Author: Shaw, Robin M ; Richmond, Bradley ; Cluntun, Ahmad A ; Hong, TingTing ; Funai, Katsuhiko ; Symons, J David ; Shaw, Kikuyo ; Li, Jing ; Bia, Ryan ; Franklin, Sarah ; Walsh, Maureen A ; Rutter, Jared

Diabetic cardiomyopathy, an increasingly global epidemic and a major cause of heart failure with preserved ejection fraction (HFpEF), is associated with hyperglycemia, insulin resistance, and intracardiomyocyte calcium mishandling. Here we identify that, in db/db mice with type 2 diabetes-induced HFpEF, abnormal remodeling of cardiomyocyte transverse-tubule microdomains occurs with downregulation of the membrane scaffolding protein cardiac bridging integrator 1 (cBIN1). Transduction of cBIN1 by AAV9 gene therapy can restore transverse-tubule microdomains to normalize intracellular distribution of calcium-handling proteins and, surprisingly, glucose transporter 4 (GLUT4). Cardiac proteomics revealed that AAV9-cBIN1 normalized components of calcium handling and GLUT4 translocation machineries. Functional studies further identified that AAV9-cBIN1 normalized insulin-dependent glucose uptake in diabetic cardiomyocytes. Phenotypically, AAV9-cBIN1 rescued cardiac lusitropy, improved exercise intolerance, and ameliorated hyperglycemia in diabetic mice. Restoration of transverse-tubule microdomains can improve cardiac function in the setting of diabetic cardiomyopathy and can also improve systemic glycemic control.

01 Jan 2022·Theranostics

Subpial delivery of adeno-associated virus 9-synapsin-caveolin-1 (AAV9-SynCav1) preserves motor neuron and neuromuscular junction morphology, motor function, delays disease onset, and extends survival in hSOD1^G93A mice

Article

Author: Wang, Shanshan ; Ichinomiya, Taiga ; Patel, Piyush M ; Li, Xiaojing ; Savchenko, Paul ; Wang, Dongsheng ; Head, Brian P ; Duong, Tiffany ; Miyanohara, Atsushi ; Li, Wenxi ; Marsala, Martin ; Bonds, Jacqueline A ; Kim, Eun Jung ; Sawada, Atsushi ; Patel, Hemal H ; Roth, David M ; Tadokoro, Takahiro

Elevating neuroprotective proteins using adeno-associated virus (AAV)-mediated gene delivery shows great promise in combating devastating neurodegenerative diseases. Amyotrophic lateral sclerosis (ALS) is one such disease resulting from loss of upper and lower motor neurons (MNs) with 90-95% of cases sporadic (SALS) in nature. Due to the unknown etiology of SALS, interventions that afford neuronal protection and preservation are urgently needed. Caveolin-1 (Cav-1), a membrane/lipid rafts (MLRs) scaffolding and neuroprotective protein, and MLR-associated signaling components are decreased in degenerating neurons in postmortem human brains. We previously showed that, when crossing our SynCav1 transgenic mouse (TG) with the mutant human superoxide dismutase 1 (hSOD1^G93A) mouse model of ALS, the double transgenic mouse (SynCav1 TG/hSOD1^G93A) exhibited better motor function and longer survival. The objective of the current study was to test whether neuron-targeted Cav-1 upregulation in the spinal cord using AAV9-SynCav1 could improve motor function and extend longevity in mutant humanized mouse and rat (hSOD1^G93A) models of familial (F)ALS. Methods: Motor function was assessed by voluntary running wheel (RW) in mice and forelimb grip strength (GS) and motor evoked potentials (MEP) in rats. Immunofluorescence (IF) microscopy for choline acetyltransferase (ChAT) was used to assess MN morphology. Neuromuscular junctions (NMJs) were measured by bungarotoxin-a (Btx-a) and synaptophysin IF. Body weight (BW) was measured weekly, and the survival curve was determined by Kaplan-Meier analysis. Results: Following subpial gene delivery to the lumbar spinal cord, male and female hSOD1G93A mice treated with SynCav1 exhibited delayed disease onset, greater running-wheel performance, preserved spinal alpha-motor neuron morphology and NMJ integrity, and 10% increased longevity, independent of affecting expression of the mutant hSOD1G93A protein. Cervical subpial SynCav1 delivery to hSOD1G93A rats preserved forelimb GS and MEPs in the brachial and gastrocnemius muscles. Conclusion: In summary, subpial delivery of SynCav1 protects and preserves spinal motor neurons, and extends longevity in a familial mouse model of ALS without reducing the toxic monogenic component. Furthermore, subpial SynCav1 delivery preserved neuromuscular function in a rat model of FALS. The latter findings strongly indicate the therapeutic applicability of SynCav1 to treat ALS attributed to monogenic (FALS) and potentially in sporadic cases (i.e., SALS).

