2019 Award


The International Prize for Scientific Research Arrigo Recordati 2019

In the last ten years Recordati made the Rare Diseases field a health priority and increased its commitment worldwide through its dedicated subsidiaries, Orphan Europe and Recordati Rare Diseases, to research, develop and market a number of treatments for orphan diseases.
To reflect its commitment, Recordati decided that the 2019 Edition of The International Prize for Scientific Research Arrigo Recordati will be dedicated to the promotion and recognition of excellence in orphan disease treatment research.
International projects within the area of rare/orphan disease treatments in all therapeutic areas (except for oncology, hematology and immunology) are eligible to apply as long the disease concerned has a prevalence of not more than 1 in 2000.
The 2019 Award is open to researchers of all nationalities who are not in any way directly affiliated with pharmaceutical or medical device companies.
The 2019 Award will be given in recognition of ongoing research projects, for which preliminary results, such as proof-of-concept and proof-of-principle, have already been obtained.

The winner project will be announced during an Award Ceremony at the Society for the Study of Inborn Errors of Metabolism Congress – (2019 SSIEM, Rotterdam, 2nd-6th September 2019).
 
The winner project will receive a research grant of € 100,000.
 


THE REVIEW COMMITTEE

For the ninth edition of the Award, the Review Committee is composed of:

 


Chairman: Robert J. Desnick Ph.D, M.D., Dean for Genetic and Genomic Medicine, Professor and Chair Emeritus Department of Genetic & Genomic Sciences, Mount Sinai School of Medicine, New York, USA.


Robert J. Desnick Ph.D, M.D.

Robert J. Desnick Ph.D, M.D.

Icahn School of Medicine at Mount Sinai
Mount Sinai Health System, New York, NY

Robert J. Desnick is Dean for Genetic and Genomic Medicine and Professor and Chairman Emeritus of the Department of Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai. In 1977, he joined the Mount Sinai faculty as the Arthur J. and Nellie Z. Cohen Professor of Pediatrics and Genetics, and Chief of Medical and Molecular Genetics. From 1993- 2011, he was the first Chairman of the Department of Genetics and Genomic Sciences at Mount Sinai. In 2011 he became the Dean for Genetics and Genomic Medicine.

Dr. Desnick’s research interests include lysosomal storage diseases (LSDs) and the inborn errors of heme biosynthesis, the porphyrias, and in particular, their treatment. His research efforts led to the Federal Drugs Administration (FDA) - and European Medicine Agency (EMA) - approval of enzyme replacement therapy (ERT) for Fabry disease (Fabrazyme) and on-going ERT clinical trials (FDA “Breakthrough” status) for NiemannPick B disease, both in partnership with Genzyme. In addition, he was a scientific founder of Amicus Therapeutics (NASDAQ; FOLD), which is developing oral pharmacologic chaperone therapy for Fabry disease (EMA-approved in 2016), Pompe disease, and other disorders. Currently, his laboratory is using gene editing technology to engineer gene therapy in the mouse model of Fabry disease with Sangamo Therapeutics.

For the porphyrias, he co-developed with Alnylam Pharmaceuticals an RNAi therapy for the acute hepatic porphyrias, performed the preclinical studies, and co-designed the ongoing clinical trials. He also served as Principal Investigator for Clinuvel Pharmaceuticals’ Phase 2 and 3 multisite clinical trials of afamelanotide, a “first-in-class” synthetic peptide for the treatment of the Erythropoietic Porphyrias, which was recently EMA-approved and pending FDA approval. He also served as the Chairman of the Scientific Advisory Committee (SAC) of Synageva Biopharma and currently serves as SAC Chair for Kiniksa Pharmaceuticals. In addition, his research includes genomics, pharmacogenomics, and personalized medicine. He has published over 740 research papers and chapters, including nine edited books. He is an elected Fellow of the American Association for the Advancement of Science and an elected member of the National Academy of Medicine.

 



Professor Francesco Emma, Director of the Division of Pediatric Nephrology, Chief of the Department of Pediatric Subspecialties, IRCCS Children’s Hospital Bambino Gesù, Rome, Italy.


Francesco Emma M.D.

Francesco Emma M.D.

Director of the Division of Pediatric Nephrology
Chief of the Department of Pediatric
Subspecialties, IRCCS Children’s Hospital
Bambino Gesù, Rome, Italy

Francesco Emma received his medical degree from the Catholic University of Louvain, Brussels, Belgium, where he specialized in Pediatrics. He subsequently completed his training in Pediatric Nephrology at Boston Children’s Hospital, Harvard Medical School, and moved to the Bambino Gesù Children’s Hospital in Rome as a staff member in the Division of Nephrology and Dialysis. There, he served as the Head of the Rare Kidney Disease Clinic before being appointed Head of the Pediatric Nephrology Division in 2005, and has set-up the Nephrology Research Laboratory. He currently holds the position of Head of the Department of Pediatric Subspecialties.

Dr. Emma’s primary research interests lie in rare renal diseases, in particular in cystinosis and nephrotic syndrome. He has authored more than 160 research articles in peerreviewed journals and is the author of several textbook chapters. He has also served in the editorial board of several scientific journals, including Pediatric Nephrology, the Journal of Nephrology and Nephrology Dialysis and Transplantation. He is an editor for the textbook “Pediatric Nephrology”.

Among his other appointments, he is the current chair of the Working Group on Inherited Renal Disorders of the European Society of Pediatric Nephrology (ESPN), has been pastsecretary of the Working Group on Inherited Kidney diseases of the European Renal Association - European Dialysis and Transplant Association (ERA-EDTA), the past Chair of the Teaching Course Committee of the International Society of Pediatric Nephrology and the past-President of the Italian Society of Pediatric Nephrology. 

