List of Cell Therapies Companies in Australia - 13

Arovella Therapeutics (ASX: ALA) is the only Australian biotech developing an iNKT cell therapy platform to create therapies for people to live longer, healthier lives.

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.

Carina Biotech An Australian clinical stage immunotherapy company established to research and develop chimeric antigen receptor T cell (CAR-T) therapies to treat solid cancers. Carina are working towards producing broad-spectrum CAR-T therapies that can be used to treat multiple solid cancers yet are patient-specific and result in little, if any, off-cancer damage. Using its proprietary platforms, Carina is also developing technologies to improve access to, and infiltration of, solid cancers, and to enhance CAR-T cell manufacturing. We are working to… 1. Expand the clinical indications for T cell therapies 2. Improve the commercial viability of T cell therapies by developing supporting technologies that make T cell therapies more effective or economically viable Headquartered in Adelaide, South Australia, Carina has strong T cell R&D capability across a network of research providers and collaborators including leading scientists at the UniSA, the Women's & Children's Hospital in Adelaide, the University of Adelaide, the Royal Prince Alfred Hospital in NSW and the Seattle Children's Hospital in the United States.

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.

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

Currus Biologics is utilising its proprietary BEAT technology to develop CAR-T cell therapies for the treatment of solid tumour cancers, traditionally difficult to treat with current CAR-T cell therapies. Currus Biologics’ proprietary Bispecific Engagers of Antigen Presenting Cells and T cells (BEAT) technology overcomes many of the challenges presented when treating solid tumours with traditional CAR-T cell therapy, demonstrating CAR-T cell proliferation and persistence, CAR-T cell trafficking to the tumour and immunological memory extending to additional antigens. Currus Biologics is a privately held biotechnology company based in Melbourne, Australia.

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.

Mesoblast (ASX:MSB; Nasdaq:MESO) is developing and commercializing allogeneic cellular medicines to treat serious and life-threatening inflammatory diseases with significant, unmet medical needs. The Company's Phase 3 off-the-shelf mesenchymal lineage cell product candidates are: • RYONCIL™ (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GVHD) • Remestemcel-L for moderate to severe acute respiratory distress syndrome (ARDS) due to COVID-19 infection • REVASCOR® for advanced chronic heart failure, and • MPC-06-ID for chronic low back pain due to degenerative disc disease. The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast’s approach to product development is to ensure rigorous scientific investigations are performed with well-characterized cell populations in order to understand mechanisms of action for each potential indication. Extensive preclinical translational studies guide clinical trials that are structured to meet stringent safety and efficacy criteria set by international regulatory agencies. All trials are conducted under the continuing review of independent Data Safety Monitoring Boards comprised of independent medical experts and statisticians. Mesoblast has an extensive patent portfolio comprising approximately 1,000 patents and patent applications with protection extending through 2040 in all major markets. This intellectual property portfolio covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells. The Company believes this patent estate provides strong global protection.

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.

S2N’s vision is a world where neuronal loss no longer means loss. Our regenerative medicine technology starts with the human hair follicle and ends with production of millions of pure neurons, all gene modification free. We aim to transform health, science and society.

VivaZome Therapeutics Pty Ltd (ABN 59 602 230 964) is a privately-held Australian biotech company, with operational headquarters at the La Trobe University in Melbourne, Australia. VivaZome aims to develop and commercialise customised exosome-based therapies for debilitating and life-threatening disorders, with a focus on neurological disorders, retinal disease and ischaemic conditions. VivaZome is developing new technologies, intellectual property and manufacturing processes that apply generically to exosome therapies. These will underpin the Company’s development of specific exosome products for its target indications. The VivaZome team has extensive expertise in the development and commercialisation of biological therapies, together with a wide network of expert contacts in the Australian and global biotech communities. VivaZome acknowledges the support of the Department of Industry and Science through the CRC-P program, and the contribution of its CRC-P partners: Australian National University, the University of Queensland, La Trobe University, Cytiva and SeerPharma Pty Ltd.