SERPINA1

Intellia Therapeutics Announces Third Quarter 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Thursday, November 9, 2023
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“2023 has been a year of remarkable progress in which Intellia received two IND clearances for investigational in vivo CRISPR therapies.

Key Points: 
  • “2023 has been a year of remarkable progress in which Intellia received two IND clearances for investigational in vivo CRISPR therapies.
  • With the imminent start of the NTLA-2001 MAGNITUDE Phase 3 trial, Intellia has now become a late-stage drug development company,” said Intellia President and Chief Executive Officer John Leonard, M.D.
  • In November, Intellia announced new positive interim results from the Phase 1 study of NTLA-2001.
  • Net Loss: The Company’s net loss was $122.2 million for the third quarter of 2023, compared to $113.2 million during the third quarter of 2022.

Krystal Biotech Announces FDA Clearance of Investigational New Drug Application for KB408 for the Treatment of Type 1 Alpha-1 Antitrypsin Deficiency

Retrieved on: 
Thursday, September 21, 2023
KRYS, Krystal, PI, Senior, NCT, Epithelium, Drug, SERPINA1, AATD, Patient, AAT, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Gene, Food, Safety, Pharmaceutical industry

PITTSBURGH, Sept. 21, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug Application (IND) for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD).

Key Points: 
  • KB408 is a modified, replication-defective, non-integrating HSV-1-derived vector carrying two full-length copies of the serpin family A member 1 (SERPINA1) gene to enable expression of alpha-1 antitrypsin (AAT).
  • KB408 is formulated for inhaled delivery to the respiratory cells of the lungs via nebulization.
  • “We are excited to advance KB408, our investigational gene therapy for patients with alpha-1 antitrypsin deficiency, into the clinic in our Serpentine-1 study,” said Hubert Chen, M.D., Senior Vice President of Clinical Development at Krystal Biotech.
  • On September 5th, the FDA granted orphan-drug designation for KB408 for the treatment of AATD.

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
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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.

Krystal Biotech Announces Orphan Drug Designation Granted to KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

Retrieved on: 
Tuesday, September 5, 2023
KRYS, Research and development, Failure mode and effects analysis, Suma, SERPINA1, AATD, Patient, Liver, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Pharmaceutical industry

KB408 IND filed on August 15, 2023

Key Points: 
  • KB408 IND filed on August 15, 2023
    PITTSBURGH, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD).
  • AATD is caused by mutations in the SERPINA1 gene that lead to decreased levels and/or decreased functionality of alpha-1 antitrypsin protein.
  • Over time, the deficiency can lead to progressive enzymatic destruction of the lung tissue, ultimately causing life-threatening pulmonary impairment and severe respiratory insufficiency.
  • KB408 is an inhaled (nebulized) formulation of the Company’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment of AATD.

Intellia Therapeutics Announces Second Quarter 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Thursday, August 3, 2023
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The Company plans to present additional data from the ATTRv-PN arm of the Phase 1 study by year-end 2023.

Key Points: 
  • The Company plans to present additional data from the ATTRv-PN arm of the Phase 1 study by year-end 2023.
  • Collaboration Revenue: Collaboration revenue decreased by $0.4 million to $13.6 million during the second quarter of 2023, compared to $14.0 million during the second quarter of 2022.
  • G&A Expenses: General and administrative expenses increased by $8.5 million to $30.7 million during the second quarter of 2023, compared to $22.1 million during the second quarter of 2022.
  • Net Loss: The Company’s net loss was $123.7 million for the second quarter of 2023, compared to $100.7 million during the second quarter of 2022.

Korro Bio and Frequency Therapeutics Announce Merger Agreement

Retrieved on: 
Friday, July 14, 2023
Science, Other Science, Research, Pharmaceutical, General Health, Health, Genetics, Other Health, DNA, Atlas Venture, Life, Liver disease, Bio, Clinical trial, SERPINA1, Jordan, RNA, Doctor of Philosophy, Piz, NEA, A1AT, Patient, Frequency, MP, Biology, Therapy, RNA editing, Video game, Vaccine, Citadel, AATD

Korro Bio, Inc., a leading RNA editing company focused on the discovery and development of novel genetic medicines, and Frequency Therapeutics, Inc. (Nasdaq: FREQ) today announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction.

