Asc Therapeutics, Inc.

Transformational in-vivo AAV gene therapy for hemophilia A Efficiently biosynthesized and secreted bioengineered factor VIII Potentially leads to lower therapeutic dose, reduced cost, and extended durability MILPITAS, Calif.--(BUSINESS WIRE)-- ASC Therapeutics, a privately-held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic and other rare disorders, today announced that the first patient has been dosed in the company’s Phase I/IIa clinical trial of the lead candidate, ASC618, at the Arkansas Children’s Hospital. ASC618 is an investigational second-generation gene therapy for patients with severe and moderately severe hemophilia A. The transformational Adeno-Associated Virus (AAV) construct of ASC618, which contains a proprietary B-domain deleted codon-optimized bioengineered chimeric Factor VIII (FVIII) gene and a minimal-length liver-specific promoter, has been shown in pre-clinical studies to produce therapeutic levels of FVIII protein at doses that are significantly lower than other constructs in expressing the native human FVIII, which have been extensively tested in clinical trials. Oscar Segurado, MD, PhD, ASC Therapeutics’ Chief Medical Officer said, “This is a special day for ASC Therapeutics and for patients with hemophilia A hoping to access a more durable and affordable second-generation gene therapy. I am grateful to our team members and the clinical research team at Arkansas Children’s Hospital involved in the successful dosing of our first patient.” Ruhong Jiang, PhD, ASC Therapeutics’ CEO added, “The successful dosing of the first patient is a testament of both our commitment to the hemophilia A community and our team's capability in advancing novel genetic therapies into the clinic.” Shelley Crary, MD, Pediatric Hematologist/Oncologist at the Arkansas Children’s Hospital, principal investigator of the ASC618 phase I/IIa clinical trial, stated, “Treating our first patient with ASC618 reinforces our focus on providing cutting edge therapeutic modalities to our patients with hemophilia A. We are now assessing in a clinical setting the relevance of a novel one-and-done gene therapy that may replace lifelong, burdensome, and expensive treatments to manage hemophilia A.” About Hemophilia A Hemophilia A accounts for most cases of hemophilia (~80%), affecting approximately 1 of every 5000 live-born males. Over a million people around the world are estimated to have hemophilia, including more than 30,000 in the United States (US). Currently, patients with hemophilia A are managed with prophylactic or on-demand therapies that either replace FVIII or bypass FVIII function. The major challenges of current treatment regimens, such the short half-life of hemophilia therapeutics with need for frequent and lifelong treatments, justify ongoing focus on gene replacement therapies. About ASC618 ASC618 is an AAV8-based gene therapy product incorporating a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant (ET3). In preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with a B domain-deleted human FVIII construct. ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells over time1. ASC618 was developed based on extensive work on a second-generation gene therapy for hemophilia A from an academic team at Emory University2. ASC Therapeutics has obtained exclusive global rights from Expression Therapeutics to develop ASC618 and has conducted IND-enabling studies in multiple animal models that further demonstrated enhanced FVIII secretion from ASC618 and lower therapeutic doses. ASC Therapeutics is conducting a phase I/IIa clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618. The program has received IND clearance and was granted Fast Track and Orphan Drug Designations by the FDA and Orphan Medicinal Product Designation by the European Commission. The study design is available at 3. About ASC Therapeutics ASC Therapeutics is a biopharmaceutical company pioneering the development of gene replacement therapies, in-vivo gene editing and allogeneic cell therapies for hematological and other rare diseases. Led by a management team of industry veterans with significant global experience in gene and cell therapy, ASC Therapeutics is developing multiple therapeutic programs based on three technology platforms : 1) In-vivo gene therapies that use hepatocytes as a protein biofactory, initially focusing on ASC618 for hemophilia A; 2) In-vivo gene editing, initially focusing on ASC518 for hemophilia A; and 3) Allogeneic cell therapy, initially focusing on Decidua Stromal Cell-based therapy for steroid-refractory acute Graft-versus-Host Disease in patients receiving bone marrow transplantation, mostly to treat hematological malignancies. To learn more please visit . References 1 Steven W. Pipe, Gil Gonen-Yaacovi, Oscar G. Segurado. Hemophilia A gene therapy: current and next-generation approaches, Expert Opinion on Biological Therapy. 2022;22:1099-1115 2 Brown HC, Wright JF, Zhou S, et al. Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery. Mol Ther Methods Clin Dev. 2014;1:14036 3 ASC618 Gene Therapy in Hemophilia A Patients.

