Remove FDA Approval Remove Immune Response Remove Laboratories
article thumbnail

A Visual Guide to Gene Delivery

Codon

For the former, clinicians extract a patient’s cells, engineer them in a laboratory, and then infuse them back into the body. And even if all these economic hurdles are overcome, there are still three main technical challenges to solve: cargo capacity, specificity, and immune response.

article thumbnail

The Long Road to End Tuberculosis

Codon

Working at the famed Pasteur Institute, the duo began by figuring out how best to culture mycobacteria — a seemingly simple, but essential first step, given how no one at the time had yet come up with an effective way of growing these bacteria in the laboratory. tuberculosis. coli bacteria from growing.

Insiders

Sign Up for our Newsletter

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

article thumbnail

A Visual Guide to Genome Editors

Codon

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Collectively, these repeat-protospacer regions are known as CRISPR arrays.

DNA
article thumbnail

New insights into the role of viral capsids in gene therapy safety

Drug Target Review

1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2. 2 Since then, the FDA has approved four more AAV-based gene therapies—Zolgensma, Hemgenix, Elevidys and Rocktavian—for treating various diseases.

article thumbnail

Moleculin Announces Annamycin Receives FDA Orphan Drug Designation for Soft Tissue Sarcomas

The Pharma Data

Moleculin is also engaged in preclinical development of additional drug candidates, including other Immune/Transcription Modulators, as well as WP1122 and related compounds capable of Metabolism/Glycosylation Inhibition. For more information about the Company, please visit [link].

FDA
article thumbnail

Sanofi and GSK to seek regulatory authorization for COVID-19 vaccine

The Pharma Data

The public health relevance of the refrigerator temperature-stable adjuvanted protein-based Sanofi-GSK vaccine is strongly supported by the induction of robust immune responses and a favorable safety profile in multiple settings. against any symptomatic COVID-19 disease in the seronegative population.

article thumbnail

Regeneron’s COVID-19 Outpatient Trial Prospectively Demonstrates that REGN-COV2 Antibody Cocktail Significantly Reduced Virus Levels and Need for Further Medical Attention

The Pharma Data

“We continue to see the strongest effects in patients who are most at risk for poor outcomes due to high viral load, ineffective antibody immune response at baseline, or pre-existing risk factors. Yancopoulos , M.D., President and Chief Scientific Officer of Regeneron. Regeneron has shared these results with the U.S.