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Today’s Big News

Dec 19, 2023

Benefit of uniQure's Huntington's gene therapy will be revealed 'in the fullness of time'

Gilead downloads solid tumor program from Compugen for $60M upfront

Otsuka pays $65M cash for European rights to Ionis’ phase 3 rare disease med

Kronos discontinues mid-stage AML trial after poor patient response

FDA rejects Checkpoint's skin cancer candidate over issue at contract manufacturer

Featured

Benefit of uniQure's Huntington's gene therapy will be revealed 'in the fullness of time'

By Annalee Armstrong

uniQure’s Huntington’s disease data were deemed “noisy and difficult to interpret” in June. While a new slice of interim results from the phase 1/2 trial may not help cut through the noise, the principal investigators says efficacy signals are “on the favorable side.”

Gilead downloads solid tumor program from Compugen for $60M upfront

By Annalee Armstrong

Gilead Sciences is uploading $60 million to Compugen’s bank account plus hefty milestones down the road in exchange for the rights to a preclinical IL-18 binding protein program that includes a solid tumor asset.

Otsuka pays $65M cash for European rights to Ionis’ phase 3 rare disease med

By Gabrielle Masson

Otsuka is paying $65 million upfront for exclusive European licensing rights to Ionis' donidalorsen, a treatment under development for a rare genetic disease known as hereditary angioedema.

Kronos discontinues mid-stage AML trial after poor patient response

By Gabrielle Masson

After a mid-stage flop, Kronos Bio is discontinuing a cancer trial for lanraplenib and seeking a partner to continue development of the SYK inhibitor—the second acute myeloid leukemia (AML) candidate to disappoint the biotech.

FDA rejects Checkpoint's skin cancer candidate over issue at contract manufacturer

By Kevin Dunleavy

In July, for Checkpoint Therapeutics, it was all about complete response rates adding momentum for its promising skin cancer candidate. Five months later, a complete response letter (CRL) has flipped the script on cosibelimab. On Monday, Checkpoint said that the U.S. regulator had rejected cosibelimab because of findings during an inspection of a facility of its third-party contract manufacturer.

After missing out on FDA voucher, bluebird quickly raises funds to support gene therapy launches

By Eric Sagonowsky

Bluebird's FDA approval for sickle cell disease gene therapy Lyfgenia left something to be desired because the agency rejected a request for a priority review voucher. Without that voucher, bluebird is moving fast to try to fund its launches.

Most doctors have not yet tried AI but are 'cautiously optimistic' about the benefits

By Heather Landi

Among those who are holding off, doctors report lack of confidence in accuracy and a lack of integration or access through existing tools — as key obstacles to adoption.

Apple plans to pause US smartwatch sales ahead of Biden ruling on pulse oximetry tech ban

By Andrea Park

With only a week left in President Biden’s review of a proposed import ban on Apple Watches containing a pulse oximetry feature, Apple is preemptively complying with the sales stoppage.

Fierce podcasts

Don’t miss an episode

'Podnosis': Breaking down barriers with adaptive recreation

Children’s Specialized Hospital, part of RWJBarnabas, is providing recreation, sports and social programs for kids with special healthcare needs. This week on "Podnosis," Anastassia Gliadkovskaya sat down with Matthew McDonald, M.D., the hospital’s president and CEO, to discuss what it takes to offer these services in-house and the future of pediatric care.

'The Top Line': A closer look at the Fierce 50

'The Top Line': Turning down the toxicity of nature’s most powerful antifungal

Resources

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