Armatus Bio LLC

March 07, 2024 - Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced a non-exclusive worldwide license and collaboration agreement with Armatus Bio for the use of Solid’s proprietary capsid AAV-SLB101 for the development and commercialization of Armatus’ vectorized RNAi candidate to treat Facioscapulohumeral muscular dystrophy (FSHD). The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells. Under the terms of the agreement, Solid granted Armatus a non-exclusive worldwide license to utilize AAV-SLB101 for treatment of FSHD and will provide Armatus AAV-SLB101 plasmid material, preclinical data characterizing AAV-SLB101, and manufacturing and regulatory know-how to enable development. In return, Solid will receive an upfront payment, payments upon the achievement of certain development and sales milestones, and tiered royalties on net sales for any products incorporating AAV-SLB101. Armatus will be responsible for the development and commercialization of licensed products incorporating AAV-SLB101. “Our transaction with Armatus Bio is the first of what we intend to be a broad out-licensing of our capsid library to both companies and academic institutions. We designed AAV-SLB101 for enhanced biodistribution to skeletal muscle, which is intended to help improve the efficacy and reduce the toxicity of AAV-delivered gene therapies for muscular disorders,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences. “AAV-SLB101 is the capsid for our next-generation Duchenne candidate, SGT-003, and we are excited to expand AAV-SLB101’s use to FSHD through this agreement with Armatus Bio.” Armatus is developing ARM-201, a potential best-in-class, single dose therapeutic used for the treatment of FSHD, a progressive genetic neuromuscular disorder caused by DUX4 overexpression. ARM-201’s proprietary miRNA payload seeks to reduce DUX4, and thus arrest muscle weakening and atrophy, prevent further degeneration, and ameliorate the amount of inflammation and oxidative stress caused by FSHD. “The use of Solid’s unique AAV-SLB101 capsid will help enable optimal patient dosing for our FSHD program. The preclinical data presented to date for AAV-SLB101 demonstrate increased muscle transduction while simultaneously reducing biodistribution in the liver. These data, combined with the data we have generated for ARM-201 give us high confidence in the potential to reduce total AAV dose levels compared to first generation capsids,” commented Brian Price, Ph.D., Chief Technology Officer at Armatus Bio. “Further, we are encouraged by the strong safety profile demonstrated by AAV-SLB101, which has a cleared IND as the capsid in Solid’s SGT-003 construct for the treatment of DMD. With this license in place, we are eager to advance our optimized ARM-201 construct as the lead candidate toward clinical evaluation.” AAV-SLB101 is a proprietary, rationally designed capsid developed for enhanced muscle tropism and reduced liver uptake. With a robust preclinical package in mice and nonhuman primates, AAV-SLB101 has demonstrated increased transduction speed, enhanced skeletal and cardiac muscle tropism, decreased liver biodistribution and improved efficiency when compared to first generation capsids. The incorporation of AAV-SLB101 into AAV delivered therapies has the potential to be a step forward in the treatment of neuromuscular and cardiac diseases. Solid Biosciences aims to license AAV-SLB101 broadly to both companies and academic institutions pursuing treatments for rare diseases. Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates for neuromuscular and cardiac programs, including SGT-003, for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, AVB-202-TT for the treatment of Friedreich’s ataxia, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Armatus Bio is a privately held late preclinical stage biotechnology innovator leveraging vectorized RNAi to address urgent unmet medical needs in genetically-driven neurological diseases. Based in Columbus, Ohio, the company is led by a seasoned team with expertise in drug development and delivery, and partnered with world renowned experts in vector biology, genomics, and neurology. The content above comes from the network. if any infringement, please contact us to modify.

