On May 2, 2022 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, reported that four abstracts, including one from a research and license collaboration, were accepted by the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) and will be presented as posters and oral presentations at the upcoming annual meeting on May 16-19, 2022 at the Walter E. Washington Convention Center in Washington, D.C (Press release, Precision Biosciences, MAY 2, 2022, View Source [SID1234613294]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Details for the poster and oral presentations can be accessed on the ASGCT (Free ASGCT Whitepaper) website at View Source

Oral Presentations:

Title: Targeting the Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo
Date and Time: Tuesday, May 17, 2022, 3:45 PM – 4:00 PM
Session Title: Gene Editing in Cancer and Complex Diseases
Location: Hall E
Abstract #: 447

Title: AAV-Meganuclease-Mediated Gene Targeting Achieves Efficient and Sustained Transduction in Newborn and Infant Macaque Liver1
Date and Time: Wednesday, May 18, 2022, 3:00 PM – 3:15 PM
Session Title: Presidential Symposium and Presentation of Top Abstracts
Location: Hall E
Abstract #: 811

Poster Presentations:

Title: Optimization of Hydroxyacid Oxidase 1 (HAO1) Targeting ARCUS Nucleases for the Treatment of Primary Hyperoxaluria Type 1 (PH1)
Date and Time: Monday, May 16, 2022, 5:30 PM – 6:30 PM
Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases I
Location & Poster Board Number: Hall D, M-120
Abstract #: 239

Title: ARCUS Gene Editing to Eliminate MELAS-associated m.3243A>G Mutant Mitochondrial DNA
Date and Time: Tuesday, May 17, 2022, 5:30 PM – 6:30 PM
Session Title: Gene Targeting and Gene Correction II
Location & Poster Board Number: Hall D, Tu-66
Abstract #: 561

1 University of Pennsylvania’s Gene Therapy Program presentation sponsored by iECURE.

On May 2, 2022 Alaunos Therapeutics, Inc. ("Alaunos" or the "Company") (Nasdaq: TCRT), a clinical-stage oncology-focused cell therapy company reported a poster presentation highlighting the Company’s membrane-bound interleukin-15 (mbIL-15) program as a potentially potent cell therapy targeting hotspot mutations expressed in solid tumors at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) being held in Washington, D.C. and virtually May 16-19, 2022 (Press release, Alaunos Therapeutics, MAY 2, 2022, View Source [SID1234613293]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are excited at the opportunity to present new preclinical data in our mbIL-15 program at the ASGCT (Free ASGCT Whitepaper) Annual Meeting. In this study we observed the potential of our Sleeping Beauty transposed mbIL-15 TCR-T cells to deepen clinical responses and improve durability of TCR-T cells targeting hotspot mutations expressed in solid tumors," stated Drew Deniger, Ph.D., Vice President, Research & Development at Alaunos Therapeutics. "We continue to be encouraged by the data and intend to file an IND application for this program in the second half of 2023."

Details of the poster presentation are as follows:

Poster Presentation: Stem-cell memory TCR-T cells targeting hotspot EGFR, KRAS and p53 neoantigens generated through co-expression of membrane-bound Interleukin-15

Session Title: Cancer – Targeted Gene and Cell Therapy I

Session Date/Time: Monday May 16, 2022, 5:30 PM—6:30 PM EST

Poster Board Number: M-234

Location: Walter E. Washington Convention Center, Hall D

Alaunos’ mbIL-15 program has been developed to augment the function of TCR-T cells by co-expression of a more potent and proprietary membrane-bound interleukin-15. In this preclinical study, Alaunos observed that mbIL-15 TCR-T cells specifically targeted and killed tumors expressing matching neoantigen and HLAs with negligible off target effects. Furthermore, Alaunos observed the potential of mbIL-15 to establish long-lived tumor-specific TCR-T cells that have the potential to survive within the circulation and the tumor microenvironment.

