Rocket puts roadblocks in the rearview

Today's Big News

Feb 7, 2023

Roche races to regulators after matching AstraZeneca's rare disease blockbuster in phase 3


Investor Syncona blames bear market's ravaging of British biotechs for ‘disappointing’ returns


Rocket, putting regulatory roadblocks in its rearview, receives show of confidence in gene therapy from FDA


Do we need new Alzheimer's scales, or a new perspective on what's meaningful to patients?


Cell therapy biotech lays off 25% of staff citing difficult funding environment


An anchor protein fine-tunes CAR cells, making them more effective against solid tumors


Fierce Biotech Fundraising Tracker '23: Garuda grabs $62M; IgGenix reels in $40M

 

Featured

Roche races to regulators after matching AstraZeneca's rare disease blockbuster in phase 3

Roche is ready to wade into the increasingly ferocious fight for the paroxysmal nocturnal hemoglobinuria space. The Big Pharma’s subcutaneous contender matched AstraZeneca’s intravenous blockbuster incumbent Soliris in phase 3, positioning it to file for regulatory approvals around the world.
 

Top Stories

Investor Syncona blames bear market's ravaging of British biotechs for ‘disappointing’ returns

Life sciences investment firm Syncona has blamed a “disappointing” drop in returns on plummeting share prices for British biotechs across its portfolio in December.

Rocket, putting regulatory roadblocks in its rearview, receives show of confidence in gene therapy from FDA

Racing toward a pivotal study, Rocket Pharmaceuticals has received a show of confidence from the FDA in the potential of its gene therapy treatment for a fatal genetic cardiac disease.

Do we need new Alzheimer's scales, or a new perspective on what's meaningful to patients?

Scales to assess Alzheimer’s disease are not nearly sensitive enough to pick up on clinically meaningful changes in progression for patients with early-stage disease. So do we throw out the scales and start over? A new study from the Alzheimer’s Association says no: we work with what we’ve got.

Cell therapy biotech lays off 25% of staff citing difficult funding environment

Cell therapy biotech Neurona has laid off a quarter of its staff, or 18 of 68 employees, a spokesperson confirmed with Fierce Biotech.

An anchor protein fine-tunes CAR cells, making them more effective against solid tumors

The new CAR design showed success in mouse models of glioma, bone cancer and lung cancer, making it a must-try in the clinic, the researchers said.

Fierce Biotech Fundraising Tracker '23: Garuda grabs $62M; IgGenix reels in $40M

Alongside the new year, we've launched a new Fierce Biotech Fundraising Tracker to keep the pulse on the industry's financing.

Led by Merck, Vertex and Lilly, pharma's share prices largely climbed in '22 despite economic angst

In a year of economic volatility and uncertainty—and one in which many other industries tanked—biopharma demonstrated its resilience. In all, 11 of the top 20 companies delivered market cap gains of at least 10%, led by Merck (45%), Vertex (33%) and Eli Lilly (32%).

Race plays a role in where patients are delivered by ambulance: study

A statistically significant difference exists when it comes to what hospitals white and nonwhite patients are transported to by ambulance, according to a study published this week in Health Affairs.

Vertex pricing under fire—again—as activists press 4 governments for Trikafta generics

A coalition of cystic fibrosis patients and their families petitioned governments in South Africa, Brazil, India and Ukraine to either revoke or suspend Vertex patents surrounding the company’s tripartite drug Trikafta.

RefleXion overcomes 'unprecedented hurdles' to land FDA nod for biology-guided radiotherapy tech

Just over a year after being recognized by the FDA for the potential “breakthrough” nature of its radiation therapy, RefleXion has gotten the go-ahead from the agency to begin rolling out the system across the U.S.
 
Fierce podcasts

Don't miss an episode

'The Top Line': What we can expect for the obesity market in 2023 and a recap of drugs approved last year

This week on "The Top Line," we discuss what we can expect from the obesity market in 2023. We also recap the drugs approved last year.
 

Resources

Whitepaper

The Power of Small & Safety: Nanoplasmid™️ Vector Platform

Discover the Power of Small with NanoplasmidTM – the next-generation plasmid for manufacturing today’s cell and gene therapies.

Whitepaper

Commercializing Gene Therapies, Part 3 – Cost Drivers

This paper explores the cost drivers behind gene therapies and how they are likely to evolve in the coming years.
Whitepaper

Unlocking the Therapeutic Potential of Previously Undruggable GPCRs

Exploring the biology of G Protein-Coupled Receptors (GPCRs) and outlining a novel approach for developing therapeutics against previously undruggable GPCRs
Whitepaper

Leveraging Archetypes for Effective Launch Strategy

Learn more about critical success factors for your next launch based on the experience of 100+ pharma product launches.
Whitepaper

Reduce control arm sizes by up to 35% for Alzheimer's disease

RCTs with digital twins require fewer control patients and shorten trial timelines for Alzheimer's disease.
Whitepaper

Quality attributes that are important for GMP-grade NTPs

What makes nucleotides suitable for current good manufacturing practices?

On-Demand Webinar

Writing the Future of Antibody Discovery Solutions

See how high-throughput DNA synthesis combined with antibody engineering expertise provides end-to-end antibody discovery solutions.

Whitepaper

Linear Compression vs. Parallel Processing: Speeding Biologics Through Development and Into Clinical Trials

Download this paper to learn considerations on how to move a biologic through clinical trials faster, advantages of Parallel Processing, and more.

Whitepaper

Intranasal Drug Product Development: Formulation to Manufacturing

What are key considerations for intranasal delivery in the treatment of acute and chronic conditions?
Whitepaper

Clinical Supply Solutions for the Asia-Pacific Region

Learn how Catalent’s extensive network across APAC can meet a wide range of clinical supply needs for sponsors conducting trials within the region.
Whitepaper

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.
Case Study

A Strategy to Standardize Process Characterization & Late Phase Development

Learn more on an approach for process characterization for the production of a monoclonal antibody, which will improve the efficiency and effectiveness of the process by targeting the right design and number of experiments.

 

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