Archive by category | Regulatory

Alnylam launches era of RNAi drugs

Alnylam launches era of RNAi drugs

On August 10, the US Food and Drug Administration approved the first RNA interference (RNAi) therapeutic, a treatment for polyneuropathy caused by transthyretin (TTR) amyloidosis from Alnylam Therapeutics. The go-ahead for Onpattro (patisiran) sees the RNAi field clear an approval hurdle considered unlikely as recently as six years ago, when pharma exited the RNAi field en masse. The US approval, with Europe expected to follow by early September, is “a major milestone,” says Anastasia Khvorova, an RNAi researcher at the University of Massachusetts in Worcester. Onpattro has an excellent safety record, but there are lingering concerns about potential long-term toxicity from newer, more potent RNAi therapeutics. And the field as a whole still faces investor skepticism in the wake of a decade of clinical trial failures.  Read more

Will the EU deregulate gene-edited plants?

Will the EU deregulate gene-edited plants?

At the beginning of the year, the advocate general of the Court of Justice of the European Union (CJEU) issued an opinion that plants created using new plant breeding techniques, including gene-editing platforms like CRISPR, TALENs and the like, are eligible for the so-called mutagenesis exemption. This exemption relates to rules the European Union uses to regulate the release and marketing of genetically modified organisms (GMOs), which are outlined in Directive (2001/18/EC), originally drafted in 2001. The exemption covers any plants considered ‘safe’ or produced using techniques that have a history of safety, including plants derived from traditional mutagenesis (hence the mutagenesis exemption).  Read more

The Developing World Needs GMOs

The Developing World Needs GMOs

The need to feed growing populations in developing countries, especially countries in Africa, must be met by increasing the yields of crops. Also, climate-change related problem such as drought continue to worsen hunger problem and humanitarian crisis in the continent. Genetically modified organisms (GMOs) could greatly help with these issues, yet resistance persists in Europe and Africa both.  Read more

Turning science and technology into a priority in Brazil

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In a previous contribution to this blog, I said that science and technology is not a priority in less developed countries, including Brazil. I recently described why this is in Scientia & Ricerca. Brazil’s government claims it cannot treat science and technology different from other areas. If it cannot double the investments in other areas, it cannot double the investment in science and technology. Since the Gross National Product (GNP) of Brazil cannot double in one year we are stuck with investments in science and technology at 1% of GNP historically.  Read more

Gottlieb on pricing, competition and new therapeutic modalities

While freelancing for Nature Biotechnology, I recently talked to US Food and Drug Administration (Rockville, MD) Commissioner Scott Gottlieb. The conversation ranged from pricing, to market competition to new therapeutic modalities like gene therapy coming down the pipeline. A more extended extract of our discussion is also available in the News Feature.  Read more

Heparin, Brazil and innovation

Heparin, Brazil and innovation

An article published at the Brazilian Journal  of Cardiovascular Surgery compared all heparins manufactured by Brazilian companies to Liquemine, manufactured by Hoffman La Roche. Heparin is a complex carbohydrate that was introduced to control thrombosis during extra-corporeal surgeries during the 1930s by Clarence Crafoord. It’s been nearly a century and there is no substitute for the drug. No surgeon performs chest surgery without heparin at hand.  Read more

GM mosquitoes fire first salvo against Zika virus

GM mosquitoes fire first salvo against Zika virus

A Brazilian city in January became the first to approve a program to grow transgenic mosquitoes for their release into the environment as a public health measure against the Zika virus outbreak. The city of Piracicaba in Brazil said it would work in collaboration with the Milton Park, UK–based Oxitec to scale up release of transgenic Aedes aegypti mosquitoes, the main vector for the Zika, dengue and Chikungunya viruses, and build a new production facility there. With no Zika vaccine in sight, government officials across the globe are pondering strategies that suppress the mosquito populations to thwart the spread of infection. US and Chinese regulators both green-lighted field tests for vector control strategies involving nontransgenic Aedes albopictus mosquitoes.  Read more

Law 13123 and access to the Zika virus

Law 13123 and access to the Zika virus

Last year I published an eBook by Schollars Press with an Last year I published an eBook by Schollars Press with an acknowledgment for this blog. The eBook was titled, Topics About Biotechnology in Brazil. One such topic was The Future of Sustainable Use of Biodiversity in Brazil.  Read more

Your input needed on rethinking US regulation

Your input needed on rethinking US regulation

With little fanfare back in the summer, the US Science and Technology Policy Office (OSTP) issued a request for information (RFI) on the need to update the US oversight of biotech products. The last time the Coordinated Framework for the Regulation of Biotechnology (51 FR 23302; June 26, 1986) was updated was in 1992 when technology, to put it mildly, looked a little different.  Read more