Volume 10 Issue 5, November 2016

BioPact’s MGMR (Medical Grade Molecular Rebar) offers a safe and scalable nanoparticle platform for controlled and targeted delivery of a wide range of therapeutics.

MeiraGTx is opening new frontiers in gene therapy by pioneering next-generation gene-regulation platforms to transform the application of gene therapy.

The potential of nucleic-acid-based drugs to halt the production of disease-associated proteins or to promote the production of therapeutic proteins has spurred a string of recent deals.

The clinical-stage biotechnology company has pioneered the development of 3D spherical nucleic acids to treat a broad range of inflammatory disorders affecting multiple organs in the body.

Over 80% of those who contract hepatitis B virus (HBV) recover from the disease, but some 350 million people worldwide are not able to control the virus and develop chronic infection that can seriously damage the liver — over 750,000 people die from complications every year.

Since the days of Watson, Crick, and Franklin, DNA has hogged the limelight, overshadowing RNA. Recently, however, RNA has claimed center stage as a multi-talented player, dialing gene expression up or down and even altering the genome through gene editing

The potential of gene silencing via RNAi has long been recognized, and innovations in the delivery of RNAi therapeutics by Dicerna Pharmaceuticals have enabled potent, long-acting gene silencing with a single subcutaneous injection for liver-associated diseases.

After safety issues dashed hopes several years ago, calcitonin-gene related peptide has re-emerged as a prominent target, catalyzing deals and potentially reinvigorating the generic-dominated migraine market.

SpineThera is developing a sustained-release injectable drug for back pain with the aims of improving safety and reducing the need for repeated epidural injections of corticosteroids.

By overcoming formulation challenges and applying its controlled-release delivery systems, Intellipharmaceutics is creating new options for pain therapies.

Voyager Therapeutics is developing potentially life-changing gene therapies to treat advanced Parkinson’s disease and other serious neurological disorders with a significant unmet medical need.

Building on success in the oncology field, Astex Pharmaceuticals is applying its fragment based drug discovery (FBDD) platform to new neurodegenerative disease targets.

AMRI offers a refined integrated drug discovery platform designed to accelerate customers’ programs from idea to investigational new drug (IND).

Cognition Therapeutics, Inc. has a first-in-class, clinical-stage, highly brain-penetrant and orally bioavailable small molecule designed to halt the initiation and progression of Alzheimer’s disease.