AAT

Korro Reports Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Tuesday, March 26, 2024
Research, Piz, Adenosine deaminase, Half-life, Patient, Alnylam Pharmaceuticals, AAT, ADAR, Mouse, RNA, Chord, N-Acetylgalactosamine, Hepatocyte, Investment, Customization, Protein, Therapy, Research and development, Administration, Liver, Vaccine

KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s platform called Oligonucleotide Promoted Editing of RNA (OPERA™).

Key Points: 
  • KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s platform called Oligonucleotide Promoted Editing of RNA (OPERA™).
  • Using CHORDs, Korro achieved greater than 50% editing using GalNAc conjugates with subcutaneous administration to the liver in a preclinical mouse model.
  • Full Year 2023 Financial Results:
    Cash Position: Cash, cash equivalents and short-term investments were $166.1 million as of December 31, 2023, compared to $55.2 million as of December 31, 2022.
  • Net Loss: Korro’s net loss was $81.2 million for the year ended December 31, 2023, as compared to $58.0 million for the year ended December 31, 2022.

Beam Therapeutics Announces Clearance of Clinical Trial Authorisation Application for BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

Retrieved on: 
Tuesday, March 26, 2024
Piz, AATD, Mutation, Biotechnology, Patient, Lung, CTA, AAT, Life expectancy, Safety, Pharmacokinetics, Liver, Medication, Pharmaceutical industry

CAMBRIDGE, Mass., March 26, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the clearance of its clinical trial authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with alpha-1 antitrypsin deficiency (AATD).

Key Points: 
  • CAMBRIDGE, Mass., March 26, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the clearance of its clinical trial authorisation (CTA) application by the United Kingdom (UK) Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with alpha-1 antitrypsin deficiency (AATD).
  • “AATD is an inherited genetic condition that can cause serious lung and liver disease, which can lead to significant debility and reduced life expectancy for patients.
  • The study design includes a dose exploration portion followed by a dose expansion portion to identify the optimal dose to take forward in a pivotal study.
  • The company expects to initiate the Phase 1/2 trial of BEAM-302 in the UK in the first half of 2024.

EQS-News: AATec Medical appoints Professor Dr. Claus Franz Vogelmeier to the Scientific Advisory Board

Retrieved on: 
Wednesday, March 13, 2024
Bronchiectasis, American Journal, Pulmonology, Asthma, AAT, European Respiratory Society, FERS, GOLD, Patient, Chronic obstructive pulmonary disease, Global Initiative, German Respiratory Society, Critical Care Medicine (journal), COPD, Pharmaceutical industry

AATec Medical appoints Professor Dr. Claus Franz Vogelmeier to the Scientific Advisory Board

Key Points: 
  • AATec Medical appoints Professor Dr. Claus Franz Vogelmeier to the Scientific Advisory Board
    The issuer is solely responsible for the content of this announcement.
  • AATec Medical appoints Professor Dr. Claus Franz Vogelmeier to the Scientific Advisory Board
    Leading pulmonology expert contributes profound clinical experience in inflammatory lung diseases such as bronchiectasis, COPD, asthma, and ARDS to AATec Medical.
  • Munich, Germany – 29.02.2024 – AATec Medical GmbH (AATec), a biotech company developing a multi-product platform technology based on recombinant alpha-1 antitrypsin (AAT), today announced the appointment of Prof. Dr. Claus Franz Vogelmeier, Professor of Medicine and Head of the Department of Pulmonary Medicine at Philipps-University in Marburg, Germany to its Scientific Advisory Board.
  • Dr. Rüdiger Jankowsky, co-founder and CEO of AATec, commented: “We are very pleased to welcome Professor Vogelmeier to our Scientific Advisory Board.

Kamada Issues 2024 CEO Letter to Shareholders

Retrieved on: 
Wednesday, March 6, 2024
Acquisition, KMDA, CMV, Hospital, Division, EMA, CSL Behring, Letter, Physician, Critical care, FDA, Technology transfer, Food, Growth, FEV1, AAT, Immunoglobulin G, Safety, University, European Medicines Agency, Food and Drug Administration, Pan American Health Organization, Patient, Plasma, Research, Partnership, Pharmaceutical industry

The recently completed 2023 was another successful period in our commercial journey as a global leader in the specialty plasma-derived field.

