Profiles in innovation.

Innovation is the cornerstone of the life sciences, as we seek to discover new diagnostics, treatments and medicines to advance our knowledge and understanding of how disease establishes and progresses. Innovation enables us to look at a problem differently, which is crucial to scientific breakthroughs.

RI Bio salutes our members’ innovations that improve health and make the world a better place. From new diagnostics to first-in-class therapeutics to regenerative medicine and new vaccines, we bring you their stories of innovation here.

Project insight that drives better decisions and better projects

THE PROBLEM

A rapidly growing bioprocessing solutions company is contemplating building a new facility but isn’t sure where to begin. They contact a few architects and general contractors for pricing, however the General Contractors can’t price the project because they have no design and the Architects can’t tell them how much it will cost as it is not their responsibility to design to a budget. What typically happens is projects are fully designed and then sent out for pricing, often coming in over budget. This requires re-design, which costs time and money. Additionally, clients want to know how much a project costs before investing in any upfront design.

Science, technology and healthcare companies are in a unique bind when it comes to construction. Without specialized construction knowledge, many companies get stuck in preconstruction analysis paralysis.

THE SOLUTION

With deep experience building healthcare, science and tech spaces, Pariseault Builders bridges the gap between science and construction for sensitive environments. A full-service construction firm, they uniquely offer upfront conceptual budgeting and facilitate Target Value Design through the preconstruction process to help clients make well-informed decisions that will positively impact their business.

Their innovative and ever-evolving service, known as a Preconstruction Services process, incorporates estimating, project management and field operations teams to provide detailed preplanning project insight. A typical process:

  • Initial meeting to better understand the client’s needs, wants, reservations, etc., including a site tour to offer any initial construction insight regarding how their project can fit the space and to identify any restraints they may incur.
  • Pariseault develops a high-level conceptual budget for the project based on historical knowledge of similar projects, current market sq./ft analysis and vendor/subtrade input. In addition, they partner with the design team to develop project scope, identify the client’s budget, and identify any constructability issues as the design develops.
  • Upon seeing the resulting “Preconstruction Service Contract,” the client quickly sees the advantage. Pariseault creates a preconstruction deliverable schedule, guides the design to a budget, identifies whether any long lead items need to be pre-ordered prior to construction, and meets with the local building department authorities to begin the permitting process. Once the final Construction Documents are completed, the owner knows for certain that the project can be constructed in their local municipality, that it is on budget, and that all long-lead items have been procured. By taking the steps to pre-plan the job, the client has saved time, money, and aggravation; and gained the peace of mind knowing much uncertainty has been removed.

Pariseault then begins the building process, drawing on their experience building sensitive environments such as an HPNE Bioprocessing Equipment Facility, modernizing Saab Technologies Autonomous and Undersea Systems, building an MRI Suite and Operating Rooms for local hospitals, and much more. From Preconstruction through Post Occupancy Services and Maintenance, they bring personal interaction and devotion to a project that results in a pleasurable construction experience.

WHAT'S NEXT?

Pariseault is planning training seminars for clients to educate them about the preconstruction process and all its advantages. To learn more about all they offer, visit pariseault.com.

World’s most brilliant compact x-ray sources.

THE PROBLEM

For over a century, humankind has benefitted from X-rays revealing hidden answers. Around the globe millions of patients depend on X-rays daily. Millions of shipments are scanned at security checkpoints. Manufacturers depend on them for millions of product quality assurance tests. But traditional X-ray tubes have reached fundamental performance limits. Even the most advanced X-rays tools leave end users with low resolution results and impractically long data acquisition times.

End users demand greater resolution and faster X-ray results. This drives X-ray equipment manufacturers to seek higher performance X-ray sources to enable next-generation solutions spanning diagnostics, materials research, industrial nondestructive testing, and life sciences.

THE SOLUTION

New laser-driven X-ray sources represent a giant leap forward. Research Instruments Corporation (RIC) has developed an ultrabrilliant microfocus X-ray source that succeeds where conventional X-ray tubes fail. It delivers 100 times greater spectral brilliance in a micrometer-scale spot beyond the limits of available X-ray tubes. This extreme spectral brilliance, ultrahigh X-ray flux and micrometer-scale spot sizes are required for advanced non-destructive testing and next-generation diagnostics.

RIC’s technology converts laser light into ultrabrilliant X-rays beyond limits of existing X-ray tubes. They have combined their proprietary hardware with TRUMPF lasers to create multiple X-ray sources that OEMs can integrate into new X-ray solutions with unprecedented X-ray capabilities with an amazingly compact suitcase-sized X-ray source.

Thanks to RIC’s innovative technology, drug discovery can accelerate with more precise molecular measurements. Patients will have access to higher-resolution, lower radiation dose X-ray diagnostics. The defense industry will gain high energy X-rays to see inside operating jet engines. Chipmakers will gain new nondestructive testing capabilities to see inside ever-shrinking semiconductors.

Already, RIC has demonstrated their innovation can make a big impact in diagnostic imaging, achieving a 20-times resolution improvement with 95 percent less radiation in mammography. This means earlier diagnosis would be possible at a fraction of the radiation patients are exposed to with typical mammograms. In addition, RIC’s technology has been implemented at National Institute of Standards and Technology in Boulder, Colorado and in Europe at ELI Beamlines, an international laser research centre outside of Prague.

WHAT'S NEXT?

RIC is accelerating development of its laser-driven X-ray platform by partnering with TRUMPF Lasers and collaborating with leading X-ray equipment manufacturers to bring laser plasma X-ray generation into the mainstream. As the company enters its next phase of commercialization, RIC is attracting technical collaborators and strategic investors to drive an ambitious plan to expand the team and lab space.

The world’s only live moss air filter.

Not your ordinary moss wall art

THE PROBLEM

35-year-old Julian was pursuing his dream career life – living in the heart of the city in a 1783 brownstone townhome and commuting to his stressful job at an architectural firm. Unfortunately, factory emissions, car exhaust, and inadequate air filtration at home aggravated his asthma. Like Julian, more than 24 million Americans suffer from respiratory diseases such as asthma and 50 million from allergies each year. Air quality is a major health issue: both indoor and outdoor pollutants from cooking, cleaning, burning fuel, and wildfires impact daily life. Julian began searching for air filtration system but was disappointed in their industrial look. Then he found Moss Pure.

 

THE SOLUTION

Moss Pure is a scientifically engineered air filter using live moss as an air filter and stress relief device. Their design has earned certified results from a U.S. laboratory showing that Moss Pure products improve air quality within minutes. Their air filter captures carbon dioxide, dust, allergens, pet dander, wildfire smoke, VOCs (toxic gases), metals, bacteria, and viruses.

Company founder and CEO Jaime Mitri is an environmental engineer and created Moss Pure during a startup competition at MIT, where it won first place. She realized the moss wall market was in demand and that live moss has tremendous potential health benefits. At the time, no one had been able to keep live moss alive indoors without continuous maintenance for more than a few days. Other moss wall companies used preserved moss which is no longer living so it cannot be a filter, is treated with chemicals and dyes and after some time will fade, deteriorate, and need replacement.

Although their design appears simple and modern, there is nothing simple about the science that goes into Moss Pure. If you were to just add live moss to a frame, it wouldn’t survive for more than a few days and it wouldn’t be an air filter. Their approach is a patent pending multi-layer of eco-friendly materials that provide needed nutrients for the live moss and that keep it alive within their product design indefinitely. Because of this approach, their products do not need watering, sunlight, or maintenance.

Stress reduction and lack of greenery are other considerations. 80% of Americans have reported feeling stressed or anxious, impacting mental and physical health, relationships, and decreasing productivity.
Lack of greenery in urban spaces also impacts physical and mental health, prompting the World Health Organization and American Psychological Association take in more nature to reduce stress.

Moss Pure’s live moss products bring nature directly into your space and smell like a fresh park or forest. They use science and product design to stimulate senses such as vision, touch, and smell, reducing stress and anxiety and increasing productivity. It’s a calming, aesthetically pleasing decor piece that immediately transforms homes and offices. Mitri knew she wanted to create a portable air filter that didn’t look like a scientific device, and has succeed with wall art pieces, tabletop décor, and the option to create a custom moss wall. That aesthetic focus is also part of what makes Moss Pure a stress reliever, and why customers like Julian love it just as much for its pleasing natural look as the health and environmental benefits.

WHAT'S NEXT?

Moss Pure plans to continue growing and expanding the business, reaching new areas across the U.S. and creating new partnerships. New collaborations include George Howell Coffee in Acton, MA, which installed Moss Pure on their manufacturing floor and in the warehouse to improve air quality and bring the beauty of nature indoors. ChopValue, another collaborator, makes frames of recycled chopsticks from Boston restaurants for Moss Pure filters. The company continues to educate customers about the science behind their products and learn from their customer feedback.  For more info, visit mosspure.com.

Bridging academia and industry.
Supporting education and innovation.

Helping today’s ideas become tomorrow’s solutions.

THE PROBLEM

A small startup led by an experienced orthopedic surgeon has developed a novel bone plate for complex pelvic fractures. Compared to the current gold standard, they believe their plate will provide enhanced fracture fixation while also cutting intraoperative time in half. They are currently working to obtain FDA approval and need to perform a cadaveric biomechanical study as part of their premarket submission. However, they are struggling to find a facility that can handle both the technical and logistical challenges that the study will entail.

 

THE SOLUTION

The RIH Orthopaedic Foundation is a 501(c)(3) non-profit and A2LA ISO 17025 accredited (Cert. # 2278.01) orthopedic research and device testing facility. Uniquely positioned between the academic and industrial sectors, it fosters collaboration between medical professionals, biomechanical researchers, and medical device manufacturers. With integrated bioskills and mechanical testing labs, RIHOF supports a wide array of projects including surgeon validation labs, non-clinical bench performance testing, and, mostly notably, biomechanical research studies.

RIHOF’s integrated setup is highly unique in the field of orthopedic research and device testing. Most other facilities fall into one of two categories: a bioskills facility without adequate testing capabilities or a mechanical testing facility that is not equipped to utilize cadaveric tissue. At RIHOF, however, the surgical necropsy tables and mechanical load frames are in adjacent, connected rooms. With this setup, RIHOF provides the perfect environment for conducting state-of-the-art biomechanical studies, particularly those utilizing cadaveric specimens. 

Successful execution of a cadaveric biomechanical study requires meticulous planning and close coordination of activities. With its integrated setup, RIHOF streamlines project workflow and controls for critical test variables such as time, temperature, and handling. This setup also facilitates the iterative and collaborative approach that is often needed when developing custom biomechanical test protocols. After test specimens are prepared in the bioskills lab, they are immediately brought into the adjacent mechanical testing lab for evaluation. By witnessing the testing and obtaining instant feedback, researchers – like the bone plate innovator mentioned above – can make adjustments as needed to refine their methodology. This ensures the efficient use of resources and the achievement of optimal results.

RIHOF’s affiliation with University Orthopedics and the Brown University Orthopedic Residency Program is an added benefit for those looking to conduct biomechanical research studies. When an expert opinion or extra set of experienced hands is needed, they have a vast network of medical professionals to draw on. During their largest and most impactful biomechanical study to date, RIHOF recruited 10+ medical students and orthopedic residents to assist with 64 total knee replacement surgeries that were performed over the course of just four days. That study – an investigation into the effects of motion during total knee cementing on tibial implant fixation strength – won the Chitranjan S. Ranawat Award at the 2022 American Association of Hip and Knee Surgeons Annual Meeting and was later published in The Journal of Arthroplasty.

WHAT'S NEXT?

RIHOF plans to expand its equipment and capabilities to better serve Rhode Island’s ever-growing biotechnology industry. Located within Providence’s Innovation & Design District, RIHOF hopes to serve as a valuable resource for local startups that might otherwise need to venture out of state for their research and testing needs.

To learn more about the RIH Orthopaedic Foundation and all it has to offer, please visit rihof.org.

Global health and economies are continually devastated by pathogens. I2Pure’s molecular iodine platform is being developed to fight back.

Microbes vs. The World

THE PROBLEM

Ivonne, in her mid-forties and in good health, was in a terrible car crash and severely burned on 20% of her body, plus suffered an open leg fracture. Her prognosis is good; however she will be in critical care for some time. Multiple systems will need to be supported throughout her recovery: likely a Foley catheter as well as a peripheral venous catheter and possibly a central venous catheter. Laser doppler could be performed to map the blood flow under the burn to identify what area will require surgical debridement and possibly a dermal scaffolding will be required to support regrowth. The open fracture will require cleaning and the bone will likely require plates and screws. She’s facing multiple surgeries and possibly stepped procedures on the same injury.

Every step of her recovery is fraught with the danger of infection. Wound care in general is difficult to manage because intrinsically the need to keep a wound pathogen-free is at odds with healing. With a complex case like this, the patient will not only will require acute and chronic wound care but will also need to interact with dozens of medical devices where pathogens can enter the body and cause systemic and local infections.

THE SOLUTION

To ensure healthy outcomes in the clinical setting, physicians typically depend on the use and application of disinfectants, most of which have significant drawbacks such as varying levels of toxicity, lack of efficacy, ineffective against biofilms and staining.

There is, however, a naturally-occurring, broad-spectrum biocidal agent that is effective against pathogens and biofilms: molecular iodine (I2). I2PURE chemists have proven expertise and experience formulating the molecular form of iodine across multiple formats. Stabilizing molecular iodine for use in its pure form removes the toxicity, acidity and the negative characteristics found in legacy iodophors. I2PURE has focused on molecular iodine because of its unique and diverse features:

  • Molecular iodine is a necessary biochemical generated by the interaction of specific enzymes in the human thyroid, mammary, salivary, and other glands.
  • The body ingests iodide (frequently as iodinated salt) and from it creates molecular iodine.
  • It is a natural weapon against pathogens.
  • It is malleable and can be integrated with polymers and coatings to eradicate pathogens and biofilms encountered during medical device usage.
  • Molecular iodine can be included in effective treatment protocols as a principal ingredient to enhance skin disinfection (ex: surgical lavage, lotions).
  • Molecular iodine is a sustainable raw material.
  • It is the only form of iodine that is a biocide.

I2PURE has submitted its molecular iodine formulations to well-respected third-party labs to test and further validate internal research findings. The results of these tests show I2PURE molecular iodine formulations to be 99.9999% effective at eradicating pathogens while leaving living tissue unharmed. Test results provided by North American Science Associates (NAMSA), the world’s leading medical contract research organization, demonstrated skin biocompatibility for I2PURE formulations containing up to 15,000 parts per million (ppm) of molecular iodine. Fifteen-thousand ppm of molecular iodine is over 3,000 times greater than the concentration found in commonly-used iodine products.

What’s more, I2PURE’s molecular iodine formulations can be impregnated into polymers to create medical devices, integrated into implant coatings, and included in topicals for the skin and liquids for irrigation. It has hundreds of uses throughout the medical practice and the continuum of patient care. Molecular iodine fills a critical void left open by existing anti-infective and disinfection solutions and offers real hope to patients such as Ivonne.

WHAT'S NEXT?

I2PURE continues to move forward in the battle against microbes to treat a variety of skin conditions and prevent infection in people and animals; as well as provide greater antiseptic capabilities for surgical use. Leadership is anticipating three products to be registered and approved over the next six to eight months in the US: Skin Prep, Dermal Regeneration Matrix, and a Wound Irrigation System.

I2PURE is anticipating the start of a PI-led Institutional Review Board for Foley Catheters to start in four to six months. The company is also anticipating a CE Marked Hydrogel for wound care available in the EU within six months.

In addition, I2PURE is developing other products with partners including:

  • Catheter anti-infective hemostatic patch
  • Central Venous Catheter with 30 days of microbicidal persistence
  • Dressings with up to 60 days of microbicidal persistence
  • Cardio-thoracic grafts with 30 days of microbicidal persistence
  • LVAD anti-infection patch
  • Polymers with up to six months of microbicidal persistence
  • Coatings for orthopedic implants with 40 days of microbicidal persistence
  • Sutures with 30 days of microbicidal persistence

The financial impact and societal costs of pathogens and biofilms grow exponentially larger each year – but I2PURE’s work could change that equation.

Quris’ Bio-AI Clinical Prediction Platform better predicts which drug candidates will safely work in humans, avoiding the tremendous costs of failed clinical trials.

Moving beyond the faulty mouse model

THE PROBLEM

Three-year-old Gabriel was a happy toddler but was falling short of developmental milestones. His non-verbal communication such as gestures, body language and facial expressions were delayed, and he struggled with language processing and problem-solving. DNA testing revealed he had Fragile X syndrome, a genetic condition involving changes in part of the X chromosome. This condition causes a range of developmental problems including learning disabilities and cognitive impairment, and can include symptoms of autism spectrum disorders. There is no cure, though therapy can help manage symptoms.

THE SOLUTION

Finding a medication to address conditions such as Gabriel’s is a costly and time-consuming process, with a high failure rate. Almost 90% of novel drugs fail in clinical trials, primarily due to inaccuracies in predicting human responses based on animal testing. This results in a significant financial loss for pharma companies, estimated at over $53 billion annually. The human cost is also enormous, as potentially life-saving treatments are delayed or never reach the market. Rare diseases generally receive inadequate funding and have even longer development and testing timelines, further jeopardized by low patient access pools for trials and regulatory roadblocks.

Quris-AI – (recently named one of the World’s Most Innovative Companies by Fast Company) – aims to solve this problem by accurately predicting which drug candidates will work safely in humans, increasing innovation to speed the introduction of safe drugs for rare diseases.

Quris-AI uses a unique approach by integrating machine learning with breakthrough patient-on-a-chip technology. This method involves testing drugs on interconnected miniaturized versions of human organs created from stem cells (patient-on-a-chip technology), providing a more accurate model of how drugs will react in the human body. The data generated from these tests are then used to train an AI model to predict drug toxicity effectively. This technology is unique because it avoids the pitfalls of traditional animal testing and can potentially save a drug developer significant time and money (since 90% of drugs fail in clinical trials, the cost would be cut by 90%) and accelerate a new drug’s path to market. Most importantly, it can help a wide range of patients, as it could be applicable to any disease that requires medication, including rare diseases that are often overlooked due to cost/trial constraints. In the future, a drug developed and/or tested with Quris-AI could hold the cure to a patient’s rare disease – rapidly finding the right drug and dosage to safely treat patients based on their own unique needs. The technology could also offer faster exploration of new ways for patients to benefit from existing drugs or drug combinations.

Since closing their seed funding, Quris-AI has been working hard to fast-track their platform development, drug pipeline R&D, and partnership initiatives. Focusing first on rare genetic diseases, they have been targeting viable drug candidates that address Fragile X syndrome (FXS), the most common inherited cause of autism and intellectual disabilities worldwide. They had hoped to move their first FXS drug candidate into the clinical phase but discontinued that push as – after intense AI analysis – their platform predicted that one of the compounds would not successfully cross the Blood-Brain-Barrier and that the combination was at high risk for toxicity … despite previous studies on mice.

As their platform proved its unique ability to predict whether a drug would pass clinical trials at an early stage, they were able to tap partnerships with various academic and industry institutions to move onto other leads targeting the activation of the Fragile X Messenger Ribonucleoprotein 1 (FMR1) gene.

To hear more about their technology, explore this video:
insidetheblueprint.com/quris-ai-artificial-intelligence-and-drug-development/

WHAT'S NEXT?

Quris-AI will stay at the forefront of the push to eliminate animal testing and move beyond the faulty mouse model in pharma and continue its mission to accelerate drastic changes to modernize and improve the drug discovery and development process.  

Quris-AI’s innovative approach has already attracted the attention of industry heavyweights. For example, Quris-AI and Merck KGaA are collaborating on a multi-year deal to examine the clinical safety prediction platform and how Quris-AI’s unique technology can benefit Merck KGaA. Quris-AI also has an exclusive partnership with The New York Stem Cell Foundation (NYSCF) Research Institute to broaden the genetic diversity of its patients-on-a-chip. These partnerships, along with the recognition received from Frost & Sullivan and Fast Company, demonstrate the potential of the Quris-AI platform to revolutionize the pharmaceutical industry.

Enhancing the sense of touch for diabetics who suffer from peripheral neuropathy

An innovative step forward for diabetic peripheral neuropathy

THE PROBLEM

A retired 63-year-old, Alex had a medical history that included peripheral vascular disease and controlled type 2 diabetes, among other issues. Alex reported he had almost no sensation in the bottom of his feet and when walking barefoot in his home, he could not feel the difference between his hardwood floors and shag carpeting. He also had difficulty balancing and would stumble into walls. Alex was diagnosed with diabetic peripheral neuropathy (DPN) in both feet. It’s the most common – and costly – complication of diabetes. As a result, Alex faced the very likely prospect of foot ulceration.

For Alex and patients like him, small cuts and abrasions often go unnoticed and can turn into larger, more grossly infected wounds called foot ulcers. Every year, over 100,000 diabetics in the U.S. will lose a toe, a foot or even a leg to their disease. According to the CDC, in 2019, direct medical expenses related to the diabetic foot exceeded $19 billion, almost all of which was attributable to treating foot ulceration.

THE SOLUTION

Current neuropathy treatments reduce the painful symptoms, but do not address the underlying pathology. Phoenix NeuroStim Therapeutics may have the solution with their SureStep™ Diabetic Insole, a patented sensory stimulation technology. SureStep looks like a typical orthotic and noninvasively imparts a proprietary mechanical stimulus to the soles of a patient’s feet to restore sensation, making use of a process known as stochastic resonance. A typical therapeutic regime consists of daily, 30-minute stimulation sessions self-administered over the course of a month.

In a recent clinical trial, SureStep was shown to improve tactile sensitivity in a population of highly neuropathic diabetics by an average of over 25%–a clinically-significant improvement–and the initial benefit not only lasted, but grew after the insoles ceased to be worn.

Specifically, the trial found that when measured one month after the end of therapy, the study group had maintained all of the therapeutic benefit observed following treatment. And, most surprisingly, after three months without any further intervention, the level of sensitivity in a majority of patients had continued to improve over and above the initially observed gains, some by as much as 25%.

In a separate study of repetitive tactile stimulation, brain scans of the participants’ somatosensory cortex–where all the peripheral nerve impulses are processed–revealed the size of the area represented by the stimulated limb had increased by over 50% and was, in a sense, reawakened. Phoenix believes that by promoting neural plasticity, stimulation with SureStep had effectively increased the brain’s ability to interpret the flow of sensory information from the periphery. The patients experienced this increase as improved sensitivity on the plantar surface.

A year later, Alex’s sensation was in the normal range for all adults his age, never mind diabetics, and says that when he curls his toes in the living room, he can feel the individual fibers in his carpet. He also recounts that following therapy he refused to return the orthotics because they had helped him so much and wanted to have a pair to take home.

WHAT'S NEXT?

Phoenix is planning another, larger clinical trial to confirm their results and prove they can reduce the incidence of foot ulcerations. With the number of diabetics in the US expected to grow by over 40% in the next ten years, there is an urgent need to reduce foot ulceration within the diabetic population from both an economic and patient health perspective.

Once SureStep is launched, the experts at Phoenix are eager to explore the technology’s use to change brain behavior, using the brain’s own neuroplasticity to treat other diseases. For instance, they want to explore the device’s impact on senior falls as loss of sensitivity in the planar surface is a major factor in senior falls.

Unravel Biosciences is seamlessly bridging target discovery with clinical efficacy to advance drugs for complex neurological diseases.

Accelerating drug development

THE PROBLEM

Chloe was a beautiful, healthy baby girl as a newborn but as she grew older, she started to exhibit a loss of coordination and ability to speak. She was diagnosed with Rett syndrome, a neurodevelopmental disease that affects brain development. What was previously thought to be a genetic mutation exclusive in females, researchers now understand is also in males, but fatal before birth. Rett syndrome symptoms include tremors, abnormal breathing patterns, muscle flaccidity, and overall motor dysfunction. There is no cure and treatment focuses on minimizing symptoms and providing supportive care.

THE SOLUTION

Unravel Biosciences is taking an innovative approach to finding hidden therapeutic targets, specifically for Rett syndrome and similar neurodevelopmental disorders. Founded by Dr. Richard Novak and Dr. Frederic Vigneault, Unravel begins with the patient’s disease state in order to trace back a potential solution to restoring health. The proprietary health model that Unravel uses is called BioNAV™, which leverages the uniqueness of every patient and computationally tests thousands of interventions. When paired with rapid pre-clinical and clinical studies, the researchers at Unravel are able to validate clinically meaningful treatments, and develop drugs best suited to treat the disease across the patient population.

Unravel looks at multi-oriented interactions in order to find the causes and effects each interaction has with one another. This begins by using patient RNA, giving researchers the molecular data to put through an unbiased screening platform to predict what kind of drugs would restore the specific patient back to health. This proprietary model enables Unravel to be more effective with therapeutic discovery, with less risk and higher success than conventional target-driven drug programs. This is a completely new and different method from conventional discovery because BioNAV™ takes a closer look at the interactions between a disease and its symptoms. In conventional methods, researchers generate compounds against a target that has only been validated in a cell culture model, hoping that it will translate to the patient population. This fails to recognize that diseases are so variable from patient to patient and that the drug that helps one patient may not help another. Therefore, a platform like BioNAV™ helps put that drug into the context of a specific patient with specific factors that differ from person to person.

BioNAV™ was developed when Drs. Novak and Vigneault started to look at what might cause the multi-oriented interactions in a diseased patient, specifically, an individual with Rett Syndrome. The team used tadpoles to create a mosaic models presenting symptoms reminiscent of Rett Syndrome, including a range of seizure severity, motor impairment, and behavior abnormalities. By testing their BioNAV™ top predictions in tadpoles, Unravel was able to identify a novel molecular pathway that significantly rescue the disease phenotypes, ultimately leading to the development of a new safe and effective drug candidate specific to treating Rett Syndrome. Thus, BioNAV™ can look at the interactions the genetic cause of Rett Syndrome has with the patient’s body and develop compounds that can rescue the disease network.

WHAT'S NEXT?

Unravel’s lead drug candidate has been successfully tested in a mouse model for Rett Syndrome and they are completing further pre-clinical development. In addition, the company is planning clinical trials in Colombia and the United States. While it has initially focused on using BioNAV™ to study Rett Syndrome, it has partnered with other institutions, foundations, and companies to discover new treatments for other diseases, including neurodevelopmental, neurodegenerative and oncology. Unravel is a trailblazer for innovation in the drug development community, as their approach enables identifying therapeutic response groups with only RNA data, which the team can test very early in the process, differentiating itself from the failure prone process of traditional target-driven development. While Rett syndrome is a difficult illness to live with and combat, Unravel is confident their work will be able to make a difference for those diagnosed with Rett Syndrome.

Stellaromics is empowering a brighter biomedical future with the world’s most powerful 3D in situ multi-omic analysis technology that can help scientists achieve breakthroughs.

Solving the mysteries of tissue analysis

THE PROBLEM

At 55, Catherine’s ovarian cancer diagnosis sidelined her from her entire life – work, family, hobbies. She underwent surgery followed by different chemotherapies. But her cancer remained resistant to therapy and she became weaker and weaker. What her particular cancer would respond to remained a mystery, like many other diseases.

Currently, the drug discovery process doesn’t have a good way to look at the mechanism of a drug’s mode of action or how different types of cells interact with the drug. To develop drugs with improved targeting profiles and treat diseases more precisely, researchers need a better picture.

THE SOLUTION

Stellaromics, founded in 2022 by a team of world renowned scientists, is on a mission to move biomedical research forward faster. They have patented a novel technology for identifying high resolution 3D transcription and translation patterns in native biological tissue using unique probe design in combination with hydrogel tissue chemistry. Their amazing new technology can empower researchers to develop novel drug treatments for previously unsolvable disease mysteries, including cancer, autoimmune disease, neurological disorders, and infectious disease.

Called STARmap (Spatially-resolved Transcript Amplicon Readout mapping), it uncovers rich gene expression patterns inside an intact tissue sample to learn how each cell works and how multiple cell types relate to each other in all dimensions. By identifying and characterizing the gene expression pattern of cells within a tissue, STARmap enables treatment target identification and evaluation for biomedical research.

Previously, scientists would need to disassociate the cell and sequence a pool of different cells as a mixture. But that would contain all different types of cells – immune cells, cancer cells, normal cells – and scientists didn’t know how they were distributed in tissue or interacted with each other. Stellaromics’ innovation opens up a new world of in situ analysis. It enables scientists to capture high resolution 3D multi-omic information from thick tissue samples, mapping gene expression patterns while preserving the spatial integrity and anatomical structure of the cells in the tissue being studied. By better defining the landscape in all different samples, including tumor samples and other neurological disease samples, STARmap can enable more precise diagnosis to stratify the patient treatment.

“From a basic biological research point of view, STARmap would help in understanding the cell interactions and other biological mechanisms underlying different disease models for patients with diverse genetic backgrounds to fully evaluate the cell states as a biomarker for discovery,” explained Ye Fu, Chief Technology Officer. “Another example would be drug development. When you treat a patient with a small-molecule drug or antibody or nucleic acid, you don’t know what types of cells get targeted, and what level of penetration gets inside a tumor or different tissues. With this method we can start with an animal model, organoids, or patient samples, and evaluate how a drug treatment actually changes the cell states for all kinds of different cells for different locations. Traditionally, scientists and pathologists have been using H&E staining, but the information that method could give is limited. With our technology we are able to actually see tens of hundreds of more types of cells, and in much thicker tissues across multiple cell layers.”

Still in the deployment stage, Stellaromics has developed a suite of products called Plexa to extend their progress by empowering others to innovate alongside them. With Plexa, any researcher or clinician can create fully annotated cellular maps for clearer understanding of disease progression and response to therapy. They offer both instruments and reagents as well as services for other biotech companies.

WHAT'S NEXT?

Stellaromics has partnered with a therapeutics company who is using the STARmap technology to develop new drugs and therapeutics as they build up their user base and data platform, mining user feedback to further improve the technology.

The next phase calls for a fully commercialized unit that will be broadly available for individual labs and pharmaceutical companies. It’s a technology that could offer faster, richer discovery so researchers can better understand tissue functions and dysfunctions, in which cells may be targeted by specific interventions. And it’s a discovery that could provide effective treatments for patients such as Catherine and millions like her.

Harnessing the healing power of our body’s natural intercellular network to treat chronic diseases and promote healthy organs.

Re-establishing a healthy balance between cells and the ECM

THE PROBLEM

Sam is a 60 year old power plant engineer looking forward to retirement and traveling across Europe, but found himself getting short of breath and easily fatigued. Testing revealed mild Pulmonary Fibrosis (PF), a currently fatal disease with no cure and an average life expectancy of three to five years. Sam was devastated but hoped current therapies could slow the disease’s progression.

The Pulmonary Fibrosis Foundation reports there are more than 200,000 Americans living with the disease. PF causes a continuous scarring or thickening of the alveolar sacs, the small air sacs that allow oxygen to enter into the blood stream. This thickening reduces oxygen exchange, making it increasingly harder to breathe and to exert normal physical activity.

THE SOLUTION

XM Therapeutics is working with the body’s extracellular matrix (ECM) to develop designer human ECM particles for tissue and organ repair for patients like Sam. Their approach is to repair tissues or organs rather than replacing them, which requires organ donors who are in short supply. Their proposition is simple and based on the observation that restoring normal ECM structure will restore normal cell behavior. For the PF patient, the hope is that the ECM particles will instruct the lung tissue to stop and even reverse the scarring process and the constant inflammation.

To understand the science behind it, we must understand that the body’s largest component is not living cells, but complex protein structures that cells create for support and sustenance. This structure is called the extracellular matrix (ECM), which is composed of thousands of proteins that are produced and eliminated constantly in response to the body’s needs.

It was previously thought that the ECM was simply a passive scaffold, but research showed that not to be true. The ECM actually drives cell behavior which allows tissues and organs to form and grow – and, importantly, to heal in response to injuries. In chronic diseases, the healing mechanism fails and the ECM grows disproportionately. This thickening is called fibrosis and it is accompanied by constant inflammation and reduced oxygen supply to cells. The ECM particles that XM Therapeutics produces in the lab, when delivered to the diseased tissue, can stop this fibrotic and inflammatory process.

Their development could be the solution for many diseases: fibrosis and inflammation are hallmarks of all chronic diseases and ECM malfunction has been characterized clearly in different organs such as in glaucoma in the eyes, heart failure, lung fibrosis, COPD, NASH in the liver, kidney failure, and in several solid tumors in cancer. XM’s first therapeutic applications are aimed at the cardiovascular and respiratory systems – or heart and lungs. Specific conditions include myocarditis, heart failure with reduced ejection fraction, and idiopathic pulmonary fibrosis.

A little background
Working with the ECM for medical purposes has been done for decades using specific proteins from animal sources. In an innovative approach, XM Therapeutics cultures various human cells in the lab to produce intact ECM particles that contain the full payload of proteins including important growth factors and cytokines that regulate cell behavior. There are several academic labs and a few companies that are developing know-how and potential approaches based on the full understanding of modern ECM biology, however, none have envisioned the practical clinical product that XM has. Once their particles are harvested, they can be stored as pharmaceuticals and injected easily and directly to diseased tissues through syringes or catheters.

The 3D cell culture technology to produce ECM particles has been in development at Dr. Jeff Morgan’s lab at Brown University for more than a decade. It is well established and scalable. The application to stop and reverse fibrosis and inflammation in heart and lungs is much more recent and builds upon academic research carried out for five to ten years. Based on modern technologies including proteomic profiling and bioinformatics, their team and collaborators are deciphering the complex molecular pathways of fibrosis.

WHAT'S NEXT?

The technology is still in the early stages of development in a process that will take several years before XM has an approved clinical product. But their expert team has worked on similar technologies before and knows the pathway well. Right now they are scaling up their production platform while carrying out pre-clinical studies such as proteomic profiling and cell-based assays for specific diseases. The plan is to build the proprietary production platform for human ECM particles in a way that allows them to design, test, and validate formulations for different tissues and diseases in a time- and capital-efficient manner. As they develop this capability, they will seek to partner with pharmaceutical companies that have the global network and capabilities to take these therapeutics through clinical efficacy trial and regulatory approval to market, considering the enormous unmet clinical need. And that will be welcome news to patients like Sam.

Cognesy Therapeutics is building a best-in-class platform technology to discover drugs that induce neuroplasticity for the treatment of neuropsychiatric diseases.

Unlocking the Potential of Neuroplasticity

THE PROBLEM

Formerly a vibrant, outgoing teenager, Suzy began experiencing symptoms of depression during the pandemic due to the ongoing stress, fear and uncertainty. She lost interest in activities and friends, began having trouble with memory and concentration and had thoughts of self-harm. Her pediatrician tried different medications, but none really helped and side effects discouraged her from continuing them.

Like Suzy, one in eight people, or 970 million, worldwide currently live with a Central Nervous System (CNS) disorder such as depression, anxiety, schizophrenia, and dementia, as well as acquired brain injuries such as stroke or trauma. While numerous medications are approved for treatment of these disorders, many people living with CNS disease are not adequately treated.

THE SOLUTION

An exciting field of study with wide implications and huge potential – neuroplasticity – may hold the answer.

Neuroplasticity is broadly defined as the ability of neurons to change, reorganize, or grow neural networks, or as some say, “rewire your brain.” Cognesy Therapeutics is focused on unlocking the full potential of neuroplasticity to develop treatments that can work for multiple CNS disorders. They are approaching discovery with neuroplasticity-first assays (a process intended to measure neuroplastic changes in living neural networks in response to a drug) along with novel chemical libraries and medicinal chemistry.

For Cognesy, drug discovery begins with tracking in vitro correlates of neuroplasticity, accomplished by measuring morphological and functional characteristics of neurons grown in a dish. This tracking then continues as the neurons age and are exposed to chemical compounds. Using those findings, Cognesy scientists are designing and synthesizing new, CNS-penetrant chemical analogs that are known to mimic existing drugs that stimulate neuronal plasticity but without the some of the undesirable side effects.

Compounds that show promise in stimulating neuroplasticity in vitro will be tested in rodents (in vivo) to confirm that the cellular effects translate into behavioral change. These in vivo phenotyping studies use animal models of human psychiatric diseases to predict whether the new compounds will be effective in treating human conditions such as depression and anxiety.

Still in the development/study phase, Cognesy has identified a set of phenotypes associated with neuroplasticity in vitro using reference compounds. Simultaneously, they have generated a chemical library of novel compounds, and are beginning to test their platform. The overall goal is to fully profile this library of compounds to identify novel molecules that induce neuroplasticity and are devoid of certain subjective effects such as altered consciousness.

WHAT'S NEXT?

Cognesy will continue to develop novel in vitro and in vivo assays to better identify neuroplastic changes and differentiate compounds based on their pharmacological characteristics and efficacy. In the future, they hope to develop compounds that induce positive brain change without the behavioral alteration that is often associated with other drugs in this class. In the near future, Cognesy’s breakthroughs could offer new pharmaceutical options to the millions of people like Suzy who are living with CNS disorders.

Based on DNA nanotechnology, Eascra’s novel family of Janus base nanomaterials provides highly effective, super biocompatible solutions that enable enhanced therapeutic delivery and regenerative medicine for articular cartilage, central nervous system disorders, solid tumors, and other chronic conditions.

Eascra’s Nanotechnology for Therapeutic Delivery

THE PROBLEM

A competitive runner, Sandra routinely ran marathons and triathlons and thought nothing of it. But lately both her knees hurt so much that she found hard to stick to her running schedule. She wrote it off as a momentary thing, training too much or simply running more than usual but at her next checkup, her doctor diagnosed her with osteoarthritis and advised her to consider other exercise options. The cartilage in both her knees had worn away from inflammation and age-related wear. At age 51, Sandra was facing increasing debilitation or possible knee replacements. Neither option suited her lifestyle.

According to the American College of Rheumatology, Osteoarthritis (OA) is the most common form of arthritis. It affects an estimated 302 million people worldwide (1–5) and is a leading cause of disability among older adults. The knees, hips, and hands are the most commonly affected appendicular joints. OA is characterized by pathology involving the whole joint, including cartilage degradation, bone remodeling, osteophyte formation, and synovial inflammation, leading to pain, stiffness, swelling, and loss of normal joint function. To date, typical available treatments focus on symptom relief until such time that joint replacement becomes the only viable treatment option.

THE SOLUTION

Eascra Biotech is creating an innovative way to treat musculoskeletal diseases such as OA with a new Janus base nanomaterial that mimics DNA. Janus base, named for the two-headed Greek god, Janus, is a versatile, new formulation that could become the basis for a whole host of new medical/therapeutic opportunities. Eascra’s technology is a flexible nanomaterial that can be assembled into various products such as a miniature cell-free scaffold or a therapeutic drug delivery container. Both are capable of delivering long term therapeutic relief to Sandra as well as maintaining her remaining knee cartilage, and in some cases, regenerating what she has lost. In early animal studies, Eascra’s Janus Base nanomaterials (JBNs) have proven to be highly effective doing just that – providing pain relief while enabling cartilage repair and regeneration in vivo.

What makes this new technology so innovative is, unlike existing lipid-based nanotechnologies, Eascra’s JBNs are temperature stable, less toxic with enhanced endosomal escape. Typically, orthopedic treatments include invasive surgeries or injected lubricants that provide short-term, temporary results. Instead, Eascra’s nanotechnology can be injected into hard-to-reach areas like cartilage for long term relief and potential tissue restoration. The successful commercialization of JBNs could provide physicians and their patients with new, less invasive, more efficacious alternatives. Eascra’s JBNs are a new chemical formulation. Janus base nanomaterials aren’t carbon-based or lipid-based like the current existing market options. Rather, JBNs mimic DNA, opening a world of options and possible treatment advantages based on JBN’s unique chemical makeup. It’s a whole new approach for age-related orthopedic diseases.

WHAT'S NEXT?

Eascra and JBNs go to space! In April 2022, Eascra received $1.86M from NASA (to conduct proof of concept studies on their new nanotechnology (JBNs). Working in microgravity will provide the Eascra team with a unique, once-in-a-lifetime opportunity to evaluate potential benefits of assembling JBNs of greater quality and consistency in a low gravity lab environment. They believe this provides a higher potential for effective commercialization. The team plans to improve the quality of life on Earth based on its versatile testing in space. With their NASA collaboration, along with their partnership with premier private space leader Axiom Space, the Eascra Team is excited to take their promising Janus base innovative technology into orbit in early spring 2023. Eascra’s goal is to provide an innovative therapeutic delivery system that can support as many medical advances as possible while also opening the way for other commercial ventures to leverage the emerging space economy to accelerate advances in science that will mean better patient care in the near future.

The nervous and immune systems are completely integrated in health and in disease. MindImmune is turning this insight into novel therapeutics targeting the immune system to treat brain disease.

MindImmune Therapeutics: Potential Alzheimer’s Therapy

THE PROBLEM

Mark was 63 when he started misplacing items around the house. His wife, Susan, started to notice his forgetfulness as he began struggling to carry out daily activities. At his next doctor’s visit, he was diagnosed with Alzheimer’s Disease. With no cure, all Susan could do was try give Mark the care he needed as his symptoms progressed. He passed away at the age of 70.

Alzheimer’s is a devastating disease that, according to the Alzheimer’s Association, affects over 6 million Americans and is projected to double by 2050. Scientists and researchers are working hard to slow the progress of the disease but there has been little success. For years, beta-amyloid plaques have been a suspected cause of the disease but there needs to be more widespread research. To find a cure, we need research on every possible factor that contributes to the brain’s neurodegeneration to bring a cure to the families and individuals affected by Alzheimer’s.

THE SOLUTION

Led by Dr. Frank Menniti, Dr. Stevin Zorn, and Dr. Robert Nelson, MindImmune Therapeutics is blazing a new trail, striving to understand the immune effector mechanisms underlying brain diseases. In looking at the immune system and its role in brain dysfunction, MindImmune discovered that innate immune cells that come in from the periphery into the brain may be a cause of synaptic damage in Alzheimer’s disease. Their goal is now to create a breakthrough drug that prevents these cells from entering the brain in the first place. This research is incredibly innovative and takes a proactive approach against the onset of Alzheimer’s.

It is also conceivable that the research at MindImmune can be applied to other chronic neuro-inflammatory diseases. Since innate immune cells can be found in other diseases such as multiple scleroses, it is possible that this is a more general pathological mechanism that can be utilized in creating treatments for other diseases. Exciting as that idea is, for the time being MindImmune is focusing on Alzheimer’s.

WHAT'S NEXT?

It has been years since the Alzheimer’s community has seen hope for an effective therapy. MindImmune’s innovative scientists are currently working through preclinical studies in mice, and it is expected that a clinical candidate will be identified in the first quarter of next year. So, though the process is long and tedious, MindImmune’s work developing a therapy that attacks peripheral immune cells could offer new hope.

Focused on creating unconstrained, Sense’s instrument-free molecular diagnostic technologies allow caregivers to open the care pathway to everyone – in a way that’s nothing short of revolutionary.

Sense Biodection, Inc.

THE PROBLEM

Jane, who lives in a nursing home, has been experiencing severe headaches and neck stiffness. While her dedicated care team correctly suspected meningitis, the official medical diagnosis was delayed due to the lengthy wait time for the lab results. In the short term, the delay prolonged Jane’s ability to get treatment with antibiotics. Had the lab work taken even longer, it is likely she would have experienced life-threatening blood poisoning and nerve damage.

While there is much focus on continuously engineering and reworking treatments to further improve healthcare, the detection of diseases and the ability to take a proactive approach to heal is equally as important. Rather than sending samples to a laboratory, there is growing need for diagnostic tests to be performed directly at the place of care to minimize time consumption and inefficiency. In nursing homes and other care facilities, the need for regular testing is even more urgent. If such tests are not easy to use or too slow with results, health can be jeopardized.

THE SOLUTION

A group of innovative scientists looked at this problem and were inspired to launch Sense Biodetction Inc., a company that strives to revolutionize diagnostic care by creating technology that can aid both caregivers and patients in the process of molecular diagnostics, which are techniques of analyzing biological markers in an individual to diagnose them. Through the Veros Instrument Free Molecular Test, accurate lab-quality test results can be delivered in just minutes. With innovations in electrochemical design and chemistry, this technology utilizes a buffer that breaks down viruses into their genetic material to amplify them via highly specialized dried amplification reagents. Powered by a single battery, this amplification then spreads up a high contrast carbon-based read-out test strip that helps to clearly detect the target and improve the readability of the results.

Sense has created hand-held testers than can be more widely used by caregivers, in hospitals, urgent care, schools, and even during travel. Not only is this innovation easy to use and a competitor to constraints put on past equipment, but also cost-effective and convenient because it works without laboratory space or special disposal protocols. Sense Biodetection Inc. saw the need for easier access to diagnostic testing and has transformed it into an incredibly accessible and efficient process.

WHAT'S NEXT?

With this redefined path for diagnostics, Sense technology can help improve healthcare drastically. If you have an innovation in the life science community, let us know and we could feature you in our Innovation Profile series. Become an RI Bio member at RI-Bio.org. We connect members to a community of like-minded professionals who are also interested in big ideas and making a difference in the world. You’ll increase your visibility and have access to professional networking, educational forums and training, and purchasing discounts.

What’s your innovation?

If you have an innovation in the life science community, let us know and we could feature you in our Innovation Profile series. Become an RI Bio member at RI-Bio.org. We connect members to a community of like-minded professionals who are also interested in big ideas and making a difference in the world. You’ll increase your visibility and have access to professional networking, educational forums, and training, and purchasing discounts.