Gene

Fate Therapeutics Announces Presentation of FT819 Proof-of-Concept Data for B cell-mediated Autoimmune Diseases at ASGCT Annual Meeting

Retrieved on: 
Monday, April 22, 2024
Cancer, Lupus nephritis, Lupus, Abstract, Cell, Patient, Gene, Society, IPSC, Autoimmune disease, SLE, Therapy, Lymphoid leukemia, Vaccine

SAN DIEGO, April 22, 2024 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases, today announced that two presentations will be featured at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, being held in Baltimore, Maryland on May 7-11, 2024.

Key Points: 
  • The Company will present data of key therapeutic mechanisms of activity for autoimmune diseases, including B cell depletion, tissue infiltration and immune reconstitution, from its Phase 1 study of FT819 in relapsed / refractory B-cell malignancies.
  • FT819 is the Company’s off-the-shelf, CD19-targeted, iPSC-derived CAR T-cell program, which is also being investigated in a Phase 1 study for patients with moderate-to-severe systemic lupus erythematosus (SLE) including lupus nephritis and extrarenal lupus (NCT06308978).
  • Accepted abstracts are available on the ASGCT Annual Meeting website .
  • Presentations details are as follows:

Revolutionary Scientists Honored for Advancements in Gene Therapy for Neuromuscular Diseases and RNA Discoveries: King Faisal Prize Laureates in Medicine, Professor Jerry Mendell, and in Science, Professor Howard Chang, Awarded

Retrieved on: 
Monday, April 22, 2024
Science (journal), Mortality, FDA, Pediatric Research, Protein, Chromatin, Cell, Limb–girdle muscular dystrophy, Research, American Philosophical Society, National Cancer Institute, Molecular biology, Life, Gene, Quran, Food, LGMD, SMA, RNA, DMD, Spinal muscular atrophy, Infant, Dystrophin, Nationwide, Duchenne muscular dystrophy, Ageing, National academy, Hospital, Cancer research, Literature, DNA, Therapy, Gene expression, Muscular dystrophy, Chromosome, USD, Stanford University, Cancer, Islamic studies, Muscle weakness, Disease, Vilcek Foundation, Patient, Assay, Society, Listeria monocytogenes non-coding RNA, Pharmaceutical industry

Genetic mutations in Duchenne muscular dystrophy (DMD) patients hinder the production of dystrophin, a crucial protein for muscle health.

Key Points: 
  • Genetic mutations in Duchenne muscular dystrophy (DMD) patients hinder the production of dystrophin, a crucial protein for muscle health.
  • Gene therapy offers a solution by addressing this genetic anomaly, allowing the body to produce dystrophin and halt muscle degeneration.
  • Ludwig Professor of Cancer Research at Stanford University, has been awarded King Faisal Prize for Science in Biology.
  • Since 1979, King Faisal Prize in its 5 different categories has awarded 295 laureates who have made distinguished contributions to different sciences and causes.

CRISPR Therapeutics to Present Oral Presentation at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting

Retrieved on: 
Monday, April 22, 2024
CRSP, CRISPR, LNP, MD, Cell, Messenger, Eye, Gene, Society, MYOC, Annual general meeting, Glaucoma

The abstract describes our proprietary capabilities to deliver to and edit genes in the eye, opening a potential new focus area.

Key Points: 
  • The abstract describes our proprietary capabilities to deliver to and edit genes in the eye, opening a potential new focus area.
  • Multiple LNPs as well as modified gRNAs and mRNAs were screened to achieve maximal editing in vivo.
  • In human primary trabecular meshwork cells, up to 95% MYOC editing and 85% protein knockdown were seen.
  • A copy of the presentation will be available at www.crisprtx.com once the presentation concludes.

Theriva™ Biologics to Present Preclinical Data Supporting the Potential Synergy of VCN-01 and First-Line Pancreatic Cancer Chemotherapy Regimens at the American Society for Cell and Gene Therapy 27th Annual Meeting

Retrieved on: 
Monday, April 22, 2024
Combination, Cancer, FOLFIRINOX, Pancreatic cancer, Poster, Animal, Irinotecan, Cell, PDAC, Real-time, Therapy, Patient, Gene, Society, Neoplasm, Oncolytic adenovirus, Pharmaceutical industry

ROCKVILLE, Md., April 22, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced the presentation of preclinical data demonstrating enhanced anti-tumor effects in human pancreatic cancer xenograft-bearing mice treated with lead product candidate VCN-01 and liposomal irinotecan. These data support the potential synergy of VCN-01 and first-line pancreatic cancer chemotherapy regimens, and will be featured in a poster presentation at the American Society for Cell and Gene Therapy (ASGCT) 27th Annual Meeting, being held both virtually and in Baltimore from May 7-11, 2024.

