Alpha-1 antitrypsin deficiency

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

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.

Korro Bio Announces Appointment of Tim Pearson to Board of Directors Upon Closing of the Proposed Merger and Promotion of Todd Chappell to Chief Operating Officer

Retrieved on: 
Wednesday, October 25, 2023

Korro Bio expects to close both the financing and merger promptly after the required approvals are obtained from Frequency’s stockholders.

Key Points: 
  • Korro Bio expects to close both the financing and merger promptly after the required approvals are obtained from Frequency’s stockholders.
  • This appointment will be effective upon completion of the merger.
  • Mr. Pearson has over 30 years of healthcare experience and has served as Chief Executive Officer of Carrick Therapeutics since July 2019.
  • Prior to that, Mr. Chappell served as Chief Executive Officer of Rasio Therapeutics, Inc. and Perceptive Navigation, LLC.

Gain Therapeutics Reports Full Year 2022 Financial Results and Business Update

Retrieved on: 
Thursday, March 23, 2023

“We look forward to commencing the clinical program for GT-02287 with the submission of the dossier for the Phase 1 clinical trial by mid-2023.

Key Points: 
  • “We look forward to commencing the clinical program for GT-02287 with the submission of the dossier for the Phase 1 clinical trial by mid-2023.
  • General and administrative (G&A) expenses for 2022 were $9.54 million, as compared to $6.82 million in 2021.
  • Net loss as of December 31, 2022 included non-cash compensation expenses of $1.53 million as compared to 2021 non-cash compensation expenses of $1.87 million.
  • As of December 31, 2022, the Company had cash, cash equivalents and marketable securities totaling $22.1 million for the year ended December 31, 2022.

Mereo BioPharma to Host Alvelestat R&D Update Call on October 31, 2022

Retrieved on: 
Monday, October 24, 2022

LONDON, Oct. 24, 2022 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO), (Mereo or the Company), a clinical-stage biopharmaceutical company focused on rare diseases and oncology, today announced that it will host a R&D update call on Monday, October 31, 2022 at 8:00 am ET on the alvelestat (MPH966) program for alpha-1-antitrypsin deficiency (AATD).

Key Points: 
  • LONDON, Oct. 24, 2022 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO), (Mereo or the Company), a clinical-stage biopharmaceutical company focused on rare diseases and oncology, today announced that it will host a R&D update call on Monday, October 31, 2022 at 8:00 am ET on the alvelestat (MPH966) program for alpha-1-antitrypsin deficiency (AATD).
  • The update will include commentary from and Q&A with leading pulmonary experts, further to the receipt of Fast Track Designation for alvelestat from the FDA announced on October 17, 2022.
  • The partnership with Ultragenyx includes potential milestone payments of up to $254 million and royalties to Mereo on Ultragenyx territories.
  • Mereo has retained EU and UK commercial rights and will pay Ultragenyx royalties on those territories.

SEI Healthcare Releases New Educational Program & Interactive Webinar on AATD

Retrieved on: 
Friday, July 22, 2022

SEI Healthcare has recently announced the successful release of an educational program & interactive webinar on alpha-1 antitrypsin deficiency (AATD)

Key Points: 
  • Lewes, Delaware--(Newsfile Corp. - July 22, 2022) - SEI Healthcare, a global, professional Independent Medical Education organization, has announced the successful release of an educational program and interactive webinar on alpha-1 antitrypsin deficiency (AATD), titled "Paradigm Shifts in AATD and Impact on Patient Management".
  • This program included the development of four patient case simulations on a live interactive webinar, which was attended by over 1900 healthcare professionals (HCPs).
  • Further details about this educational program can be found at:
    "An educational project on this topic has proven to be of extreme importance.
  • SEI Healthcare has the pleasure to continue developing educational projects bringing forth strategic insights to optimise outcomes," says Leana Ribeiro, Global Accounts Manager for SEI Healthcare.

CANbridge-UMass Chan Medical School Gene Therapy Research to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

Retrieved on: 
Wednesday, May 4, 2022

Researchers will present findings from an animal study exploring a novel second-generation gene therapy for the potential treatment of spinal muscular atrophy (SMA).

Key Points: 
  • Researchers will present findings from an animal study exploring a novel second-generation gene therapy for the potential treatment of spinal muscular atrophy (SMA).
  • This is the first data to come out of the CANbridge rare disease gene therapy research collaboration with the UMass Chan Medical School, which is focused on developing gene therapy treatments for neuromuscular conditions with unmet medical needs.
  • The Horae Gene Therapy Center faculty is interdisciplinary, including members from the departments of Pediatrics, Microbiology & Physiological Systems, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology.
  • Additionally, the University of Massachusetts Chan Medical School conducts clinical trials on site and some of these trials are conducted by the investigators at the Gene Therapy center.

