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EXTEND Technology Transfer Hub. (4/12/23). "Press Release: EXTEND Approve Investmnet on Two Projects from San Raffaele Hospital to Tackle Devastating Rare Diseases". Rome.

Region Region Milano (Milan)
  Country Italy
Organisations Organisation EXTEND (BRIDGE collaboration of Angelini Ventures + CDP Venture Capital + Evotec)
  Group Italy (govt)
  Organisation 2 San Raffaele Hospital (Ospedale San Raffaele, OSR)
  Group San Raffaele of Monte Tabor Foundation
Products Product business development (state/region)
  Product 2 gene therapy
Person Person Pingue, Claudia (Italy (govt) 202210 Fund Manager at CDP VC Technology Transfer Fund + President at EXTEND)
     


EXTEND, the Technology Transfer Hub supported by CDP Venture Capital Sgr, Angelini Ventures and Evotec, approved funding for two projects in the area of Rare Diseases after the first round of evaluation of proposals received during the very first time from inception from the Research Institutions currently part of the network.

The news has been also reported in a dedicated article published on 12th April in Affari e Finanza, the Economic insert of the Italian newspaper “la Repubblica”.

Each project will receive a total funding of about 1.5 Mio Euros to reach, through a milestones-based path, the main inflection point, consisting in the formation of a dedicated Start-up company ensuring the progression of the asset into clinical development.

The projects, originated from the scientific effort of two Teams at Ospedale San Raffaele (Milan), one of the Research Centres forming the current scientific network of EXTEND, are designed to develop innovative therapies to treat two devastating Rare Diseases, namely Rett Syndrome and Facioscapulohumeral Muscular Dystrophy (FSHD), for which there is currently no cure or even disease modifying treatment available today.

Rett syndrome is the second most important syndrome after the Down syndrome. The project led by Dr. Vania Broccoli, Group leader of Stem Cells and Neurogenesis Unit at Ospedale San Raffaele, is developing a gene therapy approach to restore the expression of a fully functional protein codified by the MeCP2 gene, which is mutated in the subjects suffering from the disease. This approach has proven to be successful pre-clinically, as demonstrated by in vitro, ex-vivo and in vivo models data.

The Team of San Raffaele led by Dr. Davide Gabellini, Group Leader of Gene Expression and Muscular Dystrophy Unit, identified a specific protein which binds to that responsible for the development of Facioscapulohumeral Muscular Dystrophy by blocking its toxic effect. Positive data in animal models have been generated and the budget received will allow an effective and efficient project progression towards the preclinical development thanks to the detailed project plan designed in collaboration of the Team of experts at Evotec.

Claudia Pingue, leading the Technology Transfer Fund at CDP and Chairman of EXTEND, commented: “Although defined “rare”, these diseases affect millions of people worldwide and Regulatory Authorities are willing to activate a Fast Track path to approval, thus representing a clear opportunity beyond the current effort. By EXTEND our mission is to fill the gaps that preclude many novel ideas to emerge and fund the most promising projects from Italian Research Institutions since the very early stages”.

   
Record changed: 2024-03-14

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