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Tools & Techniques

NCATS, FDA meeting shines light on AAV manufacturing innovation

Technologies highlighted at a NCATS-FDA AAV manufacturing workshop could ramp up the production and durability of gene therapies, but questions remain about who will test drive these innovations and where the next wave of them...

Beyond PROTACs and the proteasome: broadening the TAC toolbox

The idea of sending disease-driving proteins to the cell’s garbage can is only the first act in a field that now centers around targeted degradation. In the sequel, researchers are extending the principle to other...

Insilico offers AI-based chemistry to coronavirus response

Insilico Medicine Inc. became the second AI company this week to contribute the speed of its platform to biopharma’s mass mobilization against the 2019-nCoV crisis. As the debate continues about whether AI can transform medicinal...

Cellular fluorescence assay to assess allosteric modulator potency in drug discovery

Karolinska, Pasteur and UCSD researchers have developed a cellular fluorescence assay that can efficiently screen for potent transcription factor allosteric modulators during lead identification. Unlike compounds that work by binding targets’ active sites, allosteric modulators...

Rosenberg study opens wider door to personalized TCR therapies for cancer

A new study from TIL pioneer Steve Rosenberg adds to the momentum of personalized T cell therapies by suggesting blood can be as good a source of neoantigen-specific TCRs as tumor tissue. The results, which...

Mammoth eyes CRISPR-based therapeutics with new funding

With a $45 million series B round led by Decheng Capital, Mammoth Biosciences is capitalizing on its IP position around an alternative CRISPR enzyme and expanding its gene editing capabilities beyond diagnostics into a range...

Manufacturing roundup: Deerfield launches new CDMO, plus a gene/cell therapy JV and more

Manufacturing remains a major hurdle for companies pursuing gene and cell therapies, and as more products make it to market, the bottleneck could create problems with commercial supply. At least seven manufacturing deals and site...

Kintai reveals preclinical validation for its microbiome-inspired small molecules, prioritizes obesity

The first set of preclinical data from Kintai’s microbiome-derived small molecule platform supports the idea that manipulating the enteric signaling network with small molecules is one way to tap into the microbiome’s links to human...

JV to combine technologies into one gene and cell therapy manufacturing platform

Two manufacturing deals Tuesday underscore manufacturing's vital role in the development of next-generation gene and cell therapies. The first, a joint venture between Bio-Techne, Fresenius Kabi and Wilson Wolf, will combine the partners' scalable technologies...

Disney lab uncovers another RNA-binding small molecule for triple-negative breast cancer

Matthew Disney is attacking triple-negative breast cancer again with the latest iteration of his RNA-binding small molecule technology, which marries the concept with targeted RNA degradation to eliminate disease-causing RNA segments. In a paper published...

EPO decision strengthens UC’s CRISPR patent position in Europe as Editas looks to Cas9 alternatives

A decision at the European Patent Office has confirmed that the CRISPR IP landscape is going to look much different in Europe than it does in the U.S., contributing to the growing complexity of global...

BioNTech tackles solid tumors by pairing CAR Ts with RNA vaccines

BioNTech is marrying two of its core technologies -- RNA therapeutics and engineered cell therapies -- to solve some of the biggest challenges of bringing CAR T cells to solid tumors. It believes that together,...

Regeneron’s new biologic boosts small molecule binding

Regeneron has expanded its suite of drug discovery technologies by developing a new type of biologic capable of binding small molecules better than conventional antibodies. The company has been seeking new technologies -- such as...

Editing bacterial resistance via gene drives

In a new take on combating antimicrobial resistance, a UCSD team developed a gene drive system that could reduce resistance more efficiently than conventional CRISPR-based editing. CRISPR-based gene drive technologies have been used in diploid...

New hepatic stellate cell subset drives liver fibrosis

A team including University of Edinburgh and Gilead researchers has used single-cell sequencing to zero in on a pathogenic subpopulation of hepatic stellate cells that could be targeted to treat liver fibrosis. Reported in a...