Venture Day at Chesterfield in Durham NC

Duke’s 1st Venture Day

Venture Day Showcased 8 Duke Start-ups in Biotech & Therapeutics

On Wednesday, May 1st, over a dozen venture capital firms gathered for the first Duke Venture Day at the newly refurbished Chesterfield Building in downtown Durham.

Therapeutics Start-ups By the NumbersThe day-long event showcased therapeutic innovations currently coming out of Duke. This year’s goal was to engage the venture investing community in the drug and biotech arena to advance fundable ideas and develop deeper connections with Durham.

“What we’re seeing is more and more biotech investors interested in exploring outside the traditional biotech hubs of Boston and the Bay Area,” said Rob Hallford, Director of Duke New Ventures. “Duke Venture Day is about raising the profile of not just Duke spin-outs, but the Triangle in general as a place to find exciting new companies and put capital to work.”

Eight Duke start-up investment opportunities were presented at the Venture Day–from highly targeted radiotherapy for brain metastasis to therapies for ocular graft-versus-host disease.

As a former cigarette factory turned medical and engineering research lab, The Chesterfield set the stage to promote Durham’s transformation from a tobacco town to an innovation hub. Additionally, with a fishbowl-like view right off the open atrium, the NC Biolabs easily elucidated how its shared lab facilities can help launch a newly formed start-up from just a single bench.

4 VC chatting before Venture Day at the Chesterfield
Venture Capitals chatting before Venture Day in the Chesterfield Building in front of the BioLabs.

The Biolabs currently has 36 start-ups working in the shared lab space, 15 of them are Duke newcos. An early tenant and Duke spinout, Element Genomics, has already had a successful exit, having been acquired by UCB for $30 million.

Entrepreneur turned venture capitalist, Clay Thorp from Hatteras Venture Partners was on hand to moderate a panel on Doing Biotech Business in North Carolina. Panelists included Mark Velleca from G1 Therapeutics, Meg Booth Powell from TARGET Pharmasolutions, and Matt Kane from Duke start-up Precision Biosciences.

Venture Day Panelist
Venture Day Panelist, Meg Booth Powell, Matt Kane, Mark Velleca, with moderator Clay Thorpe.

“I firmly believe that Durham’s unique combination of a highly skilled community and relatively low cost of doing business, makes it the ideal place to start and build a biotechnology company,” said Matt Kane, CEO and co-founder of Precision Biosciences.

Precision, a genome editing company dedicated to improving life through its proprietary ARCUS genome editing platform located in downtown Durham, recently went public last month raising over $145M.

MEET THE FEATURED INNOVATORS

Each of the presenting start-up companies had the opportunity to speak in front of venture capitals, both local and from as far north as Boston.

CereiusCereius Logo, which aims to improve the lives and survival of patients facing solid tumor brain metastasis through the use of personalized and highly targeted radiotherapy, was presented by Michael Zalutsky, Professor of Neuro-Oncology Research and a Professor in the Departments of Radiology, Radiation Oncology, Pathology and Biomedical Engineering

 

GavilGavilan Biodesign logoán Biodesign, founded out of Bruce Donald’s laboratory in Computer Science, Chemistry, and Biochemistry, was recently selected as one of 11 early-stage biotech companies at the Silicon Valley biotech accelerator, IndieBio. Their technology combines state of the art physics-based modeling with a unique high accuracy AI platform to computationally screen trillions of molecules in order to find therapeutics that can overcome resistance.

 

Tellus TherapeuticsTellus Therapeutics Logo, founded by neonatologist Eric Benner, is developing novel small molecules derived from human maternal breast milk demonstrated to induce regeneration of myelin-producing oligodendrocytes and reverse white matter injury in animal models of perinatal brain injury.

 

Cellective BioTherapyCellective biotherapy logo, founded by veteran entrepreneur and noted immunologist Tomas Tedder, is developing first-in-class regulatory B10 cell-based immunotherapies for treating cancers, autoimmunity, and immunodeficiency that can either enhance or inhibit immune responses as needed for disease treatment.

 

Sisu Pharma LogoSisu Pharma, Inc. recently founded by Dennis Thiele, Professor of Molecular Genetics & Microbiology and Jiaoti Huang, Chair for the School of Medicine’s Department of Pathology, is developing drugs against a new target to treat prostate cancer in patients who have exhausted all therapeutic options.

 

 

Mastezellen Bio LogoMastezellen Bio, Inc., started by fellow pathologists, Herman Staats and Soman Abraham, is developing novel therapeutics to inhibit mast cell activation, potent proinflammatory cells that cause symptoms of allergy and anaphylaxis in a wide range of disease states such as adverse drug reactions, food allergy, mastocytosis, and seasonal rhinitis.

 

Basking Bio LogoBasking Bio, founded by endovascular neurosurgeon and research scientist, Shai Nimjee, is developing a drug-antidote pair for treatment of patients with ischemic stroke using technologies founded out of Duke Professor of Surgery, Bruce Sullenger’s Cardiovascular Biology Lab.

 

Pulsar Life Sciences LogoPulsar Life Sciences is another company started by ophthalmologist Scott Cousins developing an eye drop formulation for the treatment of inflammatory dry eye disease, including ocular graft versus host disease that occurs after hematopoietic stem cell transplantation.

dial device invented at Duke

Hungry for Innovation: Notable Project Stems from MEDx Dinner

By Alexis Kessenich

On A Chilly December Evening In Durham Guests Arrived At The Restaurant At The Durham Hotel With A Hunger For Food And Innovation. Seated around a U-shaped table, gastroenterology and engineering professionals brainstormed engineering solutions to medical problems. What started as a small working dinner hosted by MEDx quickly took on a life of its own.

Duke Medx logo

With empty plates and full minds, guests left The Durham eager to put their ideas to work. Attendees formed countless connections and projects that originated at The Durham continue to flourish. One in particular, a Duke-patented and licensed ergonomic improvement for endoscopes, embodies the collaborations MEDx was created to foster.

Students working on endoscope design

Students with prototype endoscopy dials. Photo credits, Chris Hildreth

The idea for the dinner began when Andrew Muir, M.D., chief of gastroenterology, approached Geoff Ginsburg, M.D., Ph.D., director of MEDx, for advice on how to develop collaborations between his department and the Pratt School of Engineering. Ginsburg proposed that MEDx host a dinner for faculty in each department. “One of my major goals has been to connect my people with the amazing faculty and programs at Duke,” said Muir. “This connecting is the spirit of MEDx.” Muir invited his faculty to submit ideas to improve the medical field, and Donna Crenshaw, Ph.D., executive director of MEDx, invited engineering faculty that had expertise in those areas.

A member of Muir’s team, gastroenterologist Darin Dufault, proposed improving the device used during endoscopies. During an endoscopy, a tool called an endoscope is used to examine a patient’s digestive tract. Articulating dials located on the handle of the endoscope are used to control its position during procedures. With these dials, the operator moves the tip of the endoscope up, down, left or right with one hand. But this design imposes significant physical stress on the operator, including pain, discomfort and stress-induced injuries. The handles also impose ergonomic challenges as they are not designed to adapt to varying hand sizes and grip strengths.

Dufault arrived to the MEDx dinner with an idea for a new device that would address these limitations. Over dinner, he formed a connection with Mark Palmeri, a professor of the practice of biomedical engineering, who felt he could help bring Dufault’s idea to life. Palmeri then introduced Dufault to a team of engineering graduate students to help.

“An ergonomics-based project appealed to me because we had never really done that before.” said Palmeri, “Dufault brought in anecdotal experience in how the ergonomics of their equipment is subpar, but since there’s nothing else out there to use, you are at the mercy of it.” Dufault and the team of students advised by Palmeri experimented with around 100 different iterations over the course of several months before finally settling on a design.

The winning device. This tool snaps onto the endoscope to improve functionality

The winning device, a tool that snaps on to the dials of an endoscope, addresses the current limitations with customizable attachments and extensions for the articulating dials that are compatible with standard endoscopes. “The device has two purposes,” says Dufault. “First, it reduces the reach necessary to manipulate the ‘little’ dial – the one that moves the scope from left to right – making it easier for people with smaller hands and reducing strain on the hand for all users. Secondly, it increased the radius of the ‘little’ dial, which further reduces the strain necessary to manipulate the ‘little’ dial.”

