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Peering into the Crystal Ball for 2020

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.

AGTC Grows Preclinical Pipeline with Gene Therapies for Congenital Hearing Loss, Stargardt Disease

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.

Dicerna, Novo Nordisk Eye 30+ Liver Targets in Up-to-$3.8B RNAi Partnership

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, Dicerna Launch Up-to-$1.67B Hepatitis B Collaboration

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.

On Higher Ground: The Dilemma on Gene Therapy Pricing

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.

Peripheral Vision: Cygnal Looks Beyond the CNS to Develop Drugs

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 Launches Genome Engineering for iPS Cells

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 Shares Surge after Reversal on Alzheimer’s Candidate Aducanumab

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.

Genome Editing Heads to Primetime

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.

From Poetry to Policy: An Interview with Bartha Maria Knoppers

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, NIBRT Partner on Cell Therapy, Single-Use Bioprocessing

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.

Top 10 European Biopharma Clusters

This year’s European cluster ranking includes all countries that appeared on GEN’s 2018 A-List of top 10 European biopharma clusters, but with several significant changes, including a new number-four nation and much of the bottom half of the list.

One-Stop-Shop Genome Editing Product Launched by Inscripta

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.

In AI We Trust? The Future of Genomic Medicine

Genomic medicine is borrowing from Greek theatre a device called God in the Machine. In genomic medicine, the device manifests as AI technology, but it can still resolve complicated situations.

Deep Genomics Identifies AI-Discovered Candidate for Wilson Disease

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.

Top 10 U.S. Biopharma Clusters

Disorders of the brain such as dementia, Alzheimer’s disease, and opioid addiction are most likely to shape the next generation of life sciences clusters

CRISPR-Chip Launched as “Genome Sensor”

As Cardea and Nanosens Innovations merge, CRISPR-chip was made available in an early-access program. By using the CRISPR-chip product, sold under the Nanosens brand, users will be able to use their guide RNAs to “google genomes.”

Going Viral: The Next Generation of AAV Vectors

Adeno-associated virus (AAV) is already gene therapy’s delivery vehicle of choice. And now, more efficient AAVs are being engineered using directed evolution and AI-guided workflows.

Looking Into a Crystal Ball

Gene therapy pioneer Ronald G. Crystal, MD, recalls early setbacks and triumphs, acknowledges the courage of clinical trial patients, and expresses optimism about gene therapy’s future.

Genetics of Same-Sex Behavior Revealed

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.

Merck & Co. Partners with Themis on Measles Virus Vector-Based Vaccines

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.

Top 10 Companies Leveraging Gene Editing in 2019

Venture investors and even some other biopharma giants appear much more optimistic about gene editing-based companies, given the year-over-year increases in total capital raised by many such startups.

BioNTech Boosting Pipeline, Manufacturing, Global Presence with $325M in Series B Financing

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.

PROTACs Developed with U. of Dundee Boost Boehringer Ingelheim’s Cancer Pipeline

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’s Brammer Bio Completing $100M in Expansion Projects

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.

Celyad Faces “Go-or-No-Go” Decisions on Lead CAR-T Candidates

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.

Brexit Concerns Draw Some U.K. Businesses to Ireland

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 Advances mRNA-Based Melanoma Immunotherapy ECI-006

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.

Top 10 Sequencing Companies

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.

The Risky Business of Embryo Selection

An anonymous incubator space just off the New Jersey Turnpike is the unlikely setting for Genomic Prediction, a genetic testing start-up that is daring...
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