A1AT

4DMT Reports First Quarter 2024 Financial Results and Operational Highlights

Retrieved on: 
torsdag, maj 9, 2024
FDA, Regenerative medicine, Research, Choroideremia, Biopsy, European Medicines Agency, Injection, Overalls, Inflammation, Food, Alpha-1 antitrypsin, Cystic fibrosis, Chelsea Handler, Intravitreal administration, Conference, Retina, Atypical hemolytic uremic syndrome, DME, Angiogenesis, PRISM, AMD, Safety, Security (finance), Cash, Retinitis pigmentosa, Exercise, PRIME, EMA, NHP, Clinical trial, A1AT, ASRS, ARVO, ECFS, Șaeș, Cubic foot, Patient, Society, Geographic atrophy, Pharmaceutical industry

“The first quarter of 2024 kicks off another transformative year for 4DMT with exceptional progress across our product pipeline and platform in multiple therapeutic areas, particularly our lead program 4D-150 in large market VEGF-driven retinal diseases,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.

Key Points: 
  • “The first quarter of 2024 kicks off another transformative year for 4DMT with exceptional progress across our product pipeline and platform in multiple therapeutic areas, particularly our lead program 4D-150 in large market VEGF-driven retinal diseases,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
  • R&D Expenses: Research and development expenses were $27.9 million for the first quarter of 2024, as compared to $22.4 million for the first quarter of 2023.
  • G&A Expenses: General and administrative expenses were $10.3 million for the first quarter of 2024, as compared to $8.0 million for the first quarter of 2023.
  • Net Loss: Net loss was $32.4 million for the first quarter of 2024, as compared to net loss of $28.7 million for the first quarter of 2023.

Intellia Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
torsdag, februari 22, 2024
Research, Intellia Therapeutics, Regeneron Pharmaceuticals, Fatigue, Haemophilia, NK, CTA, Liver, Cystic fibrosis, ATM, A1AT, Amyloidosis, HAE, Therapy, European Medicines Agency, ATTR, Haemophilia B, DNA, European Commission, Hereditary angioedema, Recode, Conference, Cardiomyopathy, AATD, Civil service commission, Clinical trial, Facial muscles, CRISPR, Gene editing, Food, NTLA, KLKB1, Lung, NEJM, TTR, Patient, Medicine, Disease, Tissue, Pharmaceutical industry

Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.

Key Points: 
  • Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.
  • R&D Expenses: Research and development expenses increased by $9.0 million to $109.0 million during the fourth quarter of 2023, compared to $100.0 million during the fourth quarter of 2022.
  • G&A Expenses: General and administrative expenses increased by $5.4 million to $29.0 million during the fourth quarter of 2023, compared to $23.6 million during the fourth quarter of 2022.
  • Net Loss: The Company’s net loss was $132.2 million for the fourth quarter of 2023, compared to $113.4 million during the fourth quarter of 2022.

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

Retrieved on: 
torsdag, 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.

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

Retrieved on: 
torsdag, november 9, 2023
Gene editing, Research, Food, Facial muscles, Intellia Therapeutics, European Medicines Agency, Hereditary angioedema, Disease, A1AT, AAV, International, Mortality, Liver, TTR, HAE, Clinical, EMA, Safety, Therapy, Patient, D, DNA, KLKB1, ATM, Regeneron Pharmaceuticals, Cardiomyopathy, Conference, CTA, Tissue, NK, Serum, AATD, NTLA, FDA, ATTR, CRISPR, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Lung, SERPINA1, Amyloidosis, Pharmaceutical industry, Medical device, Vaccine, Video game, IND

“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.

Korro Bio and Frequency Therapeutics Announce Closing of Merger and Private Placement of $117 Million

Retrieved on: 
fredag, november 3, 2023
ADAR, Pharmacology, De novo, Atlas Venture, RBC Capital Markets, Transcriptome, RNA, MP, CUSIP, Genetics, Doctor of Philosophy, Wardwell, Growth, Caregiver, Disease, BofA Securities, Clinical trial, Machine learning, Latham & Watkins, TD, CVR Partners, A1AT, AATD, CVR, Therapy, Investment, Patient, Chemistry, NHP, Mutation, NEA, Dietary supplement, Pharmaceutical industry, Security (finance), Citadel, Frequency

J.P. Morgan Securities LLC served as exclusive financial advisor to Korro Bio and lead placement agent on Korro Bio’s private placement.

