Category Archives: Rnai

Regenerative medicine with mRNA to rejuvenate aging cells

Studying the muscle stem cells in boys suffering fromDuchenne muscular dystrophy, Stanford scientist Helen Blau found that their telomeres–the protective caps at the end of chromosomes–were unusually short. Now, in a new study, Blau and a small group of colleagues say that they have developed a method to use modified messenger RNA to lengthen telomeres, opening a pathway to countering the effects of aging in cells–a process that might apply to a range of diseases from DMD to diabetes and heart disease.

Telomeres naturally shorten every time a … Read more

How to Find a Cancer Treatment Trial


This guide will help you look for a cancer treatment clinical trial. It does not provide medical advice and should not be used in place of advice from your doctor or other members of your health care team. Your health care team and your loved ones, if you wish, can assist you in deciding whether or not a clinical trial is right for you. The decision to take part in a clinical trial is yours alone to make.
This guide takes you through the following steps:

Step 1: Understand Clinical … Read more

Type-2 diabetes and gene therapy by deactivation of gene WDR13

CCMB scientists reveal deactivation of gene WDR13 controls type-2 diabetes & improves immune system in rats.

Research by scientists at Center for Scientific and Industrial Research-Center for Cellular and Molecular Biology (CSIR-CCMB) Hyderabad has revealed that gene WDR13 is the major cause of type-2 diabetes and it can be effectively controlled and cured by de-activating the gene.

A team of scientists working under the leadership of Dr Satish Kumar, Head Department of Animal Genetics Research Centre have found that a gene called WDR13 is responsible for causing type-2 diabetes and … Read more

Dry and Wet AMD, cure and possibility

Note – I am thankful to  eyedoc for his key opinion and note .

AMD is the leading cause of blindness in the United States, affecting 10 million Americans over age 60. It’s a condition where the central part of the retina wears out, causing vision loss. Everyone is at risk for macular degeneration as they age, but people who have a family history or who smoke are more likely to develop it.

Although there is no cure for AMD, there is a procedure which helps end-stage disease sufferers by … Read more

Rare Disease and RNAi Technology?

A disease or disorder is defined as rare in Europe when it affects fewer than 1 in 2000.
A disease or disorder is defined as rare in the USA when it affects fewer than 200,000 Americans at any given time.

One rare disease may affect only a handful of patients in the EU (European Union), and another touch as many as 245,000. In the EU, as many as 30 million people alone may be affected by one of over 6000 rare diseases existing.

80% of rare diseases have identified genetic … Read more

DSMB recommendation and HCV trial update of Benitec.

Sydney Australia, 21 July 2014: RNAi therapeutics company Benitec Biopharma
Limited (ASX: BLT, OTC: BTEBY) is pleased to provide an update regarding
recommendations from the Data Safety Monitoring Review Board (DSMB),
observations from the first patient dosed, and patient recruitment for the USJbased
Phase I/IIa TTJ034 clinical trial. TTJ034 is a ddRNAiJbased therapeutic, designed to
treat and potentially cure hepatitis C (HCV) with a single injection.
DSMB recommendation and observations from first patient dosed
Following review of data from the first patient treated with TTJ034 on 29 May 2014,
the … Read more

mRNA-based vaccines

TUBINGEN, Germany, July 1, 2014 /PRNewswire/ — CureVac, a German clinical-stage biopharmaceutical company, today announced the execution of an exclusive license agreement with Sanofi Pasteur S.A., the vaccines division of Sanofi , to develop and commercialize an mRNA-based vaccine against an undisclosed pathogen.

In 2011, CureVac and Sanofi Pasteur signed a collaboration and license option agreement for several pre-defined pathogens. CureVac met all pre-agreed milestones and acceptance criteria relating to these agreements, and therefore Sanofi Pasteur exercised its first option and extended its exclusive and non-exclusive options on all five … Read more

Alnylam and Benitec

Alnylam (ALNY) is a $5.1B company and Benitec Biopharma (BTEBY, BNIKF) is a $120M company. Both are biothech companies working in the field of RNAi (RiboNucleic Acid Interference). Both have access to a wide patent estate: Alnylam’s estate covering Small Interfering RNA (siRNA) and Benitec’s estate covering DNA Directed RNAi (ddRNAi and shRNA).

In theory they are competitors, each looking to silence genes which produce harmful levels of proteins. However, in reality this competition has not been manifest. Alnylam has concentrated its pipeline efforts primarily on rare … Read more