Remove Clinical Research Remove Gene Editing Remove Gene Therapy
article thumbnail

The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.

article thumbnail

3 Key Takeaways from Global Genes Summit 2023

Worldwide Clinical Trials

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. There were many impactful insights shared from the several sessions and amazing conversations at the #WeekInRare, but the top themes I took away are: 1.

Gene 130
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Regulatory Trends in Cell and Gene Therapies

Advarra

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

article thumbnail

New Rare Disease Drugs and Research Advancements

XTalks

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. Both therapies were awarded their approvals on the same day in December 2023.

article thumbnail

The Challenges and Promise of CAR T-Cell Therapy in Oncology

XTalks

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. So that’s really exciting,” Dr. Siciliano explained.

article thumbnail

The Human Side of Clinical Trials: Patients, Progress, and Participation

WCG Clinical

In a recent episode of WCG Talks Trials, WCG experts explored topics like the importance of community engagement, the latest developments in gene and cell therapies, and the challenges of clinical research in rare and chronic diseases like sickle cell disease and melanoma.

article thumbnail

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Worldwide Clinical Trials

This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications. For a more in-depth discussion on challenges and solutions to ALS research, read our blog.

Gene 204