article thumbnail

Antisense therapy restores fragile X protein production in human cells

Science Daily: Pharmacology News

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.

article thumbnail

Stoke Therapeutics Announces Presentations Related to the Company’s Work to Advance STK-001, the First Potential New Medicine to Target the Underlying Cause of Dravet Syndrome at the American Epilepsy Society (AES) 2020 Annual Meeting

The Pharma Data

Highlights from the Company’s presentations include: BUTTERFLY Observational Study – Baseline Analysis. A review of the study design for MONARCH, the Company’s ongoing Phase 1/2a study of STK-001 in children and adolescents with Dravet syndrome will be presented during a poster session by Linda Laux, M.D., Antiepileptic Drugs / 7B.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Driving efficiency across upstream bioprocess workflow

Drug Target Review

Transposons preferentially integrate the gene of interest into active transcriptional sites, facilitating rapid cell line development and higher titers of the protein drug. Engineering of Chinese hamster ovary cell lipid metabolism results in an expanded ER and enhanced recombinant biotherapeutic protein production.

article thumbnail

JS Bio and Etta Biotech Advancing Strategic Partnership

The Pharma Data

Etta Biotech”), to set up a high titer transient protein expression platform for high quality protein production using JS Bio’s transient transfection media. JS Bio becomes the exclusive cell culture supplier for Etta Biotech’s transient transfection high titer protein expression platform.

article thumbnail

Biogen Announces Topline Results from the Tofersen Phase 3 Study and its Open-Label Extension in SOD1-ALS

The Pharma Data

Tofersen binds to SOD1 mRNA, allowing for its declination by RNase-H in an trouble to reduce conflation of SOD1 protein product. About Tofersen Tofersen is an antisense medicine being estimated for the implicit treatment of SOD1-ALS.

article thumbnail

Eloxx Pharmaceuticals to Report Third Quarter 2020 Financial Results and Provide Business Update on November 5, 2020

The Pharma Data

Live Webcast : accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or with this link: [link]. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. International Dial-in Number: (210) 874-7715. Conference ID : 9467336. About Eloxx Pharmaceuticals.

article thumbnail

Targeting a human protein may stop Ebola virus in its tracks

The Pharma Data

Early in infection, it appears that host cellular proteins are in defense mode against Ebola virus polymerase and the viral genetic material. “This presents additional hurdles for polymerase,” says Fang. “The virus hijacks this protein and makes it do something different than its normal job in cells.

Virus 52