Skip to main content
Premium Trial:

Request an Annual Quote

In Landmark Step, Acuity Is First Firm to Begin RNAi-based Drug Study In Humans

Premium

Acuity Pharmaceuticals said on Nov. 9 that the first patients in a phase I trial of the company’s investigational siRNA-based age-related macular degeneration treatment, Cand5, have been dosed, marking the first time that an RNAi drug is being used in humans.

“The trial is underway,” Sam Reich, Acuity’s senior director of researcher and development and co-founder, told RNAi News. “Multiple patients have been dosed, and everything’s been successful to date.

“The trial is in full swing and we’re very excited,” he added.

Acuity President and CEO Dale Pfost declined to comment further on the clinical trial, but told RNAi News that “we will be reporting [on the outcome of the trial] in the latter part of next year.”

News of the patient dosing gave a shot in the arm to shares of Sirna Therapeutics, which recently filed an investigational new drug application for its own RNAi-based AMD therapy (see RNAi News, 9/10/2004); shares of Sirna climbed more than 12 percent to $2.95 on the Nasdaq exchange in mid-day trading on Thursday.

While Sirna has yet to make any public statements about the status of its AMD program, COO Nassim Usman told RNAi News that everything remains on schedule with his company’s IND.

“There are no issues” that have been raised by the US Food and Drug Administration, Usman said. “We plan on dosing [the first patient] somewhere in the next week or two.”

The start of Acuity’s phase I trial comes on the heels of the firm’s announcement that it has signed a “long-term” agreement to have pharmaceutical-grade supplies of Cand5 manufactured by Avecia Biotechnology.

Under the terms of the deal, Avecia will provide Acuity with Cand5 up through the commercial launch of the drug. Additional terms were not disclosed.

“This is the first-ever manufacturing agreement for a pharmaceutical siRNA,” Pfost said in a statement this week. “Avecia’s expertise in the manufacture of clinical-grade oligonucleotides and their manufacturing-related intellectual property will be extremely valuable to Acuity’s development program for Cand5.”

Avecia’s experience with oligo manufacture includes its status as manufacturer of Genta’s antisense-based cancer drug Genasense under a five-year contract signed in 2002.

— DM

The Scan

More Boosters for US

Following US Food and Drug Administration authorization, the Centers for Disease Control and Prevention has endorsed booster doses of the Moderna and Johnson & Johnson SARS-CoV-2 vaccines, the Washington Post writes.

From a Pig

A genetically modified pig kidney was transplanted into a human without triggering an immune response, Reuters reports.

For Privacy's Sake

Wired reports that more US states are passing genetic privacy laws.

Science Paper on How Poaching Drove Evolution in African Elephants

In Science this week: poaching has led to the rapid evolution of tuskless African elephants.