Centessa Pharmaceuticals Reports Fourth Quarter and 2021 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 30, 2022

R&D Expenses: $41.5 million for the Company for the quarter ended December 31, 2021, $95.7 million for the Successor for 2021.

Key Points: 
  • R&D Expenses: $41.5 million for the Company for the quarter ended December 31, 2021, $95.7 million for the Successor for 2021.
  • General & Administrative Expenses: $13.0 million for the Company for the quarter ended December 31, 2021, $42.9 million for the Successor for 2021.
  • Centessa Pharmaceuticals plc (Centessa) is a clinical-stage pharmaceutical company with a Research & Development (R&D) innovation engine that aims to discover, develop and ultimately deliver impactful medicines to patients.
  • We explicitly disclaim any obligation to update any forward-looking statements except to the extent required by law.

Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Epidemiology Forecast Report 2022-2032: Focus on United States, Germany, Spain, Italy, France, United Kingdom, and Japan - ResearchAndMarkets.com

Retrieved on: 
Thursday, January 20, 2022

This "Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease - Epidemiology Forecast to 2032" report delivers an in-depth understanding of the disease, historical and forecasted Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Key Points: 
  • This "Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease - Epidemiology Forecast to 2032" report delivers an in-depth understanding of the disease, historical and forecasted Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
  • The Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease epidemiology division provides insights about historical and current patient pool and forecasted trend for every seven major countries.
  • The Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease epidemiology covered in the report provides historical as well as forecasted Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease epidemiology scenario in the 7MM covering the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan from 2019 to 2032.
  • The Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease report covers a detailed overview explaining its causes, symptoms, classification, pathophysiology, diagnosis and treatment patterns
    The Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Epidemiology Report and Model provide an overview of the global trends of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease in the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan)
    The report provides insight into the historical and forecasted patient pool of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan
    What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease?

Centessa Pharmaceuticals Demonstrates Proof-of-Mechanism from First Three PiMZ Subjects Dosed in Part B of Phase 1 Study Evaluating ZF874

Retrieved on: 
Monday, November 1, 2021

BOSTON and LONDON, Nov. 01, 2021 (GLOBE NEWSWIRE) --  Centessa Pharmaceuticals plc (“Company”) (Nasdaq: CNTA), together with subsidiary Z Factor Limited (“Z Factor”), today announced proof-of-mechanism data from the first three subjects dosed in the ongoing repeat dose Phase 1 Part B study of ZF874 in subjects carrying at least one Z-mutated alpha-1-antitrypsin allele (PiXZ). This is the first demonstration that a pharmacological chaperone can provide sufficient functional Z-A1AT increases to potentially achieve greater than 11 micromolar levels in individuals with the PiZZ genotype.

Key Points: 
  • ZF-0101 is a Phase 1 study evaluating ZF874 (the Study), a novel, catalytically acting pharmacological chaperone designed to rescue the folding of the Z variant of alpha-1-antitrypsin (A1AT) to address Alpha-1 Antitrypsin Deficiency (AATD).
  • Part A of the Study was comprised of a single ascending dose study in 7 cohorts of healthy volunteers.
  • Part B of the Study was initially designed to be a 28-day repeat dose study in up to 14 PiXZ patients (including 2 placebo), assessing the safety, tolerability and pharmacokinetics of ZF874.
  • In both PiMZ subjects dosed with 15 mg/kg BID of ZF874, the observed increase in functional A1AT was between 3.5 and 6 micromolar for these subjects with one Z-gene copy.

Centessa Pharmaceuticals Announces Second Quarter 2021 Financial Results and Business Updates

Retrieved on: 
Monday, August 16, 2021

CAMBRIDGE, Mass. and LONDON, Aug. 16, 2021 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage company leveraging its innovative asset-centric business model to discover, develop and ultimately deliver impactful medicines to patients, today reported financial results for the quarter ended June 30, 2021, and provided a review of recent accomplishments and anticipated upcoming milestones.

Key Points: 
  • and LONDON, Aug. 16, 2021 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage company leveraging its innovative asset-centric business model to discover, develop and ultimately deliver impactful medicines to patients, today reported financial results for the quarter ended June 30, 2021, and provided a review of recent accomplishments and anticipated upcoming milestones.
  • $379.5 Million Initial Public Offering (IPO) Successfully Completed: In the second quarter, Centessa closed its initial public offering of 16,500,000 American Depositary Shares (ADSs).
  • The gross proceeds to Centessa from its IPO, before deducting underwriting discounts, commissions and other estimated offering expenses, totaled an aggregate of $379.5 million.
  • The Phase 2a has completed dosing and the Company anticipates sharing topline results in the third quarter of 2021.