Gene

Omega Therapeutics to Present New Preclinical Data on Epigenomic Upregulation at the American Society of Gene and Cell Therapy 27th Annual Meeting

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Tuesday, April 23, 2024

CAMBRIDGE, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- Omega Therapeutics, Inc. (Nasdaq: OMGA) (“Omega”), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, today announced it will present a poster at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, MD, May 7 – 11, 2024.

Key Points: 
  • CAMBRIDGE, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- Omega Therapeutics, Inc. (Nasdaq: OMGA) (“Omega”), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, today announced it will present a poster at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, MD, May 7 – 11, 2024.
  • The poster will feature new preclinical data that demonstrate the ability of Omega's programmable epigenomic controllers (ECs) to pre-transcriptionally upregulate gene expression across a diverse set of genes, offering a broad range of potential therapeutic applications.
  • Details for the poster presentation are as follows:
    The poster will be made available on the Omega website at https://omegatherapeutics.com/science/publications/ at the same time as the presentation.

Sania Therapeutics presents latest from AAV gene therapy platform at the American Society of Gene & Cell Therapy Annual Meeting

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Tuesday, April 23, 2024

Andy Murray, Ph.D, CEO & Co-Founder of Sania Therapeutics, said: “Since our official launch last May, we have been pressing ahead with developing truly differentiated AAV gene therapies.

Key Points: 
  • Andy Murray, Ph.D, CEO & Co-Founder of Sania Therapeutics, said: “Since our official launch last May, we have been pressing ahead with developing truly differentiated AAV gene therapies.
  • Sania’s approach, using its R-Scan platform, is focused on generating novel, targeted vectors that can be successfully and rapidly translated into the clinic.
  • It combines human induced pluripotent stem cell derived cell types and microfluidics to perform directed AAV capsid evolution in an in vitro human system.
  • It combines an intramuscular injection of AAV gene therapy with oral medicine to selectively reduce excitability in targeted motor neurons.

Sania Therapeutics presents latest from AAV gene therapy platform at the American Society of Gene & Cell Therapy Annual Meeting

Retrieved on: 
Tuesday, April 23, 2024

LONDON, UK & NEW YORK, USA – 11 April 2024, Sania Therapeutics (the “Company”), focused on developing targeted genetic medicines for prevalent disorders today announces that it will be presenting three posters at the American Society of Gene & Cell Therapy 2024 Annual Meeting, taking place from 7-11 May 2024 in Baltimore, MD.

Key Points: 
  • LONDON, UK & NEW YORK, USA – 11 April 2024, Sania Therapeutics (the “Company”), focused on developing targeted genetic medicines for prevalent disorders today announces that it will be presenting three posters at the American Society of Gene & Cell Therapy 2024 Annual Meeting, taking place from 7-11 May 2024 in Baltimore, MD.
  • Further details are below.
  • Abstracts details will be shared in due course and the posters will be made available on Sania’s website following their presentation.

Regeneron to Highlight Advances in Genetic Medicine Research at American Society of Gene and Cell Therapy (ASGCT)

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Monday, April 22, 2024

TARRYTOWN, N.Y., April 22, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that new and updated data across its genetic medicines portfolio will be presented at the American Society of Gene and Cell Therapy (ASGCT) annual conference in Baltimore, Maryland, from May 7 to 11, 2024. Data from 10 abstracts, including six oral presentations, provide insight on Regeneron’s approach to overcoming obstacles to clinical implementation of genetic medicines, from pre-dosing to delivery to long-term sustained expression. The company will also present updated data from the Phase 1/2 CHORD trial investigating DB-OTO in children with profound genetic hearing loss due to mutations of the otoferlin gene.

Key Points: 
  • Data from 10 abstracts, including six oral presentations, provide insight on Regeneron’s approach to overcoming obstacles to clinical implementation of genetic medicines, from pre-dosing to delivery to long-term sustained expression.
  • The company will also present updated data from the Phase 1/2 CHORD trial investigating DB-OTO in children with profound genetic hearing loss due to mutations of the otoferlin gene.
  • “Regeneron continues to advance methods to overcome these obstacles through our proprietary delivery approaches utilizing next-generation viral vectors, particularly specific retargeting antibodies and innovative payloads.
  • The ASGCT presentation will build on promising early results in the first patient,” said Aris Baras, M.D., Senior Vice President, Co-Head of Regeneron Genetic Medicines and Head, Regeneron Genetics Center®.

Generation Bio Announces the Acceptance of Five Abstracts and an Invited Oral Presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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Monday, April 22, 2024

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO) a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that six abstracts highlighting preclinical data from its cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms have been accepted for presentation, including one oral presentation, at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, 2024 in Baltimore, MD.

