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TEXAD – Tailoring EXosomes for Autoimmune Diseases

Periodic Reporting for period 1 - TEXAD (TEXAD – Tailoring EXosomes for Autoimmune Diseases)

Reporting period: 2019-12-30 to 2020-12-29

Autoimmune diseases (AD) are among the most prevalent group of diseases, with 23 million patients only in US. These diseases are chronic and often life-threatening, and according to AARDA (American Autoimmune Related Diseases Association), they are one of the top 10 leading causes of death in female children and women in all age groups up to 64 years of age. Current available treatments comprise chemical-based drugs such as immunosuppressant, corticosteroids and NSAIDs. Although these products are effective in controlling AD symptoms, they significantly increase the risk of getting severe infections, or developing cancer, due to the constant artificial shut down of the immune system. The long-term consequences of their use are yet to be fully understood.
Exogenus is a spin-out company dedicated to the development of new therapeutic tools based in exosomes for diseases of high unmet needs. Our proprietary therapeutic tool are exosomes secreted by Umbilical Cord Blood (UCB) cells, secreted and purified in optimized conditions. Our vesicles have anti-inflammatory and immunomodulatory properties and can act as natural modulators of the immune system towards an immunotolerant state. While there are more than 80 different ADs, and limited solutions in the market with acceptable secondary effects, Exogenus’ vesicles can be a game-changer, because they naturally rebalance the immune system and increase its tolerability in the context of an AD crisis and can be tailored for specific ADs. It is urgent for Exogenus to identify the right target diseases and define the development path, and TEXAD project was designed to address these specific aims: a) decide which ADs to pursue as potential new markets and therapeutic areas; b) implementing meaningful Proof-of-Concept (POC) studies to demonstrate the products’ therapeutic potential for the target ADs; c) develop strong Target Product Profiles (TPP), meeting the needs of the new markets; d) define the roadmap for future development of up to three products for ADs; and e) design a new Innovation Project to achieve the POC in humans for one selected AD.
Tailoring EXosomes for Autoimmune Diseases (TEXAD) was a one-year project intended to advance the field of extracellular vesicles as a therapeutic alternative for autoimmune diseases with high unmet needs. Exogenus developed a product (Exo-101), based on vesicles released by mononuclear cells from umbilical cord blood, which shows promise for regenerative medicine (namely healing of chronic wounds) and inflammatory diseases. The aim of this project was to study the latter in greater detail, by determining which disease or set of diseases would be most likely to benefit from Exo-101 treatment, and conduct pre-clinical proof-of-concept (POC) assays to support the drug’s development into clinical stage. An Innovation Associate (IA), with previous work in the field of autoimmune diseases, was hired to help guide and conduct this process. Due to the SARS-CoV-2 pandemic, Exogenus was faced with many challenges to implement the project, especially the difficulty to implement the POC studies on a timely manner. Faced with these limitations, and acknowledging the health threat imposed by the pandemic, Exogenus decided to explore the potential of Exo-101 for the treatment of lung inflammation, leveraging on the data demonstrating its anti-inflammatory and pro-regenerative effects. As such, the original plan suffered minor changes, to better fit with the new target disease. The IA hired under TEXAD was instrumental to the company during the past year, assisting in the re-definition of the Target Product Profile, and participating in fundraising, which in turn allowed Exogenus Therapeutics to retain her as an employee.
The IA contributed to the preparation of three scientific publications, one of the papers was recently published in Stem Cells Translational Medicine (Cardoso RMS and Rodrigues SC et al, 2021), and the other two are about to be submitted. During the course of the TEXAD project, participation in scientific and industry-led events was instrumental to position the company in the field of inflammatory diseases.
TEXAD was thought to be impactful in two ways: 1) by potentially providing a therapeutic alternative to patients with autoimmune conditions, who often lack effective and safe treatments (including long-term treatments); and 2) by introducing to the medical community and to the market a new class of therapeutics that, if proven effective and safe, can fundamentally change how therapies are perceived – from managing symptoms to re establishing balance in the organism, and from targeting single molecules and pathways to using a cocktail of biologic messengers to deal with complex disease mechanisms.
Due to the limitations imposed by Covid-19 pandemic to the TEXAD project and to the society, and in light of the anti-inflammatory and immunomodulatory properties of Exo-101, Exogenus decided to explore its application to diseases with lung inflammation (focusing first in ARDS - Acute Respiratory Distress Syndrome, a common hallmark of Covid-19 and many other infectious and non-infectious diseases). The impacts of this alternative application are huge: the high rates of infection seen with SARS-CoV-2 mean that a large portion of the world’s population will be affected by the virus, a phenomenon that is expected to contribute to an increase in chronic lung illness. While the priority is to stop the spreading of the virus, namely with the development of vaccines and adoption of social measures, there is a high need for treatments that prevent disease progression to ARDS, and thus prevent lung damage and death. Having already attracted governmental funds to explore the application of Exo-101 to ARDS, the company is preparing to reach clinical studies in 2022.