pharmaphorum

SEPTEMBER 1, 2021

As well as the access agreement for high-risk patients, the Novartis deal also covers a large-scale trial of the PCSK9 inhibitor and the creation of a consortium with academic groups aimed at improving the manufacturing of oligonucleotide drugs in the UK. This needs further thought,” he added.

Drugs 98

Drugs Trials Gene Silencing Genetics 98

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

RNA 40

RNA Protein Genetics Gene Expression 40

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. About Primary Hyperoxaluria Type 1 (PH1) PH1 is an ultra-rare genetic disease that affects an estimated one to three individuals per million in the United States and Europe. Oxlumo should be administered by a healthcare professional.

FDA Approval 52

FDA Approval Production RNA Medicine 52