BioCentury | Aug 5, 2020
Emerging Company Profile

Nura reveals neuroprotective lead target, first part of its neurology one-two punch

...work with fly genetics in the early 2010s uncovered the central role that SARM1, an NAD...
BioCentury | Sep 19, 2019
Distillery Therapeutics

Blocking the NAD+ biosynthesis enzyme NamPRT for PPM1D-mutant tumors

...DISEASE CATEGORY: Cancer INDICATION: Brain cancer; sarcoma; breast cancer Inhibiting NamPRT, a nicotinamide adenine dinucleotide (NAD+) biosynthesis enzyme, could...
BioCentury | Aug 8, 2019
Tools & Techniques

Checkpoint inhibitors are bringing oncolytic viruses back into the spotlight

...and to deliver the toll-like receptor adaptor molecule 1 (TICAM1; TRIF) and hydroxyprostaglandin dehydrogenase 15 NAD (HPGD...
BioCentury | Mar 29, 2019
Company News

ProQR spins out Wings Therapeutics

ProQR spun out its dystrophic epidermolysis bullosa business into Wings Therapeutics. Wings will continue to develop QR-313 to treat dystrophic epidermolysis bullosa patients with a mutation in exon 73. ProQR Therapeutics N.V. (NASDAQ:PRQR) will focus...
BioCentury | Mar 8, 2019
Clinical News

Santhera's Raxone slows loss of respiratory function in real-world DMD study

...#), idebenone (Generic), Raxone (Other), Catena (Other) Muscular Dystrophy Association Santhera Pharmaceuticals Holding AG NAD(P)H dehydrogenase quinone 1 (NQO1) (QR1) NAD(P)H dehydrogenase quinone 2 (NQO2) (QR2) Muscular...
BioCentury | Jan 26, 2019
Product Development

Raising the BD bar

Innovator biotechs say that the business development bar has been raised for companies developing new modalities, with potential partners seeking proof of mechanism on top of proof of efficacy. A host of products based on...
BioCentury | Nov 2, 2018
Company News

Ionis grants ProQR rights to QR-1123

Ionis Pharmaceuticals Inc. (NASDAQ:IONS) granted ProQR Therapeutics N.V. (NASDAQ:PRQR) exclusive, worldwide rights to QR-1123 (formerly IONS-RHO-2.5Rx) along with relevant IP. Ionis received $2.5 million in ProQR shares upfront. The partners said the shares were priced...
BioCentury | Oct 25, 2018
Preclinical News

Inhibiting metabolic enzyme to prevent NAFLD, acute kidney injury

...Pharma s.r.l. (Perugia, Italy) is developing small molecule inhibitors of a metabolic enzyme that limits NAD+...
...blocking it: prevention of non-alcoholic fatty liver disease or acute kidney injury. Low levels of nicotinamide adenine dinucleotide (NAD+)...
...that limits NAD+ production. The researchers showed that the inhibitors reduced ACMSD activity and increased NAD+...
BioCentury | Sep 13, 2018
Financial News

ProQR raises $90.6M in upsized offering

ProQR Therapeutics N.V. (NASDAQ:PRQR) raised $104.1 million on Sept. 11 through the sale of 6.6 million shares at $15.75 in a bumped-up follow-on underwritten by Citigroup, Evercore ISI, RBC Capital Markets, H.C. Wainwright and Chardan....
BioCentury | Sep 7, 2018
Clinical News

ProQR planning pivotal trial of vision loss candidate

ProQR Therapeutics N.V. (NASDAQ:PRQR) said it plans to start the pivotal Phase II/III ILLUMINATE trial of QR-110 next half to treat Leber congenital amaurosis type 10 (LCA10) caused by the p.Cys998X mutation in the centrosomal...
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