Gene targeting

Beam Therapeutics Reports Pipeline Updates and First Quarter 2023 Financial Results

Retrieved on: 
Wednesday, May 10, 2023
Well, GMP, Tissue, ESCAPE, Research and development, Drug development, Stargardt disease, Hematology, Gene targeting, Biotechnology, SCD, Clinical trial, Bone marrow, Cancer, G&A, Patient, Eye, Science, AATD, Investment, Security (finance), Administration, DSM-IV codes, Sickle cell disease, Liver, R, LNP, LNP Media Group, Services General Administration and Coordination Department, Government of Sindh, Society, Vaccine, Pharmaceutical industry, Medical imaging, Stargardt, Beam

CAMBRIDGE, Mass., May 10, 2023 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported first quarter 2023 financial results and provided an update on its BEACON clinical trial and pipeline progress.

Key Points: 
  • Beam expects to fully enroll the sentinel cohort in 2023.
  • Beam expects to initiate current good manufacturing practice compliant operations at its North Carolina manufacturing facility in late 2023.
  • Research & Development (R&D) Expenses: R&D expenses were $99.6 million for the first quarter of 2023, compared to $65.4 million for the first quarter of 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $23.5 million for the first quarter of 2023, compared to $19.2 million for the first quarter of 2022.

Ascidian Therapeutics to Present New Data From Its Lead Program Targeting ABCA4 Retinopathies at the ASGCT 2023 Annual Meeting

Retrieved on: 
Tuesday, May 9, 2023
Immortalised cell line, Bell, Trans-splicing, Gene, Macular degeneration, File, Stargardt disease, Gene targeting, Gene editing, PDT, RNA, Publication, AAV, CNS, Animal, Patient, Retinopathy, Cell, Ascidian mitochondrial code, Therapy, Society, Los Angeles Convention Center, Pharmaceutical industry, Vaccine, Stargardt, ABCA4, Ascidiacea, IND

BOSTON, May 9, 2023 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company focused on treating human diseases by rewriting RNA, announced today that Head of Research Robert Bell, Ph.D., will present new data from its lead ABCA4 program at the Twenty-Sixth Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).

Key Points: 
  • The lead program targets ABCA4-related retinopathies, including Stargardt disease, and is currently in IND-enabling studies advancing towards clinical development.
  • The oral presentation, taking place at 9:00 a.m. PDT on May 20, will highlight Ascidian's groundbreaking RNA exon editing platform.
  • Specifically, Dr. Bell will present new six-month data from Ascidian's lead ABCA4 program that demonstrate the production of full-length ABCA4 protein following a one-time treatment with a single AAV-delivered RNA exon editing development candidate in the non-human primate retina.
  • These data are the first to quantify therapeutically relevant levels of ABCA4 protein and represent the most efficient and durable RNA exon editing via trans-splicing ever demonstrated in large animals.

Epic Bio to Present Preclinical Data at American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting

Retrieved on: 
Thursday, May 4, 2023
Gene targeting, Biotechnology, DCAS, Gene, Society, Gene expression, SAN, Cell, Vaccine

SOUTH SAN FRANCISCO, Calif., May 04, 2023 (GLOBE NEWSWIRE) -- Epic BIO , a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced the acceptance of an abstract for oral presentation at the upcoming Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 16-20, 2023, in Los Angeles, Calif.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., May 04, 2023 (GLOBE NEWSWIRE) -- Epic BIO , a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced the acceptance of an abstract for oral presentation at the upcoming Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place May 16-20, 2023, in Los Angeles, Calif.
    Full details of the presentation are as follows:
    Session: Gene Targeting and Gene Correction: Hemoglobin, Muscle, and Eye

Tessera Therapeutics to Present New Data Showcasing the Broad Potential of its Gene Writing™ and Delivery Platforms at the American Society of Gene and Cell Therapy 26th Annual Meeting

Retrieved on: 
Tuesday, May 2, 2023
Gene, Gene targeting, Biotechnology, RNA, Patient, American Society of Gene and Cell Therapy, Cell, Annual general meeting, Small RNA, Therapy, Emerging technologies, Society, Vaccine, Antibiotics

SOMERVILLE, Mass., May 02, 2023 (GLOBE NEWSWIRE) -- Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic medicine known as Gene Writing™, today announced that it will present new preclinical data at the upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Los Angeles, California, May 16 – 20, 2023.

