Category: Uncategorized

For the past ten years, Austin, Texas has been swiftly growing as a high-tech hub.  Oftentimes referred to as the Silicon Valley of the South, Austin has attracted giants in technology including Google, Apple and of course Dell was founded there.  In the past few years, Austin won the contract for the new Tesla Mega Factory.  Recent reports state that Tesla electric cars will begin rolling out of the new factory by the end of the year, bringing thousands of jobs to the area.

Another sector to see rapid expansion in the Austin area is biotechnology.  With a highly educated workforce, a major university, a new medical school and the desire for people to live in Austin, there is no shortage of skilled employees.  In addition, the state of Texas has dedicated funds to support cancer called the Cancer Prevention and Research Institute of Texas (CPRIT).  Since 2008, CPRIT has funded $2.8B in grants for Research, Prevention and Product Development Research for cancer in Texas.

Some standouts in the Austin small pharma/biotech industry include Cassava Sciences, Lumos Pharma, Savara Pharmaceuticals, XBiotech, GenPrex, and Curtana Pharmaceuticals.

CASSAVA Sciences
In today’s Austin market, Cassava is the clear stand out with simufilam in two Phase III trials for Alzheimer’s Disease.  Simufilam improves the function of multiple brain receptors and exerts powerful anti-neuroinflammatory effects. A small company with a potential blockbuster on the horizon and CEO Rami Barbier and CFO Eric Shoen has done a brilliant job financing the company over the past two years. Put this company on your watchlist!

Lumos Pharma is a clinical-stage biopharmaceutical company committed to identifying, developing and commercializing life-changing therapies for patients and families living with rare diseases.  Their current candidate, LUM-201 is for PGHD – is in a Phase II trial for pediatric growth hormone deficiency.  Treatment of growth hormone deficient children is currently limited to daily injections of growth hormone. Missed injections reduce therapeutic effectiveness, resulting in suboptimal growth. Four other growth-related indications are in the pipeline. CEO Hawkins is a serial entrepreneur having built and sold several companies. 

Savara Pharmaceuticals is a rare lung disease company.  Their leading program, Molgradex, is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase III development for autoimmune pulmonary alveolar proteinosis (aPAP). CFO Dave Lowrence secured financing with Bain Capital which extends their runway beyond the Phase III readout. Savara/Molgradex could be the first FDA approved therapy for aPAP. 

XBiotech manufactures True Human™ antibodies that are derived from individuals with the most potent natural immunity to certain diseases. The company identifies unique genetic information of immune antibodies from donor blood. This genetic information is used for production of antibody therapies without modification.   This genetic information is used for production of antibody therapies without modification.   

GenPrex technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. The lead product candidate, REQORSA, will be administered with targeted therapies and with immunotherapies for NSCLC – non small cell lung cancer. 

Curtana Pharmaceuticals is developing highly targeted therapies for the treatment of brain cancer, including glioblastoma in adults and pediatric high-grade glioma and medulloblastoma in children.  They are advancing a novel small molecule therapy, CT-179, which has been shown to significantly prolong survival in relevant animal models. The target is Olig2, a cancer stem cell-associated transcription factor that is critical for tumor initiation and growth. CT-179 will enter clinical development in H1 2021.

Austin remains attractive to growing biotech companies.  Does it have the capacity to become a biotech giant?  If we factor in the relatively close proximity of its sister cities of Dallas, Houston and San Antonio, the horsepower ramps up significantly. 

World renowned universities such as The University of Texas, Texas A&M, Baylor and Rice, the top cancer center in the world (M.D. Anderson), and large pharmaceutical companies such as Allergan, AstraZeneca, Pfizer and Novartis, Austin is poised to become the hub for small to medium pharma/biotech.  Dallas takes large pharma, Houston excels in patient treatment and San Antonio leads in government facilities. 

Austin wins by tapping in to all three and building a world class platform fostering growth for smaller biotechs.  Locals like to say, “keep Austin weird” but biotech entrepreneurs should take a hard look at Austin!