Key Points: 
  • J.P. Morgan Securities LLC served as exclusive financial advisor to Korro Bio and lead placement agent on Korro Bio’s private placement.
  • BofA Securities, Piper Sandler and RBC Capital Markets also served as placement agents for Korro Bio’s private placement.
  • Goodwin Procter LLP served as legal counsel to Korro Bio and Davis Polk & Wardwell LLP served as the placement agents’ legal counsel.
  • TD Cowen served as exclusive financial advisor to Frequency Therapeutics and Latham & Watkins LLP served as Frequency’s legal counsel.

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

Retrieved on: 
torsdag, augusti 3, 2023
NTLA, Intellia Therapeutics, Retirement, Investigational New Drug, Patient, Ventilator-associated lung injury, Cardiomyopathy, Cell engineering, Disease, Tissue, Gene editing, Research, European Academy of Allergy and Clinical Immunology, NK, AATD, Liver, FDA, IND, D, DNA, SERPINA1, HAE, ATM, Therapy, CRISPR, Liver disease, KLKB1, CTA, EAACI, TTR, ATTR, A1AT, Physician, Amyloidosis, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Female, Pharmaceutical industry, Medical imaging, Fine chemical, Rare-earth element, Vaccine, Video game

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: 
fredag, juli 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: 
torsdag, maj 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.

Gain Therapeutics Announces Grant Award by Eurostars with Innosuisse for Alpha-1 Antitrypsin Deficiency Research Program

Retrieved on: 
tisdag, mars 21, 2023
University of Helsinki, DSM-IV codes, University, Hepatology, Protein folding, Research, CNS, Lung, Institute for Research in Biomedicine, A1AT, GM, IRB, Quality control, Drug discovery, Liver, AAT, EPFL, Therapy, Eurostar International Limited, Safety, Pharmaceutical industry, Medical device

This grant supports a research project to develop novel small molecule allosteric regulators against Alpha-1 Antitrypsin (AAT) Deficiency, a rare genetic condition that can result in serious lung and liver diseases.

Key Points: 
  • This grant supports a research project to develop novel small molecule allosteric regulators against Alpha-1 Antitrypsin (AAT) Deficiency, a rare genetic condition that can result in serious lung and liver diseases.
  • “We are pleased that Eurostars and Innosuisse recognize that Gain Therapeutics’ allosteric small molecule regulators provide a new approach to address AAT deficiency-related metabolic diseases.
  • We look forward to advancing our AAT program with this consortium, which combines our unique targeting approach with novel in vitro and in vivo models.
  • Consequently, these models will be used as a disease model where lead small molecules therapeutic power can be tested in a physiological setting.”

Bragar Eagel & Squire, P.C. Reminds Investors That Class Action Lawsuits Have Been Filed Against Olo, Centessa, PayPal, and Opendoor and Encourages Investors to Contact the Firm

Retrieved on: 
måndag, november 28, 2022
Observation, Restaurant, DoorDash, Tripura Merger Agreement, A1AT, Bloomberg, Risk, Autosome, Class Action, Defendant, Class, Registration statement, BID, ADS, PayPal, P.C, Farm, NNA, Patient, Prospectus, Register, CNTA, SCH, Churn, PYPL, Growth, Polycystic kidney disease, DNA, Partnership, News, NYSE, Form, Fraud, Initial, Liver, Acquisition, AATD, Complaint, IPO, AE, Holding company, Food delivery, Risk management, Audit, Medical device, Security (finance), Animal, Renewable energy, Cryptocurrency, Online shopping, Opendoor, Subway, Algorithm, Mergers and acquisitions

Throughout the Class Period, Olo touted the growth of its active locations, with Subway accounting for approximately 20% of those locations.

Key Points: 
  • Throughout the Class Period, Olo touted the growth of its active locations, with Subway accounting for approximately 20% of those locations.
  • By the first quarter of 2022, the Companys active locations had grown to approximately 82,000 a 19% increase over the prior year.
  • For more information on the Opendoor class action go to: https://bespc.com/cases/OPEN
    About Bragar Eagel & Squire, P.C.
  • The firm represents individual and institutional investors in commercial, securities, derivative, and other complex litigation in state and federal courts across the country.