BioCentury | Dec 8, 2020
Product Development

Latest data give Rocket clear path forward in rare CV disease Danon

While an adverse event in the high-dose cohort continues to outline the limits of high volume AAV dosing, Rocket believes that higher-than-expected gene expression levels at lower doses for RP-A501 to treat Danon disease give...
BioCentury | Nov 11, 2020
Product Development

Nov. 10 Quick Takes: Tezepelumab meets in asthma Phase III; plus a call for speedy trial results, updates on COVID vaccines from Sinovac, CureVac and more

AZ, Amgen rise on asthma dataTezepelumab from AstraZeneca plc (LSE:AZN; NYSE:AZN) and Amgen Inc. (NASDAQ:AMGN) met the primary endpoint of a statistically significant reduction in annualized asthma exacerbation...
BioCentury | Oct 6, 2020

White House allows FDA to release long-awaited COVID vaccine EUA guidance

In a move that is intended to bolster public confidence in FDA’s integrity, the agency Tuesday posted guidance about emergency use authorization of COVID-19 vaccines. FDA posted the guidance after...
BioCentury | May 15, 2020
Product Development

12-month data set up Pfizer DMD gene therapy for Phase III, but SAEs may give Sarepta safety edge

Pfizer believes encouraging long-term efficacy data and recently installed protocols for mitigating adverse events will help the pharma advance its DMD gene therapy into Phase III testing within the next few months as it ramps...
BioCentury | Jan 13, 2020
Clinical News

Adaptimmune rises on early promise for SPEAR TCR cell therapies in solid tumors

Cancer cell therapy play Adaptimmune gained $2.66 (200%) to $3.99 on Monday after announcing a new set of data for its SPEAR T cell therapies in four different solid tumor indications. The announcement by Adaptimmune...
BioCentury | Dec 11, 2019
Product Development

The writing has been on the wall for Sanofi’s diabetes, CV business for years

One hundred days after taking the helm as CEO, Paul Hudson has outlined a plan to get Sanofi to 30% margins by 2022 that hinges largely on the success of Dupixent and five other priority...
BioCentury | Oct 14, 2019
Clinical News

Phase III readout could position Pfizer’s JAK-1 to compete in eczema

Data from the first of two Phase III trials of Pfizer’s JAK-1 inhibitor abrocitinib to treat atopic dermatitis suggest that the oral small molecule could deliver efficacy that ranks with injectable blockbuster Dupixent dupilumab. In...
BioCentury | Aug 2, 2019
Clinical News

Aug. 2 Clinical Quick Takes: AB Sciences halts metastatic melanoma program; plus NIH, Adaptimmune and more

AB Sciences drops masitinib for melanoma AB Science S.A. (Euronext:AB) halted development of masitinib for metastatic melanoma despite reporting that the product led to a 33.3% overall response rate (ORR), the primary endpoint in the...
BioCentury | Mar 6, 2019
Clinical News

Alnylam's rare liver disease RNAi meets in Phase III

Alnylam’s givosiran met the primary endpoint in the Phase III ENVISION trial to treat acute hepatic porphyria, setting the company up to complete submission of a rolling NDA to FDA and submit an MAA to...
BioCentury | Jan 9, 2019
Distillery Therapeutics


...INDICATION: Lymphoma; colorectal cancer Cell culture and mouse studies suggest an SAE1 inhibitor could help treat...
...vitro SUMOylation of a reporter protein with an IC 50 of 0.2 μM and decreased SAE1...
...testing the SAE1 inhibitor in mouse models of colorectal cancer with high MYC expression. TARGET/MARKER/PATHWAY: SUMO1 activating enzyme subunit 1 (SAE1)...
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