On May 2, 2023 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, reported that six data presentations, including two oral presentations, highlighting the Company’s preclinical gene therapy programs and platforms will be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting, being held at the Los Angeles Convention Center in Los Angeles and virtually on May 16-20, 2023 (Press release, Poseida Therapeutics, MAY 2, 2023, View Source [SID1234630862]).

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Oral Presentations:

Title: Preclinical Proof-of-Concept: A Novel Hybrid Gene Therapy Approach to Treat Severe Early-Onset Ornithine Transcarbamylase Deficiency
Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases I
Presentation Time: Thursday, May 18, 2023, 2:30 – 2:45 PM PST
Location: Room 403 AB
Abstract Number: 127

Title: Cas-CLOVER Technology Enables Precise Gene Editing and Site-Specific Transgene Insertion in Mouse Liver
Session Title: Gene Targeting and Gene Correction: Liver
Presentation Time: Thursday, May 18, 2023 3:00 – 3:15 PM PST
Location: Room 515 AB
Abstract Number: 157

Poster Presentations:

Title: Demonstration of Human Factor VIII Expression and Activity Following Single and Repeat Dosing of a Non-Viral Integrating Gene Therapy
Session Title: Wednesday Poster Session
Session Date/Time: Wednesday, May 17, 2023, 12:00 PM PST
Location: West Hall A
Abstract & Poster Board Number: 638

Title: Development of a Novel Non-Viral Gene Therapy Platform
Session Title: Thursday Poster Session
Session Date/Time: Thursday, May 18, 2023, 12:00 PM PST
Location: West Hall A
Abstract & Poster Board Number: 945

Title: Editing of a γ-Globin (HBG1/HBG2) cis-Regulatory Element in Human Hematopoietic Stem and Progenitor Cells Using Cas-CLOVER Technology Reactivates Fetal Hemoglobin
Session Title: Thursday Poster Session
Session Date/Time: Thursday, May 18, 2023, 12:00 PM PST
Location: West Hall A
Abstract & Poster Board Number: 1212

Title: Development and Optimization of Novel Super piggyBac-Based Hybrid Gene Therapy Approach
Session Title: Friday Poster Session
Session Date/Time: Friday, May 19, 2023, 12:00 PM PST
Location: West Hall A
Abstract & Poster Board Number: 1318

On May 2, 2023 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) has accepted 14 Sangamo abstracts for presentation at the 26th ASGCT (Free ASGCT Whitepaper) Annual Meeting being held May 16-20, 2023, in-person in Los Angeles, CA and in a virtual format (Press release, Sangamo Therapeutics, MAY 2, 2023, View Source [SID1234630861]). Presentations will focus on the progression of Sangamo’s pre-clinical programs, including data from the prioritized neurology programs Nav1.7 and Prion, innovations in the epigenetic regulation platform and advances in AAV capsid engineering for delivery.

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"The data to be presented at ASGCT (Free ASGCT Whitepaper) reflect important advances in areas core to our strategy, including epigenetic regulation for neurological disorders and engineered AAV capsids to potentially address the issues of delivery," said Jason Fontenot, Ph.D., Chief Scientific Officer at Sangamo. "We are excited to share such a depth of pre-clinical data and look forward to presenting the innovation that continues to emerge from our research efforts, to potentially deliver transformative medicines to patients in need."

Data to be presented at the ASGCT (Free ASGCT Whitepaper) Annual Meeting include an oral presentation from Sangamo’s flagship neurology epigenetic regulation program Nav1.7 to treat chronic neuropathic pain. This presentation will showcase how Sangamo is using an optimized zinc finger epigenetic repressor to specifically target and seek to reduce Nav1.7 expression in dorsal root ganglions to inhibit pain sensation in diseases of chronic neuropathic pain. Beginning with Nav1.7-associated small fiber neuralgia, with an estimated prevalence of at least 43,000 patients in the US, future success in this indication could subsequently enable the broadened use of this therapy to other neuropathic pain indications, regardless of the cause of the pain.

Additional presentations at the ASGCT (Free ASGCT Whitepaper) Annual Meeting will showcase how Sangamo is advancing its proprietary zinc finger platform development efforts, including the use of zinc finger transcriptional regulators designed to reduce the expression of the Prion protein for the treatment of Creutzfeldt-Jakob disease. Sangamo will also present data from its proprietary AAV capsid discovery platform SIFTER, which enables the engineering of AAV capsids with the potential for highly improved central nervous system transduction efficiency, along with broader presentations showcasing Sangamo’s latest genomic engineering platform evolution.

