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Bluebird declares the price of its gene therapy- Zynteglo Bluebird Bio has set the price of its gene therapy Zynteglo at USD 1.8 million. Zynteglo, so far, has only been approved for treating rare blood disorders in the Europe Union. The full price of Zynteglo can be dispersed out for five years, at approx...

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transthyretin amyloidosis
Idera’s drug; CRISPR increase risk; Juvenescence grabbed USD 50M; AstraZeneca, Lilly terminates

Idera's dermatomyositis drug missed primary endpoint in phase 2 Idera’s toll-like receptor (TLR) antagonist, IMO-8400, failed to meet its primary endpoint in a phase 2 trial carried on adults with dermatomyositis. Data has shown that the drug was meant to block TLR 7, 8 and 9 but failed to beat placebo in making a s...

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transthyretin amyloidosis
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Arbor Biotechnologies shining with new CRISPR enzyme Arbor Biotechnologies is shining with its new CRISPR enzyme and has successfully bagged USD 15.6 million in series A funding. The company has recently announced its first scientific discovery of a new enzyme, Cas13d, a CRISPR-Cas13 enzyme. It could offer advantag...

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Anthera’s Sollpura flops after phase 3   Anthera cut free it’s flopped late-arrange Lilly castoff Sollpura (liprotamase), and now looks for those feared "key choices". Sollpura was out-licensed by Lilly in 2014, and the written work had all the attributes of its failure, back in December 2016 when it posted the inf...

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transthyretin amyloidosis
CRISPR to cure sickle cell; FDA rejected; Stem cells use; Celgene spinoff

Rice University researchers tested CRISPR to cure sickle cell, now preparing for upcoming challenges A team of Researchers led by Rice University has recently tested CRISPR to correct the sickle cell mutation in up to 40% of the total stem cell samples taken from affected patients. Although the results are quite pro...

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transthyretin amyloidosis
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Key to new Cancer drugs discovered in bacterial genomes Achievement is made by the scientists of The Scripps Research Institute (TSRI) Florida campus who had previously discovered LNM E1 compound used to kill prostate cancer. This time they have unlocked the capability of LNM family molecules by “mining” the inform...

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transthyretin amyloidosis
Regeneron/Sanofi’s PD-1 drug candidate; Zika targeting drugs and vaccines developments; CMO of Achillion will leave this month; Lincoln’s anti-malarial drug

Regeneron/Sanofi’s PD-1 drug candidate will be tested with ISA’s HPV immunotherapy Regeneron and Sanofi’s PD-1 candidate cemiplimab is paired with Dutch biotech ISA Pharma’s human papillomavirus (HPV) targeting drug for testing. The new drug is competing to become the sixth PD-1/PD-L1 drug on the market. Joined with...

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transthyretin amyloidosis
Roche’s Phase 2 data; Celgene and Bluebird’s CAR-T therapy; Regeneron/Sanofi gets results; Allergan on women’s health

Positive Phase 2 data for Roche’s lymphoma ADC Roche’s antibody-drug conjugate for non-Hodgkin lymphoma polatuzumab has already received breakthrough designation from the USFDA and priority medicine status in the EU, and the expectations for the same are running pretty high. Phase 2 data presented by the company at...

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KINECT 4 Study; KindredBio Announces Results; Mylan and Aspen’s Launch; EMA investigates; Amgen’s Repatha

KINECT 4 Phase III Study Demonstrating INGREZZA® Improves Tardive Dyskinesia Symptoms Neurocrine Biosciences, Inc. announced new long-term data from the KINECT 4 Phase III open-label study demonstrate that once-daily INGREZZA® (valbenazine) capsules, the first FDA approved treatment for adults with tardive dyskinesi...

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BioGen
Biohaven’s trial ; GSK integrates; FDA Approval; Phase III trial

Biohaven Completes Enrollment of Rimegepant in Second Pivotal Phase 3 Clinical Trial for the treatment of Migraine Biohaven Pharmaceutical has recently announced the completion of enrollment of their study BHV3000-302 in Phase 3. The company is examining the safety and efficacy of Rimegepant for the treatment of Mi...

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