Inc () is a global biotech group focused on unlocking the potential of personalized therapies and closed processing systems through its cell and gene therapy platform.

The Germantown, Maryland-based companys aim is to provide life-changing treatments at the point-of-care to patients at low cost.

The group's Cell and Gene Therapy Biotech platform has three key elements.The first revolves around point-of-care therapeutics, which consists of a pipeline of licensed cell and gene therapies and scientific knowhow. The second aspect relates to point-of-care technologies, which include a suite of in-licensed technologies engineered to create customized processing systems for affordable therapies.

Finally, the third component rests on a point-of-care network, which is a collaborative, international ecosystem of leading research institutes and hospitals committed to supplying cell and gene therapies at the patient bedside. It is an intricate web of affiliated pre-clinical and clinical-stage biopharmaceutical companies, research institutions and hospitals through which is able to in-license technologies or advance therapy medicinal products (ATMPs) and co-develop them with its partners.

On February 11, 2020, Or completed the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO), to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million.

The successful sale has spotlighted Orgenesis boss Vered Caplans considerable leadership skills. She has since been named one of the top 20 inspirational leaders in the field of advanced medicine by The Medicine Maker, which creates an annual Power List of top global drugmakers.

Caplan acquired Masthercell in March 2015 and grew the CDMO segment revenue from a run-rate of $3 million to a run-rate of around $30 million at the end of 2019, reflecting a compound annual growth rate of 59% under her leadership, and a sale price of more than five times the initial purchase price of around $25 million.

Caplan has indicated that she plans to use the Masthercell sale proceeds to grow Orgenesis's evolving point-of-care cell therapy business and develop advanced therapy medicinal products.

In November, Orgenesis posted third-quarter results that saw its revenue jump 40% year-over-year on the back of growth in its point-of-care cell and gene therapy network. For the period ended September 30, 2020, the company reported revenue of $1.7 million, compared to $1.2 million in the same period in 2019.

The company also maintained a solid balance sheet with $88.8 million in cash and equivalents at the end of September, reflecting the successful sale of its Masthercell Global CDMO.

Orgenesis is using the Masthercell sale proceeds to expand the companys point-of-care cell therapy business.

In October, the group completed the acquisition of Koligo Therapeutics Inc, a regenerative medicine company, including all of the assets of Tissue Genesis LLC. The agreed consideration terms include $15 million in shares of Orgenesis stock valued at $7 share which will be issued to Koligos accredited investors (with certain non-accredited investors to be paid solely in cash) and an assumption of $1.3 million in Koligos liabilities, estimated to be nearly all of Koligos liabilities.

Koligos management team will be joining Orgenesis to continue commercial and development activities. Koligo CEO Matthew Lehman is an accomplished executive in the biotech and regenerative medicine fields. Orgenesis believes the acquisition will help to expand its therapeutic and technology resources, while adding a highly experienced US team to help further bolster Orgenesis point-of-care network in the US.

On the therapeutics front, Orgenesis is focused on several key verticals, including cell-based cancer immunotherapies, treatments for metabolic and neurodegenerative diseases and tissue regeneration, as well as antiviral therapies. The groups near-term goal is to expand the availability of KYSLECEL from the recent Koligo acquisition. KYSLECEL, is made from a patient's own pancreatic islets the cells that make insulin to regulate blood sugar and is commercially available in the US for chronic and recurrent acute pancreatitis.

Like many companies, Orgenesis is also focused on the coronavirus (COVID-19) space. It is planning patient recruitment for a phase 2 randomized clinical trial of Koligos KT-PC-301, subject to US Food and Drug Administration (FDA) review and clearance of an investigational new drug (IND) application. KT-PC-301 is an autologous clinical development-stage cell therapy candidate for COVID-19-related Acute Respiratory Distress Syndrome, which Orgenesis acquired as part of the Koligo acquisition.

