Biopontis Alliance Rare Disease Foundation Inc

THE BIOPONTIS ALLIANCE RARE DISEASE FOUNDATION IS A NONPROFIT CORPORATION THAT BRINGS TOGETHER RARE DISEASE PATIENT ADVOCACY ORGANIZATIONS, ACADEMIC RESEARCH CENTERS, CONTRACT RESEARCH COMPANIES, AND THE BIOMEDICAL INDUSTRY TO TRANSLATE RESEARCH INTO POTENTIAL TREATMENTS BY FOCUSING ON THE EARLIEST STAGES OF PRODUCT DEVELOPMENT.

ALLIANCES AND PARTNERSHIPS -- DURING 2019, THE BIOPONTIS ALLIANCE ORGANIZATION MAINLY WORKED ON THE REALIZATION OF THE PROMISE OF OUR FIRST STRATEGIC PARTNERSHIP, WHICH IS THE PROJECT WITH VIB (FLEMISH INSTITUTE FOR BIOTECHNOLOGY, BELGIUM) AND THE UNIVERSITY OF ANTWERP, BELGIUM ON A CLINICAL VARIANT OF THE PERIPHERAL NEUROPATHY CHARCOT-MARIE- TOOTH DISEASE. TOGETHER WITH OUR PARTNERS, WE HAVE WORKED ON THE IDENTIFICATION OF COMPOUNDS FROM A LARGE COMPOUND BANK, OWNED BY OUR PARTNERS, THROUGH ASSAY DEVELOPMENT AND HIGH-THROUGHPUT SCREENING TECHNIQUES. THAT WORK LED TO SEVERAL 'HITS', IN OTHER WORDS, SEVERAL COMPOUNDS THAT MAY, AFTER FURTHER VALIDATION AND OPTIMIZATION, POTENTIALLY BE USED FOR THE DEVELOPMENT OF A THERAPY. THIS POSITIVE DEVELOPMENT NOT ONLY VALIDATES THE OVERALL APPROACH TAKEN BY THE BIOPONTIS ALLIANCE, BUT ALSO PROVIDES A PROMISING PLATFORM FOR FURTHER WORK TOWARDS A POTENTIAL THERAPY FOR THE INDICATED RARE DISEASE. WE ALSO WORKED WITH OUR PARTNERS ON THE DEFINITION OF THE NEXT STEPS NEEDED TOWARDS THERAPY DEVELOPMENT.

ALLIANCES AND PARTNERSHIPS -- OVER 2019, THE BIOPONTIS ALLIANCE ORGANIZATION HAS MAINLY WORKED ON THE REALIZATION OF THE PROMISE OF OUR FIRST STRATEGIC PARTNERSHIP, WHICH IS THE PROJECT WITH VIB (FLEMISH INSTITUTE FOR BIOTECHNOLOGY, BELGIUM) AND THE UNIVERSITY OF ANTWERP, BELGIUM ON A CLINICAL VARIANT OF THE PERIPHERAL NEUROPATHY CHARCOT-MARIE- TOOTH DISEASE. TOGETHER WITH OUR PARTNERS, WE HAVE WORKED ON THE IDENTIFICATION OF COMPOUNDS FROM A LARGE COMPOUND BANK, OWNED BY OUR PARTNERS, THROUGH ASSAY DEVELOPMENT AND HIGH-THROUGHPUT SCREENING TECHNIQUES. THAT WORK LED TO SEVERAL 'HITS', IN OTHER WORDS, SEVERAL COMPOUNDS THAT MAY, AFTER FURTHER VALIDATION AND OPTIMIZATION, POTENTIALLY BE USED FOR THE DEVELOPMENT OF A THERAPY. THIS POSITIVE DEVELOPMENT NOT ONLY VALIDATES THE OVERALL APPROACH TAKEN BY THE BIOPONTIS ALLIANCE, BUT ALSO PROVIDES A PROMISING PLATFORM FOR FURTHER WORK TOWARDS A POTENTIAL THERAPY FOR THE INDICATED RARE DISEASE. WE ALSO WORKED WITH OUR PARTNERS ON THE DEFINITION OF THE NEXT STEPS NEEDED TOWARDS THERAPY DEVELOPMENT. IN ADDITION, WE HAVE WORKED ON FURTHERING OUR PROJECT WITH MASS GENERAL HOSPITAL IN BOSTON, MA, BUT UNFORTUNATELY, WE HAVE NOT BEEN ABLE TO FIND THE NECESSARY FUNDING TO CONTINUE THIS PROJECT. FURTHERMORE, WE HAVE WORKED ON THE DUE DILIGENCE OF SEVERAL PROMISING CANDIDATES FOR NEW PROJECTS WITH CANDIDATE PARTNERS IN THE USA AND IN EUROPE, INCLUDING FOR DISEASES SUCH AS ALTERNATING HEMIPLEGIA, AND ATAXIA'S. FINALLY, WE HAVE CONTINUED TO PROMOTE THE TOOLS WE HAVE DEVELOPED TO INTEGRATE PATIENTS' VOICES INTO THE DRUG DISCOVERY/DEVELOPMENT PROCESS FOR RARE DISEASES THROUGH ACTIVE PARTNERSHIP WITH OTHER RARE DISEASE NON-PROFIT ORGANIZATIONS.