RNA therapy developer Proqr Therapeutics NV’s phase I/II study of adults with Usher syndrome and non-syndromic retinitis pigmentosa met all its key objectives, prompting the company to plan two parallel pivotal phase II/III studies that could start by year-end. The company wasted no time as it has already discussed next steps with the FDA to support the therapy’s registration “as soon as possible,” Aniz Girach, Proqr’s chief medical officer, told investors during a March 24 investor call. Read More
DUBLIN – Step Pharma SAS closed a €35 million (US$41.5 million) series B round to move its first-in-class cytidine triphosphate synthase 1 inhibitor into clinical trials in patients with T-cell and B-cell malignancies. Read More
With so many headlines touting drugs in development for the likes of nonalcoholic steatohepatitis and nonalcoholic fatty liver disease, an illness in the same ballpark has been back-seated: alcoholic hepatitis (AH), which is on the rise, under-recognized and brings a one-month mortality rate that averages 26%. Read More
According to the BioWorld Cancer Financings Report, 2020 proved to be a record year in terms of capital raised by biopharmaceutical companies working on therapeutics for cancer indications. Read More
LONDON – A U.K.-wide study has found that seven out of 10 patients hospitalized with COVID-19 infection were continuing to suffer physical and mental health problems five months after discharge. Read More
The continuing politicization of COVID-19 vaccines is undermining medical science and the international response to the pandemic. “Vaccine nationalism is very troubling,” Jeremy Levin, chair of the Biotechnology Innovation Organization’s (BIO) executive committee, told BioWorld. Read More
A study by Korean and U.S. geneticists has demonstrated that the newly developed Gene set analysis Association Using Sparse Signals (GAUSS) is more powerful than existing methods for detecting phenotype-associated gene sets and facilitating interpretation of gene set analysis results. Read More
New hires and promotions in the biopharma industry, including: Aura, Bellus, Celmatix, Fulcrum, Inotrem, Intravacc, Marinus, Merck, Metagenomi, Mission, Neurotrauma, Sitryx, Tango, Vividion. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Aditxt, Arch, Betterlife, Clinuvel, Exacis, Gemphire, GSK, Hutchison China Meditech, Intensity, Interna, Jeune, Kaleido, Krystal, Neurobo, Nobelpharma, Probiogen, Servier, Valence. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Akebia, Ascentage, Berg, Enterome, Horizon, RDIF, Reneo. Read More