article thumbnail

Scribe and Sanofi expand genetic therapy development deal

Pharmaceutical Technology

Scribe Therapeutics and Sanofi have expanded partnership to progress the development of in vivo genetic therapies to treat genomic diseases.

Genetics 246
article thumbnail

Single-course in vivo base editing therapy proven to lower cholesterol

Drug Discovery World

Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. Verve-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood LDL-C. “Of

In-Vivo 52
Insiders

Sign Up for our Newsletter

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

article thumbnail

Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

XTalks

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52
article thumbnail

Genome Editing Market Update: Intellia Therapeutics Presents Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy

Roots Analysis

The global genome editing market is anticipated to grow at a CAGR of 12.6% The global genome editing market is anticipated to grow at a CAGR of 12.6% The biopharmaceutical pipeline includes close to 500 gene therapies , several of which are being developed based on CRISPR technology. during the forecast period 2023-2035.

Genome 40
article thumbnail

Scribe and Sanofi partner to develop cell therapies for cancer

Pharmaceutical Technology

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Scribe is also entitled to receive potential payments worth over $1bn on meeting development and commercial milestones.

article thumbnail

Evotec and Variant Bio partner to develop fibrosis treatments

Drug Discovery World

The strategic partnership will combine Variant Bio’s genomic discovery capabilities and VB-Inference platform with Evotec’s knowledge of antifibrotic drug discovery. The strategic partnership will combine Variant Bio’s genomic discovery capabilities and VB-Inference platform with Evotec’s knowledge of antifibrotic drug discovery.

article thumbnail

Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.