Pharmaceutical Technology

OCTOBER 25, 2022

Astellas chief strategy officer Naoki Okamura said: “Gene therapy is the cornerstone of Astellas’ Primary Focus, Genetic Regulation; our goal is to bring new transformative treatment options to patients living with serious genetic diseases and limited treatment options.

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

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

pharmaphorum

SEPTEMBER 17, 2020

There are two antibodies already on the market that target PCSK9 – Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but these require subcutaneous injection every two to four weeks.

Drugs 66

Drugs Antibody Sales Branding 66

The Pharma Data

NOVEMBER 29, 2020

Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

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. The impact of CNS diseases extends beyond patients—to their families and society as well.” The promise to partners is to “move quickly.

RNA 52

RNA Antibody Life Science Protein 52

XTalks

SEPTEMBER 17, 2020

Patients with this disease have a very high level of LDL-C, which increases their risk of coronary artery disease, including heart attack, stroke and atherosclerosis. The replacement therapy has been shown to improve metabolic complications and can also be used to treat the acquired form of the disease.

Trials 98

Trials Gene Hormones Clinical Trials 98

The Pharma Data

NOVEMBER 8, 2020

“The results from the CANDOR study confirm the potential for Kyprolis to be used in combination with an anti-CD38 monoclonal antibody.”. Progeria, also called Hutchinson-Gilford Progeria Syndrome (HGPS), is an ultra-rare and fatal genetic disease of accelerated aging in children.

Trials 52

Trials Protein Gene Genetic Disease 52