COLUMBUS, Ohio, Jan. 15, 2024 /PRNewswire/ -- Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Armatus Bio to accelerate manufacturing of their gene therapy treatment for Charcot-Marie-Tooth Type 1A (CMT1A), a rare genetic neurological disease associated with independence-limiting disability and risk of fatal complications that has no approved therapies today. The partnership will seek to maximize program efficiency and speed to clinical studies of this novel, precision approach. As part of the agreement, Armatus will leverage Andelyn's extensive experience in adeno-associated virus (AAV) production and its proprietary suspension platform for development activities, plasmid manufacturing, and viral vector toxicology and GMP clinical manufacturing. The partnership strengthens the working relationship and offers great hope for patients suffering from neuromuscular disease. Both organizations, located in the well-established and growing biotech hub of Columbus, Ohio, are aligned in their belief that the rise of gene therapies will continue to advance human health. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences said, "We are extremely moved by Armatus Bio's commitment to address the urgent unmet needs in CMT1A and are impressed by its innovative therapeutic technology for the disease. We look forward to scaling up the process in our program-specific, configurable suspension platform, and accelerating the therapy into the clinic with the highest degree of quality." "Andelyn has demonstrated its strong capabilities in manufacturing complex genetic medicines with high quality and consistency, which will be critical to accelerating our development efforts," said Brian Price, PhD, Chief Technical Officer of Armatus. "We look forward to collaborating with Andelyn on this program as we work toward supporting this population that urgently awaits innovative solutions." From its time-tested experience, Andelyn is proud to offer its clients exceptional development, plasmid production, and quality manufacturing at its three Columbus, Ohio facilities. Andelyn is advancing client discoveries and manufacturing life-altering cell and gene therapies for rare and prevalent diseases. About Andelyn Biosciences, Inc. Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing curative cell and gene therapies from concept through plasmid development and manufacturing, process development, and cGMP clinical and commercial manufacturing. Andelyn's versatile capabilities include cGMP manufacturing capacity for both adherent and suspension processes up to a 2,000-liter capacity. An advanced digital model, quality system, full regulatory support and supply chain vertical integration help Andelyn accelerate the development and manufacturing of its clients' innovative cell and gene therapies. For more information, visit andelynbio.com. About Armatus Bio Armatus Bio is a privately held late preclinical stage biotechnology innovator leveraging vectorized RNAi to address urgent unmet medical needs in genetically-driven neurological diseases. Based in Columbus, Ohio, the company is led by a seasoned team with expertise in drug development and delivery, and partnered with world renowned experts in vector biology, genomics, and neurology. The company's lead candidate for Charcot Marie Tooth Type 1A (CMT1A) has received Orphan Drug Designation and Rare Pediatric Drug Designation, and is advancing toward IND-enabling studies. The company is also developing a preclinical vectorized RNAi asset for the rare neuromuscular disease Facioscapulohumeral Muscular Dystrophy (FSHD). SOURCE Andelyn Biosciences, Inc.

FIRST INVESTMENT FROM CMTA'S NEW VENTURE PHILANTHROPY ARM GLENOLDEN, PA / ACCESSWIRE / November 16, 2023 / The Charcot-Marie-Tooth Association (CMTA) today announced that it participated in the Seed Series Extension syndicate for Armatus Bio, Inc. (Armatus). Armatus is an emerging Biotechnology company that is advancing a unique gene therapy clinical candidate to target CMT type 1A, the most common form of the disease. Charcot-Marie-Tooth disease is an inherited peripheral neuropathy, principally affecting the long nerves of the hands and feet. Currently there are no FDA-approved therapies for any type of CMT. Thomas Dubensky, Ph.D., a CMTA board member commented "The CMTA is very excited to facilitate the advancement of novel therapies to be ultimately evaluated in patients living with all types of CMT. Armatus is the CMTA's first investment from its new venture philanthropy arm, which will make selected early-stage investments in Biotech companies developing unique and promising therapies against all types of CMT." Dr. Dubensky went on to say that "the venture philanthropy arm will work in tandem with STAR (Strategy to Accelerate Research), the arm of CMTA which is the largest private funder of CMT research in the world." Rachel Salzman, DVM, CEO of Armatus, commented, "Armatus is extremely grateful to the CMTA for its support, and we look forward to their continued advice and counsel as we advance ARM-101." Dr. Salzman went on to say, "Our ARM-101 development studies are particularly important for the CMT community because the data generated are designed to answer critical questions about how we can effectively deliver genetic medicines to the right parts of the body to meaningfully address this disease." She added, "As we accelerate development of ARM-101 and gain further evidence that our vectorized RNAi can normalize PMP22 in Schwann cells as a means of enabling human trials, we believe this therapy has the potential to provide benefit to the thousands of people living with CMT1A in the U.S. and around the world." Armatus Bio, based in Columbus, Ohio, is an emerging biotechnology company that is collaborating with renowned gene therapy experts to build a pipeline of novel therapeutic candidates designed to overcome the limitations of today's approaches and propel the next generation of genetic medicines. ABOUT THE CHARCOT-MARIE-TOOTH ASSOCIATION The Charcot-Marie-Tooth Association (CMTA) is the largest philanthropic funder of CMT research worldwide. The CMTA's Strategy to Accelerate Research (STAR) brings the best CMT researchers, clinicians, and experts in therapy development together with patients, pharmaceutical and biotechnology companies to expedite the development of treatments for CMT. Since 2008, the CMTA has invested more than $23.5 million via STAR. The CMTA also improves quality of life for CMT families by offering educational programs and materials, patient and professional conferences, support through youth programs, its branch system and global clinical Centers of Excellence. To learn more visit cmtausa.org and follow the CMTA on Instagram, Facebook, Twitter, TikTok, LinkedIn, and YouTube. Contact Information Marcia Semmes Media Contact, Charcot-Marie-Tooth Association marcia@cmtausa.org SOURCE: Charcot-Marie-Tooth Association View source version on accesswire.com: