Development

The Phase IIa trial will further explore the EP4 antagonist HTL0039732 in combination with immunotherapy to improve outcomes in resistant solid tumors and expand treatment options.

SynaptixBio CEO Dan Williams discusses how small biotechs drive rare disease innovation with genetic research, partnerships, and patient advocacy in a third interview installment.

CSL will fund Phase III clinical development of VMX-C001 with an option to acquire VarmX.

A collaboration between Limula and Institut Paoli-Calmettes aims to advance automated stem cell transplant processing to improve cryoprotectant removal, enhance patient outcomes, and streamline manufacturing.

A published study finds that the therapy reported specific and potent cytotoxicity against acute myeloid leukemia, T-cell acute lymphoblastic leukemia, and other rare blood cancers.

Novartis and Monte Rosa expand collaboration using AI-driven molecular glue degraders to advance drug development for immune-mediated diseases.

Telix will now include an additional, confirmatory efficacy study analysis of existing data, hoping to satisfy FDA’s request for supplemental evidence and address concerns raised in a complete response letter.

Data integrity and quality are paramount for drug discovery, manufacturing efficiency, regulatory compliance, and patient safety.

Integra has secured €10.7 million (US$12.6 million) in funding for its FiCAT gene writing platform to enhance precise DNA integration for CAR-T engineering and rare disease therapy development.

Experts to share strategies on scale-up, process intensification, and data-driven methods shaping future stem cell manufacturing and therapies.

FDA’s fast track designation may speed VMX-C001’s application review, addressing urgent surgery needs in patients on Factor Xa anticoagulants.

The partnership, which has received £118 million (US$158 million) in research funding, aims to establish a better understanding not only of how the body fights infection, but also how vaccines protect it.

A joint venture between the two entities will integrate mRNA synthesis, LNP formulation, and clinical testing to speed therapies from plasmid design to first-in-human trials.

Aptamer will create Optimer binders for Invizius’ H-Guard technology to enhance complement-targeting therapies for safer, more precise treatments for immune disorders.

The partnership expands patient access in MENA by localizing biosimilar manufacturing and distribution for gastro, neuro, and dermatology treatments.

Webinar Date/Time: Wed, Sep 24, 2025 11:00 AM EDT

Circular RNA offers enhanced stability and protein expression, making it an intriguing next-generation alternative to mRNA.

SynaptixBio CEO Dan Williams notes how rare-disease drug development is advancing on the back of FDA incentives, priority review vouchers, and a growing recognition of unmet needs.

Webinar Date/Time: Thu, Sep 25, 2025 11:00 AM EDT

Benjamin McLeod and Sebastian Lykke Skafte Andersen go behind the headlines to examine the motivations behind recent M&A activity, tariff responses, and more.

In partnership with AstraZeneca, the company previously announced approval by FDA in January 2025 for the same indications.

The agreement expands researcher access to backbone modifications that boost stability and delivery of RNA therapeutics across diverse applications.

Orlynvah is the first new, branded product for the treatment of uncomplicated UTIs to be introduced in the US in more than 25 years.

Webinar Date/Time: Tue, Sep 16, 2025 11:00 AM EDT