FDA Approval, Gene Expression, Genetic Disease and Marketing

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FDA Approval

Gene Expression

Genetic Disease

Marketing

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Drugs for dialysis, amino acid supplements, and drugs which convert blood ammonia are marketed right now as the go-to urea cycle disorder treatments.

RNA

RNA Gene Expression Gene Gene Therapy

The evolution of the RNA therapeutics landscape

Drug Discovery World

JULY 8, 2024

RNA interference: silencing gene expression The second category of RNA therapeutics is a class that has also yielded marketable drugs in recent years. Two such FDA-approved RNA aptamers are pegaptanib and avacincaptad pegol, indicated for the treatment of macular degeneration.

RNA

RNA Protein Gene Expression Vaccination

Clinical Research Informer

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

The evolution of the RNA therapeutics landscape

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