Health care and life sciences are major industry sectors in Houston driven by world-class institutions and professional talent. Houston is home to the largest medical complex in the world, the Texas Medical Center, which provides clinical health care, research and education at its 61 institutions. Houston has more than 1,760 life sciences companies, cutting edge hospitals, health facilities and research institutions with a workforce of more than 320,000 people in health care, biotech and related fields in the area. (Source)

Here are my picks for innovative biotech companies in Houston:

Coya Therapeutics, Houston, is developing disruptive, first in class, autologous regulatory T-cell (Treg) and allogeneic exosome therapies – leveraging the seminal discoveries from the laboratory of Stanley Appel M.D., in which dysfunctional Tregs modulate neurodegenerative and autoimmune diseases.  

Coya’s pipeline targets devastating diseases such as ALS, Dementia and Alzheimer’s.

CAVU Biotherapies, Houston, provides immune-based solutions to treat cancer and autoimmune diseases in dogs. Their vision is to change the world by keeping pet families intact longer while becoming the global leader in immune-guided personalized medicine.  Their initial focus is on canine cancer, and then translating the personalized immunotherapies to treat dogs, cats and horses living with allergies, cancer and autoimmune diseases.

Saranas, Houston, has developed the Early Bird Bleed Monitoring System that detects bleeds for endovascular access procedures the moment they happen, helping to preserve patient outcomes and provide an additional level of assurance for the patients most vulnerable to bleeding events.

Tvardi Therapeutics, Houston, is focused on the development of inhibitors of STAT3, a key signaling molecule positioned at the intersection of many pathways controlling gene networks integral to the survival and immune sequestration of cancer cells as well as to the pathogenesis of many inflammatory and fibrotic conditions.

The company’s lead product, TTI-101, is currently being studied in a first-in-man Phase 1 trial of patients with advanced solid tumors who have failed all lines of therapy. To date, TTI-101 has demonstrated an excellent safety profile and clinical activity.

Greenwich LifeSciences, Inc., Houston, is a public biopharmaceutical company that is developing GP2, a novel peptide immunotherapy, and is planning to commence a Phase III clinical trial.

GP2 is derived from the HER2/neu protein, which is expressed in a variety of common cancers, including breast cancer.  Tumors with elevated expression of HER2/neu protein are highly aggressive, resulting in an increased disease recurrence and a worse prognosis.  In a Phase IIb clinical trial in the HER2/neu 3+ adjuvant setting, no breast cancer recurrences were observed after median 5 years of follow-up if the patient was fully immunized.  In addition, GP2 treatment is well tolerated and no serious adverse events related to GP2 immunotherapy were reported.

ImmunoGenesis, Houston, is bringing hope to cold tumor treatment.  Nearly 40% of people will have a cancer diagnosis in their lifetime. Four of the most common cancers resulting in cancer death in the United States are those that are considered cold tumors. The few treatments that are available have poor efficacy. Their platform may change this.

Their novel PD-L1/PD-L2 dual-specific inhibitor is a platform molecule around which several promising treatments for cold tumors can be built. This is the first antibody to target PD-L2. This vision puts ImmunoGenesis at the forefront of scientific exploration.

Built utilizing the platform, their lead asset was engineered with robust effector function. Another PD-L1/PD-L2 antibody from this platform is being developed as a tumor-selective delivery vehicle for our potent stimulator of interferon genes (STING) agonist.

Japan, the Land of the Rising Sun, holds a number-two ranking in Asia, and leads the continent in biotech and pharma patents (14,414 listing at least one Japanese inventor, according to WIPO), as well as a firm number-two in R&D. (Source)

Here are my top picks for innovative biotechs:

Chordia Therapeutics, Fujisawa, is a start-up company that develops next-generation anticancer drugs based on solid scientific evidence. Their anti-cancer drug pipeline focuses on RNA regulation abnormalities that have been found as a new feature of cancer. Recent studies have newly discovered that RNA regulation is altered in cancer, and show that this RNA regulation abnormality is significantly involved in the development and progression of cancer.

Their lead asset, CLK CTX-712, carries out Phase I of First in Human.

United Immunity, Kobe, develops novel combination immunotherapies utilizing our potent anti-tumor T cell activating nano-immunotherapy “T-ignite”, we challenge conquest of intractable solid cancer, especially “cold tumor”, where conventional therapies are not effective for. The same technology is being used for the development of a next generation SARS-CoV-2 nanoparticle vaccine.

xFOREST Therapeutics, Kyoto is creating a paradigm shift in drug discovery with its unique technological assets “FOREST”, setting RNA structures as drug discovery targets. FOREST are technology platforms that integrate massively parallel biochemical analysis systems of up to 1 million species of RNA structures and in silico analysis pipelines. These comprehensive analyses include various RNA structures, regardless of their source: organ, species, or virus. As a result, the technologies have enabled the search for highly specific RNA-binders, empowering the rapid and efficient discovery of RNA-targeting drugs.

Rena Therapeutics, Tokyo, markets HDO, Hetero-duplex oligonucleotide developed by Professor Yokota Takanori of Tokyo Medical and Dental University.  This was developed to solve the inherent issues with nucleic acid drugs including:  delivery issues, toxicological issues and blood stability.

By establishing the third platform technology next to Ionis Pharmaceuticals, Inc (ASO) and Alnylam Pharmaceuticals, Inc (siRNA), we will contribute to the creation of new nucleic acid drugs, and to deliver therapeutic drugs to patients as soon as possible 

CYTLIMIC, Cytotoxic T Lymphocyte Immunotherapy in Cancer, Tokyo, is a biotechnology company developing immunotherapy products for the treatment of cancer, including a cancer peptide vaccine. CYTLIMIC’s products aim to activate immune systems to attack cancer cells while enabling patients to maintain a high quality-of-life during treatment.

France ranks third in Europe, behind the United Kingdom and Germany, in the number of biotechnology companies operating within its territory. With 400 firms employing about 6,000 people, more than half of whom work in research and development. (Source)

Here are my picks for innovative biotechs in France.

Novilase, Evry, is a leading company in image-guided laser tumor therapy.  Their vision is to establish laser therapy as a treatment alternative to surgery for tumors, benefiting the patient. Currently Novian Health is focused on providing women with a minimally invasive alternative to surgery for cancerous and non-cancerous breast tumors. The Novilase laser procedure is performed in a breast center with ultrasound imaging guidance in a manner similar to a breast biopsy.

Adocia, Lyon, BioChaperone® system optimizes the performance of therapeutic proteins.  BioChaperone® is a Molecular Delivery System for Therapeutic Proteins.Adocia designed the BioChaperone® platform technology from innovative polymers, oligomers and small organic compounds. By forming a physical complex with proteins, BioChaperone® protects them from enzymatic degradation and enhances their performance.

BioChaperone® improves “chaperoned” proteins’ efficiency through its stabilizing and solubilizing effects:

• Acceleration of action and modification of action duration
• Enhancement of absorption and bioavailability
• Decreased dosage and frequency of administration

OSE Immuno Therapeutics, Nantes, is a clinical-stage biotechnology company in immunotherapy. They are focused on developing and partnering therapies to control the immune system for immuno-oncology and autoimmmune diseases.

Three areas of expertise are being developed within our immunology research platform:

• T-cell based vaccination
• Immuno-Oncology (focus on myeloid targets)
• Auto-Immunity & Inflammation

Mnemo, Paris, is using its EnfiniT platform, an integrated drug discovery engine that combines:

• A proprietary combination of epigenetics tools assembled into powerful T cell engineering technologies
• A revolutionary target ID engine that sources newly-identified junctions between exons and transposable elements (JETs)
• Best-in-class manufacturing

Mnemo will transform the body’s immune response to overcome currently incurable diseases.

Genclis SA, Vandoeuvre-lès-Nancy, is a biotechnology company focused on developing therapies for both human and animal health using Transcription Infidelity (“TI”). TI is a patented process that begins by analyzing differences between RNA and DNA sequences to identify translated proteins capable of causing the production of either Immunoglobulin E (“IgE”), the antibody responsible for most forms of allergies or Immunoglobulin G (“IgG”), the workhorse of immunity that accounts for most of vaccine efficacy. Genclis uses its proprietary patented platform technology to develop therapies that elicit self-produced antibodies that block disease-causing proteins irrespective of their endogenous or exogenous origin.

The Indian biotechnology industry in India is divided into five major segments.  The percentage share of the biotechnology segments is:

• Bio-Pharmaceuticals: 62%
• CRO/BioIT/Research: 15%
• Bio-agriculture: 16%
• Bio-Industrial: 7%

India is also one of the first countries to have a department dedicated to the Biotechnology industry. Moreover, the Department has also set up BIRAC (Biotechnology Industry Research Assistance Council) which is a not-for-profit agency to strengthen and empower emerging Biotechnology enterprises to undertake strategic research and innovation, by handholding them from ideation to the commercialization of their products/ technologies. (Source)

With many choices, here are my top 5 picks for innovative small biotech and pharma companies.

Stempeutics Research, Bangalore,  is a late-stage life science company focused on developing and commercializing novel therapeutics based on adult stem cells. It was founded by the Manipal Education and Medical Group in 2006. Stempeutics strength lies in developing innovative stem cell products by nurturing cutting edge research and clinical applications through dedicated efforts of its highly qualified team.

Currently it is focusing on the following three innovative products: Stempeucel® drug, Stempeucare™ cosmetics and Stempeutron™ medical device.

Curadev, Noida, tumors subvert immunity in a variety of ways. They can hide their very existence from innate cellular alarm systems and circulating surveillance immune cells, frustrate the buildup and amplification of immune responses and subvert established inflammatory responses by evoking potent immunosuppressive factors.

​The recent approvals of inhibitors targeting checkpoint blockade are the first steps in the establishment of host directed immune therapies as durable methods for the treatment of a number of cancers. The coming decade will see the emergence of new generations of immune system targeted oncology drugs which can be used as monotherapy or in combination with other IO agents or existing anti-cancer drugs.

Biocon, Bangalore, pioneering spirit paved the way for biotechnology in India, and we continue to apply the same spirit in finding novel approaches to improve patient outcomes today for a better tomorrow. This drives us to continuously find new ways to treat diabetes, cancer and autoimmune diseases. Our R&D focuses on prevention, alleviation and treatment and our medicines improve the lives of millions of patients in over 120 countries, by giving them access to life-saving therapies and relief.

Bicara Therapeutics, Bangalore, Epidermal growth factor receptor (EGFR), a protein expressed on the surface of several tumor types, is one of the most extensively validated tumor-associated antigens.

Monoclonal antibodies directed at EGFR suppress the proliferation of EGFR-driven tumor cells and restrict tumor growth and metastasis. Inhibiting EGFR alone is inadequate to achieve durable therapeutic responses. Our solution: Simultaneous inhibition of EGFR and TGFβ — a signaling molecule that promotes tumor growth in the presence of EGFR and that plays a key role in suppressing the immune response in the tumor microenvironment.

Our lead program is thus a dual-action bifunctional antibody that both inhibits EGFR and disables TGFβ directly at the site of the tumor. With this approach, we hope to achieve superior anti-tumor efficacy with an improved therapeutic window.

Gangagen, Bangalore, GBPL’s “Protein Antibacterial Technology Platform (PATP)” is based on its extensive knowledge of phage biology, naturally occurring antibacterial proteins and the ability to identify and model functional domains of putative antibacterial proteins using detailed bioinformatics. The proprietary bioinformatic platform and the standardized cascade of experiments allow rapid confirmation of antibacterial activity targeting different bacterial pathogens.

What can go wrong during product commercialization?  Anyone who has ever managed products from idea through market introduction knows the complexities involved in bringing a successful product to market.

Here are my five tips for laying the ground work for successful product adoption.

1. Start with the end in mind – Knowing the strengths and limitations of your product let you take an “eyes wide open” approach.  It helps you position your product against the competition by identifying the true problem that needs to be solved and how your solution will fill that gap.  Knowing the limitations is as important as knowing the strengths. Market the strengths, acknowledge the weaknesses.
2. Talk to your customers early in the process – Customers may be buyers, influencers or both.  For a therapeutic, the customer is both the doctor and the patient.  The doctor must be well versed in the data.  What data can you show from the clinical trials?  What side effects were encountered?  How does it compare to standard of care?  The patients want to know how to use it, how much it costs and what are the short or long-term safety concerns?  Being prepared with the data and packaging it in an attractive, well thought out format ensures it will get disseminated and read as planned.
3. Make sure your financials are solid – Run your scenarios!  What is the expected reimbursement?  How will patients use their insurance?  Model out your best case and worst-case scenarios as well as an “average” scenario to see the effects of pricing differences,  discounting and dosing for the eligible population.  Always make sure to factor in current standard of care when estimating what percentage might use your solution.
4. Continually communicate with your team – Be the conductor of your orchestra and make sure everyone is playing from the same sheet music.  Stay on top of product development, manufacturing issues, regulatory, packaging and product inserts.  Make sure you approve the sales training decks and all customer facing materials.  Pull different disciplines in the company together for a team lead meeting weekly.  Make sure everyone understands how their role affects others, and keep everyone accountable.  Use a dynamic document, perhaps on SharePoint, to enable others to see what they have to do and share their progress.  Celebrate the victories along the way and always make sure to complement team members for a job well done.
5. Prepare your teams – You cannot overcommunicate with your stakeholders.  Update senior management on a regular basis.  Work closely with the sales team to prepare materials for training, customer-facing literature and sales tools.  Choose 2-3 sales representatives to be your feet on the street.  They can relay valuable information from the field and help drive peer to peer adoption Preparing the go-to-market teams ahead of approval will jump start the sales process.  

Smaller companies often bring in marketing/sales after key decisions have been made.  You can work with an Interim Chief Commercial Officer (iCCO) to get involved early and help you make critical decision that will impact the launch process.  An iCCO can save you money, often half the cost of a CCO.  They can also help the transition to a full time CCO when the time is right

The Land of Israel, also known as the Holy Land or Palestine, is the birthplace of the Jewish people, the place where the final form of the Hebrew Bible is thought to have been compiled, and the birthplace of Judaism and Christianity. (Wikipedia)  It also has a thriving biotechnology sector, fueled by an educated workforce and strong government funding initiatives.

Blockbuster prescription drugs sold worldwide that treat multiple sclerosis, cancer, Alzheimer’s and Parkinson’s diseases derive from Israeli biotechnology. Israel creates more medical devices per capita than any other country, and its life sciences exports earn more than $3 billion a year. (Madein-Israel.com)

Looking through the vast array of Biotech’s in Israel, I had many stellar companies to choose from.  I looked for innovative companies that have not yet set up operations in the U.S. to showcase Israeli-based companies.

My choices are:

Biosight Pharma, Israel, is developing innovative therapeutics for hematological malignancies and disorders.  Their lead product, aspacytarabine (BST-236), is an innovative proprietary anti-metabolite designed to enable high-dose therapy with reduced systemic toxicity.  It may serve as an ideal therapy for leukemia, particularly for delivering high doses of cytarabine to medically unfit or older adults who otherwise cannot be treated by effective doses of chemotherapy.

BST-236 is being investigated as a single-agent in a Phase 2b for first-line treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS):

OSSIO, Israel, has created bio-Integrative OSSIOfiber^®, a breakthrough fixation material technology that provides the first credible solution to the shortcomings of permanent metal hardware, conventional resorbable and allograft implants.

Strong and Bio-Integrative OSSIOfiber^® implants provide a unique matrix of fibers made from minerals like those already found in bones to support strong fixation and enable early bone attachment, gradual bone and tissue ingrowth, and complete integration, all without local or systemic adverse response.

OSSIOfiber ^® offers surgeons and patients the confidence and certainty of a fully Bio-Integrative solution that utilizes existing reimbursement and surgical techniques and provides a more natural healing environment.

Immunovative Therapies, Ltd, Jerusalem, is a clinical stage Israeli biopharmaceutical company specializing in the translation of proven immune mechanisms into immunotherapy products and protocols for treatment of cancer and infectious diseases. Our patented “Mirror Effect^TM” technology provides an immunological platform for the development of drugs that elicit the same powerful anti-tumor and anti-HIV effects of bone marrow/stem cell transplant procedures while eliminating the devastating side effects of the procedure and the need for a tissue matched donor. We have two products in clinical development: AlloStim^®, a bioengineered living cell allograft; and Chaperone-Rich Cell Lysate (CRCL), a patient-specific heat shock protein vaccine. AlloStim combines with a tumor cryoablation (killing by freezing) method to create an in-situ cancer vaccine called CryoVax^TM and combines with CRCL to create a personalized cancer vaccine called AlloVax^TM.

