Anelixis Therapeutics LLC, formed in 2013 as a for-profit subsidiary of the nonprofit ALS Therapy Development Institute (ALS TDI), has begun enrolling the phase I dose-escalation study of AT-1501, a humanized anti-CD40 ligand (CD40L) monoclonal antibody. The single-site study at Massachusetts General Hospital (MGH) is examining safety and pharmacokinetics in six cohorts of healthy volunteers and one of individuals with amyotrophic lateral sclerosis (ALS). Top-line data are expected at the end of March, according to Steven Perrin, founder, president, CEO and sole employee of the Cambridge, Mass.-based company.

The holidays are (already) here again. Much as we try to push back the calendar, those Thanksgiving doorbuster, Black Friday blowout, Small Business Saturday, Cyber Monday and Giving Tuesday pitches keep knock, knock, knocking at our doors. And those salebrations don’t even count the actual seasonal holidays, like Hanukkah, which begins at sundown on Dec. 2 to kick off an early start to this year’s festivities. Before you hit the stores – or, more likely, that “Purchase Now” icon on your phone – take a spin through these suggestions from the BioWorld team and from our fans and followers. For...

The holidays are (already) here again. Much as we try to push back the calendar, those Thanksgiving doorbuster, Black Friday blowout, Small Business Saturday, Cyber Monday and Giving Tuesday pitches keep knock, knock, knocking at our doors. Before you hit the stores take a spin through these suggestions from the BioWorld team and from our fans and followers.

In the run-up to its scheduled R&D presentation on Monday, Uniqure NV produced what H.C. Wainwright's Debjit Chattopadhyay dubbed "a compelling glimpse" of the future of hemophilia B treatment, positioning AMT-061 "to be not only the best-in-class one-and-done solution, but also, the first to market."

Moderna Inc. dropped its long-anticipated S-1 filing, allowing industry watchers to exhale. The Cambridge, Mass.-based company is seeking to raise $500 million, including overallotments – the same amount the company raised privately in February – in what many observers were calling biopharma's biggest IPO. Except that it's not, unless some qualifiers are used and provided that Moderna doesn't decide to upsize as the book-building begins.

Cue Biopharma Inc. hopes to achieve perfect harmony with LG Chem Life Sciences, the life sciences division of Korean giant LG Chem Ltd. (LGC), in a multitarget collaboration to develop its Immuno-STAT (Selective Targeting and Alteration of T cells) immuno-oncology (I-O) biologics. The deal provides LG Chem with rights in Asia to develop and commercialize Cue's lead candidate, CUE-101, and Immuno-STAT biologics that target T cells against two additional cancer antigens.

Until recent years, polycystic kidney disease (PKD) was a renal indication in search of solutions – a situation even truer for its autosomal dominant (ADPKD) and autosomal recessive (ARPKD) subgroups. That dearth of drug development in the space is beginning to change, however, thanks to greater understanding of the genetic drivers of kidney disease and of the molecular pathways that are subsequently up-regulated and cause disease.Those findings are enabling researchers to apply precision medicine approaches that are the hallmark of targeted cancer therapies to indications such as PKD. Buy-in from regulators, who have shown willingness to explore creative alternatives to historically broad trial designs, are beginning to attract both big pharma and newer entrants to the opportunities in renal disease.

On Wednesday, Revive Therapeutics Ltd., a tiny Toronto-based company that trades on the OTCQB as RVVTF, revealed that it secured FDA orphan drug designation for cannabidiol (CBD) to prevent hepatic ischemia and reperfusion injury resulting from solid organ transplantation. Under license from South Carolina Research Foundation, a unit of the University of South Carolina, Revive also is investigating REV-200, described as a cannabinoid CB1/CB2 receptor modulator, for the potential treatment of liver diseases such as autoimmune hepatitis and nonalcoholic steatohepatitis, or NASH. But the company, which markets a hemp-based CBD chewing gum branded Relicann, is primarily looking to partner the asset for development. A coveted FDA regulatory designation helps to set it apart.