Biopharma has a major presence in the Boston-Cambrige area.

Image of Boston for article on area Pharma.

Eighteen of the top 20 Biopharmas have a major presence in Boston/Cambridge, and the region continues to welcome companies of all sizes. Boston/Cambridge ranks number-one in NIH funding (4,735 awards totaling $2.457 billion), VC funding ($6.162 billion in 156 deals), and lab space (26.8 million square feet). [Source.]  

Boston/Cambridge is the home to some of the best universities and medical facilities in the world.  Mass General, Brigham and Women’s and Beth Israel Deaconess are all top ranked nationally.  For higher education, there is Harvard, MI, Boston College and Tufts University.  It is no wonder that the Boston/Cambridge area is one of the top biotech hubs in the world.

My picks for interesting and innovative biotechs are:

Entrada Therapeutics, Boston, has developed a proprietary Endosomal Escape Vehicle (EEV™) Platform that is highly versatile and modular, designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues with an improved therapeutic index.  Their novel approach is designed to address challenges associated with current therapeutics and engage targets previously considered inaccessible and undruggable.

Their current pipeline highlights trials for Duchenne Muscular Dystrophy, Myotonic Dystrophy Type 1, with preclinical products for Pompe and Neurodegenerative Diseases.

Karuna Therapeutics, Boston, strives to deliver more effective and better-tolerated treatments for large and underserved patient populations. Their lead product candidate, KarXT, is currently under development as an investigational treatment for schizophrenia and dementia-related psychosis (DRP).  KarXT, an oral modulator of muscarinic receptors that are located both in the central nervous system (CNS) and various peripheral tissues. KarXT is our proprietary product candidate that combines xanomeline, a novel muscarinic agonist, with trospium, an approved muscarinic antagonist, to preferentially stimulate muscarinic receptors in the CNS.

Checkmate Pharmaceuticals, Cambridge, is investigating the efficacy and safety of vidutolimod in combination with checkpoint inhibitors in patients with various types and stages of cancer, including melanoma and squamous cell carcinoma of the head and neck. Vidutolimod works by two complementary mechanisms that together have a unique ability to drive a strong systemic anti-tumor T cell response. First, the VLP activates an immune response to the VLP, leading to the production of antibodies that deliver the VLP into plasmacytoid dendritic cells (pDC) and other immune cells via specialized receptors called FcR . This provides an initial stimulatory signal to pDC and brings the CpG-A to TLR9 (the receptor for CpG DNA) inside the (pDC). Second, CpG-A stimulates TLR9 in a manner that induces significantly higher levels of type I interferons (IFN-α and others) in pDC resulting in a stronger anti-tumor T cell response, as compared to other innate immune activators.

Goldfinch Bio, Cambridge, is applying precision medicine to discover, develop and commercialize disease-modifying kidney disease treatments. Their approach enables them to uncover subsets of patients based on genetic and environmental factors within heterogenous kidney diseases. They have a robust pipeline of novel, clinical-stage, precision medicine product candidates, including or lead program, GFB-887 for TRPC5-mediated FSGS. All of our programs target kidney diseases with significant unmet medical needs.

oRNA, Cambridge, was built on groundbreaking research from Prof. Daniel Anderson’s lab at MIT, their oRNATM technology is the next revolution in RNA therapeutics: synthetic circular RNA efficiently generated by an elegant and proprietary molecular platform. oRNATM circular RNAs lack caps and tails and are always full-length, and are made by combining nature-driven insights with rational design. They are easily made, well-tolerated, and are engineered to express therapeutic proteins through original and proprietary mechanisms.

The potential applications of oRNA’s technology are far reaching, including cancer, autoimmune diseases, regenerative medicine, and genetic disorders. oRNAis currently evaluating where their medicines can make the biggest difference, with an initial focus on treating challenging diseases where other modalities have fallen short. As an initial focus, oRNA believes it can apply its oRNATM technology to potentially address the limitations of current immunotherapies by delivering chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (isCARTM therapy).

Inhibikase Therapeutics, Boston, has a pipeline including multiple products developed from their proprietary RAMP™ drug innovation and prodrug technology engines. Their programs are focused on halting and reversing the effects of neurodegenerative diseases inside and outside of the brain.  They are also developing host-targeted therapeutics for neurological infections in the brain. Products in the current pipeline target Parkinson’s and the GI tract, chronic myeloid leukemia, progressive multifocal leukoencephalopathy