Eikonoklastes Therapeutics, Inc.

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

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 .