RNA

Alnylam and Dicerna Form RNAi Therapeutics Collaboration on Alpha-1 Antitrypsin Deficiency-Associated Liver Disease and Complete Cross-License Agreement for Primary Hyperoxaluria Programs

Monday, April 6, 2020 - 12:00pm

Specifically, the new agreements allow for Alnylam and Dicerna to join forces in areas of common interest, namely alpha-1 liver disease and primary hyperoxaluria, said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.

Key Points: 
  • Specifically, the new agreements allow for Alnylam and Dicerna to join forces in areas of common interest, namely alpha-1 liver disease and primary hyperoxaluria, said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.
  • Under the development and commercialization agreement, Alnylams ALN-AAT02 and Dicernas DCR-A1AT, investigational RNAi therapeutics, each in Phase 1/2 development, will be explored for the treatment of alpha-1 liver disease.
  • Dicerna will select which product candidate(s) to advance in development for the treatment of patients with alpha-1 liver disease.
  • ALN-AAT02 and DCR-A1AT are investigational, subcutaneously administered RNAi therapeutics targeting alpha-1 antitrypsin (A1AT) in development for the treatment of A1AT deficiency-associated liver disease (alpha-1 liver disease).

Vir and Alnylam Expand Collaboration to Advance Investigational RNAi Therapeutics Targeting Host Factors for the Treatment of COVID-19

Thursday, April 2, 2020 - 12:00pm

As part of the original collaboration to advance investigational RNAi therapeutics to treat disease caused by coronavirus infection, Alnylam has designed and synthesized over 350 siRNAs targeting highly conserved regions of the SARS-CoV-2 genome.

Key Points: 
  • As part of the original collaboration to advance investigational RNAi therapeutics to treat disease caused by coronavirus infection, Alnylam has designed and synthesized over 350 siRNAs targeting highly conserved regions of the SARS-CoV-2 genome.
  • Upon DC selection, Vir will lead development efforts working closely with Alnylam to generate the data required to enable the potential for rapid commencement of clinical studies.
  • The biopharmaceutical industry needs to advance its full armamentarium of potential treatment approaches and strategies to address the COVID-19 pandemic.
  • Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform.

Innovations in Gene Therapy, Microbiome, Robotics, Ribozyme & Protein Therapy - 2019 - ResearchAndMarkets.com

Wednesday, April 1, 2020 - 4:21pm

The "Innovations in Gene Therapy, Microbiome, Robotics, Ribozyme, and Protein Therapy" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Innovations in Gene Therapy, Microbiome, Robotics, Ribozyme, and Protein Therapy" report has been added to ResearchAndMarkets.com's offering.
  • This edition of the Life Science, Health & Wellness TechVision Opportunity Engine (TOE) encompasses innovation insights across biosimilars, gene therapy, microbiome, robotics, ribozyme, and protein therapy.
  • The TOE also provides technological analysis for recent developments in animal protein applications, microbiome, and cell cultures.
  • The Life Science, Health & Wellness TOE will feature disruptive technology advances in the global life sciences industry.

Zymo Research Announces Partnership with Tecan for COVID-19 Viral RNA Extraction

Wednesday, April 1, 2020 - 12:38am

The DreamPrep NAP workstation, featuring Zymo Research's Quick-DNA/RNA Viral MagBead Kit, simplifies the process by providing pre-loaded scripts, optimized for viral RNA extraction, that are currently being validated for COVID-19 assays by high complexity testing labs.

Key Points: 
  • The DreamPrep NAP workstation, featuring Zymo Research's Quick-DNA/RNA Viral MagBead Kit, simplifies the process by providing pre-loaded scripts, optimized for viral RNA extraction, that are currently being validated for COVID-19 assays by high complexity testing labs.
  • Both Zymo Research and Tecan are giving the highest priority to assist researchers who are extracting viral RNA using the DreamPrep NAP workstation."
  • In the past year, Zymo Research and Tecan have worked together to optimize magnetic bead-based extraction of viral RNA using the cutting-edge DreamPrep NAP workstation.
  • The result of this collaboration is a preprogrammed, preconfigured, load-and-go system that is optimized for high-throughput viral RNA extraction.

Berkeley Lights Expands Its Platform and Introduces the OptoSeq™ 3' mRNA Library Kit

Tuesday, March 31, 2020 - 5:00pm

EMERYVILLE, Calif., March 31, 2020 /PRNewswire/ -- Today, Berkeley Lights, a leader in cell selection, announced their new OptoSeq 3' mRNA Library Kit enabling users to directly link phenotype to genotype.

