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CytRx, Artemis Pharmaceuticals and Evotec Neurosciences, PTC Therapeutics, CytRx and University of Massachusetts Medical School, and Invitrogen

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First Patients Treated in Phase I Study of CytRx HIV Vaccine

CytRx, Advanced BioScience Laboratories, and the University of Massachusetts Medical School said this week that the first patients have been enrolled and inoculated in a phase I study of an experimental HIV vaccine.

The vaccine uses technology CytRx licensed from UMMS and ABL. Under the terms of an existing agreement, CytRx will assume full ownership of the vaccine from ABL following completion of the phase I trial. ABL has an option to acquire the commercial manufacturing rights for the vaccine if it is approved by regulators.

The phase I study is set to enroll 36 healthy patients and is expected to run 18 months. Over the course of the trial, patients will receive three doses of the DNA element of the vaccine and two doses of the protein boost element of the vaccine. Trial participants will be monitored for antibody responses and cell-mediated responses, as well as adverse reactions.


Artemis To Help Evotec Develop Genetically Engineered Mice

Artemis Pharmaceuticals has signed an agreement to assist Evotec Neurosciences in generating genetically engineered mouse models, the companies said this week.

Under the deal, Artemis will apply its ArteMice Conditional gene targeting and ArteMice Speed technologies to Evotec’s efforts to develop genetically modified mice, which will be used to study the impact of genes believed to play a role in central nervous system diseases.

Financial terms of the arrangement were not disclosed.


PTC Therapeutics Begins Phase I Trial of Non-Sense Mutation Targeting Drug

PTC Therapeutics said this week that it has begun a phase I trial of PTC124, a small molecule drug that targets non-sense mutations, as a treatment for cystic fibrosis and Duchenne muscular dystrophy.

According to the company, PTC124 has shown the ability to restore full-length functional protein in genetic disease models harboring nonsense mutations. Approximately 15 percent of the cases of Duchenne muscular dystrophy and 10 percent of the cases of cystic fibrosis are due to nonsense mutations, PTC said.

Final results of the phase 1 studies of PTC124 are anticipated by the fourth quarter of this year and are expected to form the basis for initiation of phase 2 studies in early 2005.

“We plan to conduct initial patient studies in children and young adults with cystic fibrosis and Duchenne muscular dystrophy, and we hope to eventually evaluate this potential therapeutic for multiple genetic disorders,” PTC chief medical officer Langdon Miller said in a statment.


Cytrx says UMMS Collaborator Knocked Down ALS-Related Gene

CytRx reported this week that Zuoshang Xu, a University of Massachusetts Medical School researcher and company collaborator, has successfully used RNAi to knock down the gene that causes familial amyotrophic lateral sclerosis.

Xu has been working on silencing a mutant form of the gene, known as the superoxide dismutase (SOD-1) gene, in transgenic mice using short-hairpin RNA in order to determine whether RNAi can be used as a treatment for ALS.

“Our results demonstrate the high potential for applying RNAi to treat previously untreatable diseases, including ALS,” Xu said in a statement. “RNAi will be a highly effective strategy because it attacks the root of the disease.”

CytRx acquired the exclusive rights to use UMMS RNAi technology in the field of ALS therapeutics in June last year.


Invitrogen Establishes Biodefense Subsidiary, Sets Date for Release of Q2 Financial Results

Invitrogen said this week that it has formed a subsidiary focused on consolidating and advancing the company’s efforts in biosecurity.

The subsidiary, called Biological Defense Systems, will build off Invitrogen’s existing product lines, which include detection kits for airborne pathogens such as anthrax and smallpox, by developing detection systems for food and water-borne pathogens, the company said.

Invitrogen also said this week that it has scheduled the release of its second quarter financial results for July 21 before the opening of the market.

Following the release of the results, the company will hold a conference call with investors and analysts at 8:30 am Eastern.

Details about listening to the conference call can be found on the company’s website at www.invitrogen.com.

 

The Scan

Interfering With Invasive Mussels

The Chicago Tribune reports that researchers are studying whether RNA interference- or CRISPR-based approaches can combat invasive freshwater mussels.

Participation Analysis

A new study finds that women tend to participate less at scientific meetings but that some changes can lead to increased involvement, the Guardian reports.

Right Whales' Decline

A research study plans to use genetic analysis to gain insight into population decline among North American right whales, according to CBC.

Science Papers Tie Rare Mutations to Short Stature, Immunodeficiency; Present Single-Cell Transcriptomics Map

In Science this week: pair of mutations in one gene uncovered in brothers with short stature and immunodeficiency, and more.