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Info_cf_mall_market trials

Medical Sciences / Pharmaceuticals | Technology Offer Early Therapy for Cystic Fibrosis (1)
Advantages at a Glance
Neonatal Screening for CF
Early treatment with sodium channel blocker The earlier the CF diagnosis occurs, the earlier treatment can commence and the better are the chances of the patient. Following this principle, the • Reduces the thick mucus in the lung, improves Cystic Fibrosis Center at the Heidelberg University Clinic under the leadership of Professor Marcus Mall respiration, reduces lung infections and prolongs survival, because the accelerated salt and water has successfully applied a new, simple and safe absorption in the air way is effectively suppressed.
biochemical CF-test (Sommerburg et al., JIMD, 2010) Simplified registration requirements due to the and is actively campaigning in support of a regular neonatal screening program for the early detection of • Tested active substance with novel application We are searching for:
Innovative Therapy for CF
Licensees, Funding or Cooperative Partners for the In parallel with the development of the screening test, further development and clinical trials. Professor Mall and his team developed a novel preventive therapeutic strategy (Zhou et al., AJRCCM, 2008) for the early treatment of CF before any disease- Application
specific symptoms can be detected. The early therapy with amiloride commencing during the first days after Cystic Fibrosis (CF) is an inherited, non-curable and birth prevents the development of CF in animal trials. life-limiting autosomal recessive metabolic disease of Further information on this is available on page 2. the lung. Other organs can also be affected, e.g. Over 5% of the European population is carriers of the Patent Situation of the new CF Therapy
CF mutation; worldwide there are some 60,000 A European Patent (EP211440B1) has been granted people suffering from CF. Between 300 and 400 and validated in Germany, Great Britain and France. A babies with the disease are born each year in Current State of Technology
At present, only the symptoms of CF are being treated, not the actual cause. Physical therapies in TLB – Agency for the Management of
combination with mucus solvents help patients to Patents and Their Commercialisation
remove mucus from the lung. Antibiotics are used As partners of the scientific community as well as against bacterial infections of the lung. Life industry, we have been offering for more than a expectancy under current therapeutic conditions is decade customized services in invention and patent management. We search and identify world-wide potential buyers or licensees for our market-focused inventions. Our objective is to create commercial value Market Potential
The CF market is estimated to grow positively up to 2017 with a compound annual growth rate of 8.2% Your Contact within TLB
and a market value greater than US$ 2,140. The market is ready for new pharmaceuticals which Tel.: +49-721 79004-0, Fax: +49-721 79004-79 Copyright 2010 Technologie-Lizenz-Büro (TLB) der Baden-Württembergischen Hochschulen GmbH Medical Sciences / Pharmaceuticals | Technology Offer Early Therapy for Cystic Fibrosis (2)
Results of the innovative CF Therapy
Preparation of Clinical Studies
In animal trials, transgenic CF-mice (Mall et al., Nat. Clinical trials of amiloride as a preventative early Med., 2004) were treated successfully with amiloride intervention therapy against CF are already in the via nasal application, provided they were treated prior planning phase. The start-up financing of the necessary toxicological investigations and the clinical following birth (Zhou et al., AJRCCM, 2008): pilot studies of amiloride, including the request for Amiloride
Amiloride is registered as a diuretic and was introduced to the market by Merck Sharp & Dohme in 1967. Amiloride acts as an epithelial sodium channel (ENaC) blocker but achieved no positive effect in inhalation studies with patients already exhibiting CF- T ime (days )
Genetics

Figure 1:
Early treatment with amiloride immediately after
The CF mutation is located in the CFTR gene, which birth significantly reduces mortality in CF-transgenic mice encodes a transmembrane protein acting as a chloride channel and regulator of epithelial sodium channels (ENaC). In the airways, CFTR is primarily In addition, the early amiloride therapy positively responsible for the coordination of secretion and affects the mucus membrane and the formation of absorption of salt and water to maintain adequate mucus (Fig. 2) as well as the goblet cell metaplasia, hydration of airway surfaces and mucus, which is the chronic inflammation and the formation of lung required for normal mucus clearance from the lungs. emphysema. These results demonstrate that the preventive treatment with amiloride can be an wt + control CF + control CF + amiloride TLB – Agency for the Management of
Patents and Their Commercialisation
As partners of the scientific community as well as
industry, we have been offering for more than a
decade customized services in invention and patent
management. We search and identify world-wide
potential buyers or licensees for our market-focused Figure 2: The congestion of the lung with mucus in CF-
inventions. Our objective is to create commercial value transgenic mice can be effectively treated by an early amiloride therapy commencing in the first few days after birth up to 14 days after birth. AB-PAS staining. Scale bar = Your Contact within TLB
Email: uweirich@tlb.de Tel.: +49-721 79004-0, Fax: +49-721 79004-79 Copyright 2010 Technologie-Lizenz-Büro (TLB) der Baden-Württembergischen Hochschulen GmbH

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