Genome Editing

The fourth boy was the first and only patient who received the lower dose after the FDA lifted its initial clinical hold last year. According to Astellas, he was dosed over the summer and showed abnormal liver function tests (LFTs) within weeks after dosing with AT132. An initial elevation of hepatic lab values was observed within the first month of dosing, Astellas said September 1.

Both Mammoth’s therapies and diagnostics will be based on what the company touts as the largest toolbox of CRISPR proteins on earth. Through the financing, Mammoth plans to expand the toolbox and broaden its pipeline of genome-edited therapies, focusing on the smallest known CRISPR systems, based on Cas14, which targets single-stranded DNA; and Casɸ, which is encoded exclusively in the genomes of huge bacteriophages.

The team that discovered the RNA editing CRISPR system Cas13 has made another addition to the RNA editing CRISPR toolbox. Their latest paper describes Cas7-11, a single protein that consolidates the domains of more complex CRISPR systems and lacks Cas13's collateral cutting property which is toxic to cells. Taken together, Cas7-11’s properties may make it the “Cas9 of RNA” and a possible candidate for RNA therapeutics.

The company’s objectives including doubling its production capacity by 2022, expanding clinical batch production until 2025, producing clinical batches on a large scale, and provide long-term support to its gene and cell therapy customers.

CEVEC granted UCB rights and options to the ELEVECTA Technology for the research, development, and manufacturing of AAV gene therapy products.

Homology Medicines focuses on the drug product, specifically through development of formulations that enable high titers and long-term stability, thereby relieving pressure on clinical storage and supply chains.

A Stanford University-led research team has developed a compact, efficient CRISPR-Cas system which is about half the size of existing CRISPR-Cas systems, and could have broad utility for gene therapy applications as well as cell engineering, as it is easier to deliver to cells. The researchers confirmed in experiments that CasMINI could, just like its larger counterparts, delete, activate, and edit target gene sequences.

Bermingham has enjoyed a front-row seat for the conception and launch of several dynamic biotech companies. Perhaps the best known is Intellia Therapeutics, one of the first wave of public CRISPR gene editing companies. Intellia celebrated a major milestone in June when it published results on the first half-dozen patients receiving an in vivo gene editing therapy for ATTR amyloidosis, a rare liver disease.

CRISPR systems that rely on inactivated Cas enzymes—that is, dead Cas (dCas) enzymes—never looked more alive. They harness the targeting power associated with CRISPR—but not the double-strand cuts. As such, they give researchers new ways to interrogate and manipulate gene function. Tools for altering gene expression—CRISPRi, CRISPRa, CRISPRoff, and CRISPRon—and CRISPR-compatible cellular models are opening new research possibilities.

Specific and precise base changes in genomic DNA and in RNA offer hope to the millions of people suffering genetic diseases associated with point mutations. As specific as they are tidy, base editing systems spurn excess and embrace adequacy. They do just enough to correct harmful single base mutations.