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Today's Rundown

JPM22, Day 4: Vertex aims to be cystic fibrosis leader into the 2030s; Gritstone hopes for new colorectal cancer biomarker
FDA denies Denali's Takeda-partnered Alzheimer's drug from entering clinic
Editas, which caught flak in 2021 for limited gene editing data, will try to layer on the proof in 2022
Intellia stacks ATTR trial with new patient group, will weigh later-stage tweaks after BridgeBio setback
Sarepta, with pivotal data in sight, dumps Lysogene gene therapy
AstraZeneca pays Hoos-helmed Scorpion $75M to take the sting out of undruggable cancer targets
Pfizer offers a soft sell but with a strong message on vaccines in first COVID ad
Philips, still hobbled by expanded ventilator recall, braces for Q4 earnings miss
Insurers applaud CMS' proposal for Medicare to only cover Aduhelm for seniors in clinical trials
Fighting leukemia by targeting a little-known protein
AI discovery tech firm Quris completes $28M seed funding round

Featured Story

JPM22, Day 4: Vertex aims to be cystic fibrosis leader into the 2030s; Gritstone hopes for new colorectal cancer biomarker

By Annalee Armstrong,Conor Hale,Kevin Dunleavy,Kyle LaHucik

Join us for a rolling look at the top life science news coming out of this year's virtual J.P. Morgan Healthcare Conference.

FDA denies Denali's Takeda-partnered Alzheimer's drug from entering clinic

By Kyle LaHucik

The FDA has thrown Denali Therapeutics' Alzheimer's med into murky waters with a clinical hold before the drug even enters human trials. The move also impacts Takeda, which just last month moved forward on the pair's 2018 deal by opting to co-develop the drug.

Editas, which caught flak in 2021 for limited gene editing data, will try to layer on the proof in 2022

By Annalee Armstrong

Editas released a tiny drop of data last year as a proof of concept for its gene editing platform. Now, the CRISPR/Cas9 biotech will spend 2022 trying to back that up.

Intellia stacks ATTR trial with new patient group, will weigh later-stage tweaks after BridgeBio setback

By Annalee Armstrong

Intellia has always wanted its gene editing therapy NTLA-2001 to be trialed in all types of the rare disease transthyretin (ATTR) amyloidosis. Now, the CRISPR/Cas9 genome editor has accelerated those plans by slipping cardiomyopathy patients into an existing early-stage study.

Sarepta, with pivotal data in sight, dumps Lysogene gene therapy

By Nick Paul Taylor

Sarepta Therapeutics is walking away from a partnered gene therapy just months before the readout of a pivotal clinical trial. The biotech stuck with Lysogene through a tough 2020 that featured the death of a child who received their partnered gene therapy but has now decided to exit the deal.

AstraZeneca pays Hoos-helmed Scorpion $75M to take the sting out of undruggable cancer targets

By Nick Paul Taylor

The search for ways to drug the undruggable has led AstraZeneca to the door of Scorpion Therapeutics. In return for $75 million upfront, AstraZeneca has landed the chance to work with the Axel Hoos-helmed biotech on drugs against the transcription factors that control gene expression.

Pfizer offers a soft sell but with a strong message on vaccines in first COVID ad

By Sharon Klahr Coey

About a hundred years ago in 2019, “normal” was just shy of an insult—a word synonymous with “boring.” Pfizer knows that these days, “normal” (not counting the dreaded “new” normal) seems like a fantasy land.

Philips, still hobbled by expanded ventilator recall, braces for Q4 earnings miss

By Andrea Park

At the root of Philips’ troubles are the far-reaching ventilator recall, global shortages of electronic components and freight capacity and delays in the installation of its medical devices.

Insurers applaud CMS' proposal for Medicare to only cover Aduhelm for seniors in clinical trials

By Robert King

Payer groups applauded CMS' proposal to onlycover the controversial Alzheimer's drug Aduhelm for patients in clinical trials.

Fighting leukemia by targeting a little-known protein

By Angus Liu

Proteins known as phosphatases are essential to myriad cellular signaling pathways, including those that drive cancer. A research team led by scientists at Cold Spring Harbor Laboratory has just pinpointed a little-known phosphatase that acute myeloid leukemia cells appear to rely on for survival.

AI discovery tech firm Quris completes $28M seed funding round

By Gareth Macdonald

Quris raised $28 million in seed financing to scale its self-training artificial intelligence platform that predicts clinical safety and efficacy of potential new meds. The Boston and Tel Aviv startup wants to improve the drug candidate screening method.