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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.

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FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

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. If MSCs come from the patient (autologous), then an immune response shouldn’t be a hindrance.

Therapies 107
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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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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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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.

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FDA Action Alert: Merck, Aurinia and Amgen

The Pharma Data

Food and Drug Administration (FDA) are starting to pick up. People living with LN are in need of an advanced therapy that quickly drives the disease into remission and mediates kidney damage,” said Peter Greenleaf, president and chief executive officer of Aurinia, in July 2020 when the FDA accepted the NDA to review. “We Here’s a look.

FDA 52
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Patient-derived organoids in disease modelling

Drug Target Review

This is particularly pertinent in immune-based cell therapies like CAR-T, where animals do not mimic the human immune response. This was recognised in the FDA Modernization Act 2.0, In orphan and rare diseases, it is hard to get an animal model to represent a disease state that is poorly understood.

Disease 122