01 Dec 2021·Pacing and Clinical ElectrophysiologyQ3 · ENGINEERING & TECHNOLOGY

Improvement of electrical synchrony in cardiac resynchronization therapy using dynamic atrioventricular delay programming and multipoint pacing

Q3 · ENGINEERING & TECHNOLOGY

Article

Author: Lemke, Bernd ; Schiedat, Fabian ; Mügge, Andreas ; Karosiene, Zana ; Zarse, Markus ; Kloppe, Axel ; Hanefeld, Christoph ; Bogossian, Harilaos ; Mijic, Dejan

AbstractBackgroundOptimization of cardiac resynchronization therapy (CRT) is often time‐consuming and therefore underused in a clinical setting. Novel device‐based algorithms aiming to simplify optimization include a dynamic atrioventricular delay (AVD) algorithm (SyncAV, Abbott) and multipoint pacing (MPP, Abbott). This study examines the acute effect of SyncAV and MPP on electrical synchrony in patients with newly and chronically implanted CRT devices.MethodsPatients with SyncAV and MPP enabled devices were prospectively enrolled during implant or scheduled follow‐up. Blinded 12‐lead electrocardiographic acute measurements of QRS duration (QRSd) were performed for intrinsic QRSd (Intrinsic), bi‐ventricular pacing (BiV), MPP, BiV with SyncAV at default offset 50 ms (BiVSyncAVdef), BiV with SyncAV at patient‐specific optimised offset (BiVSyncAVopt), MPP with SyncAV at default offset 50 ms (MPPSyncAVdef), and MPP with SyncAV at patient‐specific optimised offset (MPPSyncAVopt).ResultsThirty‐three patients were enrolled. QRSd for Intrinsic, BiV, MPP, BiVSyncAVdef, BiVSyncAVopt, MPPSyncAVdef, MPPSyncAVopt were 160.4 ± 20.6 ms, 141.0 ± 20.5 ms, 130.2 ± 17.2 ms, 121.7 ± 20.9 ms, 117.0 ± 19.0 ms, 121.2 ± 17.1 ms, 108.7 ± 16.5 ms respectively. MPPSyncAVopt led to greatest reduction of QRSd relative to Intrinsic (−31.6 ± 11.1%; p < .001), showed significantly shorter QRSd compared to all other pacing configurations (p < .001) and shortest QRSd in every patient. Shortening of QRSd was not significantly different between newly and chronically implanted devices (−51.6 ± 14.7 ms vs. −52.7 ± 21.9 ms; p = .99).ConclusionSyncAV and MPP improved acute electrical synchrony in CRT. Combining both technologies with patient‐specific optimization resulted in greatest improvement, regardless of time since implantation.Whats newNovel device‐based algorithms like a dynamic AVD algorithm (SyncAV, Abbott) and multipoint pacing (MPP, Abbott) aim to simplify CRT optimization. Our data show that a combination of patient tailored SyncAV optimization and MPP results in greatest improvement of electrical synchrony in CRT measured by QRS duration, regardless if programmed in newly or chronically implanted devices. This is the first study to our knowledge to examine a combination of these device‐based algorithms. The results help understanding the ideal ventricular excitation in heart failure.

News (Medical) associated with SynCav-1

13 Nov 2024

·www.prnewswire.com

75+ Key Companies Charting New Frontiers in Multiple Sclerosis Therapeutic Space | DelveInsight

