Ultragenyx adjusts gene therapy dosing to boost efficacy

Today’s Big News

Oct 4, 2024

J&J drops phase 2 dengue candidate as part of ongoing move away from vaccines


Relay cuts more staff in efforts to save $50M a year


Ultragenyx tweaks gene therapy dosing to dial up efficacy after seeing signs of activity


Editas cashes in on portion of Vertex gene editing licensing agreement for $57M


Chutes & Ladders—Prothena promotes one exec while another leaves


Gene therapy pioneer Jim Wilson's Franklin Biolabs launches with suite of genetic medicine services


Breaking down BIOSECURE—Industry implications and what comes next

The Top Line Podcast: Don’t miss out on the newest episode. Listen now.

 

Featured

J&J drops phase 2 dengue candidate as part of ongoing move away from vaccines

Johnson & Johnson’s deprioritization of its infectious disease pipeline has claimed another victim in the form of its dengue virus vaccine mosnodenvir.
 

Top Stories

Relay cuts more staff in efforts to save $50M a year

Precision medicines biotech Relay Therapeutics is shedding about 10% of its workforce in efforts to streamline the organization.

Ultragenyx tweaks gene therapy dosing to dial up efficacy after seeing signs of activity

A minority of patients taking Ultragenyx Pharmaceutical’s Wilson disease gene therapy UX701 have come off standard-of-care drugs, leading the biotech to enroll a new cohort on a tweaked regimen designed to dial up the efficacy.

Editas cashes in on portion of Vertex gene editing licensing agreement for $57M

Against the backdrop of a Cas9 patent battle that refuses to die, Editas Medicine is cashing in a chunk of the licensing rights from Vertex Pharmaceuticals to the tune of $57 million.

Chutes & Ladders—Prothena promotes one exec while another leaves

Prothena, a company focused on neurodegenerative diseases, is shaking up its C-suite. The exec moves were announced just a day after the science underlying Prothena’s Parkinson’s drug prasinezumab was implicated in a massive research misconduct scandal but are unrelated, a company spokesperson told Fierce in an email.

Gene therapy pioneer Jim Wilson's Franklin Biolabs launches with suite of genetic medicine services

Gene therapy icon Jim Wilson, M.D., Ph.D., made waves in August when he announced that he was leaving the University of Pennsylvania after more than 30 years to create—and lead—two new biotech companies. One of those companies, a contract research organization focused on genetic medicines called Franklin Biolabs, officially launched this week.

Breaking down BIOSECURE—Industry implications and what comes next

This week on "The Top Line," Fierce Pharma’s Fraiser Kansteiner and Arnold & Porter’s Dan Kracov break down the BIOSECURE Act's potential impact on the life sciences industry and what comes next after the House of Representatives' recent vote.

In sustained rare disease push, Recordati lays out $825M upfront to acquire Sanofi's CAD drug Enjaymo

Recordati is floating $825 million upfront—plus another $250 million in potential commercial milestones—to get its hands on the global rights to Sanofi’s rare disease biologic Enjaymo. In 2022, Enjaymo became the first and only therapy specifically approved by the FDA for treating patients with the uncommon blood disorder cold agglutinin disease.

Leading cancer centers launch Cancer AI Alliance with $40M from big tech

Four leading cancer centers are leveraging their data resources and AI to gain ground on cancer.

Fierce Pharma Asia—Roche's $850M-plus deal with Regor; Legend's busy week; WuXi asset sales?

On the same day as Roche's Pharma Day in London, the drugs giant struck a high-dollar deal with Chinese-American biotech Regor for the latter's CDK inhibitors. Legend's momentum continued with a strong survival showing for Johnson & Johnson-partnered Carvykti and the announcement of a planned R&D facility in the U.S. WuXi AppTec and WuXi Biologics are considering hiving off certain assets amid biosecurity scrutiny. Plus more.
 
Fierce podcasts

Don’t miss an episode

Breaking down BIOSECURE—Industry implications and what comes next

This week on "The Top Line," Fierce Pharma’s Fraiser Kansteiner and Arnold & Porter’s Dan Kracov break down the BIOSECURE Act's potential impact on the life sciences industry and what comes next after the House of Representatives' recent vote.
 

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On Helix

Date: ‌4 ‌July ‌2024 ‌- ‌Location: ‌Babraham ‌Research ‌Campus, ‌Cambridge ‌UK

 

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