Key Points: 
  • EMERYVILLE, Calif., March 31, 2020 /PRNewswire/ -- Today, Berkeley Lights, a leader in cell selection, announced their new OptoSeq 3' mRNA Library Kit enabling users to directly link phenotype to genotype.
  • The OptoSeq 3' mRNA Library Kit significantly expands the capabilities of the Berkeley Lights platform.
  • "With the new OptoSeq 3' mRNA Library Kit, customers can now use the Berkeley Lights platform to refine library prep, allowing users to consolidate workflows and complete the cell journey on one system."
  • The OptoSeq 3' mRNA Library Kit includes reagents, primers, probes, and enzymes to prepare and label individual cells for cDNA sequencing.

Horizon Discovery and Pharmahungary Report Positive Early-Stage Results for Novel Micro-RNA Therapeutic for Ischemic Heart Diseases

Tuesday, March 31, 2020 - 3:09pm

Horizon Discovery Group plc (LSE: HZD) (Horizon, the Company or the Group), a global leader in the application of gene editing and gene modulation for cell line engineering, and Pharmahungary Group, a preclinical and clinical R&D company, today announced positive early-stage results for a novel micro-RNA therapeutic for ischemic heart diseases.

Key Points: 
  • Horizon Discovery Group plc (LSE: HZD) (Horizon, the Company or the Group), a global leader in the application of gene editing and gene modulation for cell line engineering, and Pharmahungary Group, a preclinical and clinical R&D company, today announced positive early-stage results for a novel micro-RNA therapeutic for ischemic heart diseases.
  • The potential therapeutic has been developed under Pharmahungarys ProtectomiR program, under which several miRNA compounds for cytoprotection in ischemic diseases, termed ProtectomiRs, have been identified.
  • The aim is to develop a curative treatment for ischemic heart disease, the number one cause of mortality worldwide.
  • We look forward to continuing our partnership with Horizon Discovery, to work toward our goal of developing novel drugs for treatment of ischemic heart diseases.

NeuBase Therapeutics Announces Positive, Preclinical Data Validating its Novel Genetic Therapy PATrOL™ Platform

Tuesday, March 31, 2020 - 12:00pm

ET

Key Points: 
  • ET
    PITTSBURGH, March 31, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq:NBSE) (NeuBase or the Company), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies to address genetic diseases, today announced positive preclinical data from its pharmacokinetics studies in non-human primates (NHPs) and in vitro pharmacodynamics data in patient-derived cell lines.
  • NeuBase Therapeutics, Inc. will discuss these data and next steps for development during a webcasted conference call with slides today, March 31, 2020, at 8:00 a.m.
  • NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides.
  • Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

Moderna Provides Update on the Impact of COVID-19 on Business Operations and Clinical Program Development

Monday, March 30, 2020 - 1:48am

Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today provided an update on the impact of the rapidly evolving COVID-19 pandemic on its business operations and clinical program development.

Key Points: 
  • Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today provided an update on the impact of the rapidly evolving COVID-19 pandemic on its business operations and clinical program development.
  • Moderna remains committed to its clinical development plans and is working closely with all stakeholders to try to mitigate the impact of the pandemic on the Companys ongoing clinical trials.
  • The Company plans to provide a detailed update on its clinical development programs during its first quarter 2020 conference call.
  • Moderna has 24 mRNA development candidates in its portfolio across all modalities, with 12 in clinical studies.

NeuBase Therapeutics Reports Financial Results for the First Fiscal Quarter of 2020

Thursday, March 26, 2020 - 8:05pm

Next week, we plan to announce the results from a pharmacokinetic study in non-human primates, as well as pharmacodynamic data in patient-derived cell lines.

Key Points: 
  • Next week, we plan to announce the results from a pharmacokinetic study in non-human primates, as well as pharmacodynamic data in patient-derived cell lines.
  • The Company believes that its current cash balance will provide sufficient capital to fund operations through the end of fiscal year 2020.
  • NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides.
  • These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements.

Dicerna Addresses Business Continuity in Response to COVID-19 Pandemic

Thursday, March 26, 2020 - 8:05pm

Dicerna Pharmaceuticals, Inc. (Nasdaq:DRNA) (the Company or Dicerna), a leading developer of ribonucleic acid interference (RNAi) therapies, today provided an overview of its response to the COVID-19 pandemic and an update on business continuity and clinical development milestones.

Key Points: 
  • Dicerna Pharmaceuticals, Inc. (Nasdaq:DRNA) (the Company or Dicerna), a leading developer of ribonucleic acid interference (RNAi) therapies, today provided an overview of its response to the COVID-19 pandemic and an update on business continuity and clinical development milestones.
  • We will continue to monitor each of the ongoing trials across our development programs with the goal of ensuring trial continuity and advancement where feasible.
  • Dicerna is instituting measures to ensure continuity of care and continued dosing of patients currently enrolled in the PHYOX2 trial.
  • To facilitate an effective remote-work policy and to ensure business continuity, Dicerna has also made available to its employees an enhanced suite of technology resources to permit secure remote access and to enable business to continue virtually.