UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

Nomlabofusp program granted Breakthrough Therapy Designation for the treatment of adults and children with FA based on FDA’s ...

Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S.

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Relay Therapeutics is approaching a pivotal clinical catalyst with the zovegalisib Phase 1/2 data readout at ESMO TAT. Check ...

Sutacimig is an investigational bispecific antibody designed to bind to endogenous factor VIIa and localize it to activated platelets, thereby promoting thrombin and fibrin formation.

Luvesilocin is a subcutaneously-administered, proprietary prodrug of the psilocybin-like compound 4-OH-DiPT designed to target the serotonin 2A receptor.

InvestigateTV+ shares a young mother’s struggle with the debilitating effects of lupus. Plus, we take an in-depth look at what could become a breakthrough treatment.

One experimental injection helped mice and pigs heal after heart attack by boosting a protective heart hormone. (CREDIT: ...

A Frisco mom’s yearslong fight led to a gene therapy breakthrough for the rare SLC6A1 disorder. Her son Maxwell is the first patient treated and is already showing promising progress.

Forty-Hz auditory stimulation increased amyloid clearance markers in aged primates and produced lasting effects, supporting its potential as a non-invasive Alzheimer’s therapy. Scientists at the Kunmi ...

The breakthrough treatment could lead to safer cancer therapies and may also help treat autoimmune diseases like lupus ...