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MDRNA, Alnylam Pharmaceuticals, Takeda Pharmaceutical, Calando Pharmaceuticals

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

Two J&J Doses

Johnson & Johnson says two doses of its SARS-CoV-2 vaccine provides increased protection against symptomatic COVID-19, CNN reports.

Pfizer-BioNTech Vaccine Response in Kids

The Pfizer-BioNTech SARS-CoV-2 vaccine in a lower-dose format appears to generate an immune response among children, according to the Washington Post.

Chicken Changes to Prevent Disease

The Guardian writes that researchers are looking at gene editing chickens to help prevent future pandemics.

PNAS Papers on Siberian Dog Ancestry, Insect Reproduction, Hippocampal Neurogenesis

In PNAS this week: ancestry and admixture among Siberian dogs, hormone role in fruit fly reproduction, and more.