European Society of Gene and Cell Therapy

Nuevocor presents pre-clinical data at ESGCT and AHA meetings

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Tuesday, November 7, 2023
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SINGAPORE, Nov. 7, 2023 /PRNewswire/ -- Nuevocor, a pre-clinical stage biotechnology company focused on developing cardiac genetic medicines through a unique platform to understand the mechanobiological causes of disease, announced today a pair of presentations at two major meetings, the European Society for Gene and Cell Therapy (ESGCT) 30th Annual Congress, and the American Heart Association (AHA) Scientific Sessions 2023.

Key Points: 
  • SINGAPORE, Nov. 7, 2023 /PRNewswire/ -- Nuevocor, a pre-clinical stage biotechnology company focused on developing cardiac genetic medicines through a unique platform to understand the mechanobiological causes of disease, announced today a pair of presentations at two major meetings, the European Society for Gene and Cell Therapy (ESGCT) 30th Annual Congress, and the American Heart Association (AHA) Scientific Sessions 2023.
  • The poster presentations showcase optimization efforts for Nuevocor's first-in-class lead programme using a mechanobiology-based approach to treat LMNA-associated dilated cardiomyopathy.
  • At the ESGCT Congress on 24 – 27 October, 2023 in Brussels, Belgium, Nuevocor co-founder and Chief Executive Officer, Dr. Yann Chong Tan, presented a poster (P613) entitled "An AAV gene therapy for LMNA dilated cardiomyopathy via disruption of the LINC complex."
  • For more information on the meetings, please visit the respective conference websites.

Sangamo Therapeutics Announces Strategic Update and Reports Third Quarter 2023 Financial Results

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Wednesday, November 1, 2023
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Regenerative Medicine Advanced Therapy, Ethics, Therapy, Pfizer, Gene, Partnership, Head, Exposition, European Society of Gene and Cell Therapy, ALTA, AAV, Haemophilia, Hope, 65th Street Yard, Workforce, ERT, DSM-IV codes, Cell, CAR, U.S. FDA, Safety, Investment, Patient, Central nervous system, Nav1.7, MAA, 22q13 deletion syndrome, Conference, Enzyme replacement therapy, Hematology, Quality of life, Mace Head Atmospheric Research Station, Novartis, Brain, European Society for Primary Immunodeficiencies, Haemophilia A, Spectrum, FDA, IND, Fabry disease, Prion, Society, Senior, SCN2A, ESGCT, STAAR, Intellectual disability, Sclerosis, BLA, Pharmaceutical industry, Management, Vaccine, Biogen, Neuroscience, Fabry, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.
  • As a result of this restructuring, Sangamo is reducing its US workforce by approximately 40%, or approximately 162 roles.
  • Sangamo expects to incur approximately $8 million-$10 million in one-time restructuring costs in the fourth quarter of 2023.
  • Revenues for the third quarter ended September 30, 2023 were $9.4 million, compared to $26.5 million for the same period in 2022.

Prime Medicine Presents First-ever Prime Editing Data in Non-human Primates Demonstrating Highly Efficient Ability of Prime Editors to Precisely Correct Disease-causing Mutation of GSD1b

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Friday, October 27, 2023
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CAMBRIDGE, Mass., Oct. 27, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology Company committed to delivering a new class of differentiated, one-time curative genetic therapies, today reported new preclinical data demonstrating the ability of liver-targeted Prime Editors to efficiently and precisely correct one of the most prevalent disease-causing mutations of glycogen storage disease 1b (GSD1b) in non-human primates (NHP) and mouse models. The data were presented today at the European Society of Gene and Cell Therapy (ESGCT) 2023 Congress in Brussels, Belgium.

Key Points: 
  • The data were presented today at the European Society of Gene and Cell Therapy (ESGCT) 2023 Congress in Brussels, Belgium.
  • “The data presented today are highly encouraging, both for patients and caregivers impacted by GSD1b, as well as for Prime Medicine and the field of gene editing,” said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of Prime Medicine.
  • “These data are the first Prime Editing data in NHPs and provide further proof-of-concept for our Prime Editing approach to potentially address a wide range of diseases, in this case, by targeting a specific gene with a liver-directed LNP.
  • In today’s presentation at ESGCT, Prime Medicine highlighted data from in vivo rodent and NHP studies with its Prime Editor targeting the p.L348fs mutation.

First Clinical Results of ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), Presented at ESGCT

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Friday, October 27, 2023
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ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).

Key Points: 
  • ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9).
  • Atamyo also announced today that Data Safety Monitoring Board (DSMB) authorized the enrollment of the second dose cohort of the ATA-001 clinical trial.
  • ATA-100 is being evaluated in a multi-center phase 1b/2b in Denmark, France, and the United Kingdom, and has received IND clearance by FDA.
  • ATA-100, a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins.

Epic Bio Reports New Data on Hypercompact Cas Proteins Optimized for Broad Therapeutic Applicability

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Thursday, October 26, 2023
Science, AAV, DCAS, Facioscapulohumeral muscular dystrophy, Biotechnology, FSHD, PAMS, Congress, Dicarboxylic acid, Cell, GEMS, Gene, ESGCT, European Society of Gene and Cell Therapy, Patient, Gene expression, SAN, Vaccine, Cas

SOUTH SAN FRANCISCO, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, is presenting new data reflecting advances in its Gene Expression Modulation System (GEMS) platform, alongside additional preclinical data supporting the company’s lead program, EPI-321, in development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). These data are being presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels, Belgium.