Multiple sclerosis (MS) is a chronic neurological disorder affecting the central nervous system (CNS), particularly the brain, spinal cord, and optic nerves. Continuous research has led to new drug developments, particularly disease-modifying therapies (DMTs) that slow MS progression. Monoclonal antibodies, oral therapies, and infusion treatments are among the most promising options and drives the multiple sclerosis market. LAS VEGAS, Nov. 13, 2024 /PRNewswire/ -- DelveInsight's ' Multiple Sclerosis Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline multiple sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the multiple sclerosis pipeline domain. Key Takeaways from the Multiple Sclerosis Pipeline Report DelveInsight's multiple sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for multiple sclerosis treatment. Key multiple sclerosis companies such as Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Promising multiple sclerosis pipeline therapies such as GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. In October 2024, Immunic, Inc. reported favorable results from the non-binding interim futility analysis of its Phase 3 ENSURE trial, which is evaluating its lead candidate, vidofludimus calcium (IMU-838), a nuclear receptor-related 1 (Nurr1) activator, for the treatment of relapsing multiple sclerosis (RMS). In October 2024, Immunic, Inc. announced a positive outcome of the non-binding, interim futility analysis of its Phase III ENSURE program, investigating lead asset, nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), for the treatment of relapsing multiple sclerosis (RMS). Based on the outcome of the interim futility analysis, an unblinded Independent Data Monitoring Committee (IDMC) has recommended that the trials are not futile and should continue as planned. In September 2024, Roche, secured FDA approval for an injectable form of its blockbuster multiple sclerosis treatment, Ocrevus, reducing patient treatment time and potentially helping the company stay ahead of increasing competition. In September 2024, Sanofi announced the outcomes of Phase III clinical trials of its investigational oral brain-penetrant BTK inhibitor, tolebrutinib, aimed at treating multiple sclerosis (MS). These studies, namely HERCULES, GEMINI 1, and GEMINI 2, have provided mixed results, with the HERCULES trial meeting its primary endpoint while the GEMINI trials did not. The HERCULES study focused on the efficacy and safety of tolebrutinib in individuals with non-relapsing secondary progressive MS (nrSPMS) compared to a placebo. In August 2024, Abata Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The FDA recently cleared ABA-101's Investigational New Drug (IND) application, and initiation of a first-in-human (FIH) Phase I study is imminent. In August 2024, TG Therapeutics announced that the FDA has cleared an investigational new drug (IND) application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for a phase 1 clinical trial in patients with progressive multiple sclerosis (MS). In January 2024, Kyverna Therapeutics, Inc. announced it received fast-track designation by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of multiple sclerosis (MS). Request a sample and discover the recent advances in multiple sclerosis treatment drugs @ Multiple Sclerosis Pipeline Report The multiple sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage multiple sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the multiple sclerosis clinical trial landscape. Multiple Sclerosis Overview Multiple sclerosis is a chronic autoimmune disorder that affects the central nervous system, leading to the deterioration or permanent damage of myelin, the protective sheath surrounding nerve fibers. While the exact cause of multiple sclerosis remains unclear, it is believed to involve a combination of genetic predisposition, environmental factors, and possibly viral infections. Common symptoms of multiple sclerosis include fatigue, difficulty walking, numbness or tingling in the limbs, muscle weakness, and vision problems, which can vary significantly among individuals and may evolve over time. Diagnosis typically involves a combination of neurological examinations, magnetic resonance imaging (MRI) to detect lesions in the brain and spinal cord, and sometimes lumbar puncture to analyze cerebrospinal fluid for inflammatory markers. While there is no cure for multiple sclerosis, treatment options aim to manage symptoms, reduce the frequency and severity of relapses, and slow disease progression. This may include disease-modifying therapies (DMTs), corticosteroids for acute exacerbations, and symptomatic treatments such as physical therapy, pain management, and lifestyle modifications. Early diagnosis and a tailored treatment plan are crucial for improving the quality of life for those living with multiple sclerosis. Find out more about multiple sclerosis treatment drugs @ Drugs for Multiple Sclerosis Treatment A snapshot of the Multiple Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging multiple sclerosis pipeline therapies @ Multiple Sclerosis Clinical Trials Multiple Sclerosis Therapeutics Assessment The multiple sclerosis pipeline report proffers an integral view of the multiple sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Multiple Sclerosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Immunomodulators, MHC class II gene modulators, Agammaglobulinaemia tyrosine kinase inhibitors, Dihydroorotate dehydrogenase inhibitors, Nuclear receptor subfamily 4 group A member 2 agonists, CD20 antigen inhibitors, Energy metabolism stimulants; Immunomodulators, Lysine-specific demethylase 1 inhibitors, Monoamine oxidase B inhibitors, Protein-arginine deiminase inhibitors, Immunostimulants Key Multiple Sclerosis Companies: Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Key Multiple Sclerosis Pipeline Therapies:GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. Dive deep into rich insights for new drugs for multiple sclerosis treatment, visit @ Multiple Sclerosis Drugs Table of Contents For further information on the multiple sclerosis pipeline therapeutics, reach out @ Multiple Sclerosis Treatment Drugs Related Reports Multiple Sclerosis Market Multiple Sclerosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key multiple sclerosis companies including Immune Response BioPharma, Inc., Clene Nanomedicine, Genzyme, ImStem Biotechnology, Rho, Inc., Bristol-Myers Squibb, TG Therapeutics, Inc., Hoffmann-La Roche, Atara Biotherapeutics, Immunic AG, Celgene, Anokion SA, Sanofi, Actelion, Biogen, Supernus Pharmaceuticals, Inc., GeNeuro SA, HuniLife Biotechnology, Inc., Emerald Health Pharmaceuticals, RemeGen Co., Ltd., Antisense Therapeutics, Biocad, AB Science, Genentech, Novartis, Pipeline Therapeutics, ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, Mapi Pharma, f5 Therapeutics, Autobahn Therapeutics, AstraZeneca, ZyVersa Therapeutics, RedHill Biopharma, Gossamer Bio, Sarepta Therapeutics, BrainStorm Cell Limited, CytoDyn, Pear Therapeutics, Solstice Neurosciences, ProJenX, among others. Multiple Sclerosis Epidemiology Forecast Multiple Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted multiple sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Relapsing Multiple Sclerosis Pipeline Relapsing Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key relapsing multiple sclerosis companies, including ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, among others. Relapsing-Remitting Multiple Sclerosis Pipeline Relapsing-Remitting Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMS companies, including TG Therapeutics, Novartis, Sanofi, Cinnagen, Polpharma Biologics, Immunic, Mapi Pharma, Biocad, Apimeds, Genentech (Roche), Merck, Immune Response BioPharma, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, Emerald Health Pharmaceuticals, GlaxoSmithKline, RedHill Biopharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultFast TrackCell TherapyImmunotherapy

