Date: 2017-10-04
Type of information: Licensing agreement
Compound: AVR-RD-04
Company: Avrobio (USA - MA) GenStem Therapeutics (USA )
Therapeutic area: Rare diseases - Genetic diseases - Lysosomal diseases
Type agreement: licensing
Action mechanism: gene therapy
Disease: cystinosis
Details:
- • On October 4, 2017, Avrobio announced the expansion of its pipeline to cystinosis. This program becomes its fourth gene therapy for lysosomal storage disorders (LSDs), and with an anticipated IND filing later this year is expected to enter the clinic in early 2018. The cystinosis program was licensed from GenStem Therapeutics. Terms of the license agreement were not disclosed.
- Cystinosis, like all lysosomal storage disorders, is a progressive and potentially fatal metabolic disorder with an estimated incidence of 1 in 100,000 to 200,000 live births. Cystinosis is caused by a single-gene defect in the CTNS gene leading to the accumulation of an amino acid called cystine in all tissues and organs of the body including the kidneys, eyes, muscles, thyroid and pancreas. Patients with cystinosis have a wide range of symptoms depending on the age of onset and disease severity including renal Fanconi syndrome and renal failure by adolescence, sensitivity to light, blindness, severe muscle weakness, difficulty swallowing, and endocrinopathies including hypothyroidism and diabetes, and neurological defects.
- The aim of AVROBIO’s investigational gene therapy to treat cystinosis is to deliver lasting and meaningful benefits for patients. The patient’s peripheral blood stem cells are extracted and genetically modified by adding a new, fully functional copy of the faulty gene. The modified cells, once delivered back into the patient via a one-time infusion, enable durable gene rescue of the progressive tissue injury, with the potential to significantly improve patient outcomes and eliminate burdensome and costly lifelong cysteamine treatments.
Financial terms:
Latest news:
Is general: Yes