Key Points: 
  • These data support the potential synergy of VCN-01 and first-line pancreatic cancer chemotherapy regimens, and will be featured in a poster presentation at the American Society for Cell and Gene Therapy (ASGCT) 27th Annual Meeting, being held both virtually and in Baltimore from May 7-11, 2024.
  • “We look forward to leveraging these findings and evaluating the combination of VCN-01 with additional first-line pancreatic cancer chemotherapy regimens, including NALIRIFOX and FOLFIRINOX.
  • In vivo: Synergy of VCN-01 plus liposomal irinotecan was observed in animals bearing subcutaneous human pancreatic tumors.
  • Combination therapy with VCN-01 + liposomal irinotecan at either dose displayed significantly reduced tumor growth compared to each treatment alone.

Intratumoral Injection of Phio’s PH-762 significantly inhibits tumor growth in murine tumor models and may generate memory-specific T cells

Retrieved on: 
Monday, April 22, 2024
Gene silencing, Neoadjuvant therapy, Cell, Gene, Society, Melanoma, Clinical trial, TME, Pharmaceutical industry

MARLBOROUGH, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL™ siRNA gene silencing technology is designed to make immune cells more effective in killing tumor cells, today announced it is presenting new data about its lead clinical product candidate, PH-762, an INTASYL compound.

Key Points: 
  • -Studies support ongoing clinical trial of PH-762 as a neoadjuvant therapy for treatment of cSCC, melanoma, or Merkel cell carcinoma
    MARLBOROUGH, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL™ siRNA gene silencing technology is designed to make immune cells more effective in killing tumor cells, today announced it is presenting new data about its lead clinical product candidate, PH-762, an INTASYL compound.
  • mPH-762-mediated silencing of PH-762 within the TME may generate memory-specific T cells, promoting IFN-γ release in the TME
    These finding support the ongoing clinical trial of PH-762’s safety and efficacy as a neoadjuvant therapy for treatment of cSCC, melanoma, or Merkel cell carcinoma
    The data, authored by Melissa Maxwell, Linda Mahoney, and Dr. Mary Spellman, will be presented at the American Society of Gene and Cell Therapy (ASGCT) on May 8th in Baltimore, Maryland.
  • Presentation Details are as follows:

Cellectis Presents Novel TALEN® Editing Processes Enabling Highly Efficient Gene Correction and Gene Insertion in HSPCs

Retrieved on: 
Monday, April 22, 2024
Brain, Euronext Growth, Pericyte, IDUA, Traffic barrier, Integrin alpha M, HSPC, Mouse, CD4, Myelocyte, CD20, Cell, Intron, DNA, Therapy, Patient, Gene, Society, BBB, DSM-IV codes, Mucopolysaccharidosis, Vaccine

NEW YORK, April 22, 2024 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ:  CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, will present first data exploring novel TALEN® editing processes in hematopoietic stem and progenitor cells (HSPCs) at the American Society of Gene and Cell Therapy (ASGCT) being held on May 7-11, 2024.

Key Points: 
  • “These two posters showcase the potential and versatility of the TALEN® technology to promote efficient gene insertion in HSPCs.
  • Cellectis has developed a TALEN® mediated promoter-less intron editing technology that enables the expression of a therapeutic transgene exclusively by monocyte derived from edited HSPCs.
  • This novel editing approach is an important addition to the HSPC gene editing toolbox that might unlock new strategies for the treatment of metabolic and neurological diseases.
  • Cellectis has developed and optimized a novel gene editing process, leveraging the TALEN® technology and circular single strand DNA template delivery, enabling highly efficient gene insertion in HSPCs.

Voyager Therapeutics to Present Broad Set of Translational Data Supporting IV-Delivered, CNS Gene Therapy Programs Advancing Toward Clinical Trials at the ASGCT 27th Annual Meeting

Retrieved on: 
Monday, April 22, 2024
TRACER, Histology, Mouse, Phenotype, Data science, Cell, AAV, CNS, Machine learning, Senior, Tauopathy, Gene, Process, Society, BBB, ALS, Central nervous system, Public, HEK293, Therapy, Centrifuge, SOD1, Vaccine

“Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system.

