XTalks

NOVEMBER 26, 2024

This progressive disease occurs when misfolded transthyretin (TTR) proteins form amyloid deposits in the heart, leading to cardiac dysfunction. BridgeBio has been highly active in the genetic diseases space.

Genetic Disease 105

Genetic Disease Genetics Protein Clinical Development 105

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

Pharmaceutical Technology

AUGUST 17, 2022

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . Merck will also make royalty payments on any approved products developed out of the partnership.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

pharmaphorum

OCTOBER 5, 2022

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that targets muscle cells. billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

RNA 105

RNA Genetics Dermatology Antibody 105

The Pharma Data

OCTOBER 14, 2020

Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

OCTOBER 27, 2020

AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. Be Biopharma .

Gene 52

Gene Gene Therapy Medicine Development 52

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