BioMarin plans hemophilia A filing as durability doubts remain
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Today's Rundown

Featured Story

JPM, Day 1: Bayer strikes a Mammoth deal, no Sunday buyouts, Pfizer delves deeper in gene editing and more

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

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Top Stories

JPM 2022: Pfizer locks in 2 partners to solidify status as mRNA powerhouse

Pfizer is determined to become an mRNA powerhouse, and it’s signing on two partners in that quest, including one that supplied tech that helped create the COVID-19 vaccine.

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BioMarin's hemophilia A gene therapy hits goal in phase 3, teeing up FDA filing despite durability doubts

Sixteen months after a stinging FDA rejection, BioMarin Pharmaceutical looks to have the data it needs to win approval for its hemophilia A gene therapy in the U.S. The study found bleeding episodes remained very rare two years after the administration of valoctocogene roxaparvovec (ValRox).

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JPM 2022: Bayer taps CRISPR science from Doudna's lab in $1B biobucks Mammoth gene therapy deal

Bayer is adding more building blocks to its cell and gene therapy ambition by tapping Mammoth Biosciences' CRISPR gene editing toolbox in a strategic collaboration with $40 million upfront and potentially worth over $1 billion.

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Maze Therapeutics navigates $190M financing round as it plots course to the clinic

Fierce 15 winner Maze Therapeutics just added $190 million to its fundraising pile in time to help its lead candidate, MZE001 for Pompe disease, make a clinical debut. Since its launch in 2019, the company ginned up approximately $400 million in investor cash.

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Novartis splashes cash on COVID-19 antiviral, plans FDA filing as Molecular Partners links drug to drop in hospitalization, death

Novartis is coming for the COVID-19 antiviral market. After failing in hospitalized patients, Molecular Partners linked ensovibep to a 78% risk reduction in an earlier-stage population, prompting Novartis to pay 150 million Swiss francs ($163 million) for an asset that could soon add to the anti-omicron arsenal. 

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JPM 2022: Moderna finds Carisma's macrophage tech compelling enough for $45M cancer research deal

Moderna is doling out a little bit of its COVID-19 vaccine cash in a $45 million oncology research deal with Carisma Therapeutics to develop up to a dozen targets.

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JPM 2022: Acadia to Stoke up pipeline with $60M central nervous system research pact

Acadia is looking to Stoke Therapeutics to breathe some air into its pipeline, with a $60 million upfront research pact to develop drugs for rare genetic diseases of the central nervous system. The upfront fee is accompanied by up to $907 million in milestones and also royalties down the line for Stoke.

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JPM22, Day 1: Bristol Myers Squibb's blockbuster losses of exclusivity, Novartis' spending plans and more

Boot up those computers and get your J.P. Morgan healthcare conference meeting links ready, because it's set to be a week full of biopharma action.

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Fierce Healthcare's Fierce 15 of 2022

Meet the winners of Fierce Healthcare's Fierce 15 of 2022.

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MeMed secures $93M to deliver tests for identifying viral, bacterial infections

While most infectious disease tests search a sample for the presence of a particular pathogen, MeMed’s diagnostic collects evidence from the immune system itself and helps identify the culprit by gauging the body’s response to an invader.

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JPM, Day 1: Teladoc 2021 revenue to hit $2B, R1 RCM picks up Cloudmed in deal valued at $4.1B and more

Advocate Aurora gave an update on the non-profit hospital chain's efforts to pivot to a consumer-related healthcare company, including new acquisitions in technology providers.

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Resources

Whitepaper: Tailored, Dedicated Support Expedites COVID-19 Vaccine Trial

Sites receiving augmentation support enroll more than 30,000 patients in just over three months. See how additional site resources can make a difference.

Research: How Buffer Prep Services Alleviate Sudden Spikes in Campaign Volume

Learn how a CDMO partnered with Thermo Fisher Scientific to manage high-volume process liquid and buffer preparation scale-up. Download the free case study from Thermo Fisher Scientific

Whitepaper: QC for ex vivo manipulated cell lines and tissues

A variety of genetic analysis methods are valuable in ensuring that ex vivo human cells to be used for cell-based therapies are correctly identified, have the correct characteristics, and are free of contaminants.

Webinar: Using Next Best to Elevate your Omnichannel Marketing Strategy

Learn how to elevate your omnichannel marketing strategy with Next Best - a combination of data and analytics, deployed to facilitate decision making.

Webinar: The Faster Path From Pre-Filled Syringe to Auto-Injector

Ypsomed and Catalent Biologics experts discuss strategies for integrated PFS manufacturing and auto-injector assembly to accelerate timelines and more in this webinar.

Whitepaper: Formulation Strategies When Transitioning From Vial to PFS

Learn how formulation development for a PFS affects manufacturing to drive the industry toward optimizing delivery of injectable products and, ultimately, improving patient care.

Whitepaper: Enhance the cost-effectiveness of your cGMP chemical supply chain

Learn how to develop a high-performing supply chain for cGMP chemicals used throughout your biologics workflow. Download the free whitepaper from Thermo Fisher Scientific

eBook: Get solutions to market faster

Read Get to Know Cloud Content Management for Life Sciences and learn how to eliminate silos across all workflows, from R&D to commercialization.

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