Gene Therapy

Latest News

A Year of DMD Gene Therapy Trial Failures

October 29th 2024

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

Different Cost-Effectiveness Models of CAR T Yield Similar Results

By Jared Kaltwasser

October 22nd 2024

Illustration of gene therapy | Image Credit: © RFBSIP - stock.adobe.com

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

By Kimberly Rath, PharmD

October 17th 2024

eye computer | Image Credit: © eevi - stock.adobe.com

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

By Karen Jacobson-Sive

October 9th 2024

Molecular model of insulin molecule | Image Credit: © Dr_Microbe - stock.adobe.com

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

By Jared Kaltwasser

October 4th 2024

More News

Sarah Boyce, president and CEO, Avidity Biosciences | Image Credit: Avidity Biosciences

October 9th 2023

Avidity Biosciences Gains FDA Orphan Drug Designation for Patients With DMD

Pearl Steinzor

Sarah Boyce, president and CEO at Avidity Biosciences, discusses her leading role at the company, as well as antibody oligonucleotide conjugate drug, AOC 1044, currently in development for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping (DMD44).

October 7th 2023

New FDA Pilot Program Will Provide Hands-On Regulatory Guidance for Cell and Gene Therapy Trials in Rare Disease

Noah Stansfield

The program will initially allow 6 sponsors of clinical trials to participate and will provide sponsors with the opportunity for frequent advice and regular communication regarding clinical trial design and other development issues.

Male hands showing protection and protection for family security, finances and health ,Insurance management planning ,property and family risk reduction ,Caring and caring for loved ones: © chaylek - stock.adobe.com

June 28th 2023

Researchers Highlight Restrictive Coverage of Gene, Cell Therapies

Jaime Rosenberg

The researchers focused on coverage trends across an array of disease states, including lymphoma, vision loss, and spinal muscular atrophy, finding discrepancies in coverage across 16 states and Medicaid Care Organizations.

June 22nd 2023

FDA Approves Delandistrogene Moxeparvovec, First Gene Therapy to Treat Duchenne Muscular Dystrophy

Mary Caffrey

The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.

May 12th 2022

A Look at the Current, Future State of Cell and Gene Therapies in the United States

Gianna Melillo

Experts evaluated the future of cell and gene therapies in the United States and offered insights on potential value-based payment models for these treatments.

May 19th 2021

Gene Therapies Present Reimbursement Challenges That Have Yet to Be Answered

Laura Joszt, MA

With more than 3000 gene therapies in development, payers will have to grapple with the challenges of paying for these innovative but expensive therapies.

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