Koligos KT-PC-301 is a cell therapy that is derived from a patients own adipose (fat) tissue. A small amount of fat is collected from the patient and sent to Koligos manufacturing facility in Indiana to make KT-PC-301. The product is made within hours and sent back to the hospital for intravenous administration. KT-PC-301 contains mesenchymal stem cells, vascular endothelial cells, and immune cells which migrate to the patients lungs and other peripheral sites of inflammation. Published nonclinical and clinical evidence demonstrates that KT-PC-301 may stabilize microcirculation to improve oxygenation; maintain T and B lymphocytes to support antibody production, and induce an anti-inflammatory effect.

In addition, Orgenesis is gearing up for a Phase 2 study of Ranpirnase for the treatment of conditions caused by human papillomavirus, pending a planned IND submission to the FDA.

Meanwhile, Orgenesis has advanced the development of a cell-based vaccine platform targeting COVID-19 and other viral diseases. It has launched BioShield to set up a first line of defense against COVID-19 through the discovery of neutralizing human antibodies to contain the spread of the viral pathogen. In addition, Orgenesis has signed an agreement with Fortune 500 science giant to develop, and potentially obtain FDA approval of Ranpirnase for treating COVID-19 patients. Ranpirnase has shown preclinical antiviral activity in diseases such as influenza, Ebola, SARS, and cytomegalovirus. Orgenesis acquired the broad-spectrum Ranpirnase antiviral as a part of its $12 million cash and stock deal to acquire Tamir Biotechnology.

Orgenesis intends to leverage its network of regional partners to advance the development of its pipeline. Towards this end, its partners have committed to funding the clinical programs. In turn, Orgenesis typically grants its partners geographic rights in exchange for future royalties, and a partnership with Orgenesis to support the supply of the targeted therapies. Caplan has said that through this unique business model, Orgenesis has already signed contracts, which could generate over $40 million in revenue over the next three years, if fully realized.

There are also plans to continue to develop, license and form partnerships around a variety of point-of-care technologies to support work in areas such as tumour-infiltrating lymphocytes, CAR-T, CAR-NK, dendritic cell therapies, and mesenchymal stem cell-based therapies.

Currently, the costs of cell and gene therapies are prohibitive, as illustrated by CAR-T therapies, which cost hundreds of thousands of dollars per patient. To lower costs, Orgenesis is switching from a high-cost centralized manufacturing model to a localized point-of-care model.

In April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous CGT Biotech platform to advance clinical trials. Under the deal, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells as a source of exosomes and other cellular products. The companies are hoping to use Orgenesiss expertise and point-of-care platform, which include automated systems, 3D printing and bioreactor technologies.

Meanwhile, Orgenesis and ExcellaBio have developed a breakthrough manufacturing process for so-called bioxomes, which are synthetically made exosomes or extracellular vesicles (EVs). The latter are what transfer DNA, RNA, and proteins to other cells, thereby altering the function of targeted cells. Until now, exosome/EV production has been based on conventional, complex and costly methods of ultrafiltration. However, the two companies have demonstrated the scale-up of bioxomes through a proprietary technique, while generating consistent and repeatable results, including uniform particles sizes.

Orgenesis has added the University of California (UC), Davis to its point-of-care network. The first project with UC Davis Health is on developing a lentiviral manufacturing system which will address a significant unmet need in the market for an efficient and scalable manufacturing process.

Orgenesis CEO Vered Caplan noted that the company has dramatically transformed in 2020 helped by the Masthercell sale.

The sale was an important step in our strategy to leverage our unique capabilities that directly address the key challenges facing the cell and gene therapy industry, Caplan said in a statement.

Our goal is to transform the delivery of cell and gene therapy through our point-of-care therapeutics, technologies and network, thereby lowering costs and unlocking the power of cell and gene through a more decentralized and integrated approach, she added.

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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Orgenesis leads the charge at an exciting time for cell and gene therapy - Proactive Investors USA & Canada

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