jondoeuk
9 months ago
They are opting out of an agreement with REGN to co-develop a factor IX gene editing therapy for haemophilia A and B. The agreement, which was signed in 2020, will terminate 180 days after NTLA provide written notice to REGN. They will continue to have obligations related to the co-development of gene-editing products directed to factor IX until the effective date of termination. Upon termination, NTLA will no longer be obligated for sharing 35% of the development costs, or be entitled to receive 35% of the profits, for gene-editing products directed to factor IX under the agreement.
Separately, the company would continue to support REGN with the development of gene-editing products directed to factor IX, as applicable, pursuant to a 2016 license and collaboration agreement between the companies. NTLA may be eligible to receive up to $320M in milestone payments and royalties in the high-single digits to low teens if REGN develops and commercialises gene-editing products under the terms of this license and collaboration deal.
Monksdream
1 year ago
That awful Cathie Wood bought more
Intellia Therapeutics Inc NASDAQ: NTLA
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Health Care : Biotechnology | Small Cap BlendCompany profile
Intellia Therapeutics, Inc. is a clinical-stage genome editing company, which is focused on developing curative therapeutics using Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) technology. CRISPR/Cas9 is a technology for genome editing, the process of altering selected sequences of genomic deoxyribonucleic acid. It is focused on leveraging its modular platform to advance in vivo and ex vivo therapies for diseases with high unmet need. Its lead in vivo candidate, NTLA-2001, is for the treatment of transthyretin (ATTR) amyloidosis, as well as NTLA-2002 for the treatment of hereditary angioedema (HAE). It is developing ex vivo applications to address immuno-oncology and autoimmune diseases. Its advanced ex vivo programs include a wholly owned chimeric antigen receptor T (CAR-T) cell candidate, NTLA-6001 targeting CD30 for the treatment of CD30-expressing hematologic cancers, including relapsed or refractory classical Hodgkin's lymphoma.
jondoeuk
3 years ago
The company will present new data at the 29th Annual Congress of the European Society of Gene & Cell Therapy meeting, taking place virtually from October 19-21.
Oral Presentations:
Title: A Novel Strategy for Off-the-shelf T Cell Therapies Evading Host T Cell and NK Cell Rejection
Abstract number: OR18
Date/Time: Wednesday, October 20, 2021, 10:45 a.m. CEST
Location: Session 2c: Immunotherapy for cancer & CAR T cells
Presenting Author: Yong Zhang, Ph.D., associate director, Cell Therapy
Title: Consecutive Genome Editing in Non-Human Primate Achieves Durable Production of Human Alpha-1 Antitrypsin at Physiologic Levels and Reduction of the Homologous Native Protein
Abstract number: OR12
Date/Time: Wednesday, October 20, 2021, 10:15 a.m. CEST
Location: Session 2b: Gene editing I
Presenting Author: Sean Burns, M.D., vice president of Intelliaβs Disease Biology and Pharmacology group
Invited Talk:
Title: Advances in CRISPR/Cas9 Therapeutic Genome Editing for In Vivo and Ex Vivo Applications
Date/Time: Friday, October 22, 2021, 11:30 a.m. CEST
Location: Session 7b: Liver and metabolic diseases II
Presenting Author: Laura Sepp-Lorenzino. Ph.D., chief scientific officer
Poster Presentation:
Title: Lipid Nanoparticles (LNPs) as a Superior CRISPR/Cas9 Delivery Modality for Highly Efficient Multiplex Gene Editing of T Cells for Adoptive Cell Therapy
Abstract number: P205
Date/Time: Tuesday, October 19, 2021, 8:00 a.m. CEST
Presenting Author: Aaron Prodeus, Ph.D., principal scientist, Cell Therapy
jondoeuk
4 years ago
In the setting of established and (rapidly) proliferating disease, there is a need to enhance and sustain T-cell function, activity, and persistence. However, too many inhibitory and not enough costimulatory signals are a major hurdle. But one way to overcome this is with immunomodulatory fusion proteins, such as this https://ashpublications.org/blood/article/130/22/2410/36564/A-CD200R-CD28-fusion-protein-appropriates-an
So I hope the company explore it.
jondoeuk
4 years ago
This trial showed that donor-derived, CD8+ T-cells engineered (using a viral vector) to express a WT1 TCR could prevent relapse of patients at high risk (post HCT) [1].
Based on it, a PhI/II trial is ongoing testing central memory vs. naive CD8+ T-cells for the treatment of high-risk AML patients post-induction chemo (over 80% will relapse within the first year [2]). As of Dec '19, seven had been treated. Four are NED (one out to over 500 days), one relapsed (but declined further treatment), and two had overt disease (one due to WT1 negative leukaemia cells).
I think the product (5001) produced by NTLA should show even better results due to a number of factors.
Refs:
1 https://www.nature.com/articles/s41591-019-0472-9
2 https://ascopubs.org/doi/full/10.1200/JCO.2014.58.3518
jondoeuk
4 years ago
Currently, the company is using CRISPR/Cas9 screening to identify edits that can improve infiltration, expansion, potency, and persistence, while also prevent exhaustion of T-cells. Using T-cells with these properties should help in their development of more effective TCR-T cell therapies in a range of solid tumours. They identified both known and novel regulators, and importantly, a number of knock-out targets that accumulate in multiple, distinct TMEs and other targets that are specific.
Data from other groups have shown that deletion of a ribonuclease augmented the capacity of T-cells to control tumour growth by enhancing their ability to infiltrate and persist within the TME [1]. Similarly, a knockout of a transcription factor lead to the improvement in the cytolytic properties of T-cells [2].
They could go further and use TCRs that target both MHC Class-I and II antigens [3], along with delivering 'payloads' to modulate TMEs [4-9].
Refs:
1 https://www.nature.com/articles/s41586-019-1821-z
2 https://www.cell.com/cell/fulltext/S0092-8674(16)31149-7
3 https://www.jci.org/articles/view/120391
4 https://www.cell.com/cancer-cell/fulltext/S1535-6108(19)30101-1
5 https://www.nature.com/articles/s41590-020-0676-7
6 https://cancerimmunolres.aacrjournals.org/content/8/6/743
7 https://cancerimmunolres.aacrjournals.org/content/8/4/518
8 https://cancerres.aacrjournals.org/content/71/17/5697.long
9 https://www.jci.org/articles/view/58814