Key Points: 
  • SOMERVILLE, Mass., May 02, 2023 (GLOBE NEWSWIRE) -- Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic medicine known as Gene Writing™, today announced that it will present new preclinical data at the upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Los Angeles, California, May 16 – 20, 2023.
  • Details of the Company’s ASGCT 26th Annual Meeting presentations are as follows:
    Session: Gene Targeting and Gene Correction: Liver
    Session: Gene Targeting and Gene Correction: New Technologies
    Title: Highly Efficient Correction of the Sickle Cell Disease Mutation in Patient HSC Using an RNA Gene Writing System, an RNA-based, Nuclease-Free Approach to Genome Editing

EdiGene to Unveil Promising Preclinical POC Data for LEAPER™ 2.0-based in vivo RNA Editing Therapies in NHP Model at the 26th Annual Meeting of ASGCT via Oral Presentation

Retrieved on: 
Tuesday, May 2, 2023
Research, Genetics, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Oncology, Other Science, Gene, Gene targeting, Algorithm, RNA, CNS, Human germline engineering, Patient, ADAR, Cell, NHP, POC, Society, Vaccine

LEAPERTM 2.0 is our proprietary exogenous protein-free RNA base editing technology that uses engineered circular ADAR-recruiting RNAs.

Key Points: 
  • LEAPERTM 2.0 is our proprietary exogenous protein-free RNA base editing technology that uses engineered circular ADAR-recruiting RNAs.
  • Data from specific NHP disease model have demonstrated the preclinical safety and efficacy of LEAPERTM 2.0-based in vivo therapeutic approach with outstanding editing efficiency.
  • With endogenous ADAR, LEAPERTM has achieved precise and highly efficient RNA base editing using only one guide RNA (arRNA).
  • Additionally, the exogenous protein-free feature significantly reduces delivery difficulty and minimizes the potential for immune response induced by foreign proteins.

Chroma Medicine to Present First Data Demonstrating Promise of Its Epigenetic Editing Platform at 26th ASGCT Annual Meeting

Retrieved on: 
Tuesday, May 2, 2023
DNA, Therapy, Gene, CSO, Gene targeting, Liver, Deletion, Gene expression, Human, Hypercholesterolemia, Chromosomal translocation, Data, LDL, Annual general meeting, Cell, DNA methylation, Society, Medicine, Antibiotics, Vaccine, Chroma

BOSTON, May 2, 2023 /PRNewswire/ -- Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced two oral data presentations that showcase the potential of its epigenetic editing platform at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16-20, 2023 in Los Angeles, California. The presentations include the first in vivo proof-of-concept data for Chroma's epigenetic editing platform along with data demonstrating the key advantages of epigenetic editing for multiplex gene regulation. Chroma plans to release data from additional pipeline programs at upcoming scientific conferences.  

Key Points: 
  • In vivo data provide compelling proof-of-concept for Chroma's epigenetic editing platform to enable highly efficient, specific, and durable gene regulation
    BOSTON, May 2, 2023 /PRNewswire/ -- Chroma Medicine , Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced two oral data presentations that showcase the potential of its epigenetic editing platform at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16-20, 2023 in Los Angeles, California.
  • The presentations include the first in vivo proof-of-concept data for Chroma's epigenetic editing platform along with data demonstrating the key advantages of epigenetic editing for multiplex gene regulation.
  • Chroma plans to release data from additional pipeline programs at upcoming scientific conferences.
  • Data presented at ASGCT demonstrate the ability of epigenetic editors to facilitate genotoxicity-free multiplexed editing in healthy donor-derived T cells, as compared to Cas9-mediated editing.

Poseida Therapeutics Announces Multiple Presentations at the American Society of Gene and Cell Therapy 26th Annual Meeting

Retrieved on: 
Tuesday, May 2, 2023
Gene targeting, American Society of Gene and Cell Therapy, Cancer, Rare, Therapy, Gene, Patient, Society, SAN, Los Angeles Convention Center, Cell, Annual general meeting, Book

SAN DIEGO, May 2, 2023 /PRNewswire/ -- 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, today announced 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) 26th Annual Meeting, being held at the Los Angeles Convention Center in Los Angeles and virtually on May 16-20, 2023.

