Date: 2022-03-09
Type of information: Patent infringement lawsuit
Product name: NTLA-5001
Compound: Autologous ex-vivo CRISPR/Cas9 engineered Wilms¿ tumor 1-specific T-cell receptor T cells
Therapeutic area: Cancer-Oncology-Rare diseases
Action mechanism: cell therapy. NTLA-5001 is an autologous TCR-T cell therapy designed to target the Wilms’ Tumor (WT1) antigen, which is highly expressed in AML and many other hematologic and solid tumors. NTLA-5001 is currently being evaluated in a Phase 1/2a study in adults with persistent or recurrent AML who have previously received first-line therapy. NTLA-5001 is an investigational CRISPR/Cas9-engineered T cell receptor (TCR)-T cell therapy in development for the treatment of all genetic subtypes of acute myeloid leukemia (AML). This autologous cell therapy candidate is designed for AML patients with the HLA-A*02:01 allele and whose tumors carry the Wilms’ Tumor 1 (WT1) antigen, which is widely overexpressed in AML and other cancers. NTLA-5001 is Intellia’s first wholly owned ex vivo therapeutic candidate, developed using its proprietary cell engineering platform for the treatment of cancer. NTLA-5001 utilizes a WT1-targeting TCR identified in collaboration with IRCCS Ospedale San Raffaele. Based on preclinical results, Intellia believes its proprietary cell engineering platform will result in a pipeline of more efficacious and safer cell-based cancer therapies.
Company: Intellia Therapeutics (USA)
Disease: acute myeloid leukemia (AML)
Latest news: • On March 9, 2022, the FDA has granted orphan drug designation for Intellia’s ex vivo investigational T cell receptor (TCR)-T cell therapy, NTLA-5001 (autologous ex-vivo CRISPR/Cas9 engineered Wilms¿ tumor 1-specific T-cell receptor T cells), for the treatment of acute myeloid leukemia (AML). A Phase 1/2a study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy. The study includes a dose escalation and expansion phase, with up to 54 total participants. The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5% blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. (NCT05066165)
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2022-03-09
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
