RNA editing

Wave Life Sciences Announces Continued Momentum in GSK Collaboration and Advancements in siRNA and RNA Editing

Retrieved on: 
Tuesday, April 23, 2024
Sequence, Small RNA, RNA editing, Potency, Durability, Health, ...Continued, RNA, MD, Doctor of Philosophy, Weight loss, IND, CNS, Obesity, Oligonucleotide, GSK, Patient, Medicinal chemistry, MBA, Liu Wei, Clinical trial, Pharmaceutical industry

CAMBRIDGE, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today provided an update on its best-in-class small interfering RNA (siRNA) and RNA editing platform capabilities.

Key Points: 
  • As part of Wave’s ongoing collaboration with GSK, GSK has selected its first two programs to advance to development candidates following achievement of target validation.
  • GSK will provide an aggregate initiation payment of $12 million to Wave for these two oligonucleotide programs.
  • Under the agreement, GSK can advance up to eight programs leveraging Wave’s PRISM™ platform and multiple RNA-targeting modalities (RNA editing, splicing, siRNA, and antisense) with target validation work ongoing across multiple therapy areas.
  • Beyond these programs, our collaboration is focusing on all Wave modalities, including RNA editing.

ProQR Announces Year End 2023 Operating and Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Trident, Eli Lilly, Exercise, ProQR, Patient, IP, Therapy, Lilly, RNA editing, Cholestasis, Rett syndrome, Research, Cardiovascular disease, Partnership, Pseudouridine, NTCP, RNA, Functional, Uridine, MECP2, Oligonucleotide, ACTB, LPL, Annual report, Conference, Patent, Biomarker, Mouse, RSRT, Eli Lilly and Company, Liver, ANGPTL3

(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.

Key Points: 
  • (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.
  • At December 31, 2023, ProQR held cash and cash equivalents of €118.9 million, compared to €94.8 million at December 31, 2022.
  • The Company experienced a net positive cash flow from operating activities in 2023 primarily due to the receipt of the Lilly up-front payment of $60 million in February 2023.
  • For further financial information for the period ended December 31, 2023, please refer to our 2023 Annual Report on Form 20-F and our Statutory Annual Report which will be available on our website, www.

Korro Bio Highlights Data for its Lead Program in Alpha-1 Antitrypsin Deficiency (AATD) and Progress Across its RNA Editing Portfolio

Retrieved on: 
Thursday, January 18, 2024
AATD, FTD, Chord, Patient, Chronic obstructive pulmonary disease, Cirrhosis, Fibrosis, Oligonucleotide, Emphysema, RNA, Mouse, RNA editing, AAT, COPD, Dicarboxylic acid, De novo, Conference, Central nervous system, Calcium channel, Piz, Protein, Inflammation, ADAR, Adenosine deaminase, Machine learning, Customization, Genetic disorder, ALS, Liver disease, Nav1.7, Vaccine

AATD can lead to severe progressive lung disease, including emphysema and chronic obstructive pulmonary disease (COPD), and severe liver disease leading to inflammation, cirrhosis, and fibrosis.

Key Points: 
  • AATD can lead to severe progressive lung disease, including emphysema and chronic obstructive pulmonary disease (COPD), and severe liver disease leading to inflammation, cirrhosis, and fibrosis.
  • Korro’s proprietary RNA editing platform, OPERA™, integrates a deep understanding of adenosine deaminase acting on RNA (ADAR) enzymology with expertise in oligonucleotide chemistry, machine learning optimization of oligonucleotides and fit-for-purpose delivery.
  • CHORDs™, or Customized High-fidelity Oligonucleotides for RNA Deamination, are single-stranded, anti-sense oligonucleotides designed to have high target efficiency and specificity by leveraging the pillars of OPERA.
  • The replay is available for 30 days following the January 9, 2024 conclusion of the presentation.

Ascidian Therapeutics Announces First-Ever IND for an RNA Exon Editor as FDA Approves Trial Plan and Fast Tracks ACDN-01 in Stargardt Disease and Other ABCA4 Retinopathies

Retrieved on: 
Monday, January 29, 2024
Retina, Patient, Therapy, DNA, Biotechnology, RNA, RNA editing, University, Stargardt disease, ABCA4, IND, Food, Interim, Safety, Fast Track, FDA, Bascom Palmer Eye Institute, Research, Pharmaceutical industry

BOSTON, Jan. 29, 2024 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company aspiring to treat human diseases by rewriting RNA, today announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application and granted Fast Track      designation for ACDN-01. ACDN-01 is the first-ever clinical-stage RNA exon editor and the only clinical-stage therapeutic targeting the genetic cause of Stargardt disease. Ascidian expects to initiate enrollment in the Phase 1/2 STELLAR study of ACDN-01 in Stargardt disease and other ABCA4 retinopathies in the first half of 2024.