Key Points: 
  • Korro Bio, Inc., a leading RNA editing company focused on the discovery and development of novel genetic medicines, and Frequency Therapeutics, Inc. (Nasdaq: FREQ) today announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction.
  • The combined company will focus on the advancement of Korro Bio’s portfolio of RNA editing programs, is expected to operate under Korro Bio, Inc. and will apply to trade on Nasdaq under the ticker symbol “KRRO”.
  • The merger and related financing are expected to close in the fourth quarter of 2023, subject to approval by Frequency Therapeutics’ stockholders and other customary closing conditions.
  • The combined company will be led by current members of the Korro Bio management team, including:
    The Board of Directors of the combined company is expected to be comprised of seven members, consisting of four members designated by Korro Bio, one member designated by Frequency Therapeutics, which will be Frequency’s Chief Executive Officer, David L. Lucchino, and two independent directors.

Intellia Therapeutics Announces First Quarter 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Thursday, May 4, 2023
NTLA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Intellia Therapeutics, Retirement, Acquisition, Patient, Ventilator-associated lung injury, ESG, Cardiomyopathy, Cell engineering, Disease, Tissue, Corporate social responsibility, Gene editing, Research, NK, Social Security Government Pension Offset, AATD, Corporation, Liver, Social, FDA, Conference, Safety, D, DNA, Health care, SERPINA1, HAE, ATM, Therapy, MHRA, CRISPR, Liver disease, Hereditary angioedema, KLKB1, TTR, ATTR, A1AT, Amyloidosis, Banc of America Securities (1998–2008), Bank, Food, Pharmaceutical industry, Vaccine, Medical imaging, Medical device, Video game, IND

Subject to regulatory feedback, the Company anticipates initiating a global pivotal trial for ATTR-CM by year-end 2023.

Key Points: 
  • Subject to regulatory feedback, the Company anticipates initiating a global pivotal trial for ATTR-CM by year-end 2023.
  • The Company plans to present additional clinical data from the ATTRv-PN arm of the Phase 1 study in 2023.
  • Based on encouraging study interest from both investigators and patients, the Company expects to complete enrollment (n=25) in 2H 2023.
  • The Company will discuss these results on a conference call today, Thursday, May 4, at 8 a.m.

Wave Life Sciences Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 22, 2023
PRISM, 50/50 (2011 film), Takeda, C9orf72, MBA, Cerebrospinal fluid, Protein, MD, Observation, RNA interference, DSM-IV codes, Disease, Duchenne muscular dystrophy, Therapy, Genetics, Aimer, Pharmacokinetics, Mutation, Research, Gene expression, Small RNA, AATD, N-Acetylgalactosamine, Episcopal conference, Muscular Dystrophy Association, Sirna Therapeutics, Liver, MDA, IND, Conference, Safety, GSK, SERPINA1, Messenger, Webcast, APP, RNA editing, HD, ICV, Central nervous system, Q&A, Genomics, RNA, CNS, CTAS, DMD, Mouse, Pharmaceutical industry, Broadcasting, Vaccine, Cryptocurrency, Video game, Wave

ET today

Key Points: 
  • ET today
    CAMBRIDGE, Mass., March 22, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the fourth quarter and full year ended December 31, 2022 and provided a business update.
  • Research and development expenses were $31.1 million in the fourth quarter of 2022 as compared to $25.8 million in the same period in 2021.
  • As of December 31, 2022, Wave had $88.5 million in cash and cash equivalents, as compared to $150.6 million as of December 31, 2021.
  • ET to discuss the fourth quarter and full year 2022 financial results and provide a business update.

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.