ASGCT Annual Meeting Presentations and Invited Sessions

Epigenetic Regulation for the Central Nervous System

Zinc Finger Transcriptional Regulator Mediated Repression of SCN9A Gene as a Therapeutic Approach for Painful Peripheral Neuropathies
Abstract No. 32
Oral Presentation – May 17; 3:45-4:00 pm PT
Engineered Zinc Finger Transcriptional Regulators Specifically Reduce Prion Expression and Extend Survival in an Aggressive Prion Disease Model
Abstract No. 490
Poster Presentation – May 17; 5:30-7:00 pm PT
Cell-Type Specific Reduction of Prion Expression in Neurons and Astrocytes Using Engineered Zinc Finger Transcriptional Regulators
Abstract No. 1561
Poster Presentation – May 19; 5:30-7:00 pm PT
Intercellular Zinc Finger Protein Delivery for Cross-Corrective Epigenetic Regulation in the CNS
Abstract No. 1485
Poster Presentation – May 19; 5:30-7:00 pm PT
Gene Activation Mediated by Zinc Finger Transcriptional Regulators (ZF-TRs) as a Therapeutic Approach for CNS Disorders
Abstract No. 1057
Poster Presentation – May 18; 5:30-7:00 pm PT
Viral Engineering for the Central Nervous System

Fitness Maturation of Engineered AAV Capsids STAC-102 and STAC-103 Enhances Central Nervous System Transduction after CSF Administration in Cynomolgus Macaques
Abstract No. 1698
Poster Presentation – May 19; 5:30-7:00 pm PT
Evolution of Blood-Brain Barrier Penetrant AAV Capsids in Non-Human Primates Using a Multiplexed Transcription Dependent Capsid Engineering Platform
Abstract No. 1203
Poster Presentation – May 18; 5:30-7:00 pm PT
Process and Platform Development for Production and Purification of CNS-Tropic Engineered AAV Capsids
Abstract No. 1460
Poster Presentation – May 19; 5:30-7:00 pm PT
Strategic Formulation Development for AAV Delivered Gene Therapies – a Case Study
Abstract No. 1186
Poster Presentation – May 18; 5:30-7:00 pm PT
Genomic Engineering Platform Evolution

Engineered Zinc Finger Transcriptional Regulators Enable Robust and Reliable Epigenetic Regulation in the Mouse Brain
Abstract No. 682
Poster Presentation – May 17; 5:30-7:00 pm PT
Zinc Finger-Transcriptional Repressors Against Immune Checkpoint Molecules to Improve Anti-Tumor Activity of Gene-Modified T Cells
Abstract No. 1680
Poster Presentation – May 19; 5:30-7:00 pm PT
A Robust and Flexible Baculovirus-Insect Cell System for AAV Vector Production with Improved Yield, Capsid Ratios and Potency
Abstract No. 471
Poster Presentation – May 17; 5:30-7:00 pm PT
Assays to Support Clinical Programs

Overcoming the Effect of Previous Enzyme Replacement Therapy on the Detection of Anti-Transgene Protein Antibodies After Treatment with Gene Therapy
Abstract No. 727
Poster Presentation – May 17; 5:30-7:00 pm PT
Development of a Competitive Ligand-Binding Assay to Detect Neutralizing Antibodies Against Chimeric Antigen Receptor of Regulatory T Cells
Abstract No. 891
Poster Presentation – May 18; 5:30-7:00 pm PT
All abstracts for the ASGCT (Free ASGCT Whitepaper) Annual Meeting are available on ASGCT (Free ASGCT Whitepaper)’s website.

On May 2, 2023 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported that Jack Khattar, President and CEO of Supernus Pharmaceuticals, will participate in a fireside chat, as well as host investor meetings, at the Bank of America 2023 Health Care Conference on Thursday, May 11, 2023, at 10:40 a.m. PT (1:40 p.m. ET) (Press release, Supernus, MAY 2, 2023, View Source [SID1234630859]).

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The conference will take place May 9-11, 2023, at the Encore Hotel in Las Vegas, Nevada. Investors interested in arranging a meeting with company management should contact the Bank of America conference coordinator. A live audio webcast of the presentation can be accessed here or by visiting Events & Presentations in the Investor Relations section on the Company’s website at www.supernus.com. An archived replay of the webcast will be available for 60 days on the Company’s website following the conference.

