A few weeks after Jennifer Doudna introduced CRISPR/Cas9 genome editing to the world, one of her old students decided to take the central part of the biology-altering invention and kill it.
CRISPR/Cas9, as the name implies, is a two-part system: a string of letters called a guide RNA, that says where to cut the DNA. And an enzyme, Cas9, that does the cutting. Often compared to molecular scissors, it was the first system that allowed researchers to cut DNA with ease and precision, promising potential cures for genetic diseases such as sickle cell and cystic fibrosis.
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For years, paper-based processes and individual point solutions dominated the clinical research landscape, and patient participation in clinical trials was largely an in-person engagement. But when the COVID-19 pandemic took a stronghold, traditional clinical trial methods emerged as inadequate, putting clinical trials and the life sciences industry at a crossroads. Practically overnight, the industry had to rapidly shift to decentralized clinical trial methods, while maintaining data quality and regulatory compliance.
Al Sandrock (Biogen via Youtube)
Two years after Al Sandrock jumped from CMO to the top post in R&D — and just months after the hyper-controversial approval of the experimental Alzheimer’s drug aducanumab (Aduhelm) — Sandrock is planning to step out of his long career at Biogen.
Late Monday evening the big biotech put out word that Sandrock, a longtime fixture in the company after a 23-year stint, is hitting the exit.
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Following a similar deal forged by Merck for its Covid-19 drug molnupiravir, Pfizer on Tuesday agreed to allow the UN-backed Medicines Patent Pool to license the company’s potential treatment Paxlovid to make it more readily available in 95 low- and middle-income countries.
The agreement allows MPP to grant sub-licenses to qualified generic drug manufacturers to facilitate additional production and distribution of the investigational antiviral, which has yet to be authorized.
Michael Yang, ViaCyte CEO
The race to develop a cell therapy for type 1 diabetes is heating up, and the team at ViaCyte and CRISPR Therapeutics appears to have pulled ahead by a hair.
ViaCyte announced Tuesday it’s putting a new therapy in the clinic derived from ‘off-the-shelf,’ gene-edited, immune-evasive stem cells, the first time such a treatment will be tested in humans. The move comes after its clinical trial application was accepted by Canadian regulators, and patient enrollment is expected to start by the end of the year.
As Merck KGaA makes moves to treat the neglected tropical disease schistosomiasis, it has announced that its treatment arpraziquantel has yielded positive Phase III trial results in children between the ages of 3 months and 6 years old, and the company will now seek regulatory approval.
A pediatric version of the standard drug praziquantel, arpraziquantel is an anti-worm medication that prevents newly hatched, parasitic worms from growing or multiplying inside a patient. The trial could offer a cure for millions.
Glen de Vries (Patrick T. Fallon/AFP via Getty Images)
Glen de Vries, the co-founder of the clinical IT software giant Medidata Solutions, died in a plane crash last week.
Emergency crews found the wreckage of a Cessna 172 in a wooded area in northern New Jersey on Thursday. De Vries was an instrument-rated private pilot, though authorities have not yet said who was piloting the plane. He was with his flight instructor Thomas Fischer, 54, and the plane was headed to Sussex Airport from Essex County Airport in Caldwell. He had started his private pilot training with Fischer in February 2016. Fischer opened the flight school with his wife Jodi in March 2012.
Chiquita Brooks-LaSure (Photo by Caroline Brehman/CQ Roll Call via AP Images)
Although sales of Biogen’s expensive new Alzheimer’s drug have been anemic since the approval in June, the prospect of CMS eventually paying for it opens up a billion-dollar can of worms, and already has the agency defending some premium and deductible increases for seniors.
CMS explained late Friday that Medicare Part B will have to increase its standard monthly premium — from $148.50 in 2021 to $170.10 in 2022 — in part because of the massive spending that could occur should the agency sign off on a national coverage decision for the drug, known as Aduhelm, and its $56,000 annual price tag next year.
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UK-based AI-assisted R&D biotech Synthace just wrapped up a Series C round, bringing in $35 million.
Per a company statement this morning, the investment will fuel commercial and go-to-market (GTM) growth as Synthace continues to expand across the Atlantic into the US. This includes plans to expand its leadership team by bringing in new VPs of marketing and sales to join US-based Guy Levy-Yurista, Synthace’s CEO.
Patrick Amstutz, Molecular Partners CEO
Molecular Partners CEO Patrick Amstutz knew going into the NIH’s ACTIV-3 study that the bar for ensovibep was ‘very high.’ A slew of Covid-19 antibodies had already flunked out of the trial — and though their Novartis-backed antibody alternative was different, he expressed cautious hope.
On Tuesday, however, he revealed that ensovibep didn’t clear that bar.
Ensovibep’s ACTIV-3 days are over after failing a futility analysis in hospitalized Covid-19 patients, the company announced. The molecule comes from a class of drugs developed by Molecular Partners that aims to perform the same functions as antibodies with far more target specificity and antiviral protection.
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