Industry Articles| Discovering Biotech Startups
Welcome to another edition of our Sponsor Atlas series, which focuses on startups and young pharmaceutical and biotechnology companies that are in early stages or stealth mode. In the next edition of Sponsor Atlas: Discovering Biotech Startups, we’re looking at Vanguard Therapeutics, exploring the current affairs and future orientation of this emerging California- based biotech startup. To accomplish this, we will give a business overview of their current operations, summarize their outsourcing needs, map out their development goals and decision-makers, and highlight their current strategies for capturing innovation. If you haven’t already read our other blogs on new biotech startups, be sure to check them out here.
Vanguard Therapeutics, Inc. is an early stage San Francisco company that is developing an orally absorbable cell adhesion-blocking drug for the prophylactic therapy of sickle cell disease and potentially more.
Vanguard Therapeutics does not currently have any registered trademarks or operating website, so we had to do some investigating on our own to gather clues about their research and potential products. The business was registered in California as a C-Corp on 09/25/2009. Their formal entity address, as noted on a California business entity search, is found at 108 Eagle Trace Drive, Half Moon Bay, CA 94019. They also have an additional location at 953 Indiana Street, San Francisco, CA 94107, which is also known as the University of California’s QB3-MBC BioLabs accelerator facility.
The MBC Biolabs location at 953 Indiana St. (formerly QB3@953), established in 2013, features 24,000 sq. ft. of lab and office space in San Francisco's Dogpatch neighborhood, just a short walk from the UCSF Mission Bay Campus. MBC BioLabs provides spaces as small as a single bench to fully-equipped labs. Scientists can conduct experiments on their first day, cutting months and millions of dollars off the time and cost necessary to gather data. MBC BioLabs’s facilities in San Francisco and San Carlos are home to 60 energetic young companies. Since opening its doors four years ago, MBC BioLabs tenant companies have raised over $1.8B dollars.
Vanguard Therapeutics is financially backed by multiple entities, with the accelerator investors primarily being BayBio FAST and California Institute for Quantitative Biosciences, and the government grant funding being from the U.S. Department of Health and Human Services.  Estimates show that Vanguard Therapeutics has received nearly $5M in funding and employs a staff of approximately 4. At this point in time their actual revenues aren’t that huge, but considering their multi-million dollar funding pipeline, we are expecting to see a lot of business activities from them in the near future.
Vanguard Therapeutics is an accelerator-backed company with operations within the JLABS incubator space located at the MBC BioLabs facility. JLABS provides a large amount of capital and a working environment for emerging companies who are on the brink of scientific discoveries and creating breakthrough products for their industry. JLABS, an accelerator firm owned by Johnson & Johnson and headquartered in San Diego, California. Ever since JLABS debuted with its flagship location in San Diego in 2012, the incubator division of Johnson & Johnson has supported dozens of early startup biotech companies that are innovating across four strategic areas of interest: consumer products, pharmaceuticals, medical devices and digital health.
The information on the outsourcing needs of Vanguard Therapeutics is few and far between, but their recent accelerator backing and substantial grant funding are very good signs they have a lot of capital to play around with for R&D, drug discovery, and even outsourcing early development work. Let’s take a look at some of their grant funding in detail to get a better picture of what they’re potentially able to outsource in the near future. Vanguard Therapeutics’s first record of NIH funding was in 2/15/2014, through an SBIR Phase I grant of $335,702 for developing reliable assays for Pentosan Polysulfate Sodium. Next in line was an SBIR Phase I grant for $226,033 in 2014 for the development of an improved oral P-selectin blocker for prophylactic sickle cell disease therapy. As a continuation of their SBIR Phase I research, Vanguard Therapeutics then received $1.9M SBIR Phase II grant on 9/19/2016, as well as another $1.6M SBIR Phase II grant on 9/21/2017. 
Keeping all of this research in mind, if your team has experience with any R&D focusing on sickle cell disease, and any new technologies surrounding that disease, it would be of tremendous benefit to Vanguard Therapeutics at this point in time. Specifically, if your team has experience with in-vivo bioanalysis of mouse tissue samples and animal model development targeting sickle cell disease, analytical method development and validations, and even formulation development and clinical trial manufacturing, then Vanguard Therapeutics should definitely be on your contact list, and you should be connecting with them for upcoming work in the near future.
Vanguard's Pipeline and R&D Focus
Vanguard Therapeutics is primarily known as a developer of therapies for sickle cell disease that targets the process that drives the disease, which is commonly known to be abnormal blood flow. The company's new oral drug blocks the cell adhesion molecule P-selectin and addresses the problem of sickle red blood cells sticking to the vascular endothelium, by helping to improve blood flow, mitigating acute painful crises, and improving the quality of life for patients afflicted with sickle cell disease. Vanguard is currently operating with a bare-bones minimum of active employees in order to keep their research management very focused during the early phases. They are also working with a very experienced group of advisors and consultants who are also leaders in the legal, scientific, pharmaceutical, and sickle cell disease fields.
Vanguard’s current program is based on the previous knowledge that clinical problems of sickle cell disease are the result of abnormal blood flow and that adhesion of sickle red blood cells to the vascular endothelium is fundamental to the abnormal blood flow condition. P-selectin on vascular endothelial cells is central to sickle red blood cell adhesion, and Vanguard’s parent compound effectively blocks P-selectin. With the support of two SBIR grants from the NIH, they are currently addressing the development of a parent compound and the identification of a lead candidate for use as an active pharmaceutical ingredient in commercial development. 
Some of their recent activities include validating P-selectin blocking activity in vivo in mice, designing and formulating dosage forms to increase absorption and prolong activity, optimizing the bioavailability pharmacokinetics and pharmacodynamics, and conducting pilot toxicology studies in experimental animals with all of the dosage forms. Current activities will advance their program toward production of high quality dosage forms for use as an optimized GMP drug substance in planned human trials and foster readiness for a pre-IND meeting with the FDA in order to facilitate the preparation for advanced clinical trials. 
- Stephen H. Embury, M.D. - Founder and CEO of Vanguard Therapeutics, has a 26-year career on the faculty at UCSF. Dr. Embury also has over ten years experience in the pharmaceutical industry, and has been primarily focused on sickle cell disease and its potential therapeutics. He received his medical degree from Northwestern University Feinberg School of Medicine and has been in practice for more than 20 years. Dr. Embury has over 148 highly influential citations and 101 scientific research papers under his belt.
A drug called pentosan polysulfate sodium (PPS) has been shown to improve blood flow in sickle cell disease (SCD) and can prevent painful debilitating symptoms of the disease. Vanguard Therapeutics is working on optimizing test methods that would allow reliable measurement of PPS in blood in order to conduct clinical trials of PPS, and ultimately develop a version of PPS that would be better absorbed as a pill. SCD is a poorly treated debilitating condition for which Vanguard Therapeutics is developing a promising, new oral prophylactic therapy. An urgent need exists on the market for this drug, as millions of patients who have inherited SCD world-wide, with over 100,000 in the US, continue to suffer with episodic pain, disability, and even premature death. Hydroxyurea treatment, a common option, has some clear-cut deficiencies, and many drugs under development target treatment rather than prevention of acute events. The cause of the most morbidity from SCD is abnormal blood flow, and subsequently the acute pain crises that result from stoppage of microvascular flow. Defective blood flow results from multiple pathophysiologies, including several that are independent of the paradigmatic sequence of deoxygenation -gt sickle hemoglobin polymerization -gt red cell sickling. Based on this evidence, Vanguard believes that endothelial P-selectin is central to ongoing impairment and acute stoppage of flow. 
Vanguard’s in vitro and preliminary clinical data show that PPS improves microvascular blood flow in SCD. However commercially available PPS is not a viable therapy because of its low oral bioavailability and limited duration of action. The research team’s plan is to develop an improved second generation PPS that will overcome these limitations. Vanguard Therapeutics’s main goal is to improve the quality of life of patients with SCD by bringing to market PPS as an oral P-selectin blocking drug for long term administration to prevent sickle red blood cells sticking to the lining of blood vessels and to improve blood flow and otherwise painful episodes in patients. In the near future, Vanguard’s IND enabling activities will further the company and a possibility to gain even more funding and partners for continuous product commercialization. 
Vanguard Therapeutics is definitely on track to have some major innovations in the biotech industry, and it’s difficult to say exactly when they will come out of start up mode, but we’re thinking it should be within the next few years, so be sure to keep them on your schedule and look out for our next edition in this series coming soon.
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