn, JD
November 2nd 2024

Medicaid Aiming to Improve Patient Access to High-Cost Therapies

Laura Joszt, MA
New legislation would clarify rules around value-based contracts in Medicaid, and a new model is expanding access to gene therapies, explained Adam Colborn, JD, of AMCP.
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.
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