Key Points: 
  • These data are being presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels, Belgium.
  • Among the data being presented are new details on Epic’s in-house engineered suite of Cas molecules derived from CasMINI, the smallest Cas protein known to work in human cells.
  • In new data at ESGCT, Epic reports that these three Cas effectors also display greater flexibility in the genetic sequences, called PAMs, that they can target.
  • “These data highlight why GEMS is an unparalleled therapeutic platform, and how our team’s unique expertise is expanding the state of the science in epigenetic editing every day.”

Generation Bio Announces Demonstration of Highly Selective T Cell Transduction In Vivo with Cell-Targeted LNP Platform

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Thursday, October 26, 2023
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Generation Bio will present these data at the European Society for Gene and Cell Therapy (ESGCT) 30th Annual Congress in Brussels, Belgium at 4:40 p.m. CEST today.

Key Points: 
  • Generation Bio will present these data at the European Society for Gene and Cell Therapy (ESGCT) 30th Annual Congress in Brussels, Belgium at 4:40 p.m. CEST today.
  • Slides from the presentation will be made available on Generation Bio’s website following the presentation.
  • “Furthermore, species translation for the stealth LNP underlying our ctLNP platform is highly encouraging as we continue to move forward with our cell-targeting work.
  • Registration information for the webcast R&D deep dive can be found on the Events page of Generation Bio’s investor website.

Orchard Therapeutics Announces Presentation of Additional Positive Data from Proof-of-concept Study of OTL-203 in MPS-IH at ESGCT 2023

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Thursday, October 26, 2023
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The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.

Key Points: 
  • The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.
  • “The complications associated with MPS-IH involve multiple organ systems and have an adverse impact on patients’ quality of life.
  • Importantly, following treatment with OTL-203, no patients reported photophobia (light sensitivity), or any other ophthalmological symptoms typically associated with MPS-IH.
  • Secondary endpoints include biochemical markers, additional clinical assessments, as well as safety and tolerability.

Excision BioTherapeutics Presents Positive Interim Clinical Data from Ongoing Phase 1/2 Trial of EBT-101 for the Treatment of HIV at ESGCT 30th Annual Congress

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Wednesday, October 25, 2023
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EBT-101 is a dual-guide, CRISPR-based investigational gene therapy for the treatment of human immunodeficiency virus type 1 (HIV-1).

Key Points: 
  • EBT-101 is a dual-guide, CRISPR-based investigational gene therapy for the treatment of human immunodeficiency virus type 1 (HIV-1).
  • The data was presented at the European Society for Gene & Cell Therapy annual meeting on October 25, 2023 in Brussels, Belgium.
  • “Excision is dedicated to developing curative, CRISPR-based therapies for people with infectious disease,” said Daniel Dornbusch, Chief Executive Officer of Excision.
  • Excision uses a multiplexed and in vivo CRISPR-based gene editing approach to excise large segments of latently integrated proviral HIV DNA.

Cellectis to Present Pre-Clinical Data on HSPC Gene Therapy Program and Comprehensive Analysis of TALE-BE at the ESGCT 30th Annual Congress

Retrieved on: 
Tuesday, October 24, 2023
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The data will be presented in three posters:

Key Points: 
  • The data will be presented in three posters:
    Presenter: Eduardo Seclen, Senior Scientist & Team Leader, Gene Editing
    Intronic editing enables lineage specific expression of therapeutics relevant for HSPC gene therapy.
  • TALEN®-mediated intron editing of the CD11b locus results in the lineage-specific expression of a reporter transgene in myeloid cells, with negligible expression in HSPC or other cellular subsets in vitro and in vivo.
  • We believe this intron editing approach could be disruptive in HSPC gene therapy and brain delivery of multiple therapeutics.
  • Using a combination of scRNA sequencing and multiple genomic read out methodologies, we demonstrate that the mutant HBB gene can be efficiently corrected in HSPCs by TALEN®-mediated gene editing coupled to non-viral gene delivery (ssODN) with a low risk of generating β-thalassemic RBCs.

Taysha Gene Therapies Presents New Preclinical In-vitro Data on TSHA-102 in Rett Syndrome Supporting miRARE Regulation of MECP2 Expression at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress

Retrieved on: 
Tuesday, October 24, 2023
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DALLAS, Oct. 24, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced new preclinical in vitro data on TSHA-102 in Rett syndrome as part of a poster presentation at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress. TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. These data demonstrate the function of the miRARE-RHD1pA regulatory element and its impact on MECP2 transgene and protein expression in human and mouse cell lines, providing further support for the regulatory control of miRARE.

Key Points: 
  • TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression.
  • These data demonstrate the function of the miRARE-RHD1pA regulatory element and its impact on MECP2 transgene and protein expression in human and mouse cell lines, providing further support for the regulatory control of miRARE.
  • “Appropriate control of MECP2 transgene expression based on cellular levels of MeCP2 is fundamental to the development of a safe and effective gene therapy for Rett syndrome, given the mosaic pattern of MECP2 silencing in females with Rett syndrome,” said Sukumar Nagendran, M.D., President, and Head of R&D of Taysha.
  • “These new in vitro data recapitulating our in vivo findings in neonatal mice further our mechanistic understanding of how the miRARE technology controls post-transcriptional MECP2 expression and reinforce the potential of TSHA-102 to address the root cause of Rett syndrome.