Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of ...

Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, ...

Swedish biotech company Gesynta Pharma AB has secured $27 million in a Series B financing round led by Innovestor Life Science in Finland.

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

MaxCyte's technology will support TG Therapeutics' efforts to develop a CD19 CAR-T cell therapy program for autoimmune diseases.

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

Atherosclerotic cardiovascular disease is a major health concern worldwide and requires effective preventive measures. Lp(a) (lipoprotein [a]) has recently garnered attention as an independent risk ...

Using “cure” to refer to “obtaining a normal life” also fits — if you ignore previously lost education, career opportunities ...

The Metachromatic Leukodystrophy Initiative (MLDi) critically responds to the findings of Zhang et al. on lentivirus-modified ...

Announcing a new article publication for BIO Integration journal. Neurologic disorders currently affect approximately 100 ...

Background Early-life inflammation has long been recognised as a key pathophysiological process in the evolution of cystic ...

With the addition of Casgevy, Children’s Hospital Los Angeles now offers two gene therapies for sickle cell, the first being Lyfgenia.