On May 2, 2023 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that management will participate in a fireside chat at the Bank of America Securities 2023 Health Care Conference on Tuesday, May 9, 2023 beginning at 11:20 a.m. PT (2:20 p.m. ET) (Press release, SpringWorks Therapeutics, MAY 2, 2023, View Source [SID1234630858]).

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To access the live webcast, please visit the Events & Presentations page within the Investors & Media section of the company’s website at View Source A replay of the webcast will be available on SpringWorks’ website for a limited time following the conference.

On May 2, 2023 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported that six abstracts highlighting preclinical data from the fusogen platform have been accepted for presentation, including two oral presentations, at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting taking place May 16-20, 2023 in Los Angeles, CA (Press release, Sana Biotechnology, MAY 2, 2023, View Source [SID1234630857]).

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Oral Presentations:

Title: A Novel Technique to Detect Peripheral Blood CAR+ T Cells Using RNAscope In Situ Hybridization (ISH) in Non-Human Primates and Mice
Summary: A novel cell-block method can be used to detect circulating CAR T cells in preclinical studies by evaluating the expression of Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element (WPRE) by Fluorescence in situ hybridization (FISH) in lieu of flow cytometry or PCR.
Session: Pharmacology/Toxicology Studies: Bio Distribution
Session Location: Petree Hall C
Session Date/Time: Friday, May 19, 2023; 3:45 p.m. – 5:30 p.m. PT
Presentation Time: 5:00 p.m. – 5:15 p.m. PT
Abstract Number: 240

Title: Target Cell and Tissue Specificity of a Novel CD8-Targeted Fusosome for Direct In Vivo Delivery of CD19 or a CD20 CAR to CD8+ T Cells
Summary: A panel of in vitro studies confirmed cell-specific transduction, CAR Expression, and target cell killing, supporting safe in vivo administration of Sana’s novel CD8-directed fusosomes for CAR T therapies.
Session: Pharmacology/Toxicology Studies: In Vitro and In Vivo Safety
Session Location: Petree Hall C
Session Date/Time: Saturday, May 20, 2023; 8:00 a.m. – 9:45 a.m. PT
Presentation Time: 9:15 a.m. – 9:30 a.m. PT
Abstract Number: 317

Poster Presentations:

Title: Advancements in Manufacturing of CD8-Targeted Fusosomes Enhance Transduction of Resting T Cells In Vitro and In Vivo
Summary: Process improvements were made to the manufacturing of Sana’s CD8 targeted fusosome for in vivo CAR T therapy, resulting in enhanced resting T cell transduction and in vitro and in vivo tumor killing.
Session: Wednesday Poster Session
Session Date/Time: Wednesday, May 17, 2023; 12:00 p.m. PT
Abstract Number: 760

Title: Temsirolimus and IL-7 Treatment Synergistically Increase Primary Resting CD8+ T Cell Transduction with CD8-Targeted Fusosomes and Enhance CD19CAR Expression
Summary: The use of IL-7 and Rapamycin analogs (Rapalogs) with Sana’s CD8 targeted fusosomes is able to increase fusosome potency and improves the ability to deliver a therapeutic CAR transgene.
Session: Thursday Poster Session
Session Date/Time: Thursday, May 18, 2022; 12:00 p.m. PT
Abstract Number: 1058

Title: In Vivo Delivery of Genetic Payloads to Human Hematopoietic Stem/Progenitor Cells
Summary: A robust strategy for establishing basal access and achieving efficient and specific in vivo delivery of genetic payloads to human hematopoietic stem and progenitor cells (HSPC) was established.
Session: Thursday Poster Session
Session Date/Time: Thursday, May 18, 2023; 12:00 p.m. PT
Abstract Number: 1017

Title: The Retargeted Universal Fusosome: A Modular Approach to Generate Fusosomes for Targeted Gene Delivery
Summary: A modular approach to developing retargetable fusosomes decouples production and targeting, offering an efficient method to identify potent candidates for cell-specific gene delivery.
Session: Thursday Poster Session
Session Date/Time: Thursday, May 18, 2023; 12:00 p.m. PT
Abstract Number: 970

The ASGCT (Free ASGCT Whitepaper) abstracts are available to the public at: View Source