Gene Therapy

Latest News
August 15th 2025

Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

July 31st 2025

Progress and Challenges With Gene Therapy in Hereditary Hearing Loss

July 25th 2025

The State and Future of Viral, Nonviral Vectors for Gene Therapy

July 22nd 2025

OneOncology Appoints Michael Byrne, DO, as Medical Director for Cellular Therapy

July 18th 2025

CGT Access Model Aims to Expand Access to Sickle Cell Treatments

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August 20th 2025

The Role of Pharmacists in Advancing Cell and Gene Therapy Integration at UC Davis Health: Jenny Craven, PharmD, BCPS

August 15th 2025

Balancing Efficacy, Safety, and Cost in Formulary Decisions for Cell and Gene Therapies: Clayton Irvine, PharmD, MBA, MS

May 6th 2025

Expanding Access to CGTs Through Innovative Payment Models: Joe DePinto, MBA

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

More News

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
July 1st 2025

FDA Grants Platform Technology Designation to Limb-Girdle Muscular Dystrophy Gene Therapy Vector

Noah Stansfield
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
June 13th 2025

DMD Gene Therapy Shows Clinical Benefits in 8- and 9-Year-Old Patients

Rose McNulty
The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD).
Participants who received RGX-202 demonstrated functional improvement and better outcomes than controls for all measures. | Image Credit: Treecha- stock.adobe.com
June 12th 2025

RGX-202 Gene Therapy Improves DMD Trajectory, Biomarkers

Rose McNulty
RGX-202 shows promising efficacy in Duchenne muscular dystrophy, improving functional outcomes and biomarker expression in early trial results.
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
May 30th 2025

Hurdles Remain Despite Progress in Gene Therapy for DMD

Rose McNulty
Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further innovations in safety, efficacy, and treatment strategies are needed.
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
May 16th 2025

Delandistrogene Moxeparvovec Shows Long-Term Safety, Tolerability in Pooled Trial Data

Rose McNulty
Delandistrogene moxeparvovec demonstrated a manageable safety profile across clinical trials for Duchenne muscular dystrophy (DMD), with most adverse events emerging within 90 days of infusion.
The new report highlights several barriers to CGT use, including cumbersome referral processes, high price tags, complex logistics, and uncertainties around reimbursement. | Image Credit: Treecha - stock.adobe.com
May 14th 2025

CGTs Have Potential to Transform Care, but Challenges Persist

Rose McNulty
Despite holding promise in a range of disease states, a variety of challenges prevent cell and gene therapies (CGTs) from more widespread use, according to a new report from Cardinal Health.
Quantitative magnetic resonance imaging seemed to show that delandistrogene moxeparvovec protects muscle from progressive damage in DMD. | Image credit: OlegKachura - stock.adobe.com
May 14th 2025

Quantitative Magnetic Resonance Outcomes Suggest Gene Therapy Slows DMD Progression

Rose McNulty
Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients with Duchenne muscular dystrophy (DMD) based on muscle quantitative magnetic resonance measures.
gene therapy | Image Credit: © RFBSIP - stock.adobe.com
April 30th 2025

Expanding Broader Patient Access to CGTs

Pearl Steinzor
Key trends and challenges are shaping the next era of advanced therapies, from FDA momentum to broader patient access.
At 6 months, 81% of wounds showed 50% or more healing vs 16% of 43 matched control wounds treated with standard of care in the VIITAL study. | Image credit: wladimir1804 - stock.adobe.com
April 29th 2025

FDA Approves Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa

Rose McNulty
Prademagene zamikeracel (Zevaskyn) is the first and only cell-based gene therapy for wound treatment in patients with recessive dystrophic epidermolysis bullosa.
The consensus guidelines aim to provide practical guidance for institutions administering gene therapy in a commercial or trial setting. | Image credit: CrazyJuke - stock.adobe.com
April 18th 2025

Consensus Guidelines Aim to Optimize DMD Gene Therapy Delivery

Rose McNulty
Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).
Real-world data suggest delandistrogene moxeparvovec is well-tolerated and effective in patients with DMD. | Image credit: natali_mis - stock.adobe.com
March 26th 2025

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

Rose McNulty
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
March 21st 2025

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

Rose McNulty
Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows Duchenne muscular dystrophy (DMD) progression.
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).
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