Duke patented the group’s device, and the patent has been licensed by a venture capital company, OBX Associates, which focuses on specialized mobile devices. Plans are already underway to begin manufacturing and distribution in the U.S. and abroad.

For Dufault and Palmeri, the collaboration won’t stop here. The pair already has plans to meet and discuss new and innovative ideas.

“Through these types of collaborations, engineers are brought problems they may otherwise not know about,” says Dufault, “and clinicians can contribute to the solution in ways that would not be possible without engineers.”

Palmeri hopes that as Duke’s design classes keep growing, medical and engineering professionals will come together regularly to figure out what’s ripe for the picking to tackle in these classes. “By getting wins like this one,” he says, “clinicians get even more excited about engaging with us. MEDx serves as a really great icebreaker for us to do so.”

“The human connection of meeting face-to-face at dinners like this one is important,” says Dufault. “No doubt in my mind it fosters collaboration and interest in working together.”

Palmeri says he appreciates that the MEDx dinner brought together junior- and senior-level people in each department to the same environment. “Young faculty are typically not folks we get the opportunity to collaborate with on projects, but this allowed us to see really neat or new ideas from the enthusiastic, young clinical faculty.”

MEDx hopes to host more dinners like this in the future. “There is so much latent potential at Duke for these types of collaborations to happen,” said Ginsburg, “which is why MEDx can be so strategic at turning a small investment on our end into potentially revolutionary designs – a small investment can result in a huge impact.”

If you are interested in having a collaborative event with members from your department and the adjacent school, contact MEDx.

lindy microglassification

Lindy Biosciences

MANAGEMENT: Deborah Bitterfield
DUKE INVENTOR: David Needham
lindybio.com

Lindy Biosciences is a development-stage protein therapeutic formulations company. Our core technology produces spherical, dense, stable particles of a therapeutic protein. These protein particles are ideal for long-term storage, or for non-standard formulations such as suspension formulations (for high-concentration delivery of antibodies), encapsulation (for sustained/controlled release), or dry powder pulmonary delivery.​

lindy biosciences logo

MICROGLASSIFICATION™

Produces dense, dry, stable protein particles ideal for suspensions or encapsulation

Microglassification™ is a process that gently removes a majority of the water from solutions of proteins, or other biologics, resulting in solid, spherical, amorphous microbeads. In this dry state, biologics are often stable enough for long-term storage, transport, or incorporation into drug delivery formulations.

The video shows one of our early experiments: Microglassification™ of a single protein microdroplet, held on the end of a glass capillary micropipette. The resulting solid microbead consists of pure protein at > 1 g/mL. Currently used methods mimic this experiment, producing protein microdroplets on a bulk scale and dehydrating those microdroplets within seconds.

High-Concentration Biologics

Biological molecules such as monoclonal antibodies often require dosing of several hundred mg. In order to allow subcutaneous administration, which can only accomodate 1-2 mL of volume, the protein must be formulated at high concentration (>200 mg/mL). However, the viscosity of solutions of these molecules increases dramatically above 100 mg/mL, making injection prohibitively difficult.

Suspensions of Microglassified protein can have a much lower viscosity than solutions of the same concentration. Preliminary formulations have been injectable through a 27G needle at >500 mg/mL.

Metacept Systems

MANAGEMENT:
DUKE INVENTOR: David Smith
metaceptsystems.com

Metacept provides design and engineering services for complete electromagnetic systems, with extensive experience in transitioning metamaterials to real-world applications. We can provide end-to-end development resources.

metacept logo

THE TECHNOLOGY

Imaging

Rethinking the problem of electromagnetic imaging from the ground up, Metacept designs hardware together with software to reach the physical limits of imaging technology.

Metamaterials

Metamaterials are a novel way to engineer material properties using micro-structural design. Metamaterials are able to intricately and precisely control many types of wave dynamics from electromagnetic to acoustic fields.

Physics-based Deep Learning

Deep learning has enabled machines to perform classification tasks. Metacept goes a step further to physically implement neural networks with electromagnetic waves to process data at the speed of light.

