4D's Fabry gene therapy marred by adverse events

Today's Big News

Jan 11, 2023

JPM23, Day 2: BioNTech goes after symptomatic COVID, Editas plots course after shake-up


JPM23, Day 3: Takeda relishing in-person meet-ups


4D's Fabry gene therapy flounders amid adverse events, further weakening Sangamo's competition


JPM23: Editas touts skinnier pipeline after major shake-up to ride out 'unfriendly' markets


JPM23: BioNTech not giving up on fighting symptomatic COVID as oncology ambitions spread


JPM23: Sage CEO says postpartum depression is no niche, as zuranolone aims to fill gaps in mental health care


JPM23: Karuna CEO says biotech can launch drugs, too, as deal rumors circulate


BioXcel, keeping cards close to its chest, teases phase 2 Keytruda combination trial data drop


Stopping 'clock' genes halts liver cancer growth

 

Featured

JPM23, Day 2: BioNTech goes after symptomatic COVID, Editas plots course after shake-up

All you need to know from the second day of the J.P. Morgan Healthcare Conference.
 

Top Stories

JPM23, Day 3: Takeda relishing in-person meetups

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

4D's Fabry gene therapy flounders amid adverse events, further weakening Sangamo's competition

4D Molecular Therapeutics has become the latest developer of a Fabry disease gene therapy to run into difficulties. After seeing three cases of a kidney condition in the first six patients, the biotech has decided to stop enrollment and collect more follow-up data before deciding on the next steps.

How MRD is accelerating effective use of biologic therapies

Advanced ctDNA analysis yields biomarkers of cancer dynamics essential in therapeutic monitoring & intervention. Learn how NeXT Personal® detects MRD, monitors variants, & delivers powerful insights.

JPM23: Editas touts skinnier pipeline after major shake-up to ride out 'unfriendly' markets

Gilmore O’Neill has certainly made an impact in his first six months as CEO of Editas Medicine, culminating in a major pipeline clear out and 20% workforce layoffs this week. But judging by his JPM comments, we shouldn’t expect things to quieten down at Editas anytime soon.

JPM23: BioNTech not giving up on fighting symptomatic COVID as oncology ambitions spread

BioNTech, the famed maker of the COVID vaccine alongside partner Pfizer, is not giving up on developing an updated shot that protects against infections. The update from CEO Ugur Sahin comes as the company's pipeline has grown to focus on oncology.

JPM23: Sage CEO says postpartum depression is no niche, as zuranolone aims to fill gaps in mental health care

Sage Therapeutics is trying to pick up pieces of the depression market that have long been left behind, including patients who are not fully served by existing meds and postpartum mothers who have never had an easy, fast option to try.

JPM23: Karuna CEO says biotech can launch drugs, too, as deal rumors circulate

Karuna Therapeutics is 24 months out from a hotly anticipated schizophrenia drug launch and the industry is buzzing with rumors that the biotech will seek a Big Pharma partnership or other deal for commercialization.

BioXcel, keeping cards close to its chest, teases phase 2 Keytruda combination trial data drop

BioXcel Therapeutics has reported “promising” top-line data including an “encouraging” response rate in its phase 2 Keytruda combination clinical trial. What is promising and encouraging about the data? That remains under wraps, with the biotech opting against providing even a smidgen of information ahead of the presentation of the full data next month.

Stopping 'clock' genes halts liver cancer growth

Scientists have figured out how disrupted cellular circadian rhythms lead to liver cancer and have shown that by inhibiting key “clock” proteins, they can stop it from growing.

ClinOne and Kayentis in pact to address DCT, hybrid trial needs

ClinOne, which develops trial site platforms, and Kayentis have inked a partnership deal in a move to address the growing needs of decentralized clinical trials and hybrid or virtual component trials.

JPM23: Is GSK’s 15-day accelerated approval withdrawal the new normal? Hear FDA commissioner Califf’s response

After GSK quickly pulled its accelerated approval for Blenrep after a confirmatory trial failure, FDA commissioner Robert Califf has weighed in on the state of the regulatory pathway.

JPM23: Ascension Health details $2.5B economic recovery plan to rebound from financial, operational headwinds

SAN FRANCISCO—Ascension Health has charted a $2.5 billion economic recovery plan that executives say has helped the health system to weather ongoing operational challenges and staffing and inflationary pressures.

JPM23: With new production lines, ResMed aims to meet CPAP machine demand amid Philips' ongoing recall

Investments in international manufacturing facilities and component contracts are setting ResMed up to better meet patient needs in 2023 and beyond.

JPM23: Sanofi has reached the 'steady state' Paul Hudson envisioned years ago, CEO says

Sanofi has two launches and 27 readouts planned for this year, and CEO Paul Hudson wants to see those numbers again. During a presentation at the J.P. Morgan Healthcare Conference, Hudson said that this is "the new cadence" for the company.

JPM23: Dexcom CEO on 'the chaos of success' and the G7 sensor's simultaneous global rollout

SAN FRANCISCO—"There’s nothing more fun than the chaos of success." Dexcom CEO Kevin Sayer discussed the worldwide launch of the newly FDA-cleared G7 glucose monitor with Fierce Medtech at the J.P. Morgan Healthcare Conference on Tuesday.
 
Fierce podcasts

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'The Top Line': 2022's rotten tomatoes, plus this week's news

This week on “The Top Line,” we discuss the biotech industry’s rotten tomatoes. Plus, we cover the drug approvals of last year, a congressional report on Biogen's "inappropriate" communications with the FDA and other top headlines from this week.

 

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Medical Affairs Metamorphosis V: The (R)evolution in Medical Communications

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Formulation Strategies When Transitioning From Vial to PFS

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Clinical Supply Solutions for the Asia-Pacific Region

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Supply Services to Support Clinical Trials in Japan & Beyond

Explore Catalent’s newest clinical supply facility in Japan, including the comprehensive services & solutions it offers to help support trials both in-country & around the world.
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A Strategy to Standardize Process Characterization & Late Phase Development

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