Key Points: 
  • “Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system.
  • Three of those programs now have development candidates selected, and we see the potential for them to enter clinical trials next year,” said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics.
  • ET
    Intravenous administration of BBB-penetrant, MAPT-Silencing, AAV gene therapy provides broad and robust CNS Tau lowering in tauopathy mouse models (#1602).
  • ET
    Intravenous delivery of AAV gene therapy for the treatment of SOD1-ALS provides broad SOD1 lowering in NHP (#1647).

Editas Medicine to Present Pre-clinical Data Demonstrating Progression of in vivo Medicines Pipeline at the American Society of Gene and Cell Therapy Annual Meeting

Retrieved on: 
Monday, April 22, 2024
Guide, LSID, Abstract, Research, Editas Medicine, Abstract (summary), Gene editing, RNA, Cell, Medicine, Gene, Society, Vaccine

The Company is presenting pre-clinical data to support its development of transformative in vivo gene editing medicines.

Key Points: 
  • The Company is presenting pre-clinical data to support its development of transformative in vivo gene editing medicines.
  • Editas Medicine presentations at ASGCT include:
    Pre-clinical data demonstrating AsCas12a gRNA modifications that enable high-potency gene editing in multiple cell types and improve gene editing outcomes in vivo, enabling the development of in vivo gene editing medicines.
  • Research on identifying potent large serine recombinases (LSRs) as a foundation to develop novel in vivo gene editing technologies for whole gene knock-in, expanding potential in vivo gene editing targets for developing medicines.
  • These in vivo data are an important step towards confirming in vivo proof of concept by the end of the year.”
    The complete list of Editas Medicine presentations is below.

Kriya Announces Six Presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting 2024

Retrieved on: 
Monday, April 22, 2024
Common, EDT, DSM-IV codes, Computational biology, Cell, AAV, Therapy, Gene, Neurology, Society, AlphaFold, PALO, Kriyā, GMP, Ophthalmology, Vaccine

PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, N.C., April 22, 2024 (GLOBE NEWSWIRE) -- Kriya Therapeutics, Inc. ("Kriya"), a biopharmaceutical company developing gene therapies to address common diseases affecting millions of people around the world, today announced six presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which will be held May 7 to 11, 2024 in Baltimore, MD.

Key Points: 
  • Kriya has established a fully integrated gene therapy product development engine including cutting-edge research, computational biology and scalable in-house GMP manufacturing deploying next-generation upstream and downstream processes and analytical characterization techniques.
  • The presentations describe core technologies and manufacturing platform capabilities that support the Company's pipeline of gene therapies in ophthalmology, metabolic disease and neurology.
  • Kriya is also sponsoring a symposium entitled “Advancing Gene Therapy for Common Diseases: from Concept to Reality”.
  • (Abstract 957)
    Title: Prequalification of analytical test methods for determining the potency of an adeno-associated virus-based gene therapy product, A. Bries et al.

GeneFab Announces Two Presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting 2024

Retrieved on: 
Monday, April 22, 2024
FDA, Phenotype, Machine learning, Cell, Protein engineering, Immunotherapy, OMICS, Gene, Society, Synthetic biology, Macrophage, Gene expression

ALAMEDA, Calif., April 22, 2024 (GLOBE NEWSWIRE) -- GeneFab, LLC (“GeneFab”), an expert contract research, development and manufacturing organization (CRDMO) today announced two poster presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which will be held May 7 to 11, 2024 in Baltimore, MD.

Key Points: 
  • ALAMEDA, Calif., April 22, 2024 (GLOBE NEWSWIRE) -- GeneFab, LLC (“GeneFab”), an expert contract research, development and manufacturing organization (CRDMO) today announced two poster presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which will be held May 7 to 11, 2024 in Baltimore, MD.
  • GeneFab employs expert genetic design and manufacturing methods to meet Advanced Therapy companies' needs.
  • With a 92,000 square foot cGMP manufacturing facility in Alameda, California that supports various customer projects, including allogeneic cell therapy, CAR-T and synthetic biology research services.
  • GeneFab’s presentations highlight two new technologies for control of payload production from cell therapies.