Crowdfunding Biopharmaceutical Innovation

EST-201 and EST-101 target the genetic root cause of ADPKD, a disease affecting more than 8 million people worldwide, with trials starting 2027-2028.

Genglycos, the gene therapy that earned the voucher, will fund new Ultragenyx therapies for other rare and ultra-rare diseases.

When large pharmaceutical companies deprioritize drug candidates, smaller developers may license or acquire them, but due diligence, deal structure, and trial planning determine whether a program can move forward.

AVT29, a proposed aflibercept biosimilar to Eylea 8 mg, could extend retinal disease treatment intervals to 16 weeks, versus 8 weeks today.

Mirikizumab (Omvoh) achieved comprehensive disease control in 27.7% of UC patients and deep remission in 20.4% with Crohn disease at 1 year.

As ArsenalBio, TScan, Bristol Myers Squibb, and others abandon ex vivo programs for in vivo engineering, early clinical data and platform differences point to both the promise and the unresolved safety, manufacturing, and regulatory questions of generating CAR-T cells inside the body.

BNT113 plus pembrolizumab failed to significantly extend progression-free survival in a 285-patient HPV16-positive head and neck cancer trial.

Viking Therapeutics has started two Phase 3 trials of once-daily oral VK2735, a dual GLP-1/GIP receptor agonist, in adults with obesity, with and without type 2 diabetes.