Remove Engineer Remove Genome Remove Genomics Remove In-Vivo
article thumbnail

Intellia Establishes Proof-of-Concept for in vivo CRISPR Bone Marrow Editing

BioSpace

Intellia Therapeutics presented preclinical data of its non-viral genome editing platform at the Keystone eSymposium: Precision Engineering of the Genome, Epigenome and Transcriptome.

In-Vivo 98
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
Insiders

Sign Up for our Newsletter

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

article thumbnail

Scribe and Sanofi partner to develop cell therapies for cancer

Pharmaceutical Technology

The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer. Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies.

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. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

article thumbnail

Gene Editing Innovations with Programmable Gene Insertion (PGI) Technology ft. John Finn, PhD, CSO, Tome Biosciences – Xtalks Life Science Podcast Ep. 160

XTalks

John Finn, PhD Chief Scientific Officer Tome Biosciences Dr. Finn has over 20 years of experience in the gene therapy space with a focus on genome editing and delivery technologies. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

article thumbnail

Gene editing: beyond the hype

pharmaphorum

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space. Zinc fingers. billion in funding.

article thumbnail

Artificial Intelligence innovation: Leading companies in in-silico drug discovery

Pharmaceutical Technology

DNA binding site prediction, peptide structure optimisation, and AI-assisted genome analysis are some of the accelerating innovation areas, where adoption has been steadily increasing. However, not all innovations are equal nor do they follow a constant upward trend.

Drugs 189