Lumacaftor (Vx-809): Lumacaftor is yet an other product for Class 2 (mainly for DDf508 or Df508 )transfer and trafficking mutation which on in-vitro and in in – vivo is showing good results. Clinical trials phase III have been a success and is expected to go for drug approvals end of year(2014). It might hit the market anytime next year 2015. http://www.reuters.com/article/2014/05/02/vertex-idUSL2N0NN1TX20140502. Lumacaftor in combination with kalydeco is the next wonder drug. Check out the below figure to understand what Lumacaftor can make.
VX – 661 : Another next generation corrector from vertex aiming the most common mutation homozygous and heterozygous DDF508.
N30 Pharma : With their amazing products such as N91115 , Lead compound and N6022, N30pharma are talking about Lung repair CFTR correction and and Anti- inflammation. Their site is a must browse for on going events. http://www.n30pharma.com/.
Their new press release states about discovery of a efficacious CFTR corrector. Interesting part of it is that Ramakrishna B an Indian Origin
is part of the team.
Gene Therapy : CF community has lots of hopes pinned on to Gene Therapy which involves correcting the faulty gene by replacing it with therapeutic one. The carriers for the therapeutic DNA are called vectors. A full fledged research is on, to find out a best vector, viral or non viral.
PTC : PTC therapatics aims at Class I CF mutations, Non Sensical muations. Their Product Atalauren is now going through Phase 3 clinical trials . This product can be used for many other problems caused by non sensical mutation. Find more details at http://ir.ptcbio.com/releasedetail.cfm ?ReleaseID=848453
Advanced Inhalation Therapies (AIT) : The Product NOxCureCF from AIT targets the Pseudomonas and the Staphylococcus aureus bacteria, using a specialized form of inhaled Nitric Oxide (NO) . Relief from Antibiotics is definitely a news worth mention.
Pulmozyme Dornase Alfa: Genentech which manufactures Pumozyme from Chinese Hamster Ovary. Pulmzyme over years has shown great results and is marketed by Roche Pharamceticals