RegenXBio plans accelerated FDA request
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Today's Rundown

Featured Story

Yes, Moderna is feeling the pressure to address monkeypox, R&D chief confirms

The pressure is on for Moderna to step up and save the day with a monkeypox vaccine, just as it did for COVID-19. But executives from the famed biotech insist the idea is still in the conceptual phase.

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Top Stories

Roche gets busy at the deal table, wagering $190M upfront on off-the-shelf CAR-Ts and ex-Biogen asset

Roche’s business development team has made a busy start to August. On Wednesday, the Swiss pharma company disclosed deals with Poseida Therapeutics and Kiniksa Pharmaceuticals that are together worth $190 million upfront.

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RegenXBio gets FDA support for Hunter syndrome gene therapy's accelerated review filing—in 2024

It’s going to be a long wait, but RegenXBio is already waving a flag to show that the Hunter syndrome gene therapy RGX-121 is headed for an accelerated approval request at the FDA.

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Clean up on IL-2: Moderna mops up autoimmune asset after peeking at early data

Another quarter, another cull for Moderna. The company snuck into its second-quarter earnings report that development of an mRNA treatment for IL-2-based autoimmune disorders has ended. 

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As cash dips below $50M, Arca kicks up search for strategic options to keep the lights on

Arca Biopharma is down to its last $50 million. With its share price still languishing in the basement, the biotech is pushing ahead with a search for strategic alternatives including the sale of the business.

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Rigel bets $230M that Forma's leukemia drug gets in formation with FDA

Rigel Pharmaceuticals has handed over $2 million in cash for exclusive licensing rights to Forma Therapeutics’ olutasidenib, an oncology treatment that currently awaits an FDA approval decision.

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Allarity, after some forced clarity from FDA, pivots lead asset hopes to combo approach

After a meeting with the FDA, Allarity Therapeutics is abandoning hope that its lead clinical asset could be sold as a monotherapy for kidney cancer.

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Eying Pfizer, Alnylam's high-stakes Onpattro heart trial delivers partial win but misses on key outcomes goal

After an investigational BridgeBio drug surprisingly failed a clinical trial that would have set up a fight against Pfizer in a rare heart condition, investors have been worrying about Alnylam because of a similar study design. Now, Alnylam has returned partially successful.

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Surgical robot maker Virtual Incision heads to space with NASA grant

Armed with a grant from NASA itself, robotic-assisted surgery will soon head for the final frontier.

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Justice Department sues Idaho to reverse 'near-total' abortion ban

The Justice Department is suing Idaho to prevent a near-total abortion ban from going into effect later this month, arguing it conflicts with a federal emergency services law.

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ResMed continues German expansion with acquisition of Mementor's insomnia digital therapeutic

With the pedal firmly to the metal in its U.S. business—thanks in no small part to rival CPAP machine maker Philips’ ongoing recall—ResMed has begun looking beyond its home turf to extend that winning streak.

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Negotiations? AbbVie CEO says current Senate proposal brings 'price controls' instead

While lawmakers are busy debating details of the reconciliation bill that may take a serious stab at drug prices, pharmaceutical companies are hustling out a defense. One notable point? The legislation wouldn't bring drug price negotiations in Medicare, a prominent pharma CEO said late last week. Instead, it would introduce price controls, he said.

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Resources

Event: Drug Development Boot Camp® 2022

The total immersion, transformative training will take place at the Harvard Club in Boston. VIRTUAL in real time is also available. Contingencies for COVID19 will apply. Register NOW for a large early bird discount www.drugstomarket.com/drugbootcamp.

Whitepaper: Genetic Insights for Biopharmaceuticals

See how integration of innovative genetic analysis techniques into drug development is driving biopharma & biotech companies ahead.

Whitepaper: Ensure Commercialization Success with TrialCard/Triangle Insights

Discover how the new partnership between TrialCard and Triangle Insights Group can help you bridge the critical gap between strategy and execution, ensuring commercialization success of your product or therapy.

Whitepaper: Through a CRO's Perspective: Innovative, Agile Solutions In Vaccine Efficacy Testing

Discover Nexelis’ first-hand perspective on how to develop innovative, agile solutions in vaccine efficacy testing in this exclusive e-book.

Whitepaper: The Most Suitable Dosage Form Technologies for the Oral Delivery of Lipid-Based Formulations

What is the most suitable dosage form technology for the Oral Delivery of Lipid-Based Formulations?

Article: 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: Intranasal Drug Product Development: Formulation to Manufacturing

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

Case Study: Versatility of Softgel Technology - Encapsulation of a Volatile Compound

Learn more about how the versatility of Softgel Technology helped a customer with a volatile compound.

Article: Lipid-Based Drug Delivery System to Bring Poorly Soluble Drugs to Market

How can Lipid-Based Drug Delivery System help bring poorly soluble drugs to market?

eBook: Orally Inhaled & Nasal Drug Delivery: A Pipeline of Opportunities

Carla Vozone discusses the significant trends in the OINDP segment including large molecules and novel indications, changes within CDMOs and various challenges facing the industry.

Whitepaper: Catalent Pulmonary & Nasal Delivery Capabilities Sheet

Did you know Catalent has newly expanded in-house capabilities spanning clinical and commercial scale manufacturing for DPIs, unit-dose, bi-dose and preserved multi-dose nasal sprays?

On-Demand 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.

Events