The firm will launch a new pivotal trial after the EMA's CHMP last year recommended against conditional marketing authorization for the gene therapy.

While bota-vec failed to meet a Phase III primary endpoint, MeiraGTx remains committed to bringing the gene therapy to patients with X-linked retinitis pigmentosa.

The firm also said this week that the NCCN added the HER2xHER3 bispecific antibody to its biliary tract cancer treatment guidelines.

The firm will use the funds to advance its KIR-CAR platform, which adapts dual receptors from NK cells to allow CAR T cells to rest when not actively targeting tumors.

The firms plan to integrate and standardize datasets and registries from patient-led groups and identify cohorts for use in clinical trials.

The firm will deliver SGT-212 to patients' brains and intravenously to try to restore frataxin levels and diminish ...

The firms submitted an application to the EMA seeking to begin a Phase IIb trial of vortosiran for stroke prevention in atrial fibrillation.

NEW YORK – PrecisionLife and Ovation.io on Wednesday said they inked an agreement to launch a consumer-facing genetic test for identifying patients' likelihood of responding to GLP-1 receptor agonists ...

Researchers believe the therapy, which targets CD83, can selectively target leukemia cells and treat side effects of stem cell transplants.

The company is hoping to seek accelerated approval in first-line HLA-A*A2:01-negative metastatic uveal melanoma based on data ...

The firm aims to develop siRNAs and antisense oligonucleotides for common diseases that require targeting tissues beyond the liver.

In the ARROW trial, researchers found that different RET fusion partners affected the durability of patients' responses, a finding that could be useful for guiding treatment.