article thumbnail

Vertex signs licence deal with CRISPR Therapeutics for diabetes therapies

Pharmaceutical Technology

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA. The system comprises the Cas9 enzyme and a guide RNA.

article thumbnail

Sanofi wagers $400m on miRecule muscular dystrophy therapy

pharmaphorum

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. billion deal in 2018.

RNA 105
Insiders

Sign Up for our Newsletter

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

article thumbnail

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

The Pharma Data

Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

article thumbnail

Boost for mRNA as Moderna to acquire OriCiro for $85 million

pharmaphorum

and OriCiro Genomics K.K. Nonetheless, Moderna has previously been involved in licensing deals to advance its technology. Biotech Moderna, Inc. have announced they have entered into a definitive agreement for Moderna to acquire OriCiro at a cost of $85 million. The deal marks 10-year-old Moderna’s first acquisition.

article thumbnail

Drug discovery quarterly review: Second quarter 2023  

Drug Discovery World

Moreover, in July 2023, AstraZeneca and Vaxess Technologies commenced a collaboration for the evaluation of a novel RNA-based pandemic influenza prototype vaccine in patch format.

Drugs 59
article thumbnail

Where is the drug discovery expertise happening in the UK?

Drug Discovery World

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. We also recently opened the Scientific Centre of Excellence for Genomic Insight at our Cambridge site.”

Drugs 76
article thumbnail

Using analytics and bioassays to de-risk your mRNA LNP drug development programme

Pharmaceutical Technology

The swift development and deployment of messenger-RNA (mRNA) vaccines against the SARS-CoV-2 virus during the COVID-19 crisis has catapulted the pharmaceuticals industry into a new paradigm. RNA has the potential to underpin breakthrough treatments for a wide variety of diseases, including many cancers, and transform medicine as we know it.

Bioassay 130