Biohealth Innovation
The great American political divide shows no sign of ending as the United States lurches toward the 2020 presidential election. However, one of the few areas where the parties have joined together in recent years has been where NIH funding is concerned.
Emergent BioSolutions Inc. (NYSE: EBS) today announced a contract award by the Office of the Assistant Secretary for Preparedness and Response (ASPR) in the U.S. Department of Health and Human Services (HHS) valued at approximately $535 million over 10 years for the continued supply of Vaccinia Immune Globulin Intravenous (VIGIV) into the U.S. Strategic National Stockpile (SNS) in support of smallpox preparedness.
REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced the first U.S. Food and Drug Administration (FDA) approval of a gene therapy that leverages REGENXBIO's proprietary NAV Technology Platform, Novartis AG's ZOLGENSMA® (onasemnogene abeparvovec-xioi; AVXS-101). ZOLGENSMA was approved by the FDA as a one-time infusion for pediatric patients with spinal muscular atrophy (SMA) who are less than two years of age.
A newborn. A fatal diagnosis. And soon, a one-time gene replacement cure in the first weeks of life.
The cost? You don’t want to know.
Today, the Biotechnology Innovation Organization (BIO) released the 2019 Emerging Therapeutic Company Trend Report, highlighting ten years (2009-2018) of biotechnology funding and deal making across five areas: venture capital, Initial Public Offerings (IPOs), follow-on public offerings, licensing, and acquisitions. The report also contains a 2019 snapshot of the industry’s clinical pipeline to highlight the significant contribution of emerging companies.
For aspiring entrepreneurs, the thought of building a company from scratch can be daunting. What if no one wants to buy what you're selling? Where will the money come from to grow the business? And what will happen if you fail?
The BIO International Convention is being held in Philadelphia from June 3-6, with more than 16,000 attendees expected from around the world. The convention draws a list of who’s who in drug discovery, biomanufacturing, genomics, biofuels, nanotechnology and cell therapy, with more than 7,000 companies and 47,000 partnering meetings.
June 12, 2019!
Register now for the 3rd annual Technology Showcase! The 2019 Technology Showcase will again highlight technologies being developed at the NCI and Frederick National Laboratory for Cancer Research (FNLCR) to encourage technology licensing and collaborations, startup company formation and the translation of NCI discoveries into solutions to benefit patients.
A Baltimore-based regenerative medicine firm called LifeSprout, which makes synthetic products used to restore soft tissue, has secured $6.5 million in financing.
A newly formed Baltimore company has raised $110 million to commercialize a cancer-detecting blood test, developed by Johns Hopkins researchers, that doctors would give patients during routine physicals.
QIAGEN (NYSE: QGEN; Frankfurt Prime Standard: QIA) today announced the immediate launch of its therascreen® PIK3CA RGQ PCR Kit (therascreen PIK3CA Kit) after it received U.S. regulatory approval as a companion diagnostic to aid in identifying breast cancer patients eligible for treatment with PIQRAY (alpelisib), a newly approved therapy developed and marketed by Novartis.
Regenxbio (RGNX) announced that a gene therapy developed based on its technology was approved by the FDA. This gene therapy product is known as Zolgensma, which Novartis (NVS) now owns after acquiring AveXis for $8.7 billion back in 2018. While Novartis does own Zolgensma, it will have to pay royalties to Regenxbio on net sales for it. That means Regenxbio can start earning revenue for one of its very first products. The company is in good shape because it has many other gene therapy programs in its pipeline. The most notable one would be RGX-314, which is being developed to treat patients with wet age-related macular degeneration.
The following funding opportunity announcements from the NHLBI or other components of the National Institutes of Health, might be of interest:
Notice of Intent to Publish a Funding Opportunity Announcement for Maximizing the Scientific Value of Existing Biospecimen Collections: Scientific Opportunities for Exploratory Research (R21 Clinical Trial Not Allowed)
(NOT-OD-19-100)National Heart, Lung, and Blood Institute
Notice of Intent to Publish a Funding Opportunity Announcement for Secondary Analyses of Existing Datasets of Tobacco Use and Health (R21 Clinical Trial Not Allowed)
(NOT-OD-19-104)National Heart, Lung, and Blood Institute
Notice of Correction to Award Budget for RFA-HL-19-024 "Short-Term Research Education Program to Increase Diversity in Health-Related Research (R25 Clinical Trial Not Allowed)"
(NOT-HL-19-698) National Heart, Lung, and Blood Institute
Notice of Change to Key Dates in RFA-RM-19-005 " NIH Directors Pioneer Award Program (DP1 Clinical Trial Optional)"
(NOT-RM-19-007) Office of Strategic Coordination (Common Fund)
Lasker Clinical Research Scholars Program (Si2/R00 Clinical Trial Optional)
(PAR-19-262) National Heart, Lung, and Blood Institute
It's the local company's first step toward establishing an international presence after it was spun out last year from MedImmune.
Rexahn Pharmaceuticals, Inc. (NYSE American: RNN), a clinical stage biopharmaceutical company developing innovative therapies to improve patient outcomes in cancers that are difficult to treat, today announced that it will voluntarily transfer its stock exchange listing to the Nasdaq Capital Market from its current listing on NYSE American.
The BioHealth Capital Region (BHCR) continues to grow rapidly with new startups and a greater number of clinical and commercial stage companies. The region expects to add approximately 2,000 life science-related jobs in the near term with more on the way. Whether it’s Kite Pharma building a new manufacturing site in Frederick County, MD, the growth of cell and gene therapy companies such as Autolus, Inc., REGENEXBIO, and RoosterBIO, Inc. or recent acquisitions of companies like Paragon BioServices, Inc., it’s evident that the BHCR is thriving.
If you need great photographer for your event, contact UZI Photography who covered the 5th Annual BioHeallth Capital Region Forum
This is the first raise for the business, which has been profitable and fueled growth organically up to this point.
A group of high school girls who wanted to create a local community for other young ladies interested in STEM subjects (science, technology, engineering and math) did so by organizing an all-girls math competition. Things turned out better than they expected at the inaugural InteGIRLS math competition on May 18 at Montgomery College in Rockville, Maryland.
At AURP’s BIO Health Caucus, we will explore trends in life science research, opportunity zone funding resources, the marriage of life science and philanthropy in global partnership opportunities. Discover the unique roles that biomedical innovation cluster and research parks play in innovation ecosystems around the globe. Translating discoveries from the lab to the market has never been more important. Connect with AURP at booth #3955 or One-on-One Partnering!
Viral safety is a critical focus during biopharmaceutical manufacturing (1–5). Although well-characterized mammalian cells such as the Chinese hamster ovary (CHO) line have been used for decades, both endogenous expression of retroviral-like particles and exogenous contamination events from viruses warrant continued vigilance (6, 7). International regulatory agencies require biomanufacturers to validate the “viral clearance” efficacy of their downstream manufacturing process steps before resulting products can be awarded clinical trial or commercial approval (8–10).
Data released May 9 by the Alliance for Regenerative Medicine (ARM) illustrates the growth of clinical activity where gene therapy is concerned.
ARM’s Quarterly Regenerative Medicine Global Data Report for the first quarter of 2019 showed 372 gene therapy clinical trials were in progress as of the end of Q1. Interestingly, a majority (217 or 58%) were studies in Phase II, followed by Phase I (123 or 33%), and Phase III (32 or 9%).
There are, undoubtedly, many factors that go into a person’s weight. A new report from the lab of Sekar Kathiresan, MD, director of the Center for Genomic Medicine at Massachusetts General Hospital (MGH), documents a clear biologic basis for the predisposition of obesity. More specifically, that genetics play a role in how heavy a person gets.
There are few, if any, more innovative, more daring, and more iconic health care executives. We'll hear about the process that drives groundbreaking invention, and her dream of permanently alleviating the shortage of transplantable organs.Martine Rothblatt, founder and CEO of United Therapeutics presents her organization's latest innovation, followed by a conversation with "Fast Money" Host and "Power Lunch" co-anchor, Melissa Lee.
The Maryland Tech Council (MTC), Maryland’s largest technology trade association, announced the winners of its 31st Annual Industry Awards during a celebration and ceremony at the Bethesda North Marriott Hotel & Conference Center attended by more than 630 business leaders from around the state.
The Food and Drug Administration has put out a call for volunteer companies to test out the its digital health pre-certification program for software as a medical device (SaMD) technologies.
Coming off the record-breaking venture financing numbers of 2018, the first quarter of 2019 displayed some cooling off, in terms of pre-money valuations across all rounds and amounts raised in Series C and later rounds, although the market remained strong by historical standards. Q1 2019 had slightly fewer up rounds for Series B and later financings than did the last two quarters of 2018. Median pre-money valuations declined moderately across all equity rounds in Q1 2019. Median amounts raised remained strong for Seed, Series A, and Series B rounds, with those medians matching or exceeding those of Q4 2018. But the median amount raised in Series C and later rounds declined significantly, falling from the historic high of $31.1 million reached in Q4 2018 to $15.3 million in Q1 2019.
Maryland Commerce is leading a delegation of 20 biohealth and life sciences companies, educational institutions, federal agencies, and city and county economic development offices to the 2019 BIO International Convention in Philadelphia next month.
Scott Gottlieb, who resigned as head of the FDA in March, has rejoined NEA as a special partner focused on healthcare investments. Gottlieb was a venture partner with the firm for 10 years before leading the FDA. Founded in 1977, the Maryland-based firm's portfolio includes Opendoor and Desktop Metal.
With expansion, Protenus recently moved to new offices in Fells Point.
The healthcare analytics company moved into a 12,000-square-foot office space that’s roughly triple the size of its previous location, according to People Operations Associate Samantha Barber, who gave us a recent tour.
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.
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.
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.
NAVAN, Inc. (NAVAN), a privately-held biotechnology company enabling the development of next generation cell and gene therapies through its novel, non-viral NanoStraw platform, announced today the appointment Heidi Hagen, Co-founder and Chief Strategy Officer for Vineti, Inc., and Howard Grimes, Ph.D., Associate Vice President and Associate Vice Provost for Institutional Initiatives at The University of Texas at San Antonio, to its Strategic Advisory Board.
Most Congressional hearings are morality plays designed to reach a predetermined outcome. It wasn’t hard to predict how the second hearing on drug pricing by the House Committee on Oversight and Reform was supposed to go. If the title, “HIV Prevention Drug: Billions in Corporate Profits after Millions in Taxpayer Investments” wasn’t enough of a clue, when Chairman Elijah Cummings (D-MD) said it was because of the “phenomenal leadership” of freshman Rep. Alexandria Ocasio-Cortez (D-NY) that the hearing was being held, any doubts evaporated. In an extraordinary gesture of deference for a new Member of Congress, Rep. Ocasio-Cortez was recognized for an opening statement before senior members of the committee.