Fabry disease

Freeline Receives Approval to Transfer to Nasdaq Capital Market

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Martedì, Novembre 29, 2022
Daimler Freeline, Fabry disease, American depositary receipt, Transgene, Gaucher's disease, Capsid, ADS, Research, EDGAR, AAV, Form 6-K, Fabry

LONDON, Nov. 29, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline) today announced that it received approval from the Nasdaq Stock Market LLC (Nasdaq) to transfer the listing of its American Depositary Shares (ADSs) representing ordinary shares of the Company from The Nasdaq Global Select Market to The Nasdaq Capital Market.

Key Points: 
  • LONDON, Nov. 29, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline) today announced that it received approval from the Nasdaq Stock Market LLC (Nasdaq) to transfer the listing of its American Depositary Shares (ADSs) representing ordinary shares of the Company from The Nasdaq Global Select Market to The Nasdaq Capital Market.
  • The Nasdaq Capital Market is a continuous trading market that operates in the same manner as The Nasdaq Global Select Market.
  • All companies listed on The Nasdaq Capital Market must meet certain financial requirements and adhere to Nasdaqs corporate governance standards.
  • In connection with the transfer to the Nasdaq Capital Market, Nasdaq granted the Company an additional 180-day period (or until May 29, 2023) to regain compliance with the minimum bid price requirement.

uniQure Highlights Therapeutic Potential of AMT-260 in Refractory Temporal Lobe Epilepsy (rTLE) at Virtual Research & Development Event

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Martedì, Novembre 29, 2022
Research, Temporal lobe epilepsy, Patient, Epilepsy, Head, Seizure, Compte rendu, Gene, Harvard Medical School, IND, Temporal lobe, Safety, Knowledge, Fabry disease, Brain, Primate, Learning, Mortality, Neoplasm, Annual report, GRIK2, GLP, Central nervous system, MD, TLE, CNS, Webcast, Disease, Risk, Health, Memory, Birth defect, Huntington's disease, Neurology, Hope, MicroRNA, Haemophilia, AAV, Hippocampus, Neuron, FDA, Movement disorders, Pharmaceutical industry, Vaccine, Medical device, Brigham

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 29, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, hosted a virtual investor event focused on AMT-260, an AAV gene therapy for refractory temporal lobe epilepsy (rTLE) and other focal epilepsies. The event featured a presentation from rTLE clinical expert Ellen Bubrick, MD, associate chair of Neurology at Harvard Medical School and director of the Epilepsy Surgery Program at Brigham and Women’s Hospital in Boston, MA on the unmet medical need of patients with rTLE. The presentation highlighted preclinical data that supports the safety and tolerability of AMT-260 and plans for its clinical development, as well as uniQure’s miQURE™ and linQURE™ technology platforms that allow the Company to use miRNAs to safely reduce the expression of genes in the brain. The Company also highlighted its progress in developing a commercial-scale AAV manufacturing platform. A replay of the investor event is available here.

Key Points: 
  • The full virtual research and development event program was webcast live under the Investors section of uniQures website at www.uniQure.com .
  • Temporal lobe epilepsy (TLE) is epilepsy that starts in the temporal lobe area of the brain.
  • About 80% of all temporal lobe seizures start in the mesial temporal lobe, with seizures often starting in or near the hippocampus which controls memory and learning.
  • Patients with refractory temporal lobe epilepsy (rTLE) experience increased morbidity, excess mortality, and poor quality of life.

4D Molecular Therapeutics to Participate in the Upcoming Evercore ISI HealthCONx 2022 Conference

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Lunedì, Novembre 21, 2022
Conference, Fabry disease, Tissue, Evercore, Ophthalmology, Choroideremia, US, Webcast, AMD, Cystic fibrosis, Entrepreneurship, Cardiology, Safety, Fabry, Vaccine

EMERYVILLE, Calif., Nov. 21, 2022 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, announced that David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT, will participate in a fireside chat at the Evercore ISI HealthCONx 2022 Conference.

Key Points: 
  • EMERYVILLE, Calif., Nov. 21, 2022 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, announced that David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT, will participate in a fireside chat at the Evercore ISI HealthCONx 2022 Conference.
  • The fireside chat will take place on Wednesday, November 30, 2022 at 3:55 p.m.
  • A live audio webcast of the fireside chat will be available by visiting the Investors & Media section of the 4DMT website at www.4dmoleculartherapeutics.com .
  • 4D Molecular Therapeutics, 4DMT, Therapeutic Vector Evolution, and the 4DMT logo are trademarks of 4DMT.

uniQure to Host Virtual Research & Development Event on Tuesday, November 29, 2022

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Giovedì, Novembre 17, 2022
EST, Harvard Medical School, Fabry disease, Virtual, Royal Commission into Aged Care Quality and Safety, Epilepsy, Safety, Patient, Website, Research, MD, Webcast, Huntington's disease, Development, Haemophilia, AAV, Quality, Broadcasting, Pharmaceutical industry, Brigham, Neurology

LEXINGTON, Ma. and AMSTERDAM, Nov. 17, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will host a Virtual Research & Development Event on Tuesday, November 29, 2022 from 8:30 a.m. to 10:30 a.m. EST.

