Apr 18 transthyretin amyloidosis

Notizia

Positive Phase 1 data supports Cyclacel’s plans for CDK2/9 inhibitor CYC065

Cyclacel faced a big failure in for its late-stage drug for leukemia just a year ago hitting hard on its shares. After that the attention was shifted towards an early-stage CDK inhibitor. As per the latest results presented by the company CDK drug has shown that it can successfully stabilize disease in advanced cancers. After positive results now the drug will further be taken into a trial in combination with AbbVie’s expected to be leukemia blockbuster Venclexta.

Lysogene delays phase 3 study of Sanfilippo gene therapy

Lysogene’s pivotal Sanfilippo syndrome trial is pushed back by a manufacturing delay. Lysogene has been working on CMC topic for quite some time but is yet to get its manufacturing setup to the point where it can possibly support the phase 2/3 trial. Due to the unavailability of a working manufacturing plant the start date for the trial has shifted to latter half of 2018.

Diagnosis of genetic disorders could further be improved by Genotype-first approach

Group of scientists from around the world had started with a genetic mutation and further tracked down the people who were suffering from it. in the process 37 individuals with an NAA15 mutation and a multitude of symptoms were discovered. Genotype-first” method could fine-tune the existing diagnostic process and help researchers devise treatments for genetic disease, saving a lot of time and improving the treatment method.

PD-1 resistance in melanoma patients successfully reversed by checkmate’s I-O combo

Checkmate Pharmaceuticals has recently unveiled the phase 1 data of its lead asset, CMP-001, in combination with Merck’s Keytruda at AACR, the combination has successfully reversed PD-1 resistance in advanced melanoma patients who had failed a prior anti-PD-1 treatment.

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