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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.

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Aevitas to sell sCFH asset rights to 4D Molecular

Pharmaceutical Technology

sCFH is an engineered and improved version of complement factor H (CFH) that can fit into adeno-associated virus (AAV) vectors with solid expression and applications confirmed in cultured human cells in vitro, in addition to various preclinical animal models in vivo.

In-Vivo 130
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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.

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Leading innovators in gene splicing using nucleases for the pharmaceutical industry

Pharmaceutical Technology

Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. However, not all innovations are equal and nor do they follow a constant upward trend.

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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.

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The Booming Potential of mRNA Technology

Roots Analysis

Unveiling The Potential of mRNA Technology mRNA is a single-stranded molecule that relays the genetic instructions needed to make proteins from DNA in the cell nucleus to ribosomes. Ribosomes are cellular machines that read mRNA sequences and produce proteins. There are several advantages to approaching vaccination this way.

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