 



Professor Norio Sakai M.D., Ph.D., Child Healthcare and Genetic Science Laboratory, Division of Health Science, Osaka University Graduate School of Medicine, Osaka, Japan.


Norio Sakai M.D., Ph.D.

Norio Sakai M.D., Ph.D.

Child Healthcare and Genetic Science
Laboratory, Division of Health Science,
Osaka University, Japan

Norio Sakai is a Professor at the Osaka University Graduate School of Medicine, in the Child Healthcare and Genetic Science Laboratory of the Health Science Division, in Osaka. Dr. Sakai graduated from the Department of Astronomy at Tokyo University in 1982 and the Osaka University Graduate School of Medicine in 1987. He completed his clinical training as a residency in 1990. He received a Ph.D from the Osaka University Graduate School of Medicine in 1994 with a paper on molecular cloning for Krabbe disease (Krabbe disease: Isolation and characterization of a full-length cDNA for human galactocerebrosidase).

He worked as a Research Fellow in the Department of Environmental Medicine, Res. Inst. Osaka Medical Center for Maternal and Child Health, Osaka for two years and moved to GSF-Institute for Mammalian Genetics in Germany. He returned to Japan in 1998 to the Department of Paediatrics at the Osaka University School of Medicine and was appointed to the position of Assistant Professor in 2005, later working as Associate Professor from 2009. He led the clinical and research group for inborn errors of metabolic disease and medical genetics in the Pediatrics Department.

Dr. Sakai has published more than 60 articles in peer-reviewed international journals and has received several awards for his research on molecular analysis for inborn errors of metabolic diseases.
His clinical work focuses on the clinical treatment of lysosomal diseases with advanced methods including enzyme replacement therapy and hematopoietic cell transplantation. His more recent research has focused on disclosing the basic molecular pathology of lysosomal diseases and developing new treatments including chaperone therapy. 

Although Recordati recognizes the value of fundamental research aimed at unraveling the disease pathophysiology/etiology, projects on orphan diseases which do not make use (or assess the potential) of an experimental treatment for a rare disease are not eligible for the Award. 
 
If the experimental treatment is based on a pre-existing treatment, the project is only eligible if the treatment considered is being repurposed in another indication or if its formulation/administration changed in a major way, in order to improve treatment quality, compliance, efficacy, safety or any other additional patient’s benefits.  
 
Both projects concerning medicinal products and medical devices are eligible for the Award as long as the disease treated is rare.

Intellectual Property Rights, when present, will be safe-guarded.

 



THE REVIEW COMMITTEE
 

 


2019 AWARD CEREMONY

The winner project will be announced during an Award Ceremony at the Society for the Study of Inborn Errors of Metabolism Congress – (2019 SSIEM, Rotterdam, 2nd-6th September 2019).
The winner project will receive a research grant of € 100,000.
There will be no University or Institutional indirect costs as a condition for this Award.


NOMINATIONS PROCEDURE

Nominations procedure will follow a two-step selection approach:
 
1) Preselection: from 10th September 2018 to 15th December 2018

  • Applications will be processed solely on-line and must be submitted in English.

  • All applications submitted in the preselection period and which will satisfy the eligibility criteria (see p 3) will be considered.

  • Applicants should submit an initial proposal composed of a Summary Page (see p 4) and a Letter of Intent (see p 5) describing the proposed project in 1200 words (plus 1 page for figures and/or tables) by uploading the requested documentation at: prize.recordati.it

  • Applicants who submit an initial proposal will receive an email confirming receipt.
    Members of the Review Committee will evaluate all the initial proposals to determine which Applicants will be invited to submit Full Applications.


2) Full Applications: from 15th February 2019 to 30st April 2019 

Selected Applicants will be invited to submit a Full Application uploading the documentation at: prize.recordati.it 
 
All members of the Review Committee will assess and select the winning project according to the pre-specified Award criteria, quality of the research as well as the therapeutic impact of the results.

The Award has “code of conduct” guidance that ensure reviews are conducted in a fair and equitable manner. Applications in which reviewers, or institutions with which they are affiliated, have an interest, will not be considered. 

The members of the Review Committee are bound by the confidentiality rules generally applicable to such bodies.

The Review Committee’s decision is final and there is no appeal mechanism.
 
Applicants who have constructive suggestions about the review of their Applications, or the process in general, are encouraged to write to: recordatiprize2019@recordati.com


2019 AWARD CRITERIA

Eligibility criteria:

  • Applied research projects on rare disease treatment (preclinical or clinical) projects have to make use of an experimental treatment approach to be eligible

  • Ongoing projects with interim results or clear milestones in order to gather sufficient data to demonstrate the effectiveness/safety

  • If the therapeutic agent already exists, the project is only eligible if the treatment is being repurposed for another rare indication or if the treatment was reformulated in order to provide an added benefit for the patient (easier to use, better compliance, safety, efficacy) or if the treatment is being used in combination with other treatments or devices.

  • Applicant CV needs to be included.

  • Applicants have to make use of the Application Form and to comply with instructions (see p 4).

Exclusion criteria:

  • Research projects aiming at developing or characterizing new models

  • Fundamental research projects which do not assess or make use of any experimental treatment or device

  • Studies with only results obtained in vitro cellular models

  • Oncology, hematology or immunodeficiency projects

  • Projects already awarded a Prize in the past

  • Projects sponsored by other pharmaceutical or medical device companies

  • Projects completed

  • Projects without Applicant’s CV

  • Projects non-compliant with the Application Form.