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.

RNA 105

RNA Genetics Dermatology Antibody 105

Delveinsight

AUGUST 26, 2021

Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease. Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases. Reduce cost.

RNA 52

RNA Antibody Life Science Protein 52

XTalks

OCTOBER 8, 2024

Apitegromab is a monoclonal antibody that selectively binds to and inhibits myostatin, a protein that limits muscle growth. Current therapies mainly address motor neuron loss, leaving a need for treatments that directly target muscle weakness. Efforts to improve SMA detection and treatment are gaining momentum.

Trials 52

Trials Antibody Genetics Medicine 52

The Pharma Data

JULY 6, 2021

Haemophilia A is a serious, inherited bleeding disorder in which a person’s blood doesn’t clot properly, as they either lack or do not have enough of a clotting protein called factor VIII. Hemlibra is a bispecific factor IXa- and factor X-directed antibody. We currently have four programs in clinical trials.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

XTalks

SEPTEMBER 17, 2020

Dietary changes, including restriction of salt and animal protein, are also recommended. Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males.

Trials 98

Trials Gene Hormones Clinical Trials 98

The Pharma Data

JANUARY 9, 2021

The most common adverse events associated with valoctocogene roxaparvovec occurred early and included transient infusion-associated reactions and transient, asymptomatic, and mild to moderate rise in the levels of certain proteins and enzymes measured in liver function tests with no long-lasting clinical sequelae. GENEr8-1 Study Description.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52