Monthly Archives

May 2019

Regenxbio building Maryland AAV plant to support gene therapies

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Regenxbio will construct a GMP facility in Maryland to produce adeno-associated viral vectors for its gene therapy programs using its NAV technology-based platform.

The good manufacturing practice (GMP) plant will be integrated into Regenxbio’s 132,000 square-foot headquarters in Rockville, Maryland, currently under construction. Financial details have not been disclosed but the manufacturing plant is expected to be operational in 2021.

“The creation of this additional manufacturing capacity using our platform suspension cell culture process will allow us to more efficiently advance our development programs from research stage to the clinic and ultimately to patients, while ensuring manufacturing capacity availability,” chief technology officer Curran Simpson said in a statement.

The plant will support the firm’s multiple gene-therapy programs through the production of Regenxbio’s NAV Technology-based vectors at scales up to 2,000 liters.

“Regenxbio’s novel and unique approach to gene therapy focuses on enabling cells in the body to produce therapeutic proteins or antibodies and targeting and correcting genetic defects,” Kenneth Mills, CEO of Regenxbio, said earlier this month during a financial conference call.

“Our NAV Technology serves as a backbone gene therapy research and is widely utilized gene therapy platform across diverse disease applications.”

The technology consists of over 100 adeno-associated viral (AAV) vectors including AAV7, AAV8, AAV9 and AAVrh10, which deliver engineered genes to cells. It has so far been used to treat over 200 patients in clinical trials for Regenexbio and its licensee partners.

Click here to read more via BioProcess International

QIAGEN’s QuantiFERON®-TB Access development program gains recognition from United Nations initiative for health innovation

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HILDEN, Germany & GERMANTOWN, Md.–(BUSINESS WIRE)–May 21, 2019–

QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) today announced that QuantiFERON ® -TB Access, a new solution under development for diagnosis of latent tuberculosis (TB) infection in low-resource regions with a high disease burden of tuberculosis, will be recognized by the Joint United Nations Programme on HIV/AIDS (UNAIDS) at the organization’s Health Innovation Exchange from May 21-23 in Geneva, Switzerland. The event will be held during the World Health Assembly and brings together ministers of health, nonprofits and industry to spotlight innovations with potential to change the world’s health landscape and benefit sustainable development.

QuantiFERON-TB Access (QFT ®  Access) is a proprietary new test designed to advance tuberculosis control in areas with limited infrastructure, including countries in Asia, Africa and Latin America. The World Health Organization (WHO) has classified 30 countries in these regions as high-burden, representing an estimated 85% of the global TB burden. QFT Access pairs highly sensitive digital detection with a complete workflow created to deliver cost-efficient results quickly and with unmatched simplicity. It eliminates the need for an extensive laboratory infrastructure while providing best-in-class QuantiFERON technology. Clinical trials are being conducted in 2019, and commercialization of QFT Access is expected to begin in 2020.

“We are pleased that UNAIDS selected QuantiFERON-TB Access for recognition at the Health Innovation Exchange. QIAGEN’s drive to develop QFT Access springs from the need for a modern, accurate test for TB infection screening in areas where the disease burden is high but resources and infrastructure are scarce,” said Thierry Bernard, Senior Vice President, Head of the Molecular Diagnostics Business Area at QIAGEN. “QFT Access builds on the success of QuantiFERON ® -TB Gold Plus, the modern gold standard for detection of latent TB infection. We have created a field-friendly Sample to Insight solution using a simple test to reduce costs and labor and avoid the need for cold chain, a computer or continuous power supply.”

Click here to read the entire press release.

Navitas Life Sciences Announces Acquisition of KAI Research, US-based Full-service Contract Research Organisation (CRO)

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PRINCETON, N.J.May 21, 2019 /PRNewswire/ — Navitas Life Sciences, a TAKE Solutions Enterprise today announced the acquisition of US based niche full service CRO and health research company, KAI Research Inc.

KAI Research brings on board top of class offerings in 3 key areas – Clinical Trial Management, Clinical Research Consultation, and Data Management & Standardization. Through this acquisition, Navitas Life Sciences expands Phase II and Phase III capabilities in North America adding to its current capabilities in Europe and APAC. KAI strengthens Navitas’ therapeutic expertise in mental health, musculoskeletal diseases, neurological disorders, pain, diabetes, infectious diseases and oncology for Pharma and medical devices. The company has differentiated offerings in the areas of patient registries and e-clinical solutions as well as long-standing relationships with clinical sites and trusted network of service providers across the United States and Africa.

Srinivasan H R, Managing DirectorNavitas Life Sciences said, “We are on track in terms of our acquisitions, KAI Research will further strengthen our capabilities in clinical services and expand our therapeutic expertise. Through this acquisition, we also gain a unique standpoint in serving Federal agencies like FDA, NIH, CDC, Department of Defense etc. Together, we intend to create a world class enterprise making meaningful strides in our ability to deliver improved trial outcomes to the bio pharma landscape. The addition of KAI’s capabilities translates to global trial capabilities across North AmericaEurope and APAC markets. Combining these operational and TA capabilities with our proprietary AI driven OneClinical platform allows us to be a great partner to small and mid-tier customers as well as to large pharma.”

Kathy Dimeo, President, KAI Research Inc said, We are happy to share that we have been acquired by Navitas Life Sciences. The complementary synergies between our companies will increase service offerings and capacity to serve our current and prospective clients and improve the quality of our service delivery, while opening our services to the global community.”

Click here to read the entire press release.

Montgomery County Economic Development Corporation (MCEDC) Launches New Bio Lab Pilot

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Helps Fund Wet Lab Space for Small Biotech Companies

The Montgomery County Economic Development Corporation (MCEDC) launches a year-long pilot program designed to help small biotech companies fund wet lab space in Montgomery County, Maryland. The Bio Lab Pilot project will assist the County’s growth-stage life sciences companies with their infrastructure needs.

The successful applicant must agree to be headquartered in the County for three years, and lease wet lab space that is 5,000 SF or less. The grant award will provide $10 per SF of lab fit-out costs, up to $30,000 per company.

“Young BioHealth companies are not always able to obtain conventional financing. These grants are designed to help fill the funding gap associated with wet lab costs,” said David Petr, President and CEO of MCEDC.

MCEDC will fund the pilot project with $250,000 from its FY19 budget, which includes $50,000 in funding from the Maryland Department of Commerce.

“Maryland Commerce is pleased to support this project, which will help offset the cost of wet lab space and encourage more small biotech companies to grow in Montgomery County,” said Kelly M. Schulz, Maryland’s Commerce Secretary.

Expanding local companies, new start-up companies, and relocating companies are eligible to apply for Bio Lab Pilot funds to fit-out newly leased lab space. Developers and building owners are ineligible to receive pilot program grants. The threshold criteria for a pilot award will be the demonstrated evidence of a funding gap that prevents the company from completing its new lab fit-out.

Click here to read the entire press release.

BioBuzz – Three Examples That Show Why The National Cancer Institute is a Goldmine of Blockbuster Technologies for Commercialization

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The 2019 Tech Showcase Focuses on Partnerships, Entrepreneurship and Business Resources for Commercializing Technologies

May 7, 2019

What do the technologies that led to the success of Kite Pharma, the creation of the pioneering immune-oncology cancer treatment avelumab (BAVENCIOⓇ) and the founding of miRecule, Inc. all have in common?

They are a few recent examples of the many technologies and companies spun out of The National Cancer Institute (NCI) in Frederick, Maryland. For years, NCI and the Frederick National Laboratory for Cancer Research (FNLCR) have been a wellspring of tech innovation that has helped create new companies and develop life-saving medicines.

On June 12th from noon to 5 pm, NCI will host its annual Technology Showcase, offering industry, inventors and entrepreneurs an invaluable opportunity to connect, explore NCI’s tech transfer process and receive expert advice on starting a new business and what it takes to commercialize a product. The event, held in cooperation with the Frederick County and Frederick City governments, will be held at the FNLCR Advanced Technology Research Center in Frederick, Maryland.

The National Cancer Institute and Frederick National Laboratory 2019 Technology Showcase


Rich Bendis, President and CEO of BioHealth Innovation, Inc. will be the keynote speaker and sessions on attracting equity investors, non-dilutive funding sources, leveraging incubators and partnering with NCI/FNLCR highlight the showcase’s increased focus on the strong business case for tech transfer collaboration. What’s more, the showcase provides an excellent opportunity for direct engagement with technology inventors.

NCI’s Tech Transfer Center and FNLCR have an outstanding track record of successful technology licensing, fruitful collaborations and successful startup formations. Here are a few highly notable examples.

Kite Pharma, a successful T-Cell therapeutics company that recently announced its plans to build a manufacturing site in Frederick County, Maryland, was founded on technology transferred out of an NCI lab. Kite, before being acquired by Gilead in 2017, collaborated with NCI to license technology developed by immuno and gene therapy pioneer Dr. Steven A. Rosenberg. This NCI technology, licensed to Kite in 2014, eventually led to the development of FDA-approved YESCARTAⓇ, Kite’s approved CAR-T immunotherapy for a type of non-Hodgkin lymphoma. In addition, a significant segment of Kite’s current pipeline is also based on this same NCI technology. The power and promise of the NCI technology licensed by Kite is evidenced by Gilead paying $11.9 billion to acquire Kite without the company having an approved product at the time.

Lab Owl Transforms Pilot Scale-up lab at top Biotech company

Avelumab, commercially known as BAVENCIO, is a pioneering Merkel cell carcinoma treatment developed as part of a co-development agreement between EMD Serono, the biopharma division of Merck KGaA, and the NCI Tech Transfer Center. In 2017, avelumab was the first checkpoint antibody approved by the FDA for this lethal form of skin cancer. During the phase II trials, NCI and EMD Serono entered into a Cooperative Research and Development Agreement (CRADA) for avelmulab. Just four years after the first NCI-led studies, avelumab received FDA approval for Merkel cell carcinoma and another indication. Merck KGaA and Pfizer entered into a collaboration to jointly develop and commercialize Bavencio® (avelumab) in a deal worth up to $2.85 billion for Merck KGaA. The collaboration has been recognized by GEN as one of the top 10 Immuno-Oncology Collaborations.

miRecule, Inc., a Gaithersburg, Maryland-based early-stage biopharmaceutical company focused on developing microRNA-based therapies for drug-resistant cancers, is another startup company spun out of NCI. The company and NCI entered into a CRADA for microRNA therapeutics for treating head and neck squamous cell carcinomas. miRecule, founded in 2016, is currently raising $1 million in seed capital and has won several grants, including a Small Business Innovation Research (SBIR) grant. As of July 2018, the company had raised $500,000 in grant funds and $250,000 from private investment (Source: Washington Business Journal). miRecule’s founder and CEO Anthony D. Saleh, who is the inventor of the company’s genomics-based drug discovery platform, is also a former NCI postdoctoral fellow and former ‘Entrepreneur in Residence’ at BioHealth Innovation.

At the 2018 Tech Showcase, Kedar Narayan, Ph.D. from the Center for Molecular Microscopy, FNLCR gave a presentation on “Nanoscale 3-D imaging of cells and tissue by FIB-SEM (focused ion beam scanning electron microscopy)”. Since that presentation, NCI has entered into an early agreement to co-develop the FIB-SEM technology. This technology provides 3D, high-resolution electron microscopic imaging of cells and tissues. The technology can scan a block of tissue layer-by-layer, for example, capturing hi-resolution images, which can then be recombined into a 3D shape without 2D data compression challenges. FEB-SEM could potentially help researchers locate drug targets and loading vehicles more quickly to accelerate R&D for life-saving treatments. This technology has received strong interest recently from a major pharmaceutical company, according to NCI.

The aforementioned success stories are just a few examples of powerful NCI/FNLCR collaborations that have led–and could lead to–powerful treatments for patients in need.

The 2019 Tech Transfer Showcase is not limited to scientists talking to scientists. This year’s showcase will feature technologies, inventors and educational sessions designed to help startup businesses navigate tech transfer, attract the right investment capital and leverage available incubator/accelerator resources to be successful.

The Technology Showcase is unique in that technical innovation is directly tied to the commercial opportunity. If you’re an entrepreneur or in industry and are looking to better understand the market landscape, economic opportunity and commercial potential associated with NCI/FNCRL technologies, mark the 2019 Technology Showcase on your calendar today.

Click here to learn more via BioBuzz.

FDA clears RemUnity drug pump co-developed by Deka, United Therapeutics

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United Therapeutics and Deka Research and Development have received FDA 510(k) clearance for the co-developed RemUnity system.

The RemUnity system is a subcutaneous delivery system for Remodulin injections. The two companies have co-developed the device that is indicated to treat pulmonary arterial hypertension.

“We developed the RemUnity system to address safety and patient convenience problems with current subcutaneous infusion pumps,” said Martine Rothblatt, chairman and CEO of United Therapeutics. “We believe the RemUnity system reduces the risk of bolus dosing due to pump failures and provides wider arrays of notifications, alerts and alarms than current pumps. Most importantly, the acoustic volume sensing technology and solid-state actuator of the RemUnity system enables it to control Remodulin flow rates without the use of a motor.”

RemUnity features a small, lightweight, durable pump that has a service life of at least three years, according to the company. It uses disposable cartridges that are connected to the pump.

“We are excited to be launching this innovative delivery technology with United Therapeutics. We are confident that it will substantially improve the lives of patients who depend on UT’s unique pharmaceutical advances,” said Dean Kamen, founder and president of Deka. “We look forward to continuing to deliver advanced solutions for patients in need.”

Click here to read more via

With promising pivotal data, Viela Bio’s lead autoimmune drug heads for FDA filing

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Viela Bio spun out of AstraZeneca last year with six autoimmune and inflammation-aimed programs and $250 million. Now, the biotech is reporting positive pivotal data for its lead drug in a rare, autoimmune disease, teeing it up for an FDA filing later this year.

Viela tested the drug, a CD19-targeting antibody called inebilizumab, in patients with neuromyelitis optica spectrum disorders (NMOSD), a disease in which the immune system attacks the optic nerves, spinal cord and brain. Current treatments fall into two groups: those used when a patient is having an attack and those used to ward off attacks. There is no treatment approved specifically for NMOSD, so the steroids and immunosuppressants prescribed to prevent attacks are used off-label.

The data, presented Tuesday at the annual meeting of the American Academy of Neurology, showed the treatment reduced the risk of attacks by 73%.

“Neuromyelitis optica attacks can be quite devastating. They can result in a rapid loss of vision up to and including blindness and paralysis. The key is to stop attacks from happening,” Viela Bio Chief Medical Officer Jorn Drappa, M.D., Ph.D., told FierceBiotech.

“None of the currently used drugs have been studied in a rigorous fashion in NMOSD—there has not been a controlled clinical trial that establishes these treatments are effective. Ours is the largest clinical trial done in neuromyelitis optica,” Drappa said.

The study enrolled 231 patients with varying severities of disease, said Viela Bio CEO Bing Yao. Most of the patients had autoantibodies against a protein called aquaporin-4 (AQP4), while a small number of them did not. These AQP4 antibodies are a biomarker of NMOSD and are believed to trigger NMOSD attacks when they bind to the nervous system.

Click here to read more via FierceBiotech.

Five winners to receive share of $250K NCC-PDI grant funding and access to unique pediatric device accelerator program

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WASHINGTONMay 1, 2019 /PRNewswire/ — The National Capital Consortium for Pediatric Device Innovation (NCC-PDI) announced five winners of its “Make Your Medical Device Pitch for Kids!” competition who will each receive $50,000 in grant funding and access to the consortium’s first-of-its-kind “Pediatric Device Innovator Accelerator Program” led by MedTech Innovator. A panel of expert judges from business, healthcare, regulatory and legal sectors selected the winners based on the clinical significance and commercial feasibility of their medical devices for children. The competition focused solely on advancing care in pediatric orthopedics and spine, a sector that the FDA identified as an emerging underserved specialty which lacks innovation.

The 2019 NCC-PDI “Make Your Medical Device Pitch for Kids!” competition winners are:

  • AMB Surgical, LLC, Dayton, Ohio – FLYTE, a device designed to reduce invasive and repetitive surgery in children and teens with orthopedic illnesses such as scoliosis and limb abnormalities
  • Auctus Surgical, Inc., San Francisco, Calif. – Auctus Surgical Dynamic Spinal Tethering System, a mechanism used to correct the scoliotic spine in pediatric patients through a tethering procedure
  • ApiFix Ltd, Boston, Mass. – ApiFix’s Minimally Invasive Deformity Correction (MID-C) System, a posterior dynamic deformity correction system for surgical treatment to provide permanent spinal curve correction while retaining flexibility
  • Children’s National Health System, Washington, D.C.– Babysteps platform to improve initial assessment of clubfoot deformity and predict the magnitude of correction
  • nView Medical, Salt Lake City, Utah – Surgical scanner using AI-based image creation to provide instant 3D imaging during surgery to improve imagery speed and accuracy

The competition was held at the University of Maryland, College Park by NCC-PDI, one of five FDA Pediatric Device Consortia grant programs that support the development and commercialization of pediatric medical devices. NCC-PDI is led by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Health System and the A. James Clark School of Engineering at the University of Maryland. The consortium recently added new accelerators BioHealth Innovation and MedTech Innovator and design firm partner, Smithwise.

Through the inaugural NCC-PDI “Pediatric Device Innovator Accelerator Program,” MedTech Innovator is providing winners with virtual in-depth, customized mentorship from some of the industry’s leading executives and investors. With its prestigious Showcase & Accelerator program, MedTech Innovator has a proven track record of identifying early-stage medical device companies with the key characteristics required for commercial success and accelerating their growth through its vast ecosystem of resources.

Click here to read the entire press release.

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