The Food and Drug Administration (FDA) has approved Lenmeldy, a gene therapy developed by Orchard Therapeutics, for the treatment of metachromatic leukodystrophy (MLD) in children. This marks a significant advancement for the 40 to 100 children born each year with MLD, a fatal neurodegenerative disease. However, the treatment comes with a record-breaking price tag of $4.25 million, making it the most expensive drug in history.
Lenmeldy: A Lifeline for MLD Patients
MLD is a rare genetic disorder that affects the brain, spinal cord, and peripheral nerves. It is caused by a deficiency of the enzyme arylsulfatase A (ARSA), which leads to the accumulation of sulfatides in the brain and other organs. This accumulation damages the myelin sheath, the protective covering around nerve cells, leading to progressive loss of motor and cognitive function. Lenmeldy works by inserting a functional copy of the ARSA gene into the patient's cells, allowing them to produce the missing enzyme and halt the progression of the disease.
Acelyrin's Antibody Treatment for Thyroid Eye Disease
In other news, Acelyrin reported preliminary positive results from an early-stage study of an antibody treatment for thyroid eye disease. The treatment is administered via a simple skin injection, offering a potentially less invasive alternative to current therapies. Further studies are needed to confirm these initial findings and assess the long-term efficacy and safety of the treatment.
Clasp Therapeutics Secures $150 Million to Advance Cancer Immunotherapies
Clasp Therapeutics, a biotech startup focused on developing novel cancer immunotherapies, has raised $150 million in venture capital. The company's approach aims to harness the body's natural defenses against cancer while minimizing the risk to healthy cells. This funding will support the development of Clasp's pipeline of immunotherapeutic candidates and advance its research efforts in the field of cancer immunology.
