Pharmaceutical Technology

OCTOBER 25, 2022

There are options in the future to possibly apply the worldwide research and development (R&D), manufacturing and marketing expertise of Astellas in gene therapy to AAV gene therapy development programmes of Taysha for genetic ailments of the central nervous system (CNS).

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Gene Therapy Gene Development Genetics 264

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105

RNA Genetics Genetic Disease Medicine 105

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. The aim will be to identify promising candidates in areas of “unmet patient need.”

RNA 101

RNA Genetics Dermatology Antibody 101

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

SEPTEMBER 17, 2020

The UK big pharma hasn’t revealed how much it is paying for rights to the drug candidate, originally developed by US biotech Dogma Therapeutics, which is still in preclinical development. At the moment no oral PCSK9 drugs have started clinical development, and AZ says it intends to start clinical testing of Dogma’s candidate next year.

Drugs 60

Drugs Antibody Sales Branding 60

The Pharma Data

NOVEMBER 8, 2020

The submission includes data from the ENLIGHTEN clinical development program in schizophrenia, and pharmacokinetic (PK) bridging data comparing ALKS 3831 and Zyprexa (olanzapine), to support an indication for schizophrenia. Bristol Myers Squibb’s Lisocabtagene Maraleucel (CAR-T) for R/R Large B-Cell Lymphoma.

Trials 52

Trials Protein Gene Genetic Disease 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. The impact of CNS diseases extends beyond patients—to their families and society as well.” Sigilon Therapeutics .

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RNA Antibody Life Science Protein 52