Key Points: 
  • ACDN-01 is the first-ever clinical-stage RNA exon editor and the only clinical-stage therapeutic targeting the genetic cause of Stargardt disease.
  • Ascidian expects to initiate enrollment in the Phase 1/2 STELLAR study of ACDN-01 in Stargardt disease and other ABCA4 retinopathies in the first half of 2024.
  • Ascidian also announced that the FDA has granted Fast Track designation for ACDN-01.
  • This designation enables Ascidian to expedite development of ACDN-01 with regular feedback from FDA through the clinical-development process.

Wave Life Sciences Announces Initiation of Dosing in RestorAATion Clinical Program Evaluating First-Ever RNA Editing Candidate, WVE-006, for Alpha-1 Antitrypsin Deficiency

Retrieved on: 
Wednesday, December 6, 2023
RNA, Liver, Messenger, AAT, Lung, Pizzicato, Chemistry, RNA editing, Disease, MBA, Aimer, MD, Human, Serum, GSK, Mutation, Biomarker, Concentration ratio, Patient, Liver disease, Therapy, Vaccine

CAMBRIDGE, Mass., Dec. 06, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced the initiation of dosing in healthy volunteers in the RestorAATion clinical trial program, which is investigating WVE-006 as a potential treatment for alpha-1 antitrypsin deficiency (AATD).

Key Points: 
  • It is designed to restore circulation of healthy, wild-type alpha-1 antitrypsin (M-AAT) protein and reduce dysfunctional Z-AAT protein, thereby potentially addressing AATD-related lung disease, liver disease, or both.
  • In preclinical studies, WVE-006 led to potent and durable RNA editing and restoration of AAT protein up to 30 micromolar, underscoring the impact of our novel chemistry.
  • Beyond WVE-006, we’re also excited to continue our collaboration with Wave Life Sciences using their best-in-class PRISM™ platform.”
    With initiation of dosing in RestorAATion, Wave has achieved its first WVE-006 milestone in its collaboration with GSK, resulting in a $20 million payment to Wave.
  • Beyond WVE-006, Wave is advancing a pipeline of wholly owned RNA editing therapeutics designed to either correct or upregulate mRNA across a range of high impact targets.

ProQR Strengthens Leading Intellectual Property Estate for ADAR-mediated RNA Editing

Retrieved on: 
Monday, November 6, 2023
Adenosine deaminase, ADAR, Patent, RNA, Adenosine, Intellectual property, Oligonucleotide, ProQR, RNA editing, European Patent Office, United States Patent and Trademark Office, Inosine, Therapy, European Patent Convention, IP, General counsel, Japan Patent Office, 3D printing, Vaccine

“The new patent that the USPTO granted to ProQR further expands the protection of RNA editing using oligonucleotides to recruit endogenous ADAR.

Key Points: 
  • “The new patent that the USPTO granted to ProQR further expands the protection of RNA editing using oligonucleotides to recruit endogenous ADAR.
  • Our leading intellectual property portfolio protects our Axiomer® ADAR-mediated RNA editing platform technology and more fundamentally the use of an oligonucleotide to recruit endogenous deaminating enzymes in the cell,” said René Beukema, Chief Corporate Development Officer and General Counsel.
  • Since then, ProQR has filed multiple additional patent applications on further improvements to form a leading patent estate that makes RNA editing with oligonucleotides that recruit endogenous ADAR proprietary to ProQR.
  • Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.

U.S. FDA AWARDS BOTH RARE PEDIATRIC DISEASE AND ORPHAN DRUG DESIGNATIONS TO HG204, A CRISPR RNA-EDITING THERAPY, FOR THE TREATMENT OF MECP2 DUPLICATION SYNDROME

Retrieved on: 
Tuesday, October 31, 2023
FDA, Ageing, Orphan Drug Act of 1983, Food, Rare, RNA, Sponsor, MDS, CRISPR, Greater China, Prevalence, MECP2, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Frasier, ODD, Speech, Neurodevelopmental disorder, Clinical trial, RNA editing, Messenger, MECP2 duplication syndrome, Therapy, Patient, GM, Pharmaceutical industry

"Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.

