RNA editing

Wave Life Sciences Reports First Quarter 2024 Financial Results and Provides Business Update

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Thursday, May 9, 2024
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ET today

Key Points: 
  • ET today
    CAMBRIDGE, Mass., May 09, 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 announced financial results for the first quarter ended March 31, 2024, and provided a business update.
  • Revenue was $12.5 million for the first quarter of 2024, as compared to $12.9 million in the first quarter of 2023.
  • Net loss was $31.6 million for the first quarter of 2024, as compared to $27.4 million for the first quarter of 2023.
  • ET to review the first quarter 2024 financial results and pipeline updates.

Wave Life Sciences Announces Appointment of Dr. Erik Ingelsson as Chief Scientific Officer

Retrieved on: 
Thursday, May 9, 2024
Stanford University, Genetics, Genomics, RNA editing, University of Oxford, Molecular epidemiology, Health, Gene editing, RNA, Doctor of Philosophy, Computational biology, Research, Employment, Obesity, Therapy, Senior, SVP, GSK, Patient, DSM-IV codes, Faculty, Cardiovascular disease, Karolinska Institute, MBA, Biology, Framingham Heart Study, RSU, Drug discovery, MASH, Insulin resistance, Pharmaceutical industry

CAMBRIDGE, Mass., May 09, 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 announced the appointment of Erik Ingelsson, MD, PhD, as Chief Scientific Officer.

Key Points: 
  • CAMBRIDGE, Mass., May 09, 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 announced the appointment of Erik Ingelsson, MD, PhD, as Chief Scientific Officer.
  • Most recently, Dr. Ingelsson served as Senior Vice President, Head of Target Discovery, at GSK.
  • Dr. Ingelsson brings to Wave deep expertise in genetics and drug discovery, as well as substantial experience in metabolic diseases.
  • He will leverage his experience to accelerate identification of new genetic insights and rapidly translate those insights into RNA medicines at Wave, including with our wholly owned INHBE program for obesity,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences.

ProQR Announces First Quarter 2024 Operating and Financial Results

Retrieved on: 
Thursday, May 9, 2024
EON, Trident, Annual general meeting, Research, Partnership, Doctor of Philosophy, Webcast, Biomarker, Knowledge, Cholestasis, NTCP, RNA editing, Oncology, European Patent Convention, Oligonucleotide, Serum, Lilly, Eli Lilly and Company, Ribose, Pseudouridine, Poster, Alnylam Pharmaceuticals, Eli Lilly, RNA, Exercise, NHP, Clinical trial, ProQR, ADAR, B4GALT1, Adenosine, EDT, Science, Patent, Patient, Liver, Eon, Metabolism, Uridine, Q&A, Pharmaceutical industry

(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 first quarter ended March 31, 2024, 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 first quarter ended March 31, 2024, and provided a business update.
  • At March 31, 2024, ProQR held cash and cash equivalents and short term financial assets of €102.7 million, compared to €118.9 million cash and cash equivalents at December 31, 2023.
  • General and administrative costs were €3.5 million for the quarter ended March 31, 2024 compared to €4.0 million for the same period last year.
  • For further financial information for the period ended March 31, 2024, please refer to the Q1 financial report filing.

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

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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
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(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

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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

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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

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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.