Autologous BCMA CAR-T Cells for the Treatment of Relapsed Refractory Light Chain Amyloidosis
Grant Award Details
Grant Type:
Grant Number:
CLIN2-15901
Investigator(s):
Award Value:
$7,999,467
Status:
Pre-Active
Grant Application Details
Application Title:
Autologous BCMA CAR-T Cells for the Treatment of Relapsed Refractory Light Chain Amyloidosis
Public Abstract:
Therapeutic Candidate or Device
NXC-201
Indication
AL Amyloidosis
Therapeutic Mechanism
Genetically modified T-cell targeting B-cell maturation antigen (BCMA)
Unmet Medical Need
Amyloid light-chain (AL) amyloidosis is a devastating, rare plasma cell disorder that results in organ deposition. In the US, there are ~3,972 diagnoses every year. Currently, there is no cure for AL amyloidosis.
Project Objective
Phase 1 trial completed
Major Proposed Activities
NXC-201
Indication
AL Amyloidosis
Therapeutic Mechanism
Genetically modified T-cell targeting B-cell maturation antigen (BCMA)
Unmet Medical Need
Amyloid light-chain (AL) amyloidosis is a devastating, rare plasma cell disorder that results in organ deposition. In the US, there are ~3,972 diagnoses every year. Currently, there is no cure for AL amyloidosis.
Project Objective
Phase 1 trial completed
Major Proposed Activities
- manufacture product to supply the proposed trial
- enrollment of the patients
- assess clinical safety of the product
Statement of Benefit to California:
Patients with AL amyloidosis face a significant reduction in their quality of life due to the debilitating symptoms and
the need for frequent medical interventions. The burden of living with a rare also places emotional and financial strains on patients and their families. According to the Journal of Comparative Effectiveness Research, from 2007 to 2015, the average cost of care AL Amyloidosis patients was over 100,000 dollars annually (Quock, 2018).