15 May 2023

·www.biospace.com

Eikonoklastes Announces Formation of Scientific Advisory Board – Gene Therapy

CINCINNATI--(BUSINESS WIRE)-- Eikonoklastes Therapeutics, a biopharmaceutical company developing a novel gene therapy platform to discover and develop critical treatments for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases, today announced the formation of a Scientific Advisory Board (SAB) - Gene Therapy, comprised of key leaders in neurology and gene therapy development. The SAB – Gene Therapy will work cohesively with management to advance the company’s gene therapy pipeline. Eikonoklastes’s gene therapy platform is built upon the pioneering work from the lab of Dr. Brian Head, Professor of Anesthesiology at University of California, San Diego. “I am very pleased to join Eikonoklastes’ Scientific Advisory Board,” said Dame Pamela Shaw, DBE, MD. “I look forward to working with this distinguished group of industry experts to advise the Eikonoklastes team through the development of its innovative technology to address enormous unmet clinical needs.” “We are fortunate to have the support of a truly world-class group of scientific and industry thought leaders on our Scientific Advisory Board, who will offer their expertise to ensure we optimally design our development program to meet the needs of patients and rapidly advance this technology through regulatory approval to treat patients with this devastating disease,” said Bruce Halpryn, Ph.D., Board Chair and CEO of Eikonoklastes. “Each member brings unique experience in technology, manufacturing, regulatory affairs, and clinical development. This collection of unparalleled expertise will serve us well as we develop our lead candidate, ET-101, initially for ALS and expanding into several other inadequately treated neurodegenerative diseases,” said Samuel Lee, MD, MBA, MPH, President and Chief Business Officer of Eikonoklastes. The founding members of the Eikonoklastes Scientific Advisory Board – Gene Therapy are: Brian Head, PhD Research Career Scientist, VA San Diego Healthcare System Professor of Anesthesiology, University of California – San Diego Russell Lonser, MD Chairman, Department of Neurological Surgery, Ohio State University Wexner Medical Center Director, Gene Therapy Institute, Ohio State University Professor of Neuroscience, Ohio State University College of Medicine Michael O’Beirne Former Executive Director, Regulatory Strategy at Sarepta Therapeutics Vaithianathan “Palani” Palaniappan, PhD Chief Technology Officer, Pioneering Medicines at Flagship Pioneering Former Head of Global Technical Operations, Sarepta Former Global Head of Biologics and New Modality Development, Takeda Seward Rutkove, MD Chair, Department of Neurology at Beth Israel Deaconess Medical Center Professor of Neurology, Harvard Medical School Dame Pamela Shaw, DBE, MD Founder and Director, Sheffield Institute for Translational Neuroscience Professor of Neurology, University of Sheffield Mark Tuszynski, MD, PhD Director, UC San Diego Translational Neuroscience Institute Distinguished Professor of Neuroscience, University of California – San Diego About Eikonoklastes Therapeutics Eikonoklastes Therapeutics is a biotech company rapidly developing a pipeline of disruptive new treatments for today’s most challenging diseases. The company’s 1st-in-class gene therapy (ET-101) has been engineered to overexpress Caveolin-1, which organizes and regulates synaptic receptors essential for neuromuscular signaling and function. Eikonoklastes is also advancing a new generation of tissue factor (TF) therapies (ET-201) for the treatment of Triple Negative Breast Cancer and other oncology indications. To learn more, visit

