The product refers to a novel add-on treatment for Glaucoma and other ophthalmological diseases based on natural growth factors from cord blood serum (CBS) and capable of slowing down disease progression through neuroprotection, therefore delaying vision loss. The product can be lyophilized and then reconstituted and administered as a simple eye collyrium, eye drops or eye gel, hereby allowing non-invasive, modular (in terms of dose/frequency of treatment) and potentially setting-neutral administration of the treatment to a large population at different conditions’ stage, with maximum impact at early/moderate stage.
Patent status
Granted
Priority number
102017000066486
Priority Date
15/06/2017
License
EUROPE
Market
Glaucoma is currently a highly widespread and sadly irreversible condition whose incidence is increasing worldwide, with 9.6 million patients in 2020 and an estimation of 111.8 million patients in 2040 (7.85 million in Europe). As it normally progresses towards blindness and is currently without a cure, potentially all patients (and mostly those at early/mid stage of the condition) could benefit of a treatment able to slow down progression, especially if this is non-invasive, 100% natural and lacking any side effect. A primary market segment to target would include people with glaucoma familiarity, early/moderate stage, uncontrolled Intra Ocular Pression (IOP), bilateral damage, and at risk of rapid progression. This segment alone represents 10% of the market corresponding to 11 million of people worldwide in 2040.
Problem
Glaucoma is a leading cause of irreversible blindness worldwide and primary open-angle glaucoma (POAG) is a major contributor. Progressive neurodegeneration of the optic nerve and the loss of retinal ganglion cells is a hallmark of glaucoma, but still etiology is unknown, and treatment is lacking. As mentioned incidence is increasing with associated high medical and social costs, both increasing proportionally to disease progression. As glaucoma is currently without a cure, there is a strong need is to slow down the progression of the disease and treat it at an early stage with effective though minimally invasive and largely available treatments. Glaucoma is a “deceptive” disease, which has no symptoms nor is it detectable at its earliest beginning, which is why it is of absolute importance to combine controls with the availability and use of treatments that act to slow down the progression starting from the very first diagnosis.
Current Technology Limits
For primary open angle glaucoma (POAG), most of the treatments currently focus on reducing aqueous humor formation, enhancing uveoscleral or conventional outflow, by lowering intraocular pressure (IOP) through medical or surgical means. However, limiting IOP levels only does not arrest of the progression of the glaucomatous damage (due to the fact that glaucomatous pathology are not exclusively due to an increase in IOP) and do not prevent patients from progressive vision loss. Therefore, other strategies are needed to reduce or reverse the progressive neurodegeneration.
Recent pathophysiological acquisitions have directed international scientific research to explore and study molecules capable of exerting a potential neuroprotective or neuro- empowering effect on retinal ganglion cells, the optic nerve and the central optic pathways, with a direct mechanism of action on the nervous structures and therefore not necessarily linked to the hypotonizing effect. Currently at the level of neuroprotection there are several molecules being tested. Some examples: substances with antioxidant, anti-apoptotic action, and substances able to reduce glutamate excitotoxicity
However, in current clinical practice neuroprotection is currently offered to patients in combination with IOP treatment only according to clinician’s judgment and mostly through administration of nutraceuticals, but significant evidence on their validity and efficacy is missing.
An effective and clinically validated neuro protective treatment accompanying IOP treatment and intervening at very early glaucoma stage, especially on young patients or patients with glaucoma familiarity would substantially change the impact of the disease.
Killer Application
The main application is a neuroprotective, CBS-based add-on treatment for glaucoma based on eye-drops which can be administered to patients in different amount/doses/treatment’ settings depending on conditions’ stage, protocols required, frequency of treatment thanks to lyophilized stocks.
Our technology and Solutions
This technology represents the first add-on neuroprotective treatment for glaucoma which brings a mix of neuroprotective factors as is pools different growth factors from cord blood serum, hereby amplifying its neuroprotective effect. The preparation of the CBS based eye-drops include production of pools of blood components derived from various cord blood donations which are unsuitable for autologous transplantation of hematopoietic stem cells (HSCs) and clinically validated, characterized in terms of Growth Factors and Cytokines, and subsequently diluted with sterile physiological solution, filtered and dispensed into dedicated devices. Conservation currently takes place at -80°C for a maximum period of two years. The whole process is performed in a class A environment (laminar flow hood) with a D background.
The product can also be successfully lyophilized, with all the associated advantages in terms of shelf-life, storage, transport and distribution together with rapid. rapid reconstitution of the product, preservation of the characteristics of the starting product (the levels of neurotrophic and neuroprotective growth factors) and possibility of dosing the neurotrophic and neuroprotective growth factors in the final preparation and being able to dispense it with dosages calibrated on the basis of clinical parameters or administration frequencies
Lyophilization is widely widely used for plasma derivatives, vaccines, antibiotics, biotechnological products.To date however, there are no alternative solutions with such a high degree of feasibility for the dispensing of blood components, other than the form of liquid storage at a temperature of -80°C.
Advantages
Roadmap
The treatment has already been tested in vivo on 10 patients and a following round of trial is currently ongoing with another 10 (Phase I/II). Given the peculiarity of legislations and registration procedures on blood-based product across countries, it is important to detect the most appropriate regulatory framework for a proper «go to market» strategy.
The roadmap currently includes:
TRL
Il team
