CTX310 showed promising safety and efficacy in a small Phase I trial, according to data presented at the American Heart ...

Investigators who designed a base-editing treatment are planning an "umbrella of umbrellas" trial, while others are developing roadmaps to guide personalized treatments.

At its annual meeting, the society discussed progress it has made in pushing ahead the Access to Genetic Counselor Services ...

The firm is preparing a protocol amendment to standardize patient monitoring and management of an immunosuppressive regimen.

The drugmaker is paying MeiraGTx $75 million upfront and $400 million in milestone payments to gain access to a treatment for LCA4 caused by AIPL1 mutations.

Trodelvy didn't improve progression-free survival versus chemo in the post-endocrine therapy first-line HR-positive, HER2-negative advanced breast cancer setting.

Intellia had already paused dosing in trials of nex-z in transthyretin amyloidosis after the patient was hospitalized due to a serious adverse event.

The UK regulator is creating a new framework for how it will approve rare disease treatments, which it intends to publish in ...

Genetic testing could improve cancer outcomes for patients, but historical research abuses and access issues have slowed ...

Researchers presented new data on Phenomix Sciences' genetic risk scores at the Obesity Society's ObesityWeek conference.

The agency is establishing a network of care centers to expand access to regenerative medicine beyond regions served by major ...

The firm had decreased Q3 sales for precision oncology drugs including Ibrance, Tukysa, and Bosulif, but sales increased for ...