On May 2, 2022 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company developing the next generation of therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported that it will present new preclinical data on PH-894 for use in adoptive cell therapy (ACT) at the American Society for Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting, which is being held May 16-19, 2022 in-person in Washington, D.C. and virtually (Press release, Phio Pharmaceuticals, MAY 2, 2022, View Source [SID1234613337]).

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Poster Details are as follows:

Title:

Self-Delivering RNAi Targeting BRD4 (PH-894) Improves the
Phenotype of HER2-CAR-T Cells During Expansion

Author:

Benjamin Cuiffo, et al.

Abstract Number:

222

Session:

Oligonucleotide Therapeutics I

Session Date and Time:

Monday, May 16, 2022 5:30 PM – 6:30 PM EDT

Location:

Walter E. Washington Convention Center, Hall D

On May 2, 2022 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported that preclinical data highlighting the use of anti-c-kit CAR-T cells, P-ckit-ALLO1 as a preconditioning agent to enable hematopoietic stem cell (HSC) transplants, reported that it will be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting, being held in Washington, D.C. and virtually on May 16-19, 2022 (Press release, Poseida Therapeutics, MAY 2, 2022, View Source [SID1234613336]).

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The Company’s anti-c-kit CAR-T program leverages its proprietary piggyBac Gene Delivery System and Cas-CLOVER Site-specific Gene Editing System to develop fully allogeneic CAR-T cells targeting human c-kit which is highly expressed on HSCs, as well as on myeloid malignancies such as acute myeloid leukemia (AML), meaning the treatment can be used for either HSC transplant conditioning or as a treatment for AML. In addition to the CAR gene, the piggyBac transposon includes a selection marker for generation of a pure CAR+ product and a proprietary fast-acting safety switch enabling rapid clearance of the reactive CAR-T cells prior to donor HSC transplant.

Presentation details:

Poster Presentation: Anti-c-kit CAR-T Cells Enable HSC Engraftment in a Humanized Model of Stem Cell Transplant Conditioning
Session Title: Cell Therapies II
Session Date/Time: Tuesday, May 17, 2022, 5:30 – 6:30 PM ET
Poster Board Number: Tu-239
Location: Walter E. Washington Convention Center, Hall D
Abstract Number: 734

On May 2, 2022 Orna Therapeutics, a biotechnology company dedicated to designing and delivering a new class of fully engineered circular RNA therapeutics, reported multiple data presentations at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting taking place in Washington, D.C., or virtually, from May 16 – 19, 2022 (Press release, Orna Therapeutics, MAY 2, 2022, View Source [SID1234613335]). Oral presentations will describe Orna’s pipeline for the first time, revealing key data on its in situ CAR (isCAR) program, amongst others, and detail the development of a powerful, new screening platform (FoRCE). Poster presentations will provide additional information on the development of delivery solutions for the isCAR platform and on the development of our muscle genetic disease program.

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Details for the presentations are shared below.

Oral Presentations:
In-situ CAR Therapy Using oRNA Lipid Nanoparticles Regresses Tumors in Mice
Presenter: Tom Barnes, Ph.D., CEO
Date/Time/Location: Monday, May 16, 2022 from 9:10 – 9:45 a.m. ET in Room 207
Session: Scientific Symposium: Function and Therapeutics Applications of Circular RNAs (circRNAs)
Summary: Orna will introduce its pipeline along with key data from its isCAR program. Data from iterative animal studies will demonstrate that oRNA lipid nanoparticles (oRNA-LNPs) can be designed to overcome current challenges of engineered cell therapies. Orna will also present progress on oRNA-LNP (non-viral) delivery of long forms of dystrophin and advances with vaccine therapies.

Discovery of Translation Initiation Elements Enabled by a Parallel Arrayed Screen of Full-length Viral UTRs in Synthetic Circular RNA
Presenter: Alexander Wesselhoeft, Ph.D., Director, Molecular Biology
Date/Time/Location: Monday, May 16, 2022 from 11:30 – 11:45 a.m. ET in Salon H
Session: Oral Abstract Session: Oligonucleotide Therapeutics
Summary: Newly discovered internal ribosome entry sites (IRES) show greater activity (vs EMCV, a common IRES) and some produce different expression levels based on cell type, granting more options for improved expression and control of oRNA.