Key Points: 
  • and AMSTERDAM, Nov. 17, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will host a Virtual Research & Development Event on Tuesday, November 29, 2022 from 8:30 a.m. to 10:30 a.m. EST.
  • Also included in this virtual event will be presentations by uniQures Research and Development team on the development of AMT-260 and a presentation by Pierre Caloz, chief operating officer at uniQure, outlining advancements in uniQures AAV manufacturing platform.
  • A replay of the webcast will be available at uniQures website for 45 days following the event.
  • uniQure is delivering on the promise of gene therapy single treatments with potentially curative results.

Freeline Reports Third Quarter 2022 Financial Results and Corporate Update

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Martedì, Novembre 15, 2022
COVID-19, Enzyme replacement therapy, Gesellschaft mit beschränkter Haftung, Patient, CMC, Fabry disease, Administration, R, Myocarditis, Transgene, Therapy, Trust, Partnership, Research, Gaucher's disease, Research and development, Capsid, Infection, Sale, Parini, ERT, Failure, Cell, EDGAR, Haemophilia, Clinical trial, AAV, CDMO, Safety, Form 6-K, Property management, Vaccine, Pharmaceutical industry, Daimler Freeline, Fabry

LONDON, Nov. 15, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today reported financial results for the third quarter of 2022 and announced its decisions to sell its CMC-focused subsidiary in Germany to Ascend Gene and Cell Therapies Ltd., streamline its organization to increase efficiency and strengthen its financial position, and prioritize the development of FLT201 in Gaucher disease and FLT190 in Fabry disease. Additionally, Freeline announced updated data from the first cohort of the MARVEL-1 Phase 1/2 clinical trial of FLT190 showing encouraging safety and durability.

Key Points: 
  • Freeline has entered into a definitive agreement to sell its subsidiary, Freeline Therapeutics GmbH, and certain related intellectual property to Ascend for $25million, subject to purchase price adjustments.
  • The transaction is expected to close in the first quarter of 2023 subject to customary closing conditions, including receipt of regulatory approvals.
  • Freeline will maintain its rights to its AAVS3 capsid, and the agreement will include a back-license to Freeline of the assigned rights necessary to develop and commercialize its current product candidates.
  • Freeline completed a thorough financial and organizational assessment to prioritize key capabilities, increase efficiencies and reduce expenses.

4D Molecular Therapeutics Announces Interim Clinical Data from On-going Phase 1/2 Clinical Trial of Intravitreal 4D-150 for Wet Age-Related Macular Degeneration (wet AMD)

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Lunedì, Novembre 14, 2022
Clinical trial, Principal, Safety, Security (finance), DME, VEGF, Diabetic retinopathy, Eye, PRISM, Partner (business rank), Blood, Webcast, AAV, Retina, Cystic fibrosis, Conference, Paganini Competition, Clinical research, Choroideremia, CNV, Inflammation, M.A, University, AMD, Fabry disease, Choroidal neovascularization, Molecule, R100, Hallucination, Macular degeneration, Therapy, US, Swelling, Severe cognitive impairment, University of Nevada, Reno, Ophthalmology, Injection, Fabry, Edema, Nobel, RNA interference, Bleeding, Tissue, Risk, Conference call, Cardiology, Scar, Patient, Medical device, Pharmaceutical industry, Vaccine, Placental growth factor

4D Molecular Therapeutics will host a conference call today, Monday November 14, 2022 at 8:00 AM E.T.

Key Points: 
  • 4D Molecular Therapeutics will host a conference call today, Monday November 14, 2022 at 8:00 AM E.T.
  • This clinical data on 4D-150 marks an important milestone for 4DMT, said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
  • We are developing 4D-150 for the treatment of large and sustainable markets in ophthalmology, including wet AMD and diabetic macular edema.
  • I am looking forward to enrolling patients in the randomized Phase 2 expansion stage of this clinical trial.