Vaxil™ Bio Ltd., is an Israeli immuno-oncology biotechnology company developing novel immunotherapies including neoantigen-like peptide products, and antibodies to treat cancer and infectious diseases. Vaxil’s products are derived from its fully owned proprietary platform VaxHit™, which in effect allows for the identification, isolation, and production of uniquely specific antigen-based immunotherapy products. Vaxil’s products are derived from its fully owned proprietary technology VaxHit™, which uses the Signal Peptide (SP) domain in selected targets as core antigens (see technology section). Vaxil’s lead product, ImMucin, which has received FDA Orphan Drug Status, was designed for the treatment of multiple myeloma (MM) cancer. Immucin™ is composed of the entire SP domain of the MUC1 tumor associated antigen (TAA), and has successfully completed a Phase I/II clinical trial in 15 MM patients demonstrating a high safety profile, robust immunity and initial hints for clinical efficacy. Vaxil™ is currently performing an additional Phase I/II study with ImMucin in metastatic breast cancer patients being treated with 1st line hormonal therapy. Vaxil also isolated SPmAb-2.1 and SPmAb-6, the only anti-MUC1 SP antibodies harboring superior therapeutic and diagnostic properties vs. other MUC1 antibodies. Vaxil further validated its VaxHit technology for anti-infective indications, isolated MTBuVax™, a multi-antigenic sub-unit LP vaccine against mycobacterium Tuberculosis (MTb) currently in pre-clinical animal studies.

ImmunoBrain Checkpoint (IBC), Israel, is a biopharmaceutical company developing novel neuroprotective immune therapies for neurodegenerative diseases. IBC licensed its platform IP from Yeda, the commercialization company of the Weizmann Institute of Science in Israel. IBC developed a proprietary fully human anti-PD-L1 antibody, engineered based on its mechanism of action to treat Alzheimer’s disease and dementia. IBC plans to start its first-in-human clinical trial in early 2022.

Emosis, Israel, addresses thrombosis, a localized clotting of the blood leading to the obstruction of the arterial or the venous circulation, is a pan-vascular issue that crosses the borders of all medical specialties. To address hemostasis and thrombosis complexity in a clinically useful way, Emosis relies on flow cytometry, a powerful technique that revolutionized clinical hematology (e.g. phenotyping of leukemia) and immunology (e.g. HIV).Conventional assays, whether related to coagulation or platelet function, are bulk assays measuring average values over heterogeneous sets of molecules or populations of cells.  In contrast, flow cytometry enables a granular assessment of platelet activation through the measurement of 10 to 20 variables per single cells (e.g. platelets), over tens of thousands of cells (platelets) within second.

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 oRNA^TM technology is the next revolution in RNA therapeutics: synthetic circular RNA efficiently generated by an elegant and proprietary molecular platform. oRNA^TM 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 oRNA^TM technology to potentially address the limitations of current immunotherapies by delivering chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (isCAR^TM 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

Toronto is the largest city in Canada.  Whether ascending the CN Tower or visiting the Hockey Hall of Fame, there are many fun places to visit and great dining in the The 6.

Toronto is one of the top cities in North America for the human health sciences, combining research, clinical and business expertise to deliver new diagnostics and therapies. The city’s life sciences sector employs nearly 30,000 professional and contributes more than $2 billion to the local economy. (Toronto.ca/business-economy 2021).

Here are my picks for exciting biotech companies in Toronto:

Bresotec is a team of health experts and entrepreneurs that are alleviating the cost, complexity and health burden of sleep apnea. Their work provides healthcare professionals and their patients with a better, simpler sleep test that reduces the cost of sleep testing without compromising lab accuracy.

RNA Diagnostics has developed The RNA Disruption Assay™ (RDA™) that will provide physicians with an evaluation of how individual patients are responding to cancer therapy. If the cancer drugs are likely to provide the patient with a benefit, the physician and patient can continue treatment with confidence. If the cancer drugs are not likely to benefit the patient, physicians may consider alternate treatments. For patients, this means avoiding harmful side effects and increasing the possibility of improved survival outcomes.

Mannin Research, is developing a unique set of molecules to treat vascular related diseases, such as Primary Open Angle Glaucoma, Pediatric Glaucoma, Cystic Kidney Disease, and Inflammation via their proprietary research platform.  Their primary indication, MAN-01, is for treatment of Primary Open Angle Glaucoma, wherein they are developing a small molecule in the form of an easy to administer eye drop to reduce intra-ocular eye pressure (IOP), a key risk factor in the progression of Glaucoma.