Key Points: 
  • SAN DIEGO, May 2, 2023 /PRNewswire/ -- 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, today announced 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) 26th Annual Meeting, being held at the Los Angeles Convention Center in Los Angeles and virtually on May 16-20, 2023.
  • Session Title: Gene Targeting and Gene Correction: Liver
    Abstract & Poster Board Number: 638
    Abstract & Poster Board Number: 945
    Abstract & Poster Board Number: 1212

Hadassah Cancer Research Institute (HCRI) Unveils a Breakthrough in Personalized Cancer Care with a Data-Driven, Gene Targeting Tool

Retrieved on: 
Monday, February 27, 2023
Cancer, MD, Disease, Gene, Risk, Research, Prognosis, Patient, Somatic mutation, HCRI, Radiation therapy, Computational biology, Laboratory, Gene targeting, Medical imaging

JERUSALEM, Feb. 27, 2023 /PRNewswire/ -- The Hadassah Cancer Research Institute at the Hadassah University Medical Center in Jerusalem, announced today that researchers have designed a genomic analysis tool for cancer patients that enables advanced selection of drugs targeting schemes for cancer genes, opening the door to improved personalized medicine in cancer treatment. The science behind this breakthrough tool is a computational biology method to assess the relative effect of each position-specific point mutation in 535 cancer genes, by quantifying the relative biological and clinical importance of point mutations. This enables the prioritization of genes for targeting and will lead to the development of new, more effective combination therapies, further improving the prognosis for cancer patients.

Key Points: 
  • JERUSALEM, Feb. 27, 2023 /PRNewswire/ -- The Hadassah Cancer Research Institute at the Hadassah University Medical Center in Jerusalem, announced today that researchers have designed a genomic analysis tool for cancer patients that enables advanced selection of drugs targeting schemes for cancer genes, opening the door to improved personalized medicine in cancer treatment.
  • This enables the prioritization of genes for targeting and will lead to the development of new, more effective combination therapies, further improving the prognosis for cancer patients.
  • "With this new gene targeting tool, we are finally able to bring the power of personalized medicine to the forefront of cancer care.
  • For more information on the Hadassah Cancer Research Institute, contact:

CRISPR and Cas Genes Market Report 2022: Growing Need For Novel Therapeutics Boosts Sector - ResearchAndMarkets.com

Retrieved on: 
Wednesday, November 30, 2022
Science, Biotechnology, Research, Pharmaceutical, Health, Infectious Diseases, Genetics, Clinical Trials, COVID-19, Gene targeting, Gene editing, CRISPR Therapeutics, Cas, Pharma, Cros, Sickle cell disease, Gene, CRISPR, Machine learning, R, Product-service system, Therapy, USD, SCD, Nanyang Technological University, Workflow, Cas9, Vertex Pharmaceuticals, Biomedical Research, Genome-wide CRISPR-Cas9 knockout screens, Research, Frontiers, Biomedicine, Agriculture, Antibiotics, Application

The global CRISPR and Cas genes market size is expected to reach USD 9.6 billion by 2030.

Key Points: 
  • The global CRISPR and Cas genes market size is expected to reach USD 9.6 billion by 2030.
  • The market is expected to register a CAGR of 17.9% during the forecast period, owing to the increasing usage of CRISPR and Cas genes systems in the biomedical research field.
  • In January 2020, Mammoth Biosciences raised USD 45 million to develop next-gen CRISPR products for therapeutics & diagnostics purposes.
  • Chapter 3 Crispr & Cas Genes Market Variables, Trends And Scope
    Chapter 7 Market Categorization: Regional Estimates & Trend Analysis by Product, Application & End-use

Ozgene and Gen-H partner on Ozgene’s new OzBIG targeted humanization capability

Retrieved on: 
Thursday, July 21, 2022
Health, Stem Cells, Genetics, Other Health, Other Science, Science, Biotechnology, Breeding, Escherichia coli, Genome, BAC, Research, CRO, Drug discovery, Partnership, Gene targeting, Disease, Mouse Models of Human Cancer database, Federal Mediation and Conciliation Service (United States), CRISPR, Gene, Technology, Science, Mouse, Antibiotics

Ozgene and Gen-H are pleased to announce our working partnership supporting Ozgenes new OzBIG capability.

Key Points: 
  • Ozgene and Gen-H are pleased to announce our working partnership supporting Ozgenes new OzBIG capability.
  • Ozgene Pty Ltd is a privately held CRO based in Perth, Australia providing custom genetically engineered mice for academic and commercial researchers worldwide.
  • Gen-H GmbH is a privately held CRO based in Heidelberg, Germany providing genetically engineered bacterial clones and E. coli strains.
  • OzBIG vastly expands the gene set available for humanization by enabling the humanization of 90% of the genes in the mouse genome, compared to 10% with standard gene targeting.