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

XTalks

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

In-Vivo 52
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News from AACR 2024: Sunday’s highlights

Drug Discovery World

Following this, Benjamin Cravatt, Professor and the Norton Gilula Chair in Biology and Chemistry at The Scripps Research Institute, discussed activity-based protein profiling (ABPP) and its use in discovering drug candidates for cancer-relevant proteins.

In-Vivo 52
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Artificial Intelligence innovation: Leading companies in in-silico drug discovery

Pharmaceutical Technology

Innovation S-curve for artificial Intelligence in the pharmaceutical industry In-silico drug discovery is a key innovation area in artificial Intelligence Drug research starts with the discovery stage that involves in vivo and in vitro models to shortlist lead drug compounds.

Drugs 189
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IRBM and Merck extend peptide therapeutics collaboration

Drug Discovery World

Italian contract research organisation IRBM has signed a new agreement with Merck & Co Inc (MSD outside the USA and Canada) to continue their collaboration in the peptide therapeutics area. We are thrilled to continue this collaboration as part of our mission to advance impactful agents into the clinic.”

In-Vivo 52
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Gene editing: beyond the hype

pharmaphorum

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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Company Spotlight: Cue Biopharma Innovates in the Immunotherapy Space with its Immuno-STAT Platform

XTalks

With several biologics in the pipeline, including one in early stage clinical trials for head and neck cancer, the company is positioned to engineer highly specific biologics against immune targets in the body. protein-based biologics) and vaccine treatments. protein-based biologics) : are those which bind to specific components (i.e.

Protein 98
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Q&A with Mark Garner: The golden age of cancer research  

Drug Discovery World

For example, in CAR-T-cell therapy, scientists can take T-cells from a patient and ex vivo (outside the body) genetically engineer them to effectively target and kill cancerous cells, then infuse them back in to the patient. Mark Garner, PhD., RA: Please can you tell us a bit more about your role at Agilent?