BioCentury | Oct 6, 2020
Management Tracks

BioMarin, Sanofi tap new execs for early R&D positions

BioMarin and Sanofi each have new leaders of their early R&D teams, as the biotech has made a high-profile hire while the French pharma has promoted from within to replace two executives who are leaving...
BioCentury | Sep 16, 2020
Deals

Sept. 15 Quick Takes: NIH consortium seeks schizophrenia biomarkers; plus Boehringer-Click, Takeda, Nestlé-Aimmune, 89bio, Cellinta, Cullinan Florentine, Bayer-Systems Oncology

NIH launches Accelerating Medicines Partnership for schizophreniaNIH has partnered with FDA, non-profits and biopharmas to launch the Accelerating Medicines Partnership Schizophrenia (AMP SCZ) initiative...
BioCentury | Aug 28, 2020
Distillery Therapeutics

Exon skipping therapy for Batten disease

...in lysosomal membrane protein CLN3 could treat CLN3...
...In neonatal mice with a deletion in CLN3...
...preclinical development for juvenile Batten disease.TARGET/MARKER/PATHWAY: Ceroid-lipofuscinosis neuronal 3 (CLN3)EXPERIMENTAL...
BioCentury | Jun 11, 2020
Deals

Cullinan adds first-in-class asset in hot NK cell checkpoint space

The latest addition to Cullinan’s oncology portfolio is a mAb against MICA that’s set to become the first compound in the clinic against the NK cell target. Through a deal announced on Thursday, Cullinan Oncology...
BioCentury | Aug 2, 2019
Clinical News

Amicus’ Batten disease gene therapy shows preserved speech, motor function

...gene therapy in the clinic is AAV-CLN3, which is in Phase I/II testing for the ceroid-lipofuscinosis neuronal 3 (CLN3)...
BioCentury | Mar 1, 2019
Company News

NICE rebuffs BioMarin's ultra-orphan therapy

The U.K.'s NICE did not recommend Brineura cerliponase alfa from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) due to uncertainty around the therapy's long-term efficacy and concerns NHS would be unable to implement the rare disease biotech's proposed...
BioCentury | Feb 22, 2019
Company News

NICE nixes BioMarin's ultra-orphan therapy for lack of long-term data

The U.K.'s NICE did not recommend Brineura cerliponase alfa from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) due to uncertainty around the therapy's long-term efficacy and concerns NHS would be unable to implement the rare disease biotech's proposed...
BioCentury | Jan 5, 2019
Finance

Ready to launch

The proposed $74 billion takeout of Celgene Corp. by Bristol-Myers Squibb Co. will not shift buysider attention away from upcoming and maturing drug launches heading into 2019, with plenty to track following a record year...
BioCentury | Nov 9, 2018
Company News

Abeona gets rights to RegenxBio gene therapy vector

...type 1 (CLN1), and Juvenile Batten Disease, also known as neuronal ceroid lipofuscinosis type 3 (CLN3...
...NASDAQ:RGNX), Rockville, Md. Abeona Therapeutics Inc. (NASDAQ:ABEO), Dallas, Texas Business: Gene therapy Allison Johnson Abeona Therapeutics Inc. RegenxBio Inc. CLN1 CLN3 MPS...
BioCentury | Nov 1, 2018
Translation in Brief

New Therapeutic Targets and Biomarkers: October 2018

New Therapeutic Targets and Biomarkers: October 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during October. Therapeutic targets are defined as any protein, gene or other molecule...
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