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Alnylam Pharmaceuticals, Isis Pharmaceuticals, Genta, Benitec, Topigen, Invitrogen and Bio Asia, and CytRx


Alnylam Presented Preclinical Data on siRNAs Targeting VEGF

Alnylam Pharmaceuticals said this week that it has presented preclinical data demonstrating the potential of its siRNAs to inhibit the expression of vascular endothelial growth factor, a target in the company’s age-related macular degeneration program.

According to the company, the siRNAs were tested in vitro for their ability to inhibit the expression of VEGF in cell lines under normal and low-oxygen conditions. Some of the siRNAs were modified, which enhanced nuclease stability, while maintaining full in vitro silencing activity, the company said. SiRNAs examined in vivo were stable and nuclease-resistant, and showed much greater stability within the vitreous of the eye and other ocular tissues over time than did similar siRNAs in unmodified form.

Alnylam said the data were presented at the 44th Annual Meeting of the American Society of Cell Biology in Washington, DC.

Isis Says Antisense Drug for Crohn’s Disease Fails in Phase III Trials

Isis Pharmaceuticals said last week that its antisense drug for Crohn’s disease, alicaforsen, failed to demonstrate statistically significant induction of clinical remissions compared with placebo in two identical phase III trials.

As a result of the data, Isis said it will no longer develop the drug for the inflammatory bowel disorder.

The company also said last week that in three phase II studies in patients with ulcerative colitis, an enema formulation of alicaforsen led to a significant improvement of symptoms.

Genta Acquires New Antisense Drug

Genta said this week that it has acquired from Temple University the global rights to a novel antisense compound, called LR3001, that targets c-myb. C-myb, said the company, is a central gene that regulates the growth of cancer cells.

LR3001 has been tested in two phase I clinical trials at the University of Pennsylvania in patients with drug-resistant myeloid leukemia, Genta said. A request for designation of the drug as an Orphan Drug for the treatment of chronic myelocytic leukemia has been submitted to US regulators, the company noted.

Genta’s acquisition of LR3001 comes as the company works to recover from the failure of its lead product candidate, Genasense, to pass muster with a US Food and Drug Administration advisory panel as a treatment for advanced melanoma in May. Shortly thereafter, Aventis pulled out of a deal to jointly develop and market the drug with Genta.

Benitec Completes Shareholder Share Purchase Plan

Benitec said this week that it has completed its shareholders’ share purchase plan.

Under the plan, a total of AU$892,100 was raised, the company said.

Topigen Says Phase I Study of Antisense Asthma Therapy Gets Green Light

Topigen said this week that it has received approval from US regulators to begin a phase I trial of its experimental antisense treatment for asthma.

The therapy, called ASM8, consists of two modified phosphorothioate antisense oligonucleotides, the company said. The oligos inhibit the expression and function of the receptors that function in two separate but related pathways for inflammation.

“As no human safety data are currently available for inhaled ASM8, the present study is designed to investigate both the safety and tolerability of this new inhaled drug for the treatment of asthma,” Paolo Renzi, chief scientific director of Topigen, said in a statement. “In this trial set to begin by the end of January 2005, healthy human male and female volunteers will be randomized to receive a single inhaled dose of nebulized ASM8. As such, standard and airway-specific assessments of safety parameters will be included in the study.”

Invitrogen Inks Deal to Acquire Chinese Reagent Firm

Invitrogen is set to acquire Bio Asia, a Chinese reagent supplier, for up to $8 million in cash, and plans to invest more than $20 million in China over the next five years, the company said this week.

Bio Asia, which is based in Shanghai and has branches in Beijing and Guangzhou, as well as 18 sales offices across China, provides sequencing reagents and custom R&D services. The company, which was founded in 1999, has approximately $5 million in revenues.

The acquisition, which was approved by both companies’ boards of directors, is pending approval from the Chinese government. It coincides with new guidelines from the World Trade Organization that will open the Chinese biotech industry to foreign-owned companies, according to Invitrogen.

CytRx Says Small-Molecule Drug Able to Help Regenerate Nerves In Animal Models

CytRx said this week that its experiment amyotrophic lateral sclerosis small-molecule drug arimoclomol was able to accelerate the regeneration of damaged nerves in experimental animals.

“These results support our belief that arimoclomol, by activating the molecular chaperone [proteins], enhances the cell’s natural ability to fix whatever is broken at the cellular level through its natural repair mechanisms,” Jack Barber, senior vice president of drug development at CytRx, said in a statement. “Given that the drug was well tolerated in two prior phase I clinical trials, we are looking forward to initiating our phase II clinical trial for ALS next year.”


The Scan

Study Finds Few FDA Post-Market Regulatory Actions Backed by Research, Public Assessments

A Yale University-led team examines in The BMJ safety signals from the US FDA Adverse Event Reporting System and whether they led to regulatory action.

Duke University Team Develops Programmable RNA Tool for Cell Editing

Researchers have developed an RNA-based editing tool that can target specific cells, as they describe in Nature.

Novel Gene Editing Approach for Treating Cystic Fibrosis

Researchers in Science Advances report on their development of a non-nuclease-based gene editing approach they hope to apply to treat cystic fibrosis.

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.