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Alnylam presents positive Phase 1 ALN-AT3 trial results in hemophilia at ISTH 2015 Congress

Additional study results from 12 hemophilia A and B subjects demonstrated that subcutaneous administration of ALN-AT3 achieved potent and dose-dependent knockdown of AT of up to 86%. AT knockdown was highly durable, with effects lasting over two months after the last dose, supporting further evaluation of a once-monthly subcutaneous dose regimen.

24 Jun 2015

RXi Pharmaceuticals receives Notice of Allowance from USPTO for novel, self-delivering RNAi platform

RXi Pharmaceuticals Corporation, a biotechnology company focused on discovering and developing innovative therapeutics, primarily in the areas of dermatology and ophthalmology, today announced that it received a Notice of Allowance from the United States Patent and Trademark Office on its novel, self-delivering RNAi platform (sd‑rxRNA).

3 Jun 2015

IDT to host instructive talk on accelerating synthetic biology at ASM General Meeting

Integrated DNA Technologies will host an instructive talk on accelerating synthetic biology at the American Society for Microbiology General Meeting in New Orleans, Louisiana on Sunday May 31, 2015 at 11am CDT.

From Integrated DNA Technologies 28 May 2015

Alnylam files clinical trial application for alpha-1 liver disease, presents data at DDW

Alnylam Pharmaceuticals, Inc. has announced that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial with ALN-AAT, a subcutaneously administered investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease).

19 May 2015

Fifth patient dosed in Benitec's TT-034 Phase I/IIa clinical trial for prevention of HCV infection

Benitec Biopharma, a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," is pleased to advise that the fifth patient in the company's 'first in man', Phase I/IIa dose escalation clinical trial of TT-034 for hepatitis C virus (HCV) infection, has today, been dosed at the Duke Clinical Research Unit.

29 Apr 2015

Alnylam announces clinical data from patisiran Phase 2 OLE study for treatment of patients with FAP

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today initial 12-month clinical data from its ongoing Phase 2 open-label extension (OLE) study with patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy (FAP).

24 Apr 2015

Myc cancer gene allows tumor cells to divide, provokes cell suicide process

Myc cancer gene empowers tumor cells to relentlessly divide but simultaneously, provokes a cell suicide process called apoptosis. Myc controls cells by commanding the expression of every tenth of the genes in the nucleus of a tumor cell. However, in spite of more than two decades of intense research, no Myc motivated killer genes have been found.

21 Apr 2015

Fourth patient dosed in Benitec Biopharma's Phase I/IIa hepatitis C trial

Benitec Biopharma, a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," today announced that the fourth patient in the company's Phase I/IIa dose escalation clinical trial of its lead program TT-034 for treating hepatitis C was dosed at the Duke Clinical Research Unit.

11 Mar 2015

New TAU study may offer hope to people diagnosed with Glioblastoma multiforme

There are no effective available treatments for sufferers of Glioblastoma multiforme (GBM), the most aggressive and devastating form of brain tumor. The disease, always fatal, has a survival rate of only 6-18 months.

25 Feb 2015

R. Bryan Miller Symposium to spotlight special focus on rare disease research

The University of California, Davis Department of Chemistry will spotlight a special focus on rare disease research with this year's 15th annual R. Bryan Miller Symposium. Experts in rare diseases will gather at the UC Davis Conference Center March 5 for a conference highlighting the opportunities and challenges in applying cutting edge technologies and "precision medicine" to better treat conditions that together affect millions of people, especially children.

16 Feb 2015

Parents can pass silenced genes to offspring through a specific mechanism

For more than a century, scientists have understood the basics of inheritance: if good genes help parents survive and reproduce, the parents pass those genes along to their offspring. And yet, recent research has shown that reality is much more complex: genes can be switched off, or silenced, in response to the environment or other factors, and sometimes these changes can be passed from one generation to the next.

4 Feb 2015

Sirnaomics submits STP705 IND Application to CFDA for skin scar treatment

Sirnaomics, Inc. and its affiliate Suzhou Sirnaomics Pharmaceutics, Co. Ltd., together with its partner Guangzhou Xiangxue Pharmaceutical, Co. Ltd., (SZSE: 300147), have formally submitted an Investigational New Drug (IND) Application to the China Food and Drug Administration for STP705, an anti-fibrosis RNA interference (RNAi) therapeutic for prevention and treatment of human skin hypertrophic scars.

5 Jan 2015

Scientists devise powerful algorithm to improve effectiveness of research technology harnessing RNAi

Scientists at Cold Spring Harbor Laboratory have devised a powerful algorithm that improves the effectiveness of an important research technology harnessing RNA interference, or RNAi.

7 Dec 2014

UNC researchers develop new approach to block KRAS oncogene

Researchers from the UNC School of Medicine and colleagues at The University of Texas MD Anderson Cancer Center have developed a new approach to block the KRAS oncogene, one of the most frequently mutated genes in human cancer.

14 Nov 2014

Researchers develop 'smart bomb' to attack B-lineage ALL

Fatih Uckun, Jianjun Cheng and their colleagues have taken the first steps towards developing a so-called "smart bomb" to attack the most common and deadly form of childhood cancer — called B-lineage acute lymphoblastic leukemia (ALL).

6 Nov 2014

Promising new approach for combating colorectal cancer

Scientists at the University of York are working on a promising new approach for tackling colorectal cancer, the second most common cause of cancer-related death.

23 Sep 2014

Internal production of hydrogen peroxide can lead cells to exit cell cycle and become senescent

What happens inside cells when they detect the activation of a cancer-inducing gene? Sometimes, cells are able to signal internally to stop the cell cycle. Such cells are able to enter, at least for a time, a protective non-growth state.

30 Aug 2014

Acromegaly and antisense therapy: an interview with Mark Diamond, CEO Antisense Therapeutics

Acromegaly is a chronic, life-threatening disease triggered by a benign tumour of the pituitary gland causing excessive growth hormone release. Oversupply of growth hormone stimulates liver, fat and kidney cells to produce excess levels of Insulin-Like Growth Factor I (IGFI), which causes abnormal growth of the bones of the hands, face and feet and bodily organs.

26 Aug 2014

Antifungal drug resistance evoked through RNAi-dependent epimutations

Microorganisms like bacteria and fungi can evade treatment by acquiring mutations in the genes targeted by antibiotics or antifungal drugs.

27 Jul 2014

Doctor maps path of discovery for developing potential anticancer agent

The journal Cancer Cell today published research led by Dr. Tak Mak mapping the path of discovery to developing a potential anticancer agent.

18 Jul 2014