Development

The regulatory progress for AAVantgarde’s AAVB-039 underscores advances in dual-vector gene therapy, as well as scalable manufacturing and efficient rare-disease clinical development.

The study by Integra Therapeutics and partners reveals how AI-engineered proteins could expand gene-editing tools and streamline development of advanced therapies.

On July 31, President Donald Trump said the federal government would “deploy every tool in our arsenal to protect American families from continued abusive drug pricing practices” should companies fail to comply.

This innovative trial guidance accelerates cell and gene therapy development for rare diseases, boosting biopharma efficiency and data maximization.

Webinar Date/Time: Wed, Oct 22, 2025 11:00 AM EDT

Novartis expands its immunology pipeline with the Tourmaline acquisition and tests direct-to-patient model to reshape drug manufacturing strategies.

Formulation and analytics are combining to advance drug development synergistically, with evolving tools and related strategies shaping quality, scalability, and innovation.

The new protein-based HDR enhancer aims to improve CRISPR precision for advancing cell and gene therapy development workflows.

Quantoom will leverage its proprietary LNP formulation technology, and MSK will have the option to expand the license agreement if there are future developments in Quantoom’s library.

Webinar Date/Time: Tue, Oct 14, 2025 10:00 AM EDT

BIO-Europe 2025 in Vienna will gather global biopharma leaders to explore investment, manufacturing, and rare disease innovation.

Pfizer will expand its obesity drug pipeline with Metsera’s clinical incretin and amylin programs, highlighting advances in biopharma development and manufacturing.

ACIP votes to separate MMR and varicella vaccines (MMRV) for children to cut febrile seizure rates.

Webinar Date/Time: Tue, Oct 7, 2025 11:30 AM EDT

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.

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.