Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

RNA 101

RNA Genetics Dermatology Antibody 101

The Pharma Data

DECEMBER 8, 2020

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases.

RNA 52

RNA Antibody Life Science Protein 52

Drug Discovery World

FEBRUARY 28, 2023

mRNA enables the delivery of a transiently expressed genetic molecule that is translated into a target protein using the machinery of the host cell. Therefore, the risk for off-target insertional mutagenesis is greatly reduced. In addition, it may be easier to target the therapeutic molecule to the correct cell or tissue.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Oxlumo should be administered by a healthcare professional.

FDA Approval 52

FDA Approval Production RNA Medicine 52