On May 7, 2024 Myeloid Therapeutics, Inc. ("Myeloid"), a clinical stage immunology company, reported multiple poster presentations at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 Annual Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually (Press release, Myeloid Therapeutics, MAY 7, 2024, View Source;cell-therapy-asgct-2024-annual-meeting-302137520.html [SID1234642817]).
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"The data presented at ASGCT (Free ASGCT Whitepaper) showcase that Myeloid continues to lead the field of in vivo immune cell engineering and RNA-enabled gene editing and delivery. The data presented today underlies our in vivo mRNA CAR, MT-302, that continues to progress well in the ongoing clinical study. We are expanding plans for MT-302’s clinical use, given several inherent product advantages for patients," said Daniel Getts, Ph.D., Chief Executive Officer of Myeloid. "In addition, our groundbreaking CREATE platform demonstrates a full-scale revolution in gene editing. CREATE provides the ability to deliver large payloads with precision and solely using mRNA, which highlights the broad potential at Myeloid to transform cancer treatments in addition to rare disease indications."
Poster presentation details and abstract highlights include:
Title: "In vivo Programming of Immune Cells Using mRNA-LNP Chimeric Antigen Receptors"
Date & Time: Thursday, May 9, 2024, 12:00 PM-7:00 PM ET
Poster Session: Targeted Gene and Cell Therapy
Location: Poster Session F3
Session Title: Cancer – Targeted Gene and Cell Therapy
Published Abstract Number: 1284
Myeloid has designed novel CARs that achieve expression and function in targeted immune cell populations. These CARs, by activating innate and adaptive immune responses following the in vivo delivery of LNP-formulated mRNA encoded CARs, are capable of eliciting anti-tumor efficacy against a range of multiple target antigens evaluated.
Myeloid has demonstrated CAR activity in human cells, and following systemic mRNA/LNP delivery in mouse and non-human primates.
MT302, an in vivo CAR targeting TROP2+ epithelial malignancies (NCT05969041), is being evaluated in a Phase 1 clinical trial to assess the candidate’s safety and preliminary efficacy.
Title: "CRISPR-Enabled Autonomous Transposable Element (CREATE) for RNA-mediated gene editing and delivery"
Date & Time: Wednesday, May 8, 2024, 12:00 PM-7:00 PM ET
Poster Session: Gene Targeting and Gene Correction New Technologies
Location: Poster Session F3, Exhibit Hall
Session Title: Cancer – Targeted Gene and Cell Therapy
Published Abstract Number: 719
Myeloid developed a proprietary, RNA-based genome editing system called CREATE – CRISPR-Enabled Autonomous Transposable Element, that combines Prime Editing with a DNA transposase to overcome the current limitations of gene delivery technologies.
CREATE merges the capabilities of CRISPR/Cas9 with human L1 retrotransposon to insert gene-sized payloads without DNA donors or double strand breaks.
Mechanistic studies reveal that the CREATE system is highly specific with no observed off-target events.
These data establish CREATE system as a programmable gene delivery platform solely based on RNA components, enabling large-scale in vivo genome engineering with broad therapeutic potential.
More information on ASGCT (Free ASGCT Whitepaper) can be found here. More information on Myeloid’s CREATE platform can be found here.