Gene Therapy

29 Nov 2022

·www.biospace.com

Eikonoklastes Therapeutics and Forge Biologics Announce Viral Vector Contract Development and AAV Manufacturing Partnership

AAV Manufacturing Partnership to Advance Eikonoklastes’ Novel Gene Therapy for Amyotrophic Lateral Sclerosis (ALS) CINCINNATI & COLUMBUS, Ohio--(BUSINESS WIRE)-- Eikonoklastes Therapeutics, a preclinical biotech company developing treatments for today’s most challenging diseases, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, today announced a manufacturing partnership that will advance Eikonoklastes’ AAV-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis (ALS). This press release features multimedia. View the full release here: Forge will provide adeno-associated viral (AAV) process development and manufacturing services for ET-101. Eikonoklastes will utilize Forge’s platform process including its proprietary HEK 293 suspension Ignition Cells™ and pEMBR™ adenovirus helper plasmid. All development and AAV manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy facility in Columbus, Ohio. “Eikonoklastes is conducting promising research into treating patients with ALS, and we are honored to work together with them during this critical phase of development to help ET-101 enter clinical trials,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge Biologics. “Our primary goal is to deliver the AAV resources, scale, technology, and expertise for our partners to help patients living with diseases like ALS with no known cure.” ALS is a rare neurological disease with significant socioeconomic impact, whose patients are desperate for novel, safe and effective therapies. ALS is the lead indication for Eikonoklastes’ ET-101, which has just received Orphan Drug Designation from the FDA—an important regulatory milestone reflecting the compelling data underlying the ET-101 program and its potential as a first-in-class treatment option for both familial and sporadic ALS. “We are excited to advance ET-101, our novel treatment for neurodegenerative diseases, towards a first-in-human clinical trial in ALS patients. Partnering our AAV manufacturing with Forge will further our ability to advance into clinical trials,” said Bruce Halpryn, Ph.D., Chair, CEO, and Co-Founder of Eikonoklastes Therapeutics. “With their end-to-end platform supporting AAV therapies, Forge is accelerating our development to reach patients suffering from ALS.” About Amyotrophic Lateral Sclerosis (ALS) ALS, also known as Lou Gehrig’s Disease, is a rare, progressive, debilitating, and universally fatal neurodegenerative disease affecting the motor neurons connecting the brain and spinal cord. Degeneration of these neurons leads to paralysis as the brain loses its ability to control muscle movement. Most people with ALS succumb to respiratory failure, usually within three to five years from when symptoms first appear. An estimated 10 percent of ALS is familial and caused by genetic mutations that are inherited. The 90%+ of sporadic ALS may be due to a combination of environment and genetic risk factors. There is currently no cure for either familial or sporadic ALS. About Eikonoklastes Therapeutics Eikonoklastes Therapeutics is a preclinical biotech company rapidly developing a pipeline of disruptive new treatments for today’s most challenging diseases. The company’s 1st-in-class gene therapy (ET-101) has been engineered to overexpress Caveolin-1, which organizes and regulates synaptic receptors essential for neuromuscular signaling and function. Eikonoklastes is also advancing a new generation of tissue factor (TF) therapies (ET-201) for the treatment of Triple Negative Breast Cancer and other oncology indications. To learn more, visit About Forge Biologics Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life changing gene therapies and help bring them from idea to reality. Forge’s 200,000 square foot facility utilizes 20 cGMP suites in Columbus, Ohio, the Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV manufacturing and hosts scalable, end-to-end manufacturing services. Offerings include process and analytical development, plasmid DNA manufacturing, viral vector manufacturing, final fill, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most. To learn more, visit .

Gene TherapyOrphan DrugClinical Study

100 Deals associated with SynCav-1

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	Preclinical	US	CavoGene LifeSciences	-
Huntington Disease	Discovery	US	Eikonoklastes Therapeutics, Inc.Startup	20 Oct 2022
Multiple Sclerosis	Discovery	US	CavoGene LifeSciences	08 Sep 2020
Parkinson Disease	Discovery	US	CavoGene LifeSciences	08 Sep 2020
Alzheimer Disease	Discovery	US	CavoGene LifeSciences	23 Oct 2018
Brain Injuries, Traumatic	Discovery	US	CavoGene LifeSciences	23 Oct 2018
Cognition Disorders	Discovery	US	CavoGene LifeSciences	23 Oct 2018
Spinal Cord Injuries	Discovery	US	CavoGene LifeSciences	23 Oct 2018

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