Four experts in life sciences and digital health development, commercialization, and investment peered into their proverbial crystal balls to offer insights on “What’s Hot in Life Sciences: A Look at the 2019 Trends and What’s on the Horizon for 2020”—the title of a panel discussion held recently at JLABS@NYC in SoHo.
Gene therapy developer Applied Genetic Technologies Corp. (AGTC) has expanded its pipeline of orphan disease treatments in recent weeks by partnering with Otonomy to develop an early preclinical candidate for congenital hearing loss, and announcing Stargardt disease as the target of its second preclinical ophthalmology program.
Novo Nordisk will apply Dicerna Pharmaceuticals’ GalXC™ platform to discover and develop RNA interference (RNAi) therapies for liver-related cardio-metabolic diseases, including chronic liver diseases, nonalcoholic steatohepatitis (NASH), type 2 diabetes, obesity, and unspecified rare diseases.
Roche will partner with Dicerna to develop and commercialize its lead product, the Phase I chronic hepatitis B virus (HBV) candidate DCR-HBVS, as well as discover and develop additional HBV treatments, through a collaboration that could generate more than $1.67 billion for Dicerna.
Katherine High, MD, the co-founder, president, and CSO of Spark Therapeutics speaks about the commercial success and renaissance of gene therapy. High is the developer of Luxturna, the first approved gene therapy for a hereditary form of blindness called Leber’s congenital amaurosis.
Cygnal Therapeutics, a spinout of Flagship Pioneering, has emerged from stealth mode to develop drugs based on pathways and targets the company has discovered in the peripheral nervous system, with an initial focus on cancer and inflammatory disease.
Synthego has launched a genome engineering service for induced pluripotent stem (iPS) cells, an expansion of automated cell editing capabilities that is designed to achieve extremely high editing efficiency of iPS cells at an industrial scale, and address the dearth of high-quality, physiologically relevant biological models needed for translational medicine.
Biogen saw its shares surge nearly 40% after surprising analysts by announcing that it plans to file for FDA approval of the Alzheimer’s disease candidate aducanumab, being co-developed with Eisai, despite halting two failed Phase III studies of the drug in March. Biogen now says a new analysis of a larger dataset from the trials showed that one study showed effective pharmacological and clinical activity, a finding supported by a subset of patients from the second study, even though it missed its primary endpoint.
A new genome editing method has been developed by David Liu’s lab at the Broad Institute. The technique, called prime editing, can change any nucleotide to any other nucleotide with no double stranded breaks and no donor DNA needed. It is more efficient, makes fewer byproducts, and has lower off-target editing than traditional CRISPR-Cas9 editing.
Canadian lawyer and professor Bartha Maria Knoppers is widely acknowledged as one of the leading authorities on legal and ethical aspects of genetics and genomics research. In the summer of 2019, she gave a talk at the annual CRISPR meeting in Quebec City, an acknowledgement of the growing importance of ethical debate following reports of human germline editing. In this interview, Knoppers discusses her journey to the frontlines of bioethics and CRISPR.
GE Healthcare (GEHC) and Ireland’s National Institute of Bioprocessing Research and Training (NIBRT) are partnering to deliver educational training programs focused on biopharmaceutical and cell therapy development and manufacturing, building on a two-year-old collaboration.
At the SynBioBeta 2019 conference in San Francisco, Inscripta launched the Onyx—a fully automated benchtop instrument for genome-scale engineering. The CRISPR-mediated, massively parallel platform—which includes software, consumables, instrument, and assays enables researchers to engineer microbial libraries containing the full breadth and scope of possible edit types, in their own labs.
Deep Genomics, a Toronto developer of treatments based on artificial intelligence, said today it has successfully used its own AI drug discovery platform to identify its first therapeutic candidate, a therapy for Wilson disease that the company plans to advance into clinical studies in 2021. DG12P1 is an oligonucleotide therapy designed to treat Wilson disease in patients who possess the exon skipping mechanism of Met645Arg.
An international group of researchers have performed the largest and most thorough investigation into the genetics of same-sex sexual behavior to date. In doing so, they lay to rest the idea of “the gay gene” and instead show a genetic contribution of many small genetic effects scattered across the genome.
The partners will develop vaccine candidates based on Themis’ measles virus vector-based platform. The vaccines are designed to deliver multiple selected antigens directly to macrophages and dendritic cells, thus triggering a specific immune response to the selected antigens.
BioNTech's $325 million Series B financing proceeds will be used not only toward advancing its pipeline of immuno-oncology candidates, but adding to its manufacturing capacity and exploring an expanded global footprint as well.
Boehringer Ingelheim says the recently-announced extension of its three-old collaboration with University of Dundee to develop new treatments targeting cancer-causing proteins reflects a commitment to open innovation as well as its ongoing focus on developing oncology as a therapeutic area.
Thermo Fisher Scientific's recently-acquired Brammer Bio is completing $100 million in capital expansion projects, as the viral vector CDMO looks to satisfy growing demand for gene therapies for clinical and commercial use.
Chimeric antigen receptor T-cell therapy developer Celyad says it expects to make some go-or-no-go decisions about its lead CAR-T candidates now in Phase I/II trials, based on data readouts expected by year’s end.
Continuing uncertainty over Brexit has compelled a handful of companies to expand operations in Ireland in recent months, with more contacting the agency that promotes foreign direct investment into the Republic, IDA Ireland.
eTheRNA immunotherapies plans to advance ECI-006 to a Phase I/II trial in the third quarter of this year following positive first-in-human data showing immune response in 35% of patients in an adjuvant setting.
Five years after the $1,000 genome introduction, the sequencing field remains rife with chatter about pushing the cost much lower. The race to drive sequencing costs ever lower may soon give way to a different kind of competition, in which companies one-up each other based on their ability to incorporate more powerful interpretive tools, support embedded applications, or tolerate adverse environments.