The firm plans to submit CMC data for DTX401 informed by the FDA's observations related to an application for its Sanfilippo syndrome type A gene therapy.

The firm will pick two lead agents targeting PSMA and CLDN18.2 later this year and a third STEAP1-targeting drug next year.

If there are no dose-limiting toxicities, the company will test the CAR T-cell therapy at higher doses in more patients with FLT3-expressing refractory AML.

SNUG01 showed favorable safety and signs of early clinical efficacy in a recently completed investigator-initiated trial in China, the firm said.

The company will instead focus resources on its last remaining clinical asset, PRT7732, a chemically distinct SMARCA2 degrader.

At least 139 funded projects have focused on precision medicine since the council began awarding the grants in 2011, ...

Sarepta will sell back $50 million worth of shares to cover part of a $100 million milestone payment and its remaining shares worth $174 million.

The firm now expects its cash runway to extend to 2028, bolstered by having recently raised $244 million via a private placement and an at-the-market issuance.

Researchers at three Boston institutions are advancing a ctDNA assay with the goal of getting more children with solid tumors into biomarker-directed therapies and trials.

The EMA's CHMP said the single pivotal US trial could serve as the basis for a marketing authorization application in the EU.

The firm is touting its AI-driven foundation model's ability to predict how any patient will respond to any drug, allowing sponsors to fine-tune their trials.

The firm will explore FORX-428's activity in patients whose tumors harbor DNA damage repair mutations or mutations that cause high DNA replication stress.