Key Points: 
  • The recently completed 2023 was another successful period in our commercial journey as a global leader in the specialty plasma-derived field.
  • Looking ahead, we expect the momentum from 2023 to extend throughout 2024, with profitability to be further increased as compared to last year.
  • These significant catalysts are propelling our continued annual double-digit profitable growth with substantial upside potential and limited downside risk.
  • On behalf of the entire Kamada team, we look forward to continuing to support patients and clinicians with the important lifesaving products that we develop, manufacture, and commercialize.

Advancing Organic Circuits: MANA Study Changes Current Computing Architecture

Retrieved on: 
Thursday, February 22, 2024
MANA, Series, Research institute, NDT, Polymer, AAT, App Store (iOS/iPadOS), JSPS, Electronics

Organic integrated circuits, constructed with small molecules or polymers, show great potential for human-friendly interactive mobile applications.

Key Points: 
  • Organic integrated circuits, constructed with small molecules or polymers, show great potential for human-friendly interactive mobile applications.
  • However, traditional organic circuits are incompatible with modern lithographic techniques and thus suffer from low integration density and poor data-processing capability.
  • The organic quaternary logic inverter circuit was constructed by connecting an AAT, comprising two "n"-type organic semiconductors and a "p"-type organic semiconductor, to a double-layered "n"-type transistor in series.
  • The proposed circuit thus advances the capabilities of organic circuits for more demanding computing applications.

EQS-News: AATec Medical starts collaboration with the German Federal Agency for Disruptive Innovation SPRIND for alpha-1 antitrypsin

Retrieved on: 
Tuesday, February 6, 2024
Partnership, Alpha-1 antitrypsin deficiency, Research, Inflammation, Lung, Acute respiratory distress syndrome, COPD, AAT, Innovation, Viral, Asthma, Pharmaceutical industry

This project is a key step in the development of the innovative ATL-105 drug platform for the treatment of respiratory diseases.

Key Points: 
  • This project is a key step in the development of the innovative ATL-105 drug platform for the treatment of respiratory diseases.
  • As part of the collaboration with SPRIND, ATL-105 will be tested in various disease models for viral diseases of the respiratory tract.
  • Sigrid Koeth, Innovation Manager at SPRIND, emphasizes the importance of the project: "With SPRIND, we want to create disruptive innovations from Germany.
  • Dr. Rüdiger Jankowsky, co-founder and CEO of AATec, comments on the collaboration: "The partnership with SPRIND enables us to accelerate our research and development work and realize our innovation potential faster.

Press Release: Sanofi to acquire Inhibrx, Inc., adding potential best-in-class rare disease asset for Alpha-1 Antitrypsin Deficiency to pipeline

Retrieved on: 
Tuesday, January 23, 2024
AATD, Serum, Development, Patient, Centerview Partners, AAT, Quality of life, Weil, Gotshal & Manges, Acquisition, Safety, Lazard, CVR, Inflammation, Research and development, Lung, Pharmacokinetics, Security (finance)

INBRX-101 is a human recombinant protein that holds the promise of allowing Alpha-1 Antitrypsin Deficiency (AATD) patients to achieve normalization of serum AAT levels with less frequent (monthly vs. weekly) dosing.

Key Points: 
  • INBRX-101 is a human recombinant protein that holds the promise of allowing Alpha-1 Antitrypsin Deficiency (AATD) patients to achieve normalization of serum AAT levels with less frequent (monthly vs. weekly) dosing.
  • AATD is an inherited rare disease characterized by low levels of AAT protein, predominantly affecting the lung with progressive deterioration of the tissue.
  • Head of Research and Development, Sanofi
    “The addition of INBRX-101 as a high potential asset to our rare disease portfolio reinforces our strategy to commit to differentiated and potential best-in-class products.
  • Lazard is acting as exclusive financial advisor to Sanofi and Weil, Gotshal & Manges LLP is acting as its legal counsel.

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.

Wave Life Sciences Highlights Recent Achievements and Upcoming 2024 Milestones

Retrieved on: 
Monday, January 8, 2024
Well, RNA, Conference, Science, Wave, Messenger, Research, AAT, Chemistry, Pizzicato, Small RNA, Lung, Weight, Health, Disease, MBA, MD, Mouse, HD, Human, Serum, GSK, Mutation, Genetics, Coronary artery disease, Obesity, Genomics, Concentration ratio, Central nervous system, Patient, Liver disease, Lof, HTT, Medicine, Therapy, Oligonucleotide, NHP, CTA, Vaccine

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.