Tag: Amyloidosis

Editas (EDIT) Focuses on Developing Gene-Editing Eye Drug

This story originally appeared on Zacks Editas Medicine, Inc. EDIT has made rapid progress in the development of its lead pipeline candidate, EDIT-101, which employs CRISPR gene editing, to treat Leber congenital amaurosis type 10 (LCA10) — a rare genetic illness that causes blindness. – Zacks The company is evaluating…

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Intellia Therapeutics Set to Enter Clinic with First Solo Ex Vivo Candidate

Courtesy Intellia Therapeutics Intellia Therapeutics is celebrating another first today as the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for NTLA-5001, its first wholly-owned ex vivo CRISPR genome editing candidate. NTLA-5001 is being developed for the treatment of acute myeloid leukemia (AML). Intellia…

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Breakout “CRISPR platform” company Mammoth Biosciences is officially a unicorn

The CRISPR-based biotech startup Mammoth Biosciences is officially a unicorn, the company says. The billion dollar valuation comes on the back of a $150 million series D round led by Redmile Group, with participation from Foresite Capital, Senator Investment Group, Sixth Street, Greenspring Associates, Mayfield, Decheng Capital, Plum Alley and…

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Multipurpose Mini CRISPR System Easier to Deliver Into Human Cells for Gene Therapy, Treat Rheumatoid Arthritis, Diverse Ailments

Bioengineers reimagine the CRISPR system as a Swiss Army knife packed with many applications. They repurposed it to make a mini version, called CasMINI, that can easily be delivered into human cells for gene therapy and treat various ailments. The new technology, described in the study titled “Engineered Miniature CRISPR-Cas…

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CRISPR-Based Therapeutics Blaze an In Vivo Path to the Clinic

Therapeutic applications of genome editing were envisioned at least as early as the mid-1990s, when the first sequence-specific genome editing technologies emerged. Initially, such applications were considered distant prospects, but by 2012, they suddenly seemed near to hand. It was at that time that CRISPR technologies emerged. CRISPR, which stands…

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Biotech Company Driven by Cutting Edge Science and Patients, Not Ego

Photo courtesy of Intellia Therapeutics Sports fans know that nothing is better for the chemistry of a team than winning, and on the CRISPR field, Intellia Therapeutics is on a winning streak.    On June 26, Intellia set the biotech world on fire, announcing the first-ever clinical data supporting the safety…

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Therapeutic Advances Using In Vivo CRISPR Genome Editing

Broadcast Date: September 16, 2021Time: 8 am PT, 11 am ET, 17:00 CET When CRISPR-Cas9 gene-editing technology exploded onto the life science stage almost a decade ago, it was widely touted as a potentially curative therapy for many genetic diseases, using various ex vivo and in vivo delivery methods. That promise is…

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Another Milestone for CRISPR-Cas9 Technology: First Trial Data for Treatment Delivered Intravenously

Unlike most other CRISPR/Cas-9 therapies that are ex vivo treatments in which cells are modified outside the body, this study was successful with an in vivo treatment Use of CRISPR-Cas9 gene editing technology for therapeutic purposes can be a boon for clinical laboratories. Not only is this application a step…

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Editas (EDIT) Up More Than 70% in Past 3 Months: Here’s Why

Shares of Editas Medicine, Inc. EDIT have rallied 78.7% in the past three months compared with the industry’s increase of 7.4%. Zacks Investment Research Image Source: Zacks Investment Research The company has made rapid progress in the development of its lead pipeline candidate, EDIT-101, in this time frame. EDIT-101 employs…

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