On May 2, 2022 Lyell Immunopharma, Inc., (Nasdaq: LYEL), a T-cell reprogramming company dedicated to the mastery of T cells to cure patients with solid tumors, reported that two abstracts have been accepted for poster presentations at the 25th Annual Meeting of the American Society of Gene & Cell Therapy, scheduled for May 16 – May 19, 2022, in Washington, DC (Press release, Lyell Immunopharma, MAY 2, 2022, View Source [SID1234613292]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The presentations will highlight preclinical data characterizing two investigational products in Phase 1 clinical development that incorporate Lyell technologies designed to address major barriers to successful Adoptive Cell Therapy (ACT): Gen-R, a genetic reprogramming technology that endows T cells with the ability to resist exhaustion, and Epi-R, an epigenetic reprogramming technology that creates populations of T cells with properties of durable stemness. T cells with properties of durable stemness are able to proliferate, persist and self-renew, as well as generate differentiated effector cell progenies to provide durable anti-tumor functionality.

The first abstract presents preclinical data for LYL797 demonstrating that Gen-R and Epi-R can enhance and prolong anti-tumor functions of ROR1-targeting CAR T-cell therapy in solid tumor model systems. The second abstract presents preclinical data for LYL132 demonstrating that Epi-R creates populations of stemlike NY-ESO-1-targeting TCR T cells that lead to products with increased proliferative capacity and prolonged functional activity in the presence of persistent antigen exposure.

Details on the presentations are below:

Preclinical Development of LYL797, a ROR1-Targeted CAR T-Cell Therapy Enhanced with Genetic and Epigenetic Reprogramming for Solid Tumors

Session: Cancer – Immunotherapy, Cancer Vaccines II
Presentation Date, Time & Location: May 17, 5:30 – 6:30 PM, Hall D, Tu-166
Abstract number: 661
Epigenetic Reprogramming (Epi-R) Yields T-Cell Receptor Products with Improved Stemness, Metabolic Fitness, and Functional Activity in the Presence of Persistent Antigen Exposure

Session: Cancer – Targeted Gene and Cell Therapy II
Presentation Date, Time & Location: May 18, 5:30 – 6:30 PM, Hall D, W-241
Abstract: 1115
About LYL797 and LYL132

LYL797 is an investigational chimeric antigen receptor (CAR) T-cell therapy for patients with receptor tyrosine kinase-like orphan receptor 1-positive (ROR1+) solid tumors. LYL797 incorporates Lyell’s novel Gen-R and Epi-R reprogramming technologies. The Phase 1 trial will assess LYL797 in patients with relapsed/refractory triple-negative breast cancer (TNBC) or non-small cell lung cancer (NSCLC). More information can be found on ClinicalTrials.gov by searching NCT05274451.

LYL132 (GSK4427296) is an investigational T-cell receptor (TCR) therapy for patients with solid tumors expressing New York esophageal squamous cell carcinoma 1 (NY-ESO-1) being developed in collaboration with GSK. LYL132 incorporates Epi-R reprogramming technology and is under investigation as a potential next-generation enhancement to letetresgene autoleucel (lete-cel), a GSK TCR therapy targeting NY-ESO-1 currently in pivotal clinical development. The Phase 1 trial will assess LYL132 in patients with NY-ESO-1+ advanced synovial sarcoma (SS) or myxoid/round cell liposarcoma (MRCLS). Lyell will manufacture LYL132 in its LyFE Manufacturing Center and GSK will conduct the Phase 1 trial. More information can be found on ClinicalTrials.gov by searching NCT04526509.

On May 2, 2022 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported two abstracts have been accepted for presentation at the American Society of Gene and Cell Therapy ("ASGCT") 25th Annual Meeting, taking place virtually and in person in Washington, D.C., May 16-19, 2022 (Press release, NeuBase Therapeutics, MAY 2, 2022, View Source [SID1234613291]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

NeuBase will present new preclinical data on the biodistribution in key tissues of the company’s lead candidate, NT-0231.F, a precision genetic medicine in development to treat myotonic dystrophy, type 1 ("DM1"). NeuBase recently presented data at the 2022 MDA Clinical & Scientific Conference demonstrating that systemic administration of NT-0231.F in the HSALR model achieves clinically relevant molecular and functional rescue, including rescue of the spliceopathy, resolution of nuclear aggregates, and reversal of myotonia (delayed muscle relaxation after contraction). Pharmacokinetic studies of NT-0231.F in wild-type BALB/C mice showed a single IV or SC dose achieved high volume of distribution.

