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MDRNA, Alnylam Pharmaceuticals, Takeda Pharmaceutical, Calando Pharmaceuticals

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MDRNA Selects Lead Candidate in Hypercholesterolemia Program
 
MDRNA announced this week that it has selected a lead candidate in its hypercholesterolemia program.
 
The molecule, called MDR-04227, is a Dicer-substrate targeting apolipoprotein B and is formulated with MDRNA’s proprietary lipid-based delivery technology.
 
“In a preclinical animal model of hypercholesterolemia, MDR-04227 was shown to be extremely potent with an IC50 of less than 100 pM,” the company said. “When formulated for systemic administration … MDR-04227 demonstrated approximately 85 percent knockdown in target messenger RNA of ApoB and a similar level of reduction of serum cholesterol following a single 1 mg/kg dose of siRNA.”
 
MDRNA first announced its hypercholesterolemia program in August (see RNAi News, 9/7/2008).
 

 
Alnylam Receives $20M Under Takeda Alliance
 
Alnylam said this week that it has received a $20 million technology-transfer payment from partner Takeda Pharmaceutical as part of the companies’ strategic alliance (see RNAi News, 5/29/2008).
 
Under that arrangement, Takeda acquired a worldwide, non-exclusive access to Alnylam’s RNAi intellectual property and technology to help develop drugs for cancer and metabolic diseases. In exchange, Alnylam is to receive $150 million upfront, $50 million in near-term technology-transfer payments, and up to $171 million in development and commercial milestones, as well as royalties.
 
With the most recent payment, Alnylam has already received $120 million of its upfront payment.
 

 
Calando Issued Patent Covering Lead RNAi Drug Candidate
 
Calando Pharmaceuticals said this week that it has been issued a US patent covering the active ingredient of its phase I siRNA-based cancer drug candidate CALAA-01.
 
The patent, No. 7,427,605, is entitled, "Inhibitors of Ribonucleotide Reductase Subunit 2 and Uses Thereof." According to Calando, it contains claims directed to inhibitory nucleic acid sequences targeting the Ribonucleotide Reductase Subunit 2 gene, as well as pharmaceutical compositions and methods for inhibiting tumor growth utilizing the sequences.
 
This summer, Calando began dosing patients in a phase I trial of CALAA-01, making it the first RNAi drug developer to begin testing a formulated siRNA therapeutic in humans (see RNAi News, 6/5/2008).

The Scan

Study Tracks Responses in Patients Pursuing Polygenic Risk Score Profiling

Using interviews, researchers in the European Journal of Human Genetics qualitatively assess individuals' motivations for, and experiences with, direct-to-consumer polygenic risk score testing.

EHR Quality Improvement Study Detects Demographic-Related Deficiencies in Cancer Family History Data

In a retrospective analysis in JAMA Network Open, researchers find that sex, ethnicity, language, and other features coincide with the quality of cancer family history information in a patient's record.

Inflammatory Bowel Disease Linked to Gut Microbiome Community Structure Gradient in Meta-Analysis

Bringing together data from prior studies, researchers in Genome Biology track down microbial taxa and a population structure gradient with ties to ulcerative colitis or Crohn's disease.

Ancient Greek Army Ancestry Highlights Mercenary Role in Historical Migrations

By profiling genomic patterns in 5th century samples from in and around Himera, researchers saw diverse ancestry in Greek army representatives in the region, as they report in PNAS.