Notch Therapeutics is improving on current T cell therapies that require a complex, patient-specific manufacturing process. The “vein-to-vein” processing of a CAR-T therapy leads to delays and variability that negatively impact patient outcomes. To fulfill the promise of a cell therapy that can be delivered as an off-the-shelf, fully characterized medicine, Notch Therapeutics is manufacturing T cells from induced pluripotent stem cells (iPSCs).  Their unique technologies and capabilities are rooted in foundational discoveries in developmental biology.

NoNO has long believed in the promise not only of reducing ischemic damage but to unlock the brain’s own capacity to recover from disease.  Their therapeutics have demonstrated potential to improve the lives of patients following ischemic stroke, traumatic brain injury, and other chronic neurological and neurodegenerative diseases.

Their lead drug candidate, nerinetide, is a first in class PSD95 inhibitor that promotes cell survival and disrupts toxic cell signals following damage to neurons. NoNO’s core technology and therapeutic targets have been validated in multiple laboratories worldwide and published in leading scientific journals.

Helix Biopharma has developed DOS47, based upon a naturally occurring enzyme isolated from the jack bean plant called urease that breaks down a natural substance found in the body, urea, into metabolites that include ammonium and hydroxyl ions.  By doing so at the site of cancerous tissues in the body, the Company believes DOS47 can modify the micro environmental conditions of cancerous cells in a manner that leads to apoptosis (programmed cell death).

Germany is famous for being the Land of Poets and Thinkers. From vital inventions to Christmas traditions, sausages and beer, Germany is home to plenty of culture, history and quirky laws! Germany is also known for its major cities, the Black Forest, the Alps and Oktoberfest. (Hey Explorer, 2020).  

Germany is also becoming one of the standout countries in biotech.  With large players such as Qiagen, Miltenyi Biotec, BioNTech and Evotec serving as anchors, smaller biotech’s are enabled to thrive. 

Germany’s biotech clusters create a variety of opportunities to transfer innovation into products, which should be attractive for investors, particularly since the EU is harmonized from a regulatory point of view and is one of the most important markets in the world. (Pinset Masons, 2021).

My picks for Germany are:

ORYX Translational Medicine, Vaterstetten, has developed strategies that bridge the gap between academic research and the pharmaceutical industry for new cancer therapies. Today, the company is developing three clinical cancer immunotherapy projects.  They have an oncolytic virus to infect and lyse tumor cells and evoke a strong anti-tumor immune response. In addition, they develop therapeutic vaccines to treat cancers by enhancing a patient’s natural defenses against the tumor.

Altyas Therapeutics, Amtsgericht Jena, is a spin-off of the University of Jena with the aim to develop novel immunological therapy concepts for the worldwide treatment of obesity, a condition with a large unmet clinical need.  The company has the relevant expertise and patents on various antigen epitopes with proven relevance to novel therapeutic immunological regulation of excessive amounts of adipocytes.

Medigene AG, Munich, is a publicly listed biotechnology company. With its scientific expertise, Medigene is working on the development of innovative immunotherapies such as T cell receptor-modified T cells (TCR‑Ts) or dendritic cell (DC) vaccines to treat cancer in fields of high medical need. The first product candidates are in clinical development.

Affectis Pharmaceuticals AG, Dortmund, is a pharmaceutical company developing novel drugs for the treatment of neurodegenerative and neuroinflammatory diseases.  Their capabilities in drug discovery and medicinal chemistry enabled the company to develop drugs with innovative mechanisms of action based on pioneering findings in the field of P2X7 receptors.  They are currently working on two products, one for neuropathic pain for MS and one for depression.

T2cure, Frankfurt, is dedicated to the development of novel regenerative therapeutics based on mononuclear cells obtained from the patient’s bone marrow. These cells have the potential to initiate repair processes in the tissue by improving blood flow and releasing messenger substances that promote cell regeneration. Such repair processes are of particular importance in patients with recent myocardial infarction or chronic cardiac insufficiency as well as in patients with general circulatory disorders, such as the arteries of the legs.

Silence Therapeutics, Berlin, are developing a deep pipeline of innovative siRNAs based on their mRNAi GOLD™ platform for diseases with a genetic basis. The depth and versatility of their liver-targeting technology gives them the opportunity to address a wide range of conditions in virtually any therapeutic area. Their wholly owned pipeline is currently focused in three therapeutic areas of high unmet need: hematology, cardiovascular disease, and rare diseases.