Lysosomal Disorders

Addressing Unmet Needs With Gene Therapy

Eric Crombez, PhD, senior vice president and chief medical officer of Ultragenyx Gene Therapy, discussed indications the company is targeting.

Severe Neurodegenerative, Epileptic Disorders the Target of Taysha Gene Therapies

With numerous targets in CNS diseases and a unique partnership with gene therapy experts at UT Southwestern, Taysha is hoping to fill significant unmet needs.

Race to Treat GM1 Gangliosidosis With Gene Therapy Continues

The first US-based patient has been dosed in Lysogene’s clinical trial of LYS-GM101, following 2 other competing trials.