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Acuity files IND; first to clinic with RNAi drug?


In line with expectations, Acuity Pharmaceuticals filed an investigational new drug application with US regulators to begin phase I testing of its siRNA-based treatment for wet age-related macular degeneration. The IND filing put the small, Philadelphia-based company on track to begin testing of the drug, called Cand5, as early as September.

Cand5 targets vascular endothelial growth factor, and has been shown in non-human primate experiments to inhibit the neovascular growth and vascular leakage associated with laser injury. These data, along with recently completed ADMET work, have now been submitted to the US Food and Drug Administration, Acuity president and CEO Dale Pfost says. The company is also adding members to its scientific advisory board to help guide Acuity through the clinical trial process.

One of those slated to join Acuity’s SAB is Alan Gewirtz, a professor at the University of Pennsylvania School of Medicine and inventor of a US patent application that forms part of the company’s core RNAi intellectual property estate. Acuity has an exclusive license to the patent application from Penn. Also expected to join the SAB is Alexander Brucker, a member of Acuity’s board of directors and professor of ophthalmology at University of Pennsylvania Health System’s Scheie Eye Institute, Pfost says.

Funding Acuity as it works to move Cand5 through phase II development is a series B round of financing, worth an expected $15 million, that as of late August was still in the process of being finalized. “Essentially, we’re down at the detail level — documents and that sort of thing — so [the financing] is in the works,” Pfost says. “We’re working towards the final portions of the close.” He declined to comment on how the series B round would impact Penn’s 25 percent stake in the company.

— Doug Macron


US Patent Application No. 20040152172. Method for Generation of a Random RNAi Library and its Application in Cell-Based Screens. Inventor: Martin Geppert, Pharmacia & Upjohn. Filed: Sept. 5, 2003.

The application claims “a method to generate a population of inhibitor sequences ready for cloning [by] extending a population of random oligonucleotide RNAi progenitors comprising a fixed primer sequence, a random oligonucleotide sequence, and a fixed stem-loop structure, via a polymerase extension reaction to produce a full hairpin random oligonucleotide RNAi progenitor.”

US Patent Application No. 20040147735. Deprotection of RNA. Lead Inventor: Laurent Bellon, Sirna Therapeutics. Filed: Jan. 6. 2004

This invention concerns a one-pot process for the deprotection of RNA molecules. It features a novel method for the removal of protecting groups from the nucleic acid base and 2''''''''-OH groups, which accelerates the process for generating synthetic RNA in a high-throughput manner, such as a 96-well format.


$7.7 million
Alnylam Pharmaceuticals’ reported net loss in the second quarter, compared to $3.7 million in the same quarter last year. The company reported revenues of $131,000 for the quarter, derived primarily from its collaboration with Merck. As of June 30, Alnylam had cash, cash equivalents, and marketable securities totaling $46.1 million.

Benitec settles its patent infringement litigation against Ambion, putting an end to one-third of a broader lawsuit filed in March. Under the terms, Ambion receives a non-exclusive, worldwide license to make and sell research tools and products using Benitec’s patented, DNA-directed RNAi technology.

Alnylam Pharmaceuticals takes an exclusive license to patents and patent applications held by Hybridon related to using RNA interference molecules targeting vascular endothelial growth factor.

Dharmacon and Genospectra sign an agreement to collaborate on the commercialization of a genome-wide line of assays specifically designed to measure RNA changes from cell lysates, fresh tissue, and archived tissue samples.

AVI BioPharma signs a five-year cooperative R&D agreement with the US Army Medical Research Institute of Infectious Diseases under which the company’s antisense compounds will be tested against several viruses, bacteria, and toxins that may be used as bioterror agents.

Qiagen announces a supply agreement with Novartis for consumables related to nucleic acid stabilization, separation, purification, and handling, as well as siRNA and instrumentation.

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