Clinical Stage Pipeline
Transthyretin amyloidosis (ATTR)Hereditary angioedema (HAE)
Sickle Cell Disease
Acute Myeloid Leukemia
Transthyretin amyloidosis (ATTR)
Transthyretin amyloidosis is a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. These protein deposits most frequently occur in the peripheral nervous system, which is made up of nerves connecting the brain and spinal cord to muscles and sensory cells that detect sensations such as touch, pain, heat, and sound. Protein deposits in these nerves result in a loss of sensation in the extremities (peripheral neuropathy)
Gene Defect: Mutations in the TTR gene cause transthyretin amyloidosis. The TTR gene encodes a protein called transthyretin that transports vitamin A and thyroid hormone. The disease is inherited in autosomal dominant manner.
Treatment Approach: NTLA 2001 is a systemically delivered investigational CRISPR/Cas9 therapy that targets and abolish the mutant TTR gene, and reduce the synthesis pathogenic mutant protein
Clinical Phase: CTA submitted, Phase 1
Hereditary angioedema (HAE)
Hereditary angioedema is a disorder characterized by recurrent episodes of severe swelling (angioedema). The most common areas of the body to develop swelling are the limbs, face, intestinal tract, and airway. Swelling in the airway can restrict breathing and lead to life-threatening obstruction of the airway. About one-third of people with this condition develop a non-itchy rash called erythema marginatum during an attack.Gene Defect: Mutations in the SERPING1 gene cause hereditary angioedema type I and type II. The SERPING1 gene provides encodes C1 inhibitor protein, which is important for controlling inflammation. C1 inhibitor blocks the activity of certain proteins that promote inflammation. Lack of C1 inhibitor results in increased levels of bradykinin, a protein that leads to swelling.
Treatment Approach: NTLA-2002 is a CRISPR/Cas9 based gene therapy product. It aims to prevent the unregulated production of bradykinin by knocking out the prekallikrein B1 (KLKB1) gene.
Clinical Phase: IND in 2021
Other Treatment Program
Sickle Cell Disease
Acute Myeloid Leukemia
Treatment Platform: CRISPR/Cas9 Gene Editing with Lipid Nanoparticle (LNPs) Delivery System
Advantages of LNPs:
- Clinically-proven delivery to liver,
- Large cargo capacity
- Transient expression
- Biodegradable
- Low immunogenicity
- Well-tolerated
- Redosing capability
- Scalable synthetic manufacturing
- Tunable
Comments
Post a Comment