The Skyfront Perimeter UAV taking off from the Alyeska trans-Alaska pipeline right of way near Fox for the first true beyond-visual-line-of-sight commercial flight, approved by the Federal Aviation Administration under the small UAS rule. The UAV flew 3.87 miles along the pipeline corridor.

Echodyne’s Role in 1st FAA Approved Out-of-Site Drone Flight

A public-private consortium led by the University of Alaska has conducted the first-ever federally-authorized test flight of a drone beyond the operator’s line of sight without on-the-ground observers keeping watch – with Echodyne, the radar venture that’s backed by Microsoft co-founder Bill Gates and headquartered in Kirkland, Wash., playing a supporting role.

echodyne radar product

Autonomous flight beyond visual line of sight will be key to the kinds of drone delivery operations envisioned by Amazon, Walmart and other retailers.

During Wednesday’s flight, a Skyfront Perimeter multirotor drone inspected a 3.87-mile stretch of Trans-Alaska Pipeline infrastructure as part of the University of Alaska’s Unmanned Aircraft Systems Integration Pilot Program, one of 10 such programs that won approval from the Federal Aviation Administration last year.

The big thing about this flight is that the drone made use of Iris Automation’s Casia onboard detect-and-avoid system, paired up with Echodyne’s ground-based MESA airspace management radar system, without having a human on the route.

Current FAA regulations limit drone flights to the operator’s visual line of sight. Pilot projects have been experimenting with technologies that can ensure safe operations beyond the visual line of sight, known as BVLOS. But until now, the FAA’s waivers still required a ground-based observer to look out for non-cooperative aircraft coming into the test area.

This week’s flight of a drone totally on its own was authorized after it flew the same route with visual observers.

“The test mission designed by the team at the University of Alaska at Fairbanks is an excellent demonstration of the potential for commercial UAS,” Eben Frankenberg, the founder and CEO of Echodyne, said in a news release. “With Iris Automation and Echodyne sensor technologies, routine commercial missions like linear inspection and medical deliveries to remote communities are both practical and safe.”

The radar system developed by Echodyne relies on metamaterials technology, which uses specially structured electronics to bend electromagnetic waves. Circuits based on metamaterials can allow for the construction of flat-panel radar devices that match the performance of larger, more expensive phased array antennas.

 

READ THE FULL STORY HERE

 

[Originally posted by Geek Wire — August 2, 2019]

artistic idea of quantum computing

Accessible Quantum Computing Just Around the Corner

Immense computing power on the cloud may not be far off, says IonQ COO.

Look at the vast machines being developed by IBM and Google, and it’s hard to imagine that quantum computing will ever be available to companies beyond FTSE 500s with multi-million-dollar budgets.

But, while it’s unlikely your server room will feature a superconductor quantum computer within the next three years, a world where medium and large businesses may have access to infinitely more powerful machines may not be far off.

ionq logo

According to Allied Market Research, a demand for quantum computing could drive a market worth US$5.8 billion by 2025, representing a compound annual growth rate of more than 30 percent.

But for Stewart Allen, COO of quantum computing startup IonQ, that figure may be an underestimation.

“What I would say is that in the next 18 to 24 months, when someone hits upon a real problem that they’re solving with real results from QC, then that could open the floodgates that makes [that figure] look small.

“In the next 12 months, it will be select groups [with access to quantum computers],” Allen told TechHQ. “12 months out from that, it will be ‘broad brush’, with everyone rushing out to train themselves– [access] will be across all industries.”

Smaller quantum contenders

Founded in 2015, IonQ has been built with “modest budgets”. But its technology– based on the use of ‘natural’ qubits– is attracting the interest of world-leading organizations.

Other ‘close’ rivals– or quantum computing companies of IonQ’s size– tend to use manufactured qubits made from silicon. IonQ is one of very few employing the method of ion trapping where qubits are made of natural particles such as atoms, electrons or protons.

 

READ THE FULL STORY HERE

 

[Originally posted by T_HQ — July 23, 2019]


About IonQ

We’re building the world’s best quantum computers to solve the world’s hardest problems.

We believe useful quantum computers will look as different from the laptops and smartphones we use every day as classical computers appear next to an abacus. And we believe the best way to build a quantum computer is by starting with nature’s qubit: the atom. Accurate, powerful, and flexible, ionized atoms are the heart of our quantum systems.

After decades of research, IonQ was founded in 2015 by Chris Monroe and Jungsang Kim with $2 million in seed funding from New Enterprise Associates, a license to core technology from the University of Maryland and Duke University, and the goal of taking trapped ion quantum computing out of the lab and into the market. The next year, we raised an additional $20 million from GV, Amazon Web Services, and NEA, and built two of the world’s most accurate quantum computers.

Phitonex Launches NovaFluor Dyes Enabling High-Res Analysis

Phitonex, Inc. launched their new suite of NovaFluor dyes today at CYTO2019, the 34th Congress of the International Society for Advancement of Cytometry, the largest industry conference in single cell biology.

Phitonex logo

New dyes developed on the NovaFluor platform were shown, which enable researchers to radically increase in the number of scientific questions they can answer, accelerating discoveries in biomarkers and treatments for life-threatening diseases.

“Our NovaFluor dyes address key unmet needs across the spectrum of cell analysis and help researchers answer substantially more questions per cell on extant flow cytometry instrumentation. We are incredibly excited to get our NovaFluor dyes into the hands of researchers and move forward with our game-changing InfiniFluor dyes,” said Michael Stadnisky, Ph.D, CEO of Phitonex.

Presentations describing the new dyes and the Phitonex platform technology were presented at CYTO Innovation and the Futures panel discussion by CEO Michael Stadnisky. Additionally, Phitonex won the CYTO Innovation Technology showcase based on its transformative technology, team, market opportunity, and business approach.

The Phitonex platform enables the deterministic engineering of optical properties to provide high-resolution analysis of single cells by flow cytometry, and in the future, other applications. Lower noise, less spectral overlap, and fluorescence-by-design means that Phitonex dyes immediately unlock a higher number of parameters across current instrumentation and provide unmatched cell population resolution to drive enhanced biological insight.

“By leveraging DNA as a structural tool, our platform technology allows us to customize fluorescent labels with a remarkable degree of flexibility,”  Craig LaBoda, Co-Founder and CTO said.

 

READ THE FULL STORY HERE

[Originally posted by Yahoo Finance — June 24, 2019]

Chip Lets Robots “Imagine” Their Actions Before Moving

Robots that can rapidly plan out their movements could accelerate factory automation—and help keep fragile humans safe.

Putting your hand in front of an industrial robot arm is not, generally, a good idea. These machines might move quickly and precisely, but they are so blind and stupid that they’ll gladly break a limb without so much as an “oops.”

realtime robotics logo

So it took a little courage to try this trick with a robot arm being tested at Realtime Robotics, a startup located in Boston’s Seaport neighborhood. I reached forward to intercept its movement as it grasped a widget from a table and moved to put it in a box. Thankfully, the robot paused, moved deftly around my outstretched arm, and then neatly deposited the item in its box. No broken limbs today.

This kind of graceful adaptability could prove incredibly useful for the robotics industry. There are some robots that can work alongside people, but they tend to be low-power, imprecise, and of limited use. The most capable, and powerful, industrial machines still have to work in very precisely controlled environments, away from soft, breakable humans.

“Even if you’re not worried about having humans next to the robot, you might want to modify your cell without incurring the cost of bringing in a technician,” says Sean Murray, a robotics engineer and cofounder at Realtime Robotics who showed me around.

The movement problem

A number of companies are trying to find ways around this problem. Some are testing sensors that will stop a powerful robots in its tracks if it spots an obstacle. Realtime Robotics is trying to go further, by giving robots the kind of low-level intelligence needed to move through the real world. This is the physical awareness that humans and animals take for granted whenever they move an arm or a leg.

In several different rooms at Realtime, industrial robot arms are testing the capabilities of a new chip that the company has developed to make this possible. When hooked up to 3D sensors, this chip lets the machines rapidly consider a range of different actions, effectively “imagining” the outcome, before choosing the one best suited to the task at hand. In one room, I watched as two robots performed balletic feats of teamwork, gliding around one another and occasionally handing over items.

“The fundamental challenge is that robots are so stupid,” says George Konidaris, founder and chief roboticist at Realtime as well as an assistant professor at Brown University in Providence, Rhode Island. “We have this basic motor competence and robots don’t.”

Motion planning is deceptively difficult for a robot, partly because each joint adds an extra dimension to the calculations that must be performed.

Make your move

The company’s chip supercharges the mathematical computations behind a relatively simple motion-planning algorithm developed by Konidaris and others while he was at Duke University. By running the computations in parallel, the dedicated chip can perform them more than 10,000 times more quickly than a regular computer chip, while also using less power.

“The approach is very clever,” says Tomás Lozano-Pérez, a professor at MIT who advised Konidaris when he was a graduate student.

READ THE FULL STORY HERE

[Originally posted by MIT Technology Review — June 17, 2019]

ABOUT REALTIME ROBOTICS

We are transforming automation in its broadest sense by enabling machines to recognize, respond and decide how and where to move in milliseconds, even in variable environments. Our RapidPlan processor harnesses cutting-edge computer processing and software to end the trade-off between speed and safety that’s holding automation back today.

Enzyvant: FDA Acceptance of Biologics License Application

Enzyvant Announces FDA Acceptance of Biologics License Application (BLA) and Priority Review Status for RVT-802, a Novel Investigational Tissue-Based Regenerative Therapy for Pediatric Congenital Athymia

RVT-802, a one-time therapy, leverages Enzyvant’s T cell generation platform designed to treat profound immunodeficiencies

Left untreated, congenital athymia is uniformly fatal, with death typically occurring in first 24 months of life

Company to present at Roivant Pipeline Day in New York City on June 6, 2019

enzyvant logo

CAMBRIDGE, Mass. & BASEL, Switzerland–(BUSINESS WIRE)–Enzyvant, a biopharmaceutical company focused on developing and commercializing transformative therapies for patients with rare, often fatal conditions, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its Biologics License Application (BLA) for RVT-802, a novel investigational tissue-based regenerative therapy designed to treat pediatric congenital athymia, and granted Priority Review. Congenital athymia is a rare and deadly condition associated with complete DiGeorge Anomaly (cDGA), CHARGE syndrome, and FOXN1 deficiency. At this time, the FDA is not planning to hold an Advisory Committee meeting to discuss the application, and Enzyvant anticipates a regulatory decision in December 2019.

“We look forward to the potential of RVT-802 becoming available as an approved regenerative medicine to all families and patients who could benefit from it.”

Children with congenital athymia are born without a thymus, resulting in a severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. Approximately 20 infants are born each year in the United States with congenital athymia, which is fatal if untreated. Death typically occurs in the first 24 months of life due to susceptibility to infection. Currently, there are no FDA-approved therapies for this condition. RVT-802 stimulates and facilitates the body’s production of naive, immunocompetent T cells, with the goal of bolstering the immune system and restoring the body’s ability to fight infection. Investigational RVT-802 is designed to be administered as a single treatment.

“We are proud to be advancing RVT-802, a regenerative therapy that embodies bold, transformative science. The intense urgency to treat infants and young children who would otherwise succumb to congenital athymia drew us to forge a partnership with Duke University and continues to motivate us to advance toward a potential approval with focus and speed,” said Rachelle Jacques, Chief Executive Officer of Enzyvant. “The long-term data for RVT-802 as a one-time treatment reinforces the potentially life-saving value and durable impact of this therapy. We are committed to working collaboratively with payers to establish a value-based reimbursement model that accelerates access for patients.”

The BLA filing for RVT-802 included clinical data that demonstrated long-term durability of treatment with RVT-802. At the time of the BLA filing, a total of 93 patients received RVT-802 across multiple clinical studies, including 85 patients who met the criteria for inclusion in the efficacy analysis. The Kaplan-Meier estimates of survival [95% confidence interval] at year one and year two post treatment were 76% [66 – 84] and 75% [66 – 83], respectively. For patients surviving 12 months post-treatment, there was a 93% probability of surviving 10 years post-treatment. During clinical development, the most commonly (≥ 5%) reported RVT-802 related adverse events included thrombocytopenia (11%), neutropenia (8%), pyrexia (5%), and proteinuria (5%).

“The journey of this therapy has involved the dedication and contributions of so many and, most notably, the bravery of patients and their families,” said Dr. Louise Markert, Professor of Pediatrics at Duke University School of Medicine, whose pioneering work at Duke led to the development of RVT-802. “It is gratifying to see this therapy advance a significant step closer to a potential FDA approval. We are hopeful we can look to a future of continuing to save children’s lives.”

“We congratulate the Enzyvant team on this important milestone, as well as Dr. Markert and her colleagues at Duke for their remarkable scientific accomplishments and dedication to athymic patients and their families,” said Myrtle Potter, Vant Operating Chair at Roivant Pharma, and Chair of Enzyvant’s Board of Directors. “We look forward to the potential of RVT-802 becoming available as an approved regenerative medicine to all families and patients who could benefit from it.”

Ms. Jacques will be presenting at Roivant Pipeline Day in New York City tomorrow, June 6, at 4:20 p.m. ET. To request access to the webcast or to learn more about Roivant Pipeline Day, please email pipelineday@roivant.com.

About RVT-802

RVT-802 is a novel investigational tissue-based regenerative therapy designed to treat primary immune deficiency resulting from pediatric congenital athymia. In a healthy, functioning immune system, T cells that start as stem cells in bone marrow become fully developed in the thymus. RVT-802 is designed to replicate this process in the absence of a thymus.

Derived from infant thymus tissue, RVT-802 is processed and cultured prior to implantation into a patient’s quadricep muscle. The patient’s bone marrow stem cells migrate to the implanted tissue product, where they are trained to become naïve, immunocompetent T cells. With the renewed ability to generate T cells, immune system function can be restored.

RVT-802 has been granted Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Orphan Drug designations by the FDA.

In 2016, Enzyvant entered into an exclusive worldwide licensing agreement with Duke University to develop RVT-802. M. Louise Markert, M.D., Ph.D., Professor of Pediatrics at Duke University School of Medicine, has led research on the treatment of immunodeficiency in patients with congenital athymia. The findings of Dr. Markert and her research team have been published in the New England Journal of Medicine as well as numerous other peer-reviewed scientific journals and clinical publications.

About Enzyvant

Enzyvant, a wholly owned subsidiary of Roivant Sciences, is a biotechnology company focused on developing transformative therapies for patients with rare diseases. Enzyvant leverages the Roivant platform to develop therapies that address high unmet medical needs while driving greater efficiency in research, clinical development, and commercialization. The FDA has accepted Enzyvant’s Biologics License Application submission for RVT-802, a novel investigational tissue-based regenerative therapy for the treatment of congenital athymia and granted Priority Review. Enzyvant anticipates a regulatory decision in December 2019. The company is also preparing to initiate a clinical trial of RVT-801, an investigational enzyme replacement therapy for the treatment of Farber disease. For more information, please visit www.enzyvant.com.

About Roivant

Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. Roivant does this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. For more information, please visit www.roivant.com.

About Roivant Pharma

Roivant Pharma is the biopharmaceutical business unit of Roivant Sciences. Roivant Pharma is focused on end-to-end biopharmaceutical company creation, launch, and oversight. Roivant Pharma companies include Altavant, Aruvant, Axovant, Dermavant, Enzyvant, Genevant, Immunovant, Metavant, Myovant, Respivant, Urovant, and Arbutus.

About Roivant Pipeline Day

Roivant Pipeline Day will be held on Thursday, June 6, 2019 in New York City. The event will feature presentations and Q&A sessions from executives across the Roivant family of companies highlighting new clinical data and investments in technology. The event is scheduled to begin at 1:00 p.m. ET and will continue until approximately 4:30 p.m. ET. A live webcast will be available to interested parties. To request access to the webcast or to learn more about the event, please email pipelineday@roivant.com.

Contacts

Media:
Liz Melone
liz@scientpr.com

ORIGINALLY POSTED HERE