Archive by category | News

Biotech leaders call for free press

To the Editor: We, the undersigned, are biotechnology executives, entrepreneurs, academic leaders and investors. We are gravely concerned about trends in the United States that are undermining our news media, such that more than 300 news publications across the country recently found it necessary to run coordinated editorials in defense of the First Amendment’s guarantee of freedom of the press.  Read more

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

First approval in sight for Novartis’ CAR-T therapy after expert panel vote

Emily Whitehead, 12, the first child to receive CAR-T cell therapy has been cancer-free for five years.

On July 12, in a historic move, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 10–0 in favor of approving CTL019 (tisagenlecleucel), a CD19-targeting chimeric antigen receptor (CAR) T-cell therapy developed by Novartis for treating relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) in children and young adults. The committee’s unanimous backing opens the door to approval of the therapy by the US Food and Drug Administration (FDA) on or before October 3. The US, which has lagged behind Europe in approving both gene therapy and cell therapy, has now taken a lead in adopting an innovative immuno-oncology treatment that combines elements of each modality—and which addresses a real and sizeable unmet need.  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

CRISPR germline editing reverberates through biotech community

CRISPR germline editing reverberates through biotech community

The organizers of a recent meeting in Napa, California, to consider the broad societal implications of clustered, regularly interspaced, short palindromic repeats (CRISPR) genome editing have succeeded in their primary goal of stimulating public debate on the ethical issues raised by the technology. Although the event, held on January 24, took place behind closed doors, a subsequent commentary from its leading participants—plus two influential non-attendees, George Church of Harvard Medical School in Boston, and Martin Jinek of the University of Zurich—prompted widespread media coverage (Science 348, 36–38, 2015). The group has called for a broadly based discussion of the potential merits and risks of the technology and a global moratorium on germline applications, until such time, if ever, responsible uses can be identified.  Read more

Nonbrowning GM apple cleared for market

Nonbrowning GM apple cleared for market

The US Department of Agriculture (USDA) on February 13 approved the first genetically modified (GM) apple developed to resist browning.  The Arctic apple’s go-ahead is a notable achievement for Canadian firm Okanagan Specialty Fruits, as the small biotech is one of the few to have successfully moved a GM plant through the regulatory process on its own. Indeed, two weeks after the apple’s launch, Okanagan announced its acquisition by Germantown, Maryland-based Intrexon, a synthetic biology company. Okanagan’s stockholders will receive $10 million in upfront cash and $31 million in Intrexon common stock. Industry observers now question whether the Arctic apple will carve a niche in the fruit tree industry or get caught up in the GM labeling debate.  Read more

$1-million price tag set for Glybera gene therapy

$1-million price tag set for Glybera gene therapy

The first gene therapy approved in the Western world is set to go on sale in Germany at a cost close to $1 million per treatment. The record-breaking price tag came to light in November 2014, when Amsterdam-based Uniqure and its marketing partner Chiesi, of Parma, Italy, filed a pricing dossier with German authorities to launch Glybera. A few weeks later, the focus on gene therapies sharpened further when Cambridge, Massachusetts–based Bluebird Bio presented striking early clinical data from four beta-thalassemia patients treated with its Lentiglobin BB305 gene therapy, at the American Society of Hematology meeting in San Francisco. Within three months, these patients had begun producing sufficient hemoglobin to reduce or eliminate the need for blood transfusions.  Read more

First US biosimilar edges toward market

First US biosimilar edges toward market

Sandoz’s biosimilar drug is about to become the first drug approved under the FDA’s biosimilar pathway. On January 7, the US Food and Drug Administration’s (FDA’s) oncologic drugs advisory committee voted unanimously to recommend approval of Basel-based Sandoz’s granulocyte colony stimulating factor filgrastim, a biosimilar of Thousand Oaks, California–based Amgen’s $1.2-billion seller Neupogen. Six years after Sandoz’s biosimilar filgrastim was approved in Europe, with 7.5 million patient-exposure days to support approval, it would seem a reasonable bet that the drug, whose proposed brand name is Zarxio, will get a green light on March 8, as Nature Biotechnology goes to press.  Read more