CANbridge Pharmaceuticals Granted Orphan Drug Designation for CAN 106 for the Treatment of Myasthenia Gravis

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Mercoledì, Novembre 16, 2022
Oncology, Health, FDA, General Health, Clinical Trials, Pharmaceutical, Biotechnology, FDA, Pompe, Weakness, Spinal muscular atrophy, Muscle contraction, Paroxysmal nocturnal hemoglobinuria, American Society of Gene and Cell Therapy, Acetylcholine, C5a, European Society for Medical Oncology, Glycogen storage disease, Ventilation, Glycogen storage disease type II, Neuromuscular disease, Degenerative disease, WuXi Biologics, University of Massachusetts, Patient, US, C3b, European Society of Gene and Cell Therapy, Muscle, Cell, C3a, SMA, Hospital, Glioblastoma, Research, MAC, Safety, Woman, Hunter syndrome, Drug, C3, Myasthenia gravis, Disease, Complement component 5, US FDA, Gene, C5, Congress, PNH, University, Haemophilia, Society, Fabry disease, Orphan, Pharmaceutical industry, Vaccine

Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.

Key Points: 
  • Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.
  • We continue to advance our global development strategy for CAN106 and look forward to developing CAN106 for myasthenia gravis and other complement-mediated diseases.
  • CAN106 is a clinical-stage investigational novel, long-acting recombinant humanized monoclonal antibody that binds to and neutralizes C5, a key component of the complement system.
  • CAN106 acts downstream of C3 in the complement pathway, preserving the generation of C3a and C3b, which are important for innate immunity.

4D Molecular Therapeutics Interim Clinical Data from the On-going Phase 1/2 Clinical Trial of 4D-150 for Wet AMD to be Released Monday, November 14, 2022

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Giovedì, Novembre 10, 2022
Investor, GLOBE, Private Securities Litigation Reform Act, Scar, Hallucination, Infrared spectroscopy correlation table, Risk, U.S. Securities and Exchange Commission, Macular degeneration, Swelling, Choroideremia, Nasdaq, Webcast, Tissue, Blood, Cystic fibrosis, Fabry, Bleeding, Ophthalmology, Therapy, US, Severe cognitive impairment, AMD, Choroidal neovascularization, Safety, RNA interference, VEGF, Molecule, Edema, Retina, Clinical trial, Patient, Security (finance), Cardiology, Percentage, CNV, Fabry disease, Pharmaceutical industry, Vaccine, Placental growth factor

4D Molecular Therapeutics also announced that interim clinical data from cohort 1 of this Phase 1/2 clinical trial will be released at 7:30 am E.T., on Monday, November 14, 2022.

Key Points: 
  • 4D Molecular Therapeutics also announced that interim clinical data from cohort 1 of this Phase 1/2 clinical trial will be released at 7:30 am E.T., on Monday, November 14, 2022.
  • 4D will host a conference call and live webcast on November 14th, 2022, at 8:00 am E.T.
  • 4D Molecular Therapeutics will host a conference call and live webcast on November 14, 2022, at 8:00 am E.T.
  • 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements.

4D Molecular Therapeutics Reports Third Quarter 2022 Financial Results

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Mercoledì, Novembre 9, 2022
GLOBE, Security (finance), Private Securities Litigation Reform Act, Risk, U.S. Securities and Exchange Commission, Interim, Choroideremia, Nasdaq, Research, Tissue, Cystic fibrosis, Fabry, Ophthalmology, Therapy, US, AMD, Safety, Molecule, AAV, D, Program, Clinical trial, Conference, Patient, Cardiology, COVID-19, Fabry disease, Pharmaceutical industry, Vaccine, Lease

EMERYVILLE, Calif., Nov. 09, 2022 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics, Inc. (Nasdaq: FDMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, today reported third quarter 2022 financial results.

Key Points: 
  • Revenue: Total revenue for the quarter ended September 30, 2022, was $0.5 million, as compared to $1.4 million for the quarter ended September 30, 2021.
  • Net Loss: Net loss was $25.7 million for the quarter ended September 30, 2022, as compared to $22.2 million for the quarter ended September 30, 2021.
  • 4D Molecular Therapeutics, 4DMT, Therapeutic Vector Evolution, and the 4DMT logo are trademarks of 4DMT.
  • Our results for the quarter ended September 30, 2022, are also not necessarily indicative of our operating results for any future periods.

Freeline to Present at the Stifel Healthcare Conference

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Mercoledì, Novembre 9, 2022
Investor, GLOBE, Haemophilia, Transgene, Capsid, Nasdaq, Gaucher's disease, Senior, AAV, Conference, Fabry disease, Vaccine

LONDON, Nov. 09, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that Chief Executive Officer Michael Parini will participate in a fireside chat at the Stifel Healthcare Conference being held in New York.

Key Points: 
  • LONDON, Nov. 09, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that Chief Executive Officer Michael Parini will participate in a fireside chat at the Stifel Healthcare Conference being held in New York.
  • Senior management will also participate in one-on-one investor meetings during the conference.
  • A live webcast of the fireside chat will be available on the Investors section of Freelines website at www.freeline.life.
  • An archived replay of the fireside chat will be available for approximately 90 days following the fireside chat.