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Sangamo Therapeutics Announces Data From Novel Proprietary Neurotropic AAV Capsid Demonstrating Industry-leading Blood-brain Barrier Penetration and Brain Transduction in NHPs

Retrieved on: 
Wednesday, March 13, 2024
Research, Neurology, Genetics, Clinical Trials, Health, Pharmaceutical, General Health, Other Science, Science, STAC, AAV, Sangamo Therapeutics, Gene, Brain, Capsid, Gene expression, ALS, BBB, Central nervous system, Novartis, CNS, CTA, DRG, Traffic barrier, Prion, NHP, Tropism, IND, Nav1.7, Neuron, Biogen, Tauopathy, Excipient, Therapy, DSM-IV codes, Spinal cord, Liver, Animal, Vaccine

Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.

Key Points: 
  • Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.
  • In NHP studies when administered intravenously at clinically relevant doses, STAC-BBB demonstrated its potential to be a leading neurotropic capsid.
  • Exhibited 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published neurotropic capsid variants evaluated in the study.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.

Xenon Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, February 29, 2024
Research, FOS, FDA, Pain, Food, Drug discovery, SCN8A, Epilepsy, Safety, SCN1A, Neurocrine Biosciences, Nav1.7, Webcast, Depression, Patient, OLE, KV7, DSM-IV codes, MDD, IND, NDA, Ageing, Neurology, Pharmaceutical industry

VANCOUVER, British Columbia, Feb. 29, 2024 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today reported financial results for the fourth quarter and full year ended December 31, 2023 and provided a corporate update.

Key Points: 
  • We also continue to evaluate other neurological indications that present a potential fit for XEN1101’s novel mechanism and product profile.
  • Other income was $8.7 million for the fourth quarter of 2023, and $31.4 million for the year ended 2023, compared to $7.1 million and $3.9 million for the same periods in 2022.
  • Net loss was $44.7 million for the fourth quarter of 2023, and $182.4 million for the year ended 2023, compared to $37.4 million and $125.4 million for the same periods in 2022, respectively.
  • Xenon will host a conference call and webcast today at 4:30 pm Eastern Time (1:30 pm Pacific Time) to discuss its fourth quarter and full year 2023 results.

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.

Xenon Pharmaceuticals Outlines Key Milestone Opportunities for 2024

Retrieved on: 
Monday, January 8, 2024
SCN1A, Drug discovery, DSM-IV codes, IND, Nav1.7, Depression, Epilepsy, FOS, Pain, SCN8A, Ageing, KV7, Food, Neurocrine Biosciences, MDD, Patient, Safety, OLE, Channel modulator, Pharmaceutical industry

VANCOUVER, British Columbia, Jan. 08, 2024 (GLOBE NEWSWIRE) --  Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today outlined progress within its pipeline programs and key milestones for 2024.

Key Points: 
  • VANCOUVER, British Columbia, Jan. 08, 2024 (GLOBE NEWSWIRE) --  Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today outlined progress within its pipeline programs and key milestones for 2024.
  • Xenon anticipates that patient enrollment in X-TOLE2 will be completed in the second half of 2024.
  • Xenon is actively assessing various clinical and regulatory pathways to support late-stage clinical development of XEN1101 in MDD and expects to initiate the Phase 3 clinical program in 2024.
  • The near-term focus is on development candidates targeting Kv7, Nav1.1 and Nav1.7 where Xenon expects multiple candidates will enter IND-enabling studies in 2024 and 2025.

