Remove FDA Remove Immune Response Remove Trials
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FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

Therapies 103
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Overcoming challenges in biosimilar analytical characterization

Drug Patent Watch

Additionally, the immunogenicity of biosimilars, which is the ability of the drug to trigger an immune response, can be highly variable and impact the safety and efficacy of the drug. Robust Clinical Trial Design : Clinical trials for biosimilars should be designed to demonstrate equivalence or non-inferiority to the reference product.

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A research team searches for every gene that helps tumors evade immunotherapy

Broad Institute

By then, a few cancer immunotherapies had entered clinical trials and only a handful had been approved by the FDA. As a first step, Manguso set his sights on treatments called PD-1 checkpoint inhibitors, which at the time had shown tremendous promise in clinical trials and would receive their first FDA approval in 2014.

Research 137
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Demonstrated a Durable Immune Response and Elicited Dual Mechanisms of Protection Against Delta and Other SARS-CoV-2 Variants of Concern in Data Published in New England Journal of Medicine

The Pharma Data

Antibody and T-cell immune responses strong and stable at eight months after immunization Demonstrated neutralizing antibody activity against the Delta variant (B.1.617.2) In addition, the T-cell responses are especially strong and stable over time, which is also potentially important for activity against these variants.”.

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Novavax COVID-19 Vaccine Granted Fast Track Designation by U.S. FDA

The Pharma Data

Food and Drug Administration (FDA) has granted Fast Track Designation for NVX-CoV2373, the Company’s COVID-19 vaccine candidate. Novavax expects to begin its pivotal Phase 3 clinical trial in the United States and Mexico by the end of November. and globally.”. About NVX-CoV2373. and Australia. Fast Track Designation by the U.S.

Vaccine 52
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FDA Action Alert: Merck, Aurinia and Amgen

The Pharma Data

Food and Drug Administration (FDA) are starting to pick up. The NDA was built on data from the Phase III VICTORIA trial. We will continue to collaborate with the FDA during their review process and in parallel build our commercial readiness for a potential approval and commercial launch in the first quarter of 2021.”.

FDA 52
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Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

Can they deliver healing genes without triggering an overactive immune response? The details are disturbingly reminiscent of the famous case of Jesse Gelsinger , who died from a ferocious immune response to experimental gene therapy in September 1999. An immune response is two-tiered.