Pizzicato

Wave Life Sciences Highlights Recent Achievements and Upcoming 2024 Milestones

Retrieved on: 
Monday, January 8, 2024
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We expect 2024 will be an inflection year that will drive significant value for Wave, our shareholders, and most importantly, for the patients who will benefit from our research,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences.

Key Points: 
  • We expect 2024 will be an inflection year that will drive significant value for Wave, our shareholders, and most importantly, for the patients who will benefit from our research,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences.
  • Expected milestones: Wave plans to deliver proof-of-mechanism data in individuals with AATD in 2024.
  • Expected milestones: Wave expects to deliver data, including dystrophin protein expression from muscle biopsies at 24 weeks, in the third quarter of 2024.
  • A live webcast of this presentation will be available on the Investor Relations page of the Wave Life Sciences website at http://ir.wavelifesciences.com .

Beam Therapeutics Highlights Progress Across Base Editing Portfolio and Outlines 2024 Anticipated Milestones

Retrieved on: 
Monday, January 8, 2024
SCD, LNP, Liver, Beam Therapeutics, Pfizer, Conference, Genotype, Sickle cell disease, Gene editing, Second wave, Survival, ESCAPE, Biotechnology, IND, Research, AAT, Pizzicato, Mortality, G6PC, Therapy, Third wave, Clinical trial, Glucose, DSM-IV codes, GMP, Trial of the century, AATD, Transplant, Growth, Investment, Hematology, Stem cell, HBF, Safety, Liver disease, Piz, CTA, Genetic distance, Beam

CAMBRIDGE, Mass., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported progress across the company’s hematology and genetic disease portfolios and provided updates on anticipated upcoming milestones.

Key Points: 
  • “Our vision is to establish Beam as a sustainable, fully integrated company pioneering a new class of genetic medicines with base editing.
  • Preclinical models suggest base editing could lead to improved HbF induction and lower residual disease-causing hemoglobin S compared to existing gene therapy options.
  • The company is on-track to report initial data on multiple patients from the BEACON trial in the second half of 2024.
  • Beam expects to report an initial clinical dataset for BEAM-201 in the second half of 2024.

Korro Announces Selection of its First Development Candidate for the Potential Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

Retrieved on: 
Thursday, December 7, 2023
LNP, RNA, Liver, ADAR, Pizzicato, Disease, SNV, Mouse, Doctor of Philosophy, Human, A1AT, Mutation, AATD, Patient, Piz, Vaccine

KRRO-110 is a proprietary RNA editing oligonucleotide delivered to liver cells using clinically validated LNP technology licensed from Genevant.

Key Points: 
  • KRRO-110 is a proprietary RNA editing oligonucleotide delivered to liver cells using clinically validated LNP technology licensed from Genevant.
  • Preclinical development of KRRO-110 is ongoing in preparation for a potential regulatory filing in the second half of 2024.
  • “Selecting a development candidate for our AATD program is an important milestone for Korro, but more importantly, for patients.
  • “Korro’s RNA editing approach has the potential to be transformative for AATD patients and Korro is well-positioned to deliver a best-in-class therapeutic.

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.

Wave Life Sciences Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, November 9, 2023
Research, Q&A, Dystrophin, DSM-IV codes, Biomarker, Therapy, Obesity, CSF, RNA, Pizzicato, Human, HD, MD, Coronary artery disease, Liver, CNS, Exon, MBA, Liver disease, Oligonucleotide, Pharmacokinetics, CTAS, Clinical trial, Webcast, Messenger, GSK, Serum, AATD, Safety, DMD, N-Acetylgalactosamine, Patient, Small RNA, NHP, Mutation, Vaccine, Medical imaging, Cryptocurrency, Pharmaceutical industry, Takeda, Wave

ET today

Key Points: 
  • ET today
    CAMBRIDGE, Mass., Nov. 09, 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 financial results for the third quarter ended September 30, 2023, and provided a business update.
  • Wave expects to initiate dosing with WVE-006 in healthy volunteers in the fourth quarter of 2023 and deliver proof-of-mechanism data in individuals with AATD in 2024.
  • Wave remains on track to initiate dosing in FORWARD-53 in 2023 and enroll up to 10 boys in the trial.
  • ET to review third quarter 2023 financial results and pipeline updates.

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.

Beam Therapeutics Presents Preclinical Data Highlighting Utility of BEAM-302 to Correct an Alpha-1 Antitrypsin (AAT) Deficiency Disease-Causing Mutation

Retrieved on: 
Thursday, September 7, 2023
Infection, Rat, Serum, Biotechnology, Liver, NSG, Mutation, Toxicity, SERPINA1, Inflammation, Lung, Piz, AATD, HIV disease progression rates, Pizzicato, Mouse, AAT, LNP, Dietary supplement, Pharmaceutical industry, Vaccine, Beam

The data are featured in an oral presentation titled “BEAM-302: Targeting AATD Liver and Lung Disease with Base Editing” at the Alpha-1 Antitrypsin Deficiency 2023 Meeting in Naples, Italy.