Key Points: 
  • "Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.
  • The FDA defines rare pediatric diseases as rare diseases (with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals aged under 18.
  • When the FDA awards rare pediatric disease designation (RPDD), the Sponsor of the trial firstly is granted a 6-months accelerated review of the drug candidate irrespective of indication, and which may be sold to another company.
  • "MDS is a rare and fatal childhood disease with the progressive of neurological regression such as loss of motor skills and speech.

U.S. FDA AWARDS BOTH RARE PEDIATRIC DISEASE AND ORPHAN DRUG DESIGNATIONS TO HG204, A CRISPR RNA-EDITING THERAPY, FOR THE TREATMENT OF MECP2 DUPLICATION SYNDROME

Retrieved on: 
Tuesday, October 31, 2023
FDA, Ageing, Orphan Drug Act of 1983, Food, Rare, RNA, Sponsor, MDS, CRISPR, Greater China, Prevalence, MECP2, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Frasier, ODD, Speech, Neurodevelopmental disorder, Clinical trial, RNA editing, Messenger, MECP2 duplication syndrome, Therapy, Patient, GM, Pharmaceutical industry

"Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.

Key Points: 
  • "Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.
  • The FDA defines rare pediatric diseases as rare diseases (with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals aged under 18.
  • When the FDA awards rare pediatric disease designation (RPDD), the Sponsor of the trial firstly is granted a 6-months accelerated review of the drug candidate irrespective of indication, and which may be sold to another company.
  • "MDS is a rare and fatal childhood disease with the progressive of neurological regression such as loss of motor skills and speech.

Rett Syndrome Research Trust's Genetic Medicines Summit Sets the Stage for Accelerated Therapeutic Development

Retrieved on: 
Wednesday, October 11, 2023
Office of Rare Diseases Research, Food, Doctor of Philosophy, Biomarker, National Center for Advancing Translational Sciences, RSRT, Critical Path Institute, RNA, Harvard University, MD, DNA, Therapy, Summit, Center for Biologics Evaluation and Research, MPH, Roger Hawkins (drummer), IND, MacNamara, JD, Biotechnology, P.C, Rare, United States Department of the Army, MECP2, EEG, FDA, Broad Institute, Hyman, MIT, RNA editing, Clinical trial, Wuhan Institute of Biological Products, Bangladesh Technical Education Board, Regulation of tobacco by the U.S. Food and Drug Administration, COA, Rett syndrome, Pharmaceutical industry, Medical imaging, Rett, Research

TRUMBULL, Conn., Oct. 11, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts. This landmark event gathered distinguished scientists, clinicians, regulatory experts, and over 40 leading biopharma companies. The goal of the Summit was to foster dialogue among scientific, clinical, industry, and regulatory stakeholders to facilitate collaboration, identify best practices, and refine expectations to expedite pre-clinical and clinical development programs focused on genetic medicines for children and adults with Rett syndrome.

Key Points: 
  • The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts.
  • TRUMBULL, Conn., Oct. 11, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts.
  • RSRT's Rett Syndrome Genetic Medicines Summit underscores the organization's unwavering commitment to curing Rett syndrome.
  • RSRT remains steadfast in its mission to accelerate the development of genetic therapies and ultimately find a cure for Rett syndrome.

Wave Life Sciences Highlights Growth Strategy for Building the Leading RNA Medicines Company in Annual R&D Day

Retrieved on: 
Thursday, September 28, 2023
HD, GSK, DSM-IV codes, Human, Obesity, Duchenne muscular dystrophy, Therapy, Genetics, Serum, Deep learning, Novel, Weight loss, Wave, Mutation, MBA, Part, RNA, Biomarker, Doctor of Philosophy, CNS, Messenger, Chemistry, Odds ratio, Protein, DIO, DMD, MD, Pizzicato, Patient, Map, Kidney, Webcast, Mouse, Liver, Exon, RNA editing, Vaccine, Pharmaceutical industry, Soft drink, Dietary supplement, Duchenne, Small RNA, AATD

“We are bringing an exciting, genetics-based approach to the metabolic disease and obesity space with our first siRNA program targeting INHBE.

Key Points: 
  • “We are bringing an exciting, genetics-based approach to the metabolic disease and obesity space with our first siRNA program targeting INHBE.
  • In addition to high-impact silencing targets like INHBE, we have a near-term pipeline focus on protein restoration and repair with our RNA editing and splicing capabilities.
  • Growing pipeline with high-value RNA editing targets:
    Beyond WVE-006, Wave highlighted several undisclosed RNA editing targets which span prevalent and rare liver, kidney and lung diseases.
  • The multidose portion of the SELECT-HD clinical trial is ongoing and has been enrolling with high demand.