Key Points: 
  • CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO) a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that six abstracts highlighting preclinical data from its cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms have been accepted for presentation, including one oral presentation, at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, 2024 in Baltimore, MD.
  • “We are delighted to have a robust presence at this year’s ASGCT meeting and showcase some of the innovative technical work underpinning the breakthroughs we have achieved for our cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms,” said Matt Stanton, Ph.D., chief scientific officer.
  • “We’ve made rapid progress across our platforms and look forward to presenting further updates at the meeting.”

Arbor Biotechnologies to Present Data Supporting Therapeutic Programs in PH1 and ALS, and the Discovery of a Novel Type V Nuclease at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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Monday, April 22, 2024

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Arbor Biotechnologies®, a biotechnology company discovering and developing the next generation of genetic medicines, today announced four upcoming presentations at the 2024 American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, taking place May 7-11 in Baltimore, Maryland.

Key Points: 
  • Arbor will present in vivo NHP data supporting clinical development of ABO-101, its most advanced gene editing therapeutic candidate designed to address primary hyperoxaluria type 1 (PH1) through inactivation of the HAO1 gene in the liver.
  • The company also will present data supporting its end-to-end nuclease development platform in a third presentation outlining the discovery and optimization of a unique, compact nuclease, termed ABR-004.
  • Proof-of-concept data in non-human primates show potent, therapeutically relevant silencing of PCSK9 in vivo with the novel nuclease, signaling opportunities for broader therapeutic applications.
  • Together, the suite of presentations will demonstrate the utility of Arbor’s proprietary discovery and development approach for enabling efficient identification and optimization of novel nucleases.

TScan Therapeutics Announces Upcoming Presentations at the American Society of Gene and Cell Therapy 27th Annual Meeting

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Monday, April 22, 2024

WALTHAM, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, today announced the acceptance of one abstract for oral presentation and four abstracts for poster presentation at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting being held May 7-11 in Baltimore, MD as well as virtually.

Key Points: 
  • WALTHAM, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, today announced the acceptance of one abstract for oral presentation and four abstracts for poster presentation at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting being held May 7-11 in Baltimore, MD as well as virtually.

Excision BioTherapeutics to Present Positive Data from its HSV-1 Keratitis Program, EBT-104, at the ASGCT 2024 Annual Meeting

Retrieved on: 
Monday, April 22, 2024

Herpes Simplex Keratitis (HSK), caused by the infection of herpes simplex virus type 1 (HSV-1) in the cornea, is a major cause of blindness worldwide.

Key Points: 
  • Herpes Simplex Keratitis (HSK), caused by the infection of herpes simplex virus type 1 (HSV-1) in the cornea, is a major cause of blindness worldwide.
  • Although current anti-HSV-1 therapies interfere with viral DNA replication, they do not eliminate HSV-1 reservoirs or prevent recurrence.
  • CRISPR/Cas-mediated gene editing can potentially address the underlying causes of the disease by directly eliminating the latent HSV-1 reservoirs.
  • The 440 guide RNA pairs formed with these 42 PlmCasX guide RNAs were tested in tissue culture for DNA excision efficiency.

Tessera Therapeutics to Present New Data Across Preclinical Programs for its Gene Writing™ and Delivery Platforms at the American Society of Gene and Cell Therapy 27th Annual Meeting

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Monday, April 22, 2024

SOMERVILLE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Tessera Therapeutics, a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, today announced six presentations of new preclinical data at the upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Baltimore, Maryland, May 7 – 11, 2024.

Key Points: 
  • SOMERVILLE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Tessera Therapeutics, a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, today announced six presentations of new preclinical data at the upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place in Baltimore, Maryland, May 7 – 11, 2024.
  • “We look forward to presenting multiple new datasets at this year’s ASGCT Annual Meeting, including our progress towards in vivo therapies for phenylketonuria, alpha-1 antitrypsin deficiency, sickle cell disease and Wilson’s disease, as well as our advancements in novel T-cell based therapies,” said Michael Severino, M.D., CEO of Tessera Therapeutics.
  • “We continue to focus on the development of transformative genetic medicines through the applications of our Gene Writing and non-viral delivery platforms.”
    Details of the Company’s ASGCT 27th Annual Meeting presentations are as follows:

Excision BioTherapeutics Announces Oral Presentation Highlighting Positive Data from its HBV Program, EBT-107, at the Upcoming ASGCT 2024 Annual Meeting

Retrieved on: 
Monday, April 22, 2024

HBV is one of the most prevalent infectious diseases worldwide that lacks curative therapies.

Key Points: 
  • HBV is one of the most prevalent infectious diseases worldwide that lacks curative therapies.
  • While existing antiviral and immunomodulator treatments slow liver damage by reducing viral load, they fail to eliminate covalently closed circular DNA (cccDNA) that enables persistent viral infection.
  • Excision’s lead product candidate for the treatment of HBV infection, EBT-107, uses dual guide RNAs to effectively deactivate the virus and prevent the emergence of escape variants.
  • Testing a selected combination of editing nucleases and paired guide RNAs in HBV-infected cells, we observed decreased copies of total HBV DNA.