Poster Presentations:
Improved Immune Cell Expression with Circular RNA (oRNA) in vivo
Presenter: Kevin Kauffman, Ph.D., Principal Scientist
Date/Time/Location: Monday, May 16, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics I
Summary: oRNA lipid nanoparticles (oRNA-LNPs) show higher splenic T cell expression and biodistribution to the spleen in vivo with improved formulation characteristics compared to their linear mRNA-LNP counterparts.

Systemic Delivery of Circular RNA Encoding Partial Dystrophins and Expression in Skeletal Muscle
Presenter: Tatiana Fontelonga, Ph.D., Scientist
Date/Time/Location: Tuesday, May 17, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics II
Summary: Micro and mini versions of the dystrophin gene can be encoded in a high capacity oRNA, delivered via LNP, and properly expressed in primary human cells and the mdx mouse model of Duchenne muscular dystrophy.

On May 2, 2022 Ambrx Biopharma Inc., or Ambrx, (NYSE: AMAM), a clinical stage biopharmaceutical company using an expanded genetic code technology platform to create Engineered Precision Biologics, reported that it filed its annual report on Form 20-F (the "Form 20-F") for the year ended December 31, 2021 with the U.S. Securities and Exchange Commission ("SEC") on April 26, 2022 (Press release, Ambrx, MAY 2, 2022, View Source [SID1234613334]). The Form 20-F can be accessed by visiting either the SEC’s website at www.sec.gov or the Company’s website at www.ambrx.com.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The Company will provide a hard copy of the annual report containing its audited consolidated financial statements, free of charge, to its shareholders and holders of its American Depositary Shares upon request. Requests should be directed in writing by email to [email protected].

On May 2, 2022 Catamaran Bio, Inc., a biotechnology company developing off‑the-shelf chimeric antigen receptor (CAR)-NK cell therapies to treat cancer, reported that the company will present two posters at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, being held in Washington, DC, on May 16-18, 2022 (Press release, Catamaran Bio, MAY 2, 2022, View Source [SID1234613333]). Catamaran scientists will present data supporting CAR‑NK cell therapy design strategies to neutralize the immunosuppressive effects of the tumor microenvironment, including use of synthetic biology-enabled chimeric switch receptors which convert the inhibitory signal of TGF-β into positive NK cell activation signals.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"We look forward to sharing our progress in engineering CAR-NK cells to confer them with the functional attributes to enable durable efficacy in solid tumors," said Vipin Suri, PhD, MBA, Chief Scientific Officer of Catamaran Bio. "Our presentations at ASGCT (Free ASGCT Whitepaper) describe approaches to mitigate the effects of TGF-β, a highly immunosuppressive cytokine known to reduce the anti-tumor activity of immune cells. We will show data on the activities of multiple TGF-β based synthetic receptors that confer a range of benefits to CAR-NK cells and enhance anti-tumor activity in high TGF-β environments."

Details of the poster presentations are as follows:

Presentation Title: Engineered allogeneic CAR natural killer cells resist tumor microenvironment immunosuppression by expression of a TGF-βR2 dominant negative receptor
Session Title: Cancer – Immunotherapy, Cancer Vaccines I
Session Date & Time: Monday May 16, 2022, 5:30 p.m. ET
Location: Hall D
Abstract Number: 313
Poster Board Number: M-194

Presentation Title: A synthetic biology approach to address the immunosuppressive tumor microenvironment: Novel TGF-β switch receptors convert inhibitory signals to enhance NK cell activity
Session Title: Cancer – Immunotherapy, Cancer Vaccines I
Session Date & Time: Monday May 16, 2022, 5:30 p.m. ET
Location: Hall D
Abstract Number: 331
Poster Board Number: M-212

The abstracts will be available on the ASGCT (Free ASGCT Whitepaper) conference website.