Volume 13 Issue 2, June 2019

At one of its most exciting phases of growth, the cell and gene therapy market has gained momentum in dealmaking, product sales and big pharma interest, all of which are analyzed in this feature.

Over the past year, the biopharma industry has witnessed some landmark approvals, high-value merger and acquisition deals and phenomenal venture funding in the US. In this feature, we chart the key trends in biopharma dealmaking since June 2018.

Plentiful acquisitions, increased financing and pioneering digital health approvals all shaped the medtech industry in 2018. With nontraditional players such as Apple and Google engaging in the field, the industry looks set to evolve further.

Acting as the commercialization platform in partnership with major academic and research institutions, Aptorum has in-licensed a number of infectious-disease-targeted compounds and methods from Hong Kong academic institutions.

Evox Therapeutics is harnessing the natural delivery capabilities of extracellular vesicles to develop an entirely novel class of biotherapeutics.

Relmada Therapeutics is developing REL-1017, a phase 2, noncompetitive N-methyl-D-aspartate receptor antagonist for the treatment of depression and Rett syndrome.

ENT-01, an orally administered compound in phase 2 trials, is being developed to repair the dysfunctional gut–brain axis in patients with neurodegenerative disease.

TetraGenetics’ breakthrough technology produces unprecedented levels of correctly folded and functional membrane-spanning proteins that enable the discovery of novel therapeutic antibodies.

Athira Pharma, Inc. is advancing a novel, potentially transformative approach for the treatment of Alzheimer’s disease and other neurodegenerative diseases. Unlike other products approved and in development, Athira’s drug candidates hold the potential to improve dementia symptoms and reverse or halt the degenerative process.

Fortuna Fix develops ethical, directly reprogrammed neural cell therapeutics manufactured on proprietary platform robotics allowing end-to-end manufacturing at high precision and scale.

Over the past two decades, Wallonia has emerged as a global powerhouse of cell therapy and regenerative medicine research and development.

With two innovative products in late-stage development for the treatment of delayed-union fractures, spinal fusion and knee osteoarthritis, Bone Therapeutics is expanding its pipeline, including through research and development and commercialization partnerships, to help patients improve mobility.

Using a multipronged strategy, Celyad is driving the development of next-generation chimeric antigen receptor-T cell-based therapies to treat cancer.

Promethera Biosciences’ HepaStem platform offers the first allogeneic off-the-shelf, liver-derived stem cell therapy for severe liver diseases that could become an alternative to liver transplantation.

PDC*line Pharma is developing cancer vaccines derived from a proprietary allogeneic human plasmacytoid dendritic cell line.

CORESTEM launched the world’s first stem cell therapy for amyotrophic lateral sclerosis in South Korea in 2015. The company is now seeking out-licensing partners as it works toward product approvals in the US and Europe.

Nikon, a leading provider of advanced optical solutions, offers a vast array of optical OEM components to enable superior imaging system performance.

Legend Biotech is a global, clinical-stage biopharmaceutical company with integrated end-to-end capabilities focused on discovery and development of novel cell therapies.

enGene has developed a proprietary nonviral gene delivery platform and Gene Pill technology to deliver orally administered gene therapies to mucosal tissues, creating new opportunities for treating hard-to-reach tissues across many indications.

With burgeoning research into cell-based therapies, Longboat Amniotics has developed a novel process for the retrieval and processing of tissue-specific neonatal quality stem cells from amniotic fluid.

Building on its history of successful collaborations, Sanofi Pasteur, the vaccines global business unit of Sanofi, is seeking partners with a common drive for excellence and pursuit of innovation.

Sobi is an integrated biopharmaceutical company with capabilities throughout the value chain and a strong track record of commercializing medications in complex markets. While Sobi continues to develop a sustainable research and development base, its proven value-driven platform for launching products for rare diseases offers potential partners strengths in the field. Sobi is looking for opportunities to inlicense late-stage biologics.

Combine your knowledge with GSK’s to help develop innovative vaccines together.

TAGCyx Biotechnologies uses its proprietary Xenoligo technology platform to generate novel DNA aptamers containing an artificial nucleotide that have high affinity and selectivity for target proteins. TAGCyx is building a pipeline of Xenoligo aptamers and aptamer–drug conjugates, and is looking for global pharmaceutical partners.

Harnessing its unique CriPec technology, Cristal Therapeutics is improving the treatment options for patients with a range of solid tumors by creating transiently stable nanomedicines.

Elucida Oncology is developing ultrasmall (<10 nm) hybrid silica particles, C'-Dots, for guiding highly targeted image-directed surgical treatment and/or efficient delivery of small-molecule therapeutic agents and radio therapeutics.

Oncopeptides is developing melflufen, a first-in-class peptide-conjugated alkylator for the treatment of relapsed and refractory multiple myeloma.

YUMAB offers fully human antibody development from target discovery to lead. Building on 30 years of successful research and development by its founders and six years in the market, the company is looking for additional partners interested in customized business and research service solutions for the development of antibody therapeutics.

Building on a century of experience in developing advanced optical solutions, Nikon embarked on a quest in 2017 to help accelerate clinical and diagnostic imaging innovation.

Digital health company Medable has created the first Human Digitome, a digital platform that represents human health and disease and is built to provide real-world data for personalized and predictive medicine.

AI Personalized Medicine

Avectas is developing Solupure, a next-generation cell-engineering tool for ex vivo immune cell therapy.

The first artificial intelligence drug discovery company with an in-market commercially validated platform.