List of Gene Therapy Companies in Australia - 10

Beroni Group is an international biotechnological company listed on the National Stock Exchange (Australia) and traded on the OTC markets in the U.S.A. with business presence in Australia, China, Japan and the USA. It currently has four core businesses – cell therapies, developing new anti-cancer drugs, e-commerce platform for pharmaceutical and healthcare products, and detection & diagnosis of infectious diseases. Beroni Group’s overall strategic goal is to become a world’s leading enterprise in the biotechnology, life sciences, and environmental science industries.

Cartherics works to create precisely-defined immunotherapy treatments to combat a variety of cancers. We take a multi-platform approach combining the best components of immune defences utilising gene editing and CAR technology. Our team has strong backgrounds in research, development, and product commercialisation. And we partner with leading commercial and academic groups with complementary technology to push the boundaries of cell therapy for cancer.

Cell Therapies Pty Ltd is a contract development and manufacturing organization (CDMO) that manufactures and deploys advanced cell-based therapies to the global market. Established in 2003, Cell Therapies is one of the most experienced GMP compliant manufacturers of cell therapies, gene therapies, cellular immunotherapies, and regenerative medicine products globally. We have a 13-cleanroom GMP facility that meets global regulatory and international organisation for standardisation (ISO) standards benchmarked by the Pharmaceutical Inspection Convention/Cooperation Scheme (PIC/S). Our services cover the complete manufacturing process from development to deployment, providing needle-to-needle control with clinical integration. Our facility is co-located with the Peter MacCallum Cancer Centre in Melbourne, Australia, providing access to hospitals, research institutes, and universities to support product development, translation to the clinic, and patient access. Please contact us through our website to discuss potential collaborations.

Celosia Therapeutics is a privately held pre-clinical stage gene therapy company, developing solutions for neurodegenerative diseases that have limited alternative therapeutic options.

Chimeric Therapeutics is a clinical stage cell therapy company focused on bringing the promise of cell therapy to life for more patients with cancer. We believe that cellular therapies have the promise to cure cancer not just delay disease progression. To bring that promise to life for more patients, Chimeric’s world class team of cell therapy pioneers and experts is focused on the discovery, development and commercialization of the most innovative and promising cell therapies.chm

CSL is a leading global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency, dialysis and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our businesses, CSL Behring, CSL Seqirus, CSL Plasma and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest.

Cynata Therapeutics Limited (ASX: CYP) is an Australian clinical-stage stem cell and regenerative medicine company focused on the development of therapies based on Cymerus™, a proprietary therapeutic stem cell platform technology. Cymerus™ overcomes the challenges of other production methods by using induced pluripotent stem cells (iPSCs) and a precursor cell known as mesenchymoangioblast (MCA) to achieve economic manufacture of cell therapy products, including mesenchymal stem cells (MSCs), at commercial scale without the limitation of multiple donors. Cynata’s lead product candidate CYP-001 met all clinical endpoints and demonstrated positive safety and efficacy data for the treatment of steroid-resistant acute graft-versus-host disease (GvHD) in a Phase 1 trial. Clinical trials of Cymerus MSC products in osteoarthritis (Phase 3) and in severe complications arising from COVID-19 (Phase 2) are currently ongoing. Planning is also underway for further clinical trials of Cymerus MSC products in GvHD (through licensee Fujifilm), critical limb ischemia, idiopathic pulmonary fibrosis, renal transplantation, and diabetic foot ulcers. In addition, Cynata has demonstrated utility of its Cymerus MSC technology in preclinical models of numerous diseases, including the clinical targets mentioned above, as well as asthma, heart attack, sepsis, acute respiratory distress syndrome (ARDS) and cytokine release syndrome.

Immutep is a globally active biotechnology company that is a leader in the development of LAG-3 related immunotherapeutic products for the treatment of cancer and autoimmune disease. Immutep is dedicated to leveraging its technology and expertise to bring innovative treatment options to market for patients and to maximize value to shareholders.

Lateral Pharma Pty Ltd Is a privately-owned Melbourne AU-based biotechnology company that commenced operations in May 2015 to repurpose LAT8881, a Phase 2 ready orally-available compound with extensive toxicology and human safety data, into new high potential pain and respiratory indications. Lateral aims to demonstrate efficacy in a human clinical Phase 2 proof-of-concept study using LAT8881 to treat neuropathic pain within the next 12 months. Lateral Pharma has discovered a new class of naturally-derived peptide motifs and their novel target family of proteins that restore normal tissue function in mammals under conditions of damage and cellular stress. Small synthetic peptides derived from growth hormone and at least three other proteins are active in animal models of neuropathic pain, osteoarthritis, obesity, COPD, severe influenza A and recently in initial data in SARS-CoV-2. Lateral has filed multiple patents protecting its intellectual property Lateral is focussed on developing its drugs for the treatment of Neuropathic pain but subject to additional funding will also explore development in respiratory diseases such as Influenza, SARS-CoV-2, COPD, Steroid resistant asthma, and Post-COVID Syndrome (Long-COVID).

Prescient Therapeutics (ASX: PTX) is a clinical stage oncology company developing personalised medicine approaches to cancer, including targeted and cellular therapies. Cell Therapies OmniCAR: is a universal immune receptor platform enabling controllable cell activity and multi- antigen targeting with a single cell product. OmniCAR’s modular CAR system decouples antigen recognition from the cell signalling domain. It is the first universal immune receptor allowing post- translational covalent loading of binders to immune cells. OmniCAR is based on technology licensed from Penn and Oxford University; and other assets. Prescient is developing OmniCAR-T cells programs for next-generation CAR-T therapies for AML; Her2+ solid tumours, including breast, ovarian and gastric cancers; and GBM. CellPryme: Prescient's novel, ready-for-the-clinic, CellPryme-M technology enhances adoptive cell therapy performance by shifting T and NK cells towards a central memory phenotype, improving persistence, and increasing the ability to find and penetrate tumours. CellPryme-M is a 24-hour, non-disruptive process during cell manufacturing. Targeted Therapies PTX-100 is a first in class compound with the ability to block an important cancer growth enzyme known as geranylgeranyl transferase-1 (GGT-1). It disrupts oncogenic Ras pathways by inhibiting the activation of Rho, Rac and Ral circuits in cancer cells, leading to apoptosis (death) of cancer cells. PTX-100 is now in a Phase 1b expansion cohort study in T cell lymphomas, where it has shown encouraging efficacy signals and safety. PTX-200 is a novel PH domain inhibitor that inhibits an important tumour survival pathway known as Akt, which plays a key role in the development of many cancers, including breast and ovarian cancer, as well as leukemia. Unlike other drug candidates that target Akt inhibition, PTX-200 has a novel mechanism of action that specifically inhibits Akt without non-specific kinase inhibition effects.