DMD gene therapy death examined

Today's Big News

May 19, 2023

Moderna, looking beyond vaccines, guides first mRNA protein replacement therapy past early clinical test


DMD gene therapy death exposes risks of treating older patients


ASGCT 2023: Rocket’s hematology programs blast off toward regulators 


Fierce Biotech Fundraising Tracker '23: Myeloid snags $73M A2; Ray raises $100M


Fierce Biotech M&A Tracker 2023: Biodexa to acquire oncology biotech Varian; Gilead nabs PARP1 specialist, Bellus


Chutes & Ladders—Gilead finds Christi Shaw replacement, taps Roche exec to fly Kite

 

Featured

FDA committee backs Pfizer's RSV shot for maternal population, though safety concerns remain

Pfizer’s RSV shot has won the support of an FDA advisory committee for use in pregnant people to protect infants, but lingering concerns remain from a few members that couldn’t back the vaccine’s safety.
 

Top Stories

Moderna, looking beyond vaccines, guides first mRNA protein replacement therapy past early clinical test

Moderna has early clinical evidence that its mRNA technology can treat a rare disease by replacing intracellular proteins. Participants in the phase 1/2 trial experienced a 66% overall relative risk reduction in the frequency of life-threatening events, encouraging the biotech to keep studying the candidate.

DMD gene therapy death exposes risks of treating older patients

Cure Rare Disease has shared a deep dive into the death of the only participant in a gene therapy trial. The nonprofit and its collaborators tied the death of the Duchenne muscular dystrophy patient to their immune reaction to the viral vector, raising concerns about dosing older, more advanced people.

Understanding Venture Debt Funding for Life Sciences Companies

Venture debt is a type of debt financing obtained by early and growth-stage companies and typically used as a complementary method to equity financing.

ASGCT 2023: Rocket's hematology programs blast off toward regulators

As the American Society of Gene & Cell Therapy annual meeting draws to a close, Rocket Pharmaceuticals spotlighted three ongoing clinical programs for patients with blood disorders, two of which are nearing regulators’ desk. 

Navigating uncertainty in modern clinical development

Developing a new life-saving drug or medical device is fraught with challenges. But while challenges often vary from program to program, one in particular is guaranteed: uncertainty. In this article, Dr Kim, CMO at Cytel, discusses the nature and consequences of the uncertainty challenge, as well as the options available to help pharmaceutical and biotechnology companies navigate it.

Fierce Biotech Fundraising Tracker '23: Myeloid snags $73M A2; Ray raises $100M

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

Fierce Biotech M&A Tracker 2023: Biodexa to acquire oncology biotech Varian; Gilead nabs PARP1 specialist, Bellus

Fierce Biotech's M&A Tracker, your one-stop shop for dealmaking in the biopharma universe.

SCOTUS hands win to Sanofi, Regeneron in long-running PCSK9 feud with Amgen

After numerous twists and turns, a highly divisive patent infringement case between Amgen and partners Sanofi and Regeneron has come to an end at the U.S. Supreme Court.

Abbott snags FDA nod for insertable cardiac monitor that can stay in place for up to 6 years

Many insertable cardiac monitors, known as ICMs or loop recorders, are already able to stay in place under the skin for several years at a time, enabling long-term tracking of irregular heartbeats—but Abbott has upped the ante.

A key House panel approved a slew of health policies. Here's a look at 4 of them

A key House panel advanced a slew of healthcare bills during a busy Wednesday on the Hill for health policy.

'The Top Line': 2023's biopharma layoffs, plus this week's headlines

This week on "The Top Line," we''ll discuss the recent biopharma layoff trends, plus Elizabeth Holmes, Sarepta Therapeutics, Abbott's FDA approval, and the rest of the week's headlines.

Chutes & Ladders—Gilead finds Christi Shaw replacement, taps Roche exec to fly Kite

Gilead taps longtime Roche exec to lead Kite. Billy Dunn exits FDA's revolving door. Recursion names a new CMO.

Fierce Pharma Asia—Takeda's 'challenging' year; AZ's cancer buy; Astellas' menopause drug nod

Takeda is bracing for two important patent expirations this year. AstraZeneca has in-licensed an antibody-drug conjugate from Chinese company LaNova Medicines. Astellas' menopause drug fezolinetant has won a delayed FDA approval. And more.
 
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'The Top Line': 2023's biopharma layoffs, plus this week's headlines

This week on "The Top Line," we''ll discuss the recent biopharma layoff trends, plus Elizabeth Holmes, Sarepta Therapeutics, Abbott's FDA approval, and the rest of the week's headlines.
 

Resources

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ChatGPT in Drug Discovery: Rise of Large Language Models

ChatGPT shows promise in drug discovery for information extraction, scientific text authoring, hypothesis prediction, and chemical entity prediction. This whitepaper explores its potential to streamline research, delving into its technology, applications, and limitations.
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Discovering High-Affinity, Functional Anti-GLP-1R Antibodies

Learn how a GPCR-focused library with a cell-based biopanning strategy led to the discovery of 13 high-affinity and selective anti-GLP-1R antibodies with highly developable therapeutic properties.
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Decentralized Clinical Trials: The Complete Guide for 2023

Discover a game-changing approach to clinical research with Hybrid and Decentralized Clinical Trials (DCTs). Learn about the benefits, challenges, and key elements for success.
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How Lilly’s Legal and Library Services Empower Teams Across the Drug Development Pipeline

What role do the Library and Legal teams play in the drug development pipeline at Lilly? An import one – find out how.

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Medical Affairs Metamorphosis VI: The Changing Face of Stakeholder Engagement

This paper focuses on stakeholder engagement and how Medical Affairs teams’ responsibilities, goals, tools, and techniques are changing.
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Maximizing Clinical Trial Success

Strategies for Optimal Site Selection and Feasibility Research in Novel Therapies and Geographies
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Running Decentralized Trials at Scale

Explore how clinical supply can be scaled up as decentralized programs progress, and how technological solutions can accelerate trial deployment.
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Key Clinical Supply Issues Sponsors Should Consider with their CRO

As sponsors increasingly look to CROs to support their clinical studies, making and addressing certain clinical supply considerations at the outset of the relationship can reduce the risk of potentially unforeseen impact to budget and time later. Discover key issues sponsors should consider to help their CRO or partner of choice better understand their clinical supply needs.

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Specialized Expertise for Management of Clinical Supply Budgets

For sponsors, cost is a key concern when it comes to managing the supply of their clinical trials. A financial project analyst (FPA) is a specialized expert who can help control clinical supply costs and keep the project within budget. Explore the critical role that a Financial Project Analyst plays in clinical trial supply and budget management.
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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.
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Designing an Optimal APAC Clinical Supply Chain

Discover key considerations that every trial sponsor should know when navigating the complex clinical supply distribution landscape across Asia-Pacific.
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Roles and Responsibilities of Specialized Clinical Supply Experts

How can specialized expertise help you optimize your clinical supply chain? Learn about the roles and responsibilities of unique experts such as clinical supply management, project management, and financial project analysis.
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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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Questions You Need to Ask Your CRO About Clinical Supply

Planning on using a CRO to support your next clinical study? This infographic outlines critical areas to address at the start of the relationship in order to ensure that budget and timelines keep on track for the duration of the trial.
 

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