A California-based CDMO will be looking to expand its capabilities in its home state to keep up with market demand. Avid Bioservices is expanding its laboratories in Tustin, California around its capacity for its mammalian cell business. The investment is priced at $6 million, and the 4,500-square-foot expansion is expected to be completed by the end of the year. No outside funds were necessary and the company used cash on hand to fund the project. This expansion tails other investments the company has made to increase process development capacity in 2019 and 2022. Expanding its laboratories could support an additional $20 million in annual process development revenue, doubling the company’s current process development capacity, according to a statement. “Avid is focused on ensuring that it always has available capacity to meet the needs of its customers (current and future),” said Nick Green, CEO of Avid, in an email to Endpoints News. “This latest project will generate additional upstream and downstream process development capacity at the front-end of the mammalian cell business, which is critical for the efficient on-boarding of new customer projects.” This comes on the heels of Avid opening an analytical and process development suite within the company’s cGMP manufacturing facility. The launch of these is to expand its CDMO service offering into the rapidly growing cell and gene therapy market, with the company looking to fulfill those capabilities in 2023. Massachusetts-based Charles River Laboratories and ASC Therapeutics, a privately held biopharma that specializes in gene therapies, have agreed to manufacture ASC618, a gene therapy for hemophilia A. Financial details and a timeline of the arrangement were not disclosed. Kerstin Dolph, SVP for biologics solutions management at Charles River, said that the company has been working with ASC since 2019, to help establish a manufacturing process for their preclinical research. Together both companies have established a process for AAV8 production. Dolph also said that the plasmid manufacturing and testing, GMP-upstream and -downstream processes, and the fill/finish for ASC are happening at their facility in Rockville, MD. After acquiring Pfizer-owned Hospira Adelaide , the San Diego CDMO BioCina has expanded its service offerings to include the manufacture of plasmid DNA (pDNA) in a new GMP suite. While no financial or size details were presented, the suite is now open and has up to 300 L single-use fermentation capacity and scaled downstream processing equipment to complement their manufacturing capabilities. “With our extremely strong history of quality in manufacturing, an exemplary regulatory inspection history, and our customer-centric business approach, BioCina is in a strong position to add significant value to companies working in the RNA and gene and cell therapy space that are struggling to find reliable and collaborative outsourcing partners,” said BioCina CEO Ian Wisenberg. According to the company, its decision to expand its service offering into pDNA development and manufacturing was driven by the demand for GMP-grade pDNA. Swiss drug manufacturer Lonza and French pharmaceutical group Pierre Fabre announced on Wednesday that the companies have entered into a manufacturing agreement. While no financial details were revealed, the collaboration will be centered around manufacturing W0180, a monoclonal antibody discovered by Pierre Fabre targeting the VISTA checkpoint. The asset is being investigated as a single agent and in combination with pembrolizumab in Phase I clinical trial in various solid cancer tumors. The deal will have Lonza provide cGMP drug product manufacturing services for clinical supply from its fill and finish facility in Stein, Switzerland. Lonza has been on the move across the globe recently, as it has not only recently collaborated with a VC in Israel, but has also completed a manufacturing facility in Bend, Oregon.

June 13, 2022 12:00 UTC Expanded relationship will build upon established processes initiated in 2019 to meet ASC Therapeutics clinical program WILMINGTON, Mass.--(BUSINESS WIRE)-- Charles River Laboratories International, Inc. (NYSE: CRL) and ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in vivo gene replacement, gene editing and allogeneic cell therapies, today announced they have agreed to manufacture ASC618, a second-generation gene therapy for hemophilia A. Hemophilia A is caused by the lack of the blood clotting factor VIII (FVIII), a protein whose instructions are provided by F8, a gene that is mutated in people with the disease. ASC618 is designed to deliver a shortened, but optimized version of the gene to liver cells. ASC Therapeutics has received IND clearance, as well as key regulatory designations in the U.S. and Europe for its ASC618 program. Viral Vector Manufacturing Services Since 2019, ASC Therapeutics has worked with Charles River, previously Vigene Biosciences, to leverage the Company’s industry-leading expertise in Good Manufacturing Practice (GMP)-virus manufacturing, and established processes for AAV production and purification. Together, Charles River and ASC Therapeutics have established a high-yield upstream process for AAV8 production, optimized the downstream purification methods to result in a more predictable drug product output, and fine-tuned a scalable manufacturing process in both upstream and downstream. The collaboration will take advantage of Charles River’s end-to-end CDMO capabilities, building on the Company’s acquisitions of Cognate BioServices, Cobra Biologics, and Vigene Biosciences in 2021 that expanded its comprehensive cell and gene therapy portfolio to span each of the major CDMO platforms – cell therapy, viral vector and plasmid DNA production. Approved Quotes “We are proud to work with ASC Therapeutics as they progress their second-generation gene therapy program for hemophilia A to clinical stage. For three years, our collaboration has relied upon a strong and transparent relationship to support a seamless transition between process development and GMP production, and we’re excited to expand that work.” – Kerstin Dolph, Senior Vice President, Global Biologics, Charles River “Our work with Charles River has a strong foundation of trust—strengthening our shared outlook for the future and building confidence in our internal processes and oversight of external capabilities. As we moved toward the next phase of our therapeutic development, we are excited to work with Charles River to further maximize our manufacturing know-how.” -- Ruhong Jiang, PhD, Chief Executive Officer, ASC Therapeutics About Charles River Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit . About ASC Therapeutics ASC Therapeutics is a biopharmaceutical company pioneering the development of gene replacement therapies, in vivo gene editing and allogeneic cell therapies for hematological, metabolic, and other rare diseases. Led by a management team of industry veterans with significant global experience in gene and cell therapy, ASC Therapeutics is developing multiple therapeutic programs based on four technology platforms: 1) In-vivo gene therapy of inherited blood clotting disorders, initially focusing on ASC618, second generation gene therapy for hemophilia A, for which U.S. FDA IND clearance, Orphan Drug and Fast Track Designations were received; 2) In-vivo gene therapy in metabolic disorders, initially focusing on Maple Syrup Urine Disease, in collaboration with the Universities of Massachusetts and Pennsylvania; 3) In-vivo gene editing, initially focusing on ASC518 for hemophilia A; and 4) Allogeneic cell therapy, the first indication with a Decidua Stromal Cell-based therapy for steroid-refractory acute Graft-versus-Host Disease, for which U.S. FDA IND clearance and Orphan Drug Designation were received. To learn more please visit .