Sangamo Therapeutics Announces Strategic Update and Reports Third Quarter 2023 Financial Results

Retrieved on: 
Wednesday, November 1, 2023
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Regenerative Medicine Advanced Therapy, Ethics, Therapy, Pfizer, Gene, Partnership, Head, Exposition, European Society of Gene and Cell Therapy, ALTA, AAV, Haemophilia, Hope, 65th Street Yard, Workforce, ERT, DSM-IV codes, Cell, CAR, U.S. FDA, Safety, Investment, Patient, Central nervous system, Nav1.7, MAA, 22q13 deletion syndrome, Conference, Enzyme replacement therapy, Hematology, Quality of life, Mace Head Atmospheric Research Station, Novartis, Brain, European Society for Primary Immunodeficiencies, Haemophilia A, Spectrum, FDA, IND, Fabry disease, Prion, Society, Senior, SCN2A, ESGCT, STAAR, Intellectual disability, Sclerosis, BLA, Pharmaceutical industry, Management, Vaccine, Biogen, Neuroscience, Fabry, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.
  • As a result of this restructuring, Sangamo is reducing its US workforce by approximately 40%, or approximately 162 roles.
  • Sangamo expects to incur approximately $8 million-$10 million in one-time restructuring costs in the fourth quarter of 2023.
  • Revenues for the third quarter ended September 30, 2023 were $9.4 million, compared to $26.5 million for the same period in 2022.

Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2023 Financial Results

Retrieved on: 
Tuesday, August 8, 2023
Biotechnology, Pharmaceutical, Genetics, Health, Pfizer, Central nervous system, Therapy, Gene, Haemophilia A, BLA, Biogen, Safety, Fabry disease, Phytoene synthase, Research, Brain, AAV, Science, Fast track (FDA), U.S. FDA, Sodium, Phase 3, Nav1.7, Novartis, Patient, Prion, Primate, Annual general meeting, Cell, American Society for Cell Biology, Eli Lilly and Company, MAA, FDA, STAAR, Enzyme replacement therapy, IND, Neurology, Haemophilia, Society, Pharmaceutical industry, Medical device, Vaccine, Fabry, Chroma, Sanofi, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and second quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and second quarter 2023 financial results.
  • We are pleased to have executed several business development deals, demonstrating the excitement in Sangamo’s science and platform,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • Revenues for the second quarter ended June 30, 2023 were $6.8 million, compared to $29.4 million for the same period in 2022.
  • Additionally, revenues relating to our collaboration agreement with Kite decreased by $5.1 million, reflecting a reduction in collaboration activities during the quarter.

Sangamo Therapeutics to Present Pre-clinical Data From Its Genomic Engineering Platform at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Tuesday, May 2, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Creutzfeldt–Jakob disease, Central nervous system, Gene, Prevalence, DSM-IV codes, Research, Flexible, AAV, Pain, Nav1.7, Patient, Neuropathic pain, Engineering, Cell, Therapy, Society, Vaccine, Powder metallurgy, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 14 Sangamo abstracts for presentation at the 26th ASGCT Annual Meeting being held May 16-20, 2023, in-person in Los Angeles, CA and in a virtual format.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 14 Sangamo abstracts for presentation at the 26th ASGCT Annual Meeting being held May 16-20, 2023, in-person in Los Angeles, CA and in a virtual format.
  • Presentations will focus on the progression of Sangamo’s pre-clinical programs, including data from the prioritized neurology programs Nav1.7 and Prion, innovations in the epigenetic regulation platform and advances in AAV capsid engineering for delivery.
  • Additional presentations at the ASGCT Annual Meeting will showcase how Sangamo is advancing its proprietary zinc finger platform development efforts, including the use of zinc finger transcriptional regulators designed to reduce the expression of the Prion protein for the treatment of Creutzfeldt-Jakob disease.
  • Sangamo will also present data from its proprietary AAV capsid discovery platform SIFTER™, which enables the engineering of AAV capsids with the potential for highly improved central nervous system transduction efficiency, along with broader presentations showcasing Sangamo’s latest genomic engineering platform evolution.

Sangamo Therapeutics Announces Strategic Update and Reports Preliminary First Quarter 2023 Financial Results

Retrieved on: 
Wednesday, April 26, 2023
Biotechnology, Pharmaceutical, Genetics, Health, Change, Pfizer, Gene, Head, Biogen, Haemophilia, GAAP, Research, AAV, American Society of Gene and Cell Therapy, Trial of the century, Gene editing, Nav1.7, Novartis, Patient, Investment, Annual general meeting, Cell, Kidney transplantation, MAA, Bank, American Society for Cell Biology, FDA, Therapy, STAAR, Kite, Environment, IND, Fabry disease, Haemophilia A, Society, BLA, Conference, Pharmaceutical industry, Renewable energy, Vaccine, Fine chemical, Video game, Cryptocurrency, Beauty salon, Fabry, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including a strategic pipeline prioritization and restructuring, and reported certain preliminary first quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including a strategic pipeline prioritization and restructuring, and reported certain preliminary first quarter 2023 financial results.
  • In addition, R. Andrew Ramelmeier, Ph.D., Executive Vice President, Technical Operations will be leaving the company on July 10, 2023.
  • Sangamo expects to incur approximately $5 million - $7 million in one-time restructuring costs in the second and third quarters of 2023.
  • Sangamo’s full first quarter 2023 financial results will be reflected in a Quarterly Report on Form 10-Q which is expected to be filed no later than May 10, 2023.

AlgoTx Progresses ATX01 in Two Indications

Retrieved on: 
Thursday, March 30, 2023
Biotechnology, FDA, Health, Clinical Trials, Oncology, ACT, Exercise, Erythromelalgia, Mayo Clinic, EU, Spinal cord, University of Erlangen–Nuremberg, Skin, Hope, Brain, CIPN, EMA, Pain, Nav1.7, Patient, Swelling, EASE, Clinical professor, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Red, Food, Nav1.8, Peripheral nervous system, Pharmaceutical industry

Meanwhile, the first patients have been randomized into the “ATX01 for the pain of ChemoTherapy” (ACT) study in Chemotherapy-Induced Peripheral Neuropathy (CIPN).

Key Points: 
  • Meanwhile, the first patients have been randomized into the “ATX01 for the pain of ChemoTherapy” (ACT) study in Chemotherapy-Induced Peripheral Neuropathy (CIPN).
  • Both programs have been granted Fast Track Designation by the FDA.
  • AlgoTx is conducting the ACT study as part of its US / EU Phase 2 program in the indication of CIPN.
  • AlgoTx has obtained an Orphan Disease Designation from both FDA and EMA to support development of ATX01 in this indication.

Q-State Biosciences Announces New Peer Reviewed Publication Introducing Its Next Generation Swarm™ Optogenetic Measurement Technology System

Retrieved on: 
Wednesday, July 13, 2022
Science, Neurology, Cardiology, Other Science, Biotechnology, Research, Pharmaceutical, Health, Nav1.7, Drug discovery, Ion, Frontiers, Patient, Paper, Brain, Therapy, Collection, High-throughput screening, Pain, Research, Neurological disorder, CNS, Drug development, Medical device

Q-State Biosciences (Q-State), a discovery technology and therapeutics company advancing programs for the treatment of serious central nervous system disorders, announced today the publication of a peer reviewed original research article in Frontiers in Molecular Neuroscience.

Key Points: 
  • Q-State Biosciences (Q-State), a discovery technology and therapeutics company advancing programs for the treatment of serious central nervous system disorders, announced today the publication of a peer reviewed original research article in Frontiers in Molecular Neuroscience.
  • The paper, titled Highly Parallelized, Multicolor Optogenetic Recordings of Cellular Activity for Therapeutic Discovery Applications in Ion Channels and Disease-associated Excitable Cells , highlights Q-States proprietary Swarm technology system.
  • The publication is part of a special research topic collection entitled Targeting Ion Channels for Drug Discovery: Emerging Challenges for High Throughput Screening Technologies.
  • The paper details the design and operating capabilities of the new Swarm system, including its use applications in neuronal and cardiac model systems to broadly quantify cellular activity.