Trans-splicing

Ascidian Therapeutics Raises $40 Million in Series A Extension Financing from Apple Tree Partners and Appoints Michael Ehlers as Interim CEO

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星期三, 十一月 8, 2023
FDA, IND, AAV, Trans-splicing, Food, Therapy, ATP, RNA, ABCA4, Retinopathy, Ascidian mitochondrial code, Exon, Regulation of tobacco by the U.S. Food and Drug Administration, Society, Cell, Stargardt disease, Risk, Gene, Animal, Drug discovery, DNA, Pharmaceutical industry, Vaccine, Stargardt, Ascidiacea

BOSTON, Nov. 8, 2023 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company focused on treating human diseases by rewriting RNA, today announced $40 million in Series A extension funding committed by Apple Tree Partners (ATP). The funds will be used to advance Ascidian's lead program, an RNA exon editor to halt the progression of Stargardt disease or other ABCA4 retinopathies, and additional programs in the company's pipeline. The company also announced that its founding CEO Michael Ehlers, M.D., Ph.D., will return as interim President and Chief Executive Officer, effective immediately. Dr. Ehlers, who also chairs Ascidian's Board of Directors and serves as Chief Scientific Officer at ATP as well as a venture partner at the firm, succeeds Romesh Subramanian, Ph.D., who is departing Ascidian.

Key Points: 
  • The company also announced that its founding CEO Michael Ehlers, M.D., Ph.D., will return as interim President and Chief Executive Officer, effective immediately.
  • Dr. Ehlers, who also chairs Ascidian's Board of Directors and serves as Chief Scientific Officer at ATP as well as a venture partner at the firm, succeeds Romesh Subramanian, Ph.D., who is departing Ascidian.
  • Today's announced financing follows ATP's initial $50 million Series A investment in Ascidian announced in October 2022.
  • Beyond its lead program, Ascidian is advancing programs with first-in-class potential in neurological and neuromuscular disorders.

EQS-News: Nature Communications publication reveals highly efficient AAV delivery of large genes using ViGeneron´s REVeRT technology

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星期四, 十月 26, 2023
Gene, Tissue, Genome, Nature Communications, AAV, Trans-splicing, Messenger, Vaccine, Reversion

The data demonstrate the delivery of genes larger than the usual AAV packaging size with high reconstitution efficiency, enabling gene supplementation or simultaneous knockout and transcriptional activation of different genes.

Key Points: 
  • The data demonstrate the delivery of genes larger than the usual AAV packaging size with high reconstitution efficiency, enabling gene supplementation or simultaneous knockout and transcriptional activation of different genes.
  • Researchers in today’s Nature Communications paper report that REVeRT was validated both in vitro and in vivo for the efficient reconstitution of disease relevant genes and gene editing modules larger than 5kb.
  • The REVeRT technology achieves high expression levels of selected genes without generating alien proteins as side products, thereby mitigating potential immunogenicity concerns.
  • We are actively exploring strategic partnerships in various disease areas to accelerate the delivery of these transformative treatments to patients.”

Nature Communications publication reveals highly efficient AAV delivery of large genes using ViGeneron’s REVeRT technology

Retrieved on: 
星期三, 十月 25, 2023
Gene, Tissue, Genome, Nature Communications, AAV, Trans-splicing, Messenger, Vaccine

The data demonstrate the delivery of genes larger than the usual AAV packaging size with high reconstitution efficiency, enabling gene supplementation or simultaneous knockout and transcriptional activation of different genes.

Key Points: 
  • The data demonstrate the delivery of genes larger than the usual AAV packaging size with high reconstitution efficiency, enabling gene supplementation or simultaneous knockout and transcriptional activation of different genes.
  • Researchers in today’s Nature Communications paper report that REVeRT was validated both in vitro and in vivo for the efficient reconstitution of disease relevant genes and gene editing modules larger than 5kb.
  • The REVeRT technology achieves high expression levels of selected genes without generating alien proteins as side products, thereby mitigating potential immunogenicity concerns.
  • We are actively exploring strategic partnerships in various disease areas to accelerate the delivery of these transformative treatments to patients.”

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

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星期二, 五月 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.

Ascidian Therapeutics to Present Data From Its Lead Program Targeting ABCA4 Retinopathies at the Retinal Cell and Gene Therapy Innovation Summit and ARVO 2023 Annual Meeting

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星期二, 四月 18, 2023
ARVO, Trans-splicing, Gene, Stargardt disease, Visual impairment, Foundation Fighting Blindness, Biotechnology, Gene editing, Stargardt, Retina, Disease, RNA, DSM-IV codes, Publication, AAV, ABCA4, Retinopathy, Oregon Health & Science University, Patient, Physician, OHSU, School of Life Sciences (University of Dundee), Association for Research in Vision and Ophthalmology, Research, Therapy, Summit, Pharmaceutical industry, Vaccine, Ophthalmology, Ascidiacea, IND

BOSTON, April 18, 2023 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company focused on treating human diseases by rewriting RNA, announced today that Chief Medical Officer Jay Barth, M.D., will present at the Eighth Annual Retinal Cell and Gene Therapy Innovation Summit on April 21, 2023, and Head of Molecular Biology Shimyn Slomovic, Ph.D., will present at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting on April 26, 2023. Their sessions will highlight Ascidian's groundbreaking RNA exon editing platform and the company's lead program targeting ABCA4 retinopathies, including Stargardt disease, which is currently in IND-enabling studies. 

Key Points: 
  • Their sessions will highlight Ascidian's groundbreaking RNA exon editing platform and the company's lead program targeting ABCA4 retinopathies, including Stargardt disease, which is currently in IND-enabling studies.
  • Ascidian's first-of-its-kind RNA exon editing platform is designed to expand the therapeutic possibilities of genetic medicine and treat diseases not addressable by today's gene therapy and gene editing technologies.
  • Data to be shared at ARVO demonstrate ABCA4 RNA exon editing in multiple model systems, including cell lines, human retinal explants, and non-human primates at time points extending to six months.
  • Dr. Barth's presentation at the Retinal Cell and Gene Therapy Innovation Summit will provide an update on Ascidian's lead program and the promise of RNA exon editing for patients with Stargardt disease and other ABCA4-related retinopathies.