The presentations are listed below, and the full abstracts are available on the ASGCT (Free ASGCT Whitepaper) meeting website. All times are listed in Eastern Time (ET).

Title: Pharmacokinetics, Biodistribution, and CNS Penetration of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy-Type 1 Following Systemic Administration Systemic Administration in Mice
Presenter: Noel Monks, Ph.D.
Session Date/Time: Tuesday, May 17, 5:30 PM – 6:30 PM
Poster Board Number: Tu-90
Session Title: Oligonucleotide Therapeutics II
Room: Hall D
Abstract Number: 585

Title: Pharmacology, Biodistribution and Tolerability of a PATrOL-Enabled Investigational Genetic Therapy for Myotonic Dystrophy, Type 1
Presenter: Sandra Rojas-Caro, M.D.
Session Date/Time: Wednesday, May 18, 5:30 PM – 6:30 PM
Poster Board Number: W-175
Session Title: Musculo-skeletal Diseases
Room: Hall D
Abstract Number: 1049

About NeuBase’s DM1 Program
Patients with DM1 suffer from cognitive deficits and muscle pathology caused by a trinucleotide expansion in the DMPK gene which, when transcribed, results in an RNA hairpin structure that sequesters RNA splice proteins. NeuBase’s DM1 investigational genetic therapy, NT-0231.F, targets mutant DMPK pre-mRNA with a novel peptide-nucleic acid ("PNA") pharmacophore and is designed to selectively engage with the toxic RNA hairpin structure, release the splicing proteins, and restore RNA splicing and downstream protein production. The PNA pharmacophore is conjugated to NeuBase’s novel delivery technology that is designed for broad distribution, including into the deep brain, with the potential for a whole body, disease-modifying solution for DM1. For more information, visit www.neubasetherapeutics.com/pipeline/.

On May 2, 2022 Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo for patients with solid and hematologic malignancies, reported that it will have an oral presentation and two posters at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting, to be held May 16-19, 2022 (Press release, Umoja Biopharma, MAY 2, 2022, View Source [SID1234613290]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The oral presentation will include preliminary safety data of UB-VV100, a combination of VivoVec and RACR technology platforms. VivoVec is an in vivo lentiviral vector-based CAR T-cell platform developed for off-the-shelf use, while RACR is a rapamycin-activated cytokine receptor designed to drive in vivo CAR T-cell survival without the requirement for lymphodepletion through administration of rapamycin. The poster presentations will include descriptions of VivoVec platform advancements that expand particle mechanisms of action and enhance particle manufacturing processes.

Presentation details:

Oral Presentation Title: Preclinical Activity and Safety of UB-VV100, A Novel Lentiviral Vector Product Designed for Selective and Effective In Vivo Engineering of Therapeutic Anti-CD19 CAR T Cells for B cell Malignancies
Abstract Number: 1242
Session: Cell-Based Cancer Immunotherapies III
Presenting Author: Alissa Brandes, Ph.D., Principal Scientist, Umoja Biopharma
Presentation Date, Time: Thursday May 19, 2022; 10:15 AM – 12:00 PM ET

Poster Presentation Title: A Lentiviral-Based In Vivo CAR T Cell Generation Platform with Viral Particle Surface Engineering Incorporating Anti-CD3 Single Chain Variable Fragment and T Cell Costimulatory Molecules
Abstract Number: 879
Session: RNA Virus Vectors
Presenting Author: Christopher Nicolai, Ph.D., Senior Scientist, Umoja Biopharma
Presentation Date, Time: Wednesday May 18, 2022; 5:30 – 6:30 PM ET

Poster Presentation Title: Development of a Scalable, Suspension Cell Culture-Based Manufacturing Process for VivoVec, a Lentiviral Vector Platform for In Vivo CAR-T Cell Generation
Abstract Number: 1166
Session: Vector Product Engineering, Development or Manufacturing III
Presenting Author: Jeff Plomer, Senior Director Process Development, Umoja Biopharma
Presentation Date, Time: Wednesday May 18, 2022; 5:30 PM – 6:30 PM ET

Presentations can be accessed from the ASGCT (Free ASGCT Whitepaper) website at View Source