(NASDAQ: CRSP) Therapeutics AG is a Switzerland-based
gene-editing company. This is focusing on the development of transformative
gene-based medicines for serious diseases. The Company is developing products
using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9
gene-editing platform.
This is allowing for precise directed changes to genomic
deoxyribonucleic acid (DNA). Sangamo Therapeutics (SGMO)CRSP stock news at https://www.webull.com/quote/nasdaq-crsp
company
is a clinical-stage
biotech company and it is focusing on the research and development of genomic
medicine across 4 distinct technology platforms. These are including gene
therapy, cell therapy, genome editing, and genome regulation. Sangamo is a
naturally occurring class of transcription factor proteins found in humans and
other species
Ø Clinical Pipeline
Sangamo is developing a series of clinical programs, which are either
wholly-owned or partnered with well-established pharma and biotech companies.
This is focusing on 3 therapeutics areas in inherited metabolic disease
("IMDs"), central nervous system ("CNS), and inflammatory and
autoimmune diseases.
Ø
Gene Therapy Programs
The company's most advanced program is an investigational gene therapy
for severe hemophilia A, SB-525. This is developed under a global collaboration
with Pfizer (PFE), of which the rights of SB-525 have been transferred to Pfizer to run a phase 3
trial. Both companies are announced updated initial data from the
phase1/2 trial of SB-525. It was generally well-tolerated and demonstrated a
sustained increase in Factor VIII activity. SB-525 has been granted RMAT,
Orphan Drug, and Fast Track designation by the FDA as well as Orphan Medicinal
Product Designation by the European Medicines Agency.
Ø
Cell Therapy Programs
Sangamo is working with Sanofi (NASDAQ: SNY) to develop ex vivo gene-edited cell therapies, ST-400
and BIVV-003, for transfusion-dependent beta-thalassemia ("TDT") and
sickle cell disease ("SCD") respectively. Both ST-400 and BIVV-003
are related product candidates using the same technology. It is involving gene
editing of a patient's own hematopoietic stem progenitor cells using non-viral
delivery of ZFN technology.
Ø
Genome Regulation Programs
The company also has several preclinical programs evaluating their
ZFP-TF technology as a novel therapeutic and this is approaching for CNS
diseases. In December, Sangamo has announced a collaboration with Biogen (BIIB) to develop and commercialize ST-501
for tauopathies including Alzheimer's disease, ST-502 for synucleinopathies.
These are including Parkinson's disease, a third undisclosed neuromuscular
disease target, and up to 9 additional neurological disease targets. This
company is working with Pfizer to evaluate ALS and frontotemporal lobar
degeneration that is linked to the mutations in the C9ORF72 gene. You can gain more VIX ETFsinformation at https://www.webull.com/quote/etf_morelist/index_panic .
