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I've just added an Ataluren update to my blog (relates to mutations ending in X), including investor presentation images & clinicaltrials.gov links to the upcoming Ataluren trials: http://sixtyfiverosesblog.wordpress.com/2014/05/22/may-2014-ataluren-update/ There is also more information &...

I've just summarised the May 1 Vertex investor conference, includes info about Kalydeco, VX809 & VX661: http://sixtyfiverosesblog.wordpress.com/2014/05/04/may-2014-vertex-update/ Also, it was announced on May 1 that the trial adding VX661 to patients stable on Kalydeco (with G551D/F508del)...

I've just summarised the Feb 25 Vertex investor conference, includes info about Kalydeco, VX809, VX661 and second generation correctors: http://sixtyfiverosesblog.wordpress.com/2014/02/27/february-2014-vertex-update/ Also, last week it was announced that Kalydeco received FDA approval for...

I recently wrote a blog post about the latest Vertex news (includes info from the Dec and Jan press releases & investor conferences). Since I wrote this post it has also been announced that the combination therapy VX661/Ivacaftor has been granted breakthrough FDA designation...

I've recently added info to my blog about the current gene therapy trial, as well as info about a future trial that involves a new approach: http://sixtyfiverosesblog.wordpress.com/2014/01/20/uk-gene-therapy-update/

Hi all, The Australian Kalydeco campaign has a new petition (started last week) & we would appreciate as many signatures as possible. There are 200+ Australians still waiting for Kalydeco who need your help. Thanks...

I just added two blog posts, one about Ataluren, with information about the upcoming trial & EMA application, and the other about a new potential approach for nonsense mutations (mutations that end in X). New Potential Approach for Nonsense Mutations November Ataluren Update

Kalydeco is still not available to the 200 Australians with G551D. The reimbursement has been delayed as meetings and negotiations continue between Vertex and the Australian PBAC committee. We have a campaign through facebook and twitter, with the new YesToKalydeco page...

I summarised a scientific article released in August that discusses how VX809 helps F508del, how the folding can be improved further and other mutations that were helped by VX809 in this study: http://sixtyfiverosesblog.wordpress.com/2013/10/18/how-does-vx809-help-f508del-part-2/

Novoteris recently announced that the FDA has granted orphan drug designation for inhaled nitric oxide as a treatment for Cystic Fibrosis. The pilot trial demonstrated an 8.8% absolute improvement from baseline after 2 weeks. Phase 2 trials are planned for 2014. More info here...

ProQR Therapeutics recently received orphan drug designation for their Cystic Fibrosis therapy that targets the F508del mutation. This approach is different to the CFTR modulation approach (potentiators and correctors), which targets the defective protein. ProQR are targeting the Cystic Fibrosis...

PTC had their first investor conference yesterday, here is a brief summary: - Second phase 3 Ataluren study planned to start in the first half of 2014 with CF nonsense mutations - Plan to apply to the EMA (Europe) this year - Potential for combination with Vertex's Kalydeco discussed during...

I was just looking through my blog and realised I have not posted about Calista here. Calista Therapeutics are developing medications that target F508del. Pre clinical studies are underway and they are looking for funding for a phase 2a trial. The lead candidate is Procaftor, which appears...

Proteostasis have identified multiple compounds that improve the folding and trafficking of F508del. These compounds have a similar level of activity to VX809, and when added to VX809, about 40% of normal function is reached. Trials are planned to start in 2015. I've added more info and some...

I've summarised the latest Vertex investor conference and added some images from the conference here: http://sixtyfiverosesblog.wordpress.com/2013/07/31/july-vertex-update/ Main points: G551D A new study with VX661 & Kalydeco is planned for the second half of 2013 with patients who have...

A recent study found that Ataluren failed to enhance read through past stop codons (they tested all the possible stop codon combinations in different situations), instead they suggested that Ataluren interacted with the drug screening system, the luciferase enzyme. I've summarised the...

I have added several new posts to my blog and thought I would add links here. The first post has new graphs from vertex showing that 1) When a second generation corrector is added to VX809 & Kalydeco, heterozygotes (1 copy F508del) increase to about 33% of normal function (based on data with...

Galapagos are developing new CFTR potentiators and correctors. The image below suggests that there is superior CFTR opening with their potentiator (GLPG1) compared to Kalydeco with F508del, and that GLPG1 may be more potent with G551D than Kalydeco. Galapagos has a “large pipeline of four...

The phase 2 VX661/Kalydeco results have just been released: Statistically significant improvements in lung function were seen in this study with CFs who have two copies of F508del "Treatment with combination of VX-661 and ivacaftor for 28 days in two highest dose groups resulted in mean...

N30 has started a phase 1b/2a trial with CFs who have 2 copies of F508del. The N6022 is both anti-inflammatory and a CFTR corrector. "N6022 is the first of a novel class of N30 Pharma's drugs aimed at conservation of S-nitrosoglutathione (GSNO) a key, endogenous, signaling molecule that has...