OrgenesisInc () is a global biotech groupfocused 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. It's Cell and Gene Therapy Biotech (CGT) 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 Orgenesis is able to in-license technologies or advanced therapy medicinal products (ATMPs) and co-develop them with its partners.

On February 11, 202, Orgenesis 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 the worlds top drugmakers.

Caplan acquired Masthercell in March 2015 and grew the CDMO segment revenue from a run-rate of just $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 theMasthercell sale proceeds to grow the groups evolving point-of-care cell therapy business and develop advanced therapy medicinal products.

Orgenesis posted strong financial results for the year ended December 31, 2019, driven by its rapidly expanding point-of-care cellular therapy platform and new collaborations.

Gross profit jumped by 92% to $15 million in fiscal 2019, compared to $7.8 million for fiscal 2018. Gross profit soared 92% to $15 million in FY2019, compared to $7.8 million for FY 2018. The companys growth strategy is working as the point-of-care platform generated $3.1 million in sales, compared to nothing in FY 2018.

The company had cash and equivalents of $11.4 million at the end of 2019, which did not include the $127 million from the sale of its CDMO business in February.

Orgenesis is using theMasthercell sale proceeds to expand the companys point-of-care cell therapy business. 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, per year. To lower costs, Orgenesis is switching from a high-cost centralized manufacturing model to a localized point-of-care model.

The biotech is currently focused on therapies which span a wide range of treatments, such as cell-based immunotherapies, treatments for metabolic and neurodegenerative diseases and tissue regeneration.

At the start of April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a new joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous Cell and Gene Therapy 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 (mdMSC) as a source of exosomes and various other cellular products. Orgenesis and RevaTis are hoping to build on RevaTiss early success in animals to develop therapies and advance human trials using 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 ultracentrifugation or 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.

Orgenesisrecently completed the acquisition of Tamir Biotechnology and its broad spectrum antiviral platform, ranpirnase in a cash and stock deal for roughly $21 million. The company will use ranpirnase to target human papillomavirus (HPV), which causes genital warts. A topical version of ranpirnase was evaluated in Phase 1/2 clinical trials and it demonstrated a clear clinical effect and a good safety profile, the group noted.

Going forward, Orgenesis said it plans to move the program through a Phase 2b trial in the US. Additionally, the company will undergo a new clinical trial targeting anal dysplasia, a precusor to anal cancer driven by the HPV virus. Ranpirnase is a member of the superfamily of enzymes that cause the degradation of RNA and mediate biological activities, including cell death.

Orgenesis CEO Vered Caplan noted that the company has dramatically transformed since the beginning of 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 onTwitter:@UttaraProactive

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