Spanish biotech developing novel recombinant live biotherapeutic products for respiratory diseases is looking for SMEs with expertise in developing animal models for safety and efficacy tests, and also in the field of health treatment formulation and delivery processes to carry out the preclinical development and manufacturing/formulation studies. The company wants to submit the project to the next Eurostars call once it is published.
Chronic obstructive pulmonary disease (COPD) is a group of progressive lung diseases characterized by inflammation in the lungs, damaged lung tissue and a narrowing of the airways. COPD is the third leading cause of death worldwide (>3M of deaths/year; 5% of all deaths).
Some cases of emphysema (a chronic lung disease characterized by progressive, irreversible expansion of the alveoli with eventual destruction of alveolar tissue, causing obstruction to airflow) are caused by a genetic condition called alpha-1-antitrypsin (A1AT) deficiency, which is more prevalent in young patients (aged =45 years) and non-smokers.
A1AT (SERPINA1) is the dominant systemic serine protease inhibitor and is produced mostly by hepatocytes. A1AT’s preferred target is Neutrophil Elastase (NE), a protease released by activated neutrophils during an inflammatory process. When A1AT serum levels are not sufficient to protect the alveoli from NE, this results in progressive alveolar destruction and eventually emphysema.
Treatment options of COPD include inhaled bronchodilators and steroids, pulmonary rehabilitation, long-term oxygen therapy, antibiotics and/or steroids during exacerbations. A1AT deficient patients are usually offered the same treatment options as other COPD patients. Although intravenous administration of recombinant A1AT has been tested in the clinic, this intervention did not lead to a significant benefit on pulmonary function. However, a more recent trial provided evidence that A1AT therapy slows the progression of lung destruction.
Higher local A1AT levels in the airway epithelial lining fluid have been achieved via inhalation route . However, a Phase II/III study of inhaled human A1AT did not demonstrate changes in time to exacerbation of symptoms.
Alternatively, local production in the lung of A1AT by a living system (i.e. bacteria) represents an attractive approach to reduce the production costs and possible undesired effects associated with systemic administration. For this reason, the company wants to engineer a live biotherapeutic product that expresses A1AT and other factors that promote tissue regeneration. The company will also engineer and test a product that vaccinates against infections associated with emphysema exacerbations.
Their aim is to use EUREKA Eurostars funding (call probably in February 2021) in a 2 years project to engineer these products and carry out the relevant preclinical development and manufacturing / formulation studies.
They are seeking SME partners with expertise in developing animal models for safety and efficacy tests, and also in the field of health treatment formulation and delivery processes.
Call Deadline: not published yet
Deadline for EOI: November 30, 2020
- Specific area of activity of the partner: The company is seeking partners (SMEs) for research cooperation agreements to:
1 –Develop a murine model of emphysema caused by A1AT deficiency. It requires ability to work with Mycoplasma (BSL2) and deliver it through inhalation. This model will be used to test the efficacy and safety of our product(s). Dose/response experiments, quantification and stability of A1AT production by strain product in vivo as well as effectiveness and toxicology studies will have to be performed.
2 - Develop scaled-up fermentation and formulation/delivery processes. Requires ability to work with Mycoplasma (BSL2) and to test intra pulmonary delivery.
Under development/lab tested
Patent(s) applied for but not yet granted - Existing patent applications protect the tools used for genetic engineering of Mycoplasma, the genetic alterations of the Mycoplasma chassis strains, etc. Yhe company will apply for additional patents covering the A1AT expressing product and methods for scale up production, formulation and delivery.