Key Points: 
  • The data are featured in an oral presentation titled “BEAM-302: Targeting AATD Liver and Lung Disease with Base Editing” at the Alpha-1 Antitrypsin Deficiency 2023 Meeting in Naples, Italy.
  • “In today’s presentation, we shared – for the first time – a full summary of preclinical in vivo data for BEAM-302, our lead candidate for the potential treatment of AATD.
  • BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to precisely correct the PiZ mutation, a single-letter genetic error.
  • Beam plans to submit a regulatory application for authorization to initiate clinical trials of BEAM-302 in the first quarter of 2024.

Wave Life Sciences Announces Submission of First Clinical Trial Application for WVE-006, the First-ever RNA Editing Clinical Candidate, and Plans for Upcoming Virtual “R&D Day”

Retrieved on: 
Tuesday, September 5, 2023
GSK, Liver disease, Serum, Wave, Mutation, CTA, MBA, SERPINA1, RNA, Messenger, AATD, MD, Pizzicato, Webcast, AAT, RNA editing, Genome, Vaccine

CAMBRIDGE, Mass., Sept. 05, 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 submission of its first clinical trial application (CTA) for WVE-006 in alpha-1 antitrypsin deficiency (AATD). WVE-006 is a first-in-class, GalNAc-conjugated RNA editing oligonucleotide (“AIMer”) and is designed to correct the single base mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele, thereby enabling restoration and circulation of functional, wild-type alpha-1 antitrypsin (M-AAT) protein. The WVE-006 clinical program will be highlighted in Wave’s virtual “R&D Day” on September 28, 2023 at 10:00 a.m. ET, among other programs.

Key Points: 
  • The WVE-006 clinical program will be highlighted in Wave’s virtual “R&D Day” on September 28, 2023 at 10:00 a.m.
  • “With the submission of the first CTA for WVE-006, we have officially initiated clinical development of the industry’s first-ever RNA editing therapeutic candidate,” said Anne Marie Li-Kwai-Cheung, Chief Development Officer at Wave Life Sciences.
  • A live webcast of the event can be accessed by visiting “Investor Events” on the investor relations section of the Wave Life Sciences website: https://ir.wavelifesciences.com/events-and-presentations .
  • Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website.

Beam Therapeutics Reports Progress Across Base Editing Portfolio and Outlines Key Anticipated Milestones

Retrieved on: 
Monday, January 9, 2023
IND, Disease, Pizzicato, Cell, Blood, Toxicity, Thalassemia, Tissue, HSC, Biotechnology, Fetal hemoglobin, LNP, Safety, Glucose, FDA, SCD, Mutation, Security, Starch, Gene editing, Toxic epidermal necrolysis, ASH, Conference, CD7, Immune system, Patient, G6PC, Research, Cancer, Clinical trial, Hematology, Science, Liver, Society, Sickle cell disease, AATD, Investment, Genetic disorder, ESCAPE, Pharmaceutical industry, Vaccine, Medical imaging, Beam

“Beam enters 2023 with significant momentum across all of our core pipeline areas and an expanding leadership position in the next generation of gene editing,” said John Evans, chief executive officer of Beam.

Key Points: 
  • “Beam enters 2023 with significant momentum across all of our core pipeline areas and an expanding leadership position in the next generation of gene editing,” said John Evans, chief executive officer of Beam.
  • Platform Opportunity: Beam is advancing its Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning strategy in an effort to bring base editing treatments to more patients.
  • In December 2022, Beam presented in vivo proof-of-concept data at the American Society of Hematology Annual Meeting and Exposition (ASH) highlighting its potential.
  • BEAM-201: In December 2022, Beam received clearance from the FDA for its Investigational New Drug (IND) application for BEAM-201.

Wave Life Sciences and GSK Announce Collaboration to Drive Discovery and Development of Oligonucleotide Therapeutics Focusing on Novel Genetic Targets

Retrieved on: 
Tuesday, December 13, 2022
RNA, Enzyme, Inosine, Chemistry, Security (finance), Guanine, Webcast, PN, MD, Lung, Degenerative disease, MBA, Investment, COVID-19, Liver, AAT, Aimer, Liver disease, Clinical trial, Genetics, Sirna, Liver transplantation, PRISM, Drug development, Adenine, Organization, SERPINA1, Fatty liver disease, Genome, Allele, Oligonucleotide, Therapy, Genetic disorder, Protein, Drug discovery, Liposome, GSK, DNA, NASH, ADAR, AATD, Disease, Mutation, Tissue, Patient, Cell, Hepatocyte, Genomics, Pizzicato, History, IND, Risk, Genotype, Q&A, Pharmaceutical industry, Vaccine, Fine chemical, Wave

Wave’s PRISM platform is the only oligonucleotide platform offering three RNA-targeting modalities (editing, splicing, and silencing, including siRNA and antisense).

Key Points: 
  • Wave’s PRISM platform is the only oligonucleotide platform offering three RNA-targeting modalities (editing, splicing, and silencing, including siRNA and antisense).
  • Importantly, these modalities incorporate novel chemistry, including PN backbone chemistry and control of stereochemistry, to optimize the pharmacological properties of therapeutic oligonucleotides.
  • Pairing GSK's genetic expertise with the best-in-class PRISM™ platform enables us to accelerate drug discovery for newly-identified targets, by matching target to modality.
  • Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases.