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Nightstar files for $86M IPO to fund gene therapy trials – FierceBiotech

Posted: September 7, 2017 at 2:46 pm

Nightstar Therapeutics has filed to raise up to $86 million in a Nasdaq IPO. The money will equip Nightstar to complete a phase 3 trial of its choroideremia gene therapy and advance two other eye disease candidates through early-stage clinical studies.

London-based Nightstar, also known as NightstaRx, is set to move the choroideremia asset into phase 3 in the first half of next year. The therapy, NSR-REP1, is advancing into the 140-patient trial on the strength of data on 32 subjects treated in investigator-sponsored studies. Those trials found 90% of patients either maintained or improved their visual acuity in the year after receiving the gene therapy.

Given choroideremia causes currently-untreatable progressive vision loss, Nightstar sees the data as supporting further development. The asset is moving forward with a fairly clean safety profile in the 50 people treated to date. Investigators have seen one adverse eventtransient intraocular inflammationthat may have stemmed from treatment with NSR-REP1.

Challenges await Nightstar as it scales up for the phase 3 trial and potentially commercial sales, though. The biotech acknowledges the administration of NSR-REP1 requires significant skill and training, potentially creating a bottleneck to use of the gene therapy. And as a small, unpartnered player in a new field, any number of events could knock it off course.

What Nightstar does have is a head start. Spark Therapeutics has the most advanced challenger to NSR-REP1 but its program is yet to move past phase 1/2. The field is similarly clear for Nightstars follow-up candidate NSR-RPGR, which moved into the clinic just ahead of MeiraGTxs rival X-linked retinitis pigmentosa gene therapy. AGTCs Biogen-partnered candidate is close behind.

Nightstar has reached this point using money from a succession of private rounds, starting with the 12 million Syncona invested when the biotech spun out of the University of Oxford in 2014. The biotech pulled in a further 5 million when it named former Johnson & Johnson VP David Fellows as CEO early in 2015. A $35 million series B round followed late in 2015. And Nightstar broadened its investor base and raised a further $45 million in a series C round a few months ago.

Along the way, Nightstar has built out a team in preparation for the broadening of its clinical trial program and life on public markets. Last month, Ex-Pfizer clinical lead Tuyen Ong, M.D., left PTC Therapeutics to serve as chief development officer. And in April, Nightstar hinted at its IPO plans by recruiting the man who led Intercept Pharmaceuticals repeated public raises, Senthil Sundaram.

The question now is how receptive public investors are to gene therapy biotechs. The companies in the sector to go public to date have delivered mixed returns, with the successes of bluebird bio and Spark offset by the steady decline of uniQure and implosion of Dimension Therapeutics.

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Nightstar files for $86M IPO to fund gene therapy trials – FierceBiotech

Recommendation and review posted by Fredricko

Ortho Regenerative Technologies’ CSO, Michael Buschmann, PhD, Appointed Bioengineering Chair at George Mason … – Markets Insider

Posted: September 7, 2017 at 2:46 pm

KIRKLAND, QC, Sept. 6, 2017 /CNW/ -Ortho Regenerative Technologies Inc. (“Ortho RTi” or the “Company”), an emerging Orthopaedic and Sports Medicine Technology company, wishes to congratulate its Chief Scientific Officer, Michael Buschmann, PhD, on his recent appointment as Bioengineering Chair, Professor and Eminent Scholar (an award from Virginia’s Center for Innovative Technology) at George Mason University (“Mason”) in Fairfax, VA.

Dr. Buschmann’s selection as the next chair of Bioengineering at Mason resulted from a highly competitive and rigorous recruitment process. Prior to his appointment, Dr. Buschmann established and led a multidisciplinary research program at Ecole Polytechnique in Montreal, QC, focusing on the use of biomaterials to repair cartilage, meniscus and bone and to deliver plasmid DNA and small interfering RNA.

Dr. Buschmann earned his PhD in 1992 in Medical Engineering and Medical Physics from the Massuchusetts Institute of Technology in the Harvard-MIT Division of Health Sciences and Technology and conducted his postdoctoral studies at the ME Mueller Institute of Biomechanics, University of Bern, Switzerland. He became a faculty member at cole Polytechnique in 1994, becoming a full professor in 2001. His research achievements include over 150 peer-reviewed articles, over 330 conference proceedings, 5 book chapters, over 75 invited presentations, 19 patent applications (7 granted), over 12,000 citations, and an h-index of 56. He has graduated 20 PhD students, 17 MSc students, and supervised 14 postdoctoral fellows. During his academic career at cole Polytechnique, he obtained over $50 million in external research funding as principal investigator.

Dr. Buschmann’s research work has been recognized by 19 prizes/awards and his abilities as an educator have earned him 6 teaching awards at cole Polytechnique. Dr. Buschmann has received numerous awards for his research, including the prestigious Canada Research Chair Tier 1 in 2001 and in 2008, the Melville Medal of the American Society of Mechanical Engineering (“ASME”) in 1997, and Article of the Year for ASME Journal of Biomedical Engineering in 1996. In addition to Ortho RTi, Dr. Buschmann has been the driving force behind several biotech startup companies as founder or principal inventor.

“On behalf of the Board and staff at Orthi RTi, I would like to congratulate Michael on this prestigious appointment, said the Company’s Executive Chairman and CEO, Dr. Brent Norton. “It is very well deserved. Michael is a world-class researcher who has made fundamental and translational contributions to the fields of biomechanics, biomaterials, and nanomedicine. On a personal note, I look forward to continuing to work with him to bring products based on Ortho RTi’s proprietary biopolymer platform successfully to market.”

About Ortho Regenerative Technologies Inc.

Ortho RTi is an emerging Orthopaedic and Sports Medicine technology company dedicated to the development of novel therapeutic tissue repair devices to dramatically improve the success rate of sports medicine surgeries. We are committed to improving patients’ lives through increasing the success rates of surgeries for soft tissue injuries. Our proprietary biopolymer has been specifically designed to increase the healing rates of sports related injuries to ligaments, tendons and cartilage. The polymer can be directly placed into the site of injury by a surgeon during a routine operative procedure without significantly extending the time of the surgery and without further intervention. Visit us on the internet at

Forward-Looking Statements

This news release may contain certain forward-looking statements regarding the Corporation’s expectations for future events. Such expectations are based on certain assumptions that are founded on currently available information. If these assumptions prove incorrect, actual results may differ materially from those contemplated by the forward-looking statements contained in this press release. Factors that could cause actual results to differ include, amongst others, uncertainty as to the final result and other risks. The Corporation disclaims any intention or obligation to publicly update or revise any forward- looking statements, whether as a result of new information, future events or otherwise, other than as required by security laws.

SOURCE Ortho Regenerative Technologies Inc.

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Ortho Regenerative Technologies’ CSO, Michael Buschmann, PhD, Appointed Bioengineering Chair at George Mason … – Markets Insider

Recommendation and review posted by Guinevere Smith

Working Lung Model with Intact Vasculature Likely to Aid Research, Lung Transplants – Lung Disease News

Posted: September 7, 2017 at 2:46 pm

Researchers at Columbia University have found a way to bioengineer aworking lung a very complex structure with a viable and intactblood vessel network that can support studies of lung cell repair and stem cell transplants, aiding both research into lung diseases and, potentially, the availability of donor lungs.

The team, led by Gordana Vunjak-Novakovic, director of the Laboratory for Stem Cells and Tissue Engineering, recently published itfindings inthe journal Science Advances in a study titled Functional vascularized lung grafts for lung bioengineering.

We developed a radically new approach to bioengineering of the lung, Vunjak-Novakovic, who is also aprofessor of medical sciences at Columbia, said in a news release.

With more than 40 different cell types and a large airway and vasculature surface area, the lung is an incredibly complex organ. It has been a challenge to find ways to promote lung repair to treat advanced lung diseases, the third leading cause of death worldwide.

In contrast toprevious bioengineering projects that required an extensive reconstruction of the lungs vasculature, theteamshowed itis possible to recreate the pulmonary epithelium while preserving its main structural elements, including such supporting such as fibroblasts, myocytes, chondrocytes, and pericytes. The epithelium is tissue that lines the cavities and surfaces of organs and blood vessels.

We reasoned that an ideal lung scaffold would need to have perfusable and healthy vasculature, and so we developed a method that maintains fully functional lung vasculature while we remove defective epithelial lining of the airways and replace it with healthy therapeutic cells, Vunjak-Novakovic said. This ability to selectively treat the pulmonary epithelium is important, as most lung conditions are diseases of the epithelium.

The research team used an ex vivo lung perfusion system (EVLP) in a rodent, and delivered to the lung a mild detergent solution to remove lung tissue-specific cells while protecting the remaining structures and other types of cells.

EVLP works in ways similar to theextracorporeal membrane oxygenation (ECMO) system used to support patients incardiovascular and respiratory failure. EMCObypasses the lungs to provide the body with necessary oxygen and promote gas exchange in an externally controlled system.

Using its system, the team created a lung scaffold with functional bronchial and vascular architecture. These structures were able to support the attachment and growth of human adult and stem cell-derived pulmonary cells.

Researchers think the bioengineered lung model can help with lung repair, andalso help to improve the number of transplantable lungs by makingdonor lungsmore resilient and durable, said Matthew Bacchetta, at associate professor of surgery at the Columbia University Medical Center, and astudyco-author.

The team is nowtesting itsapproach to study lung development and repair in disease, andto develop new targeted therapeutics. They are also focused on developing new imaging-guided lung evaluation strategies for clinical applications.

This research project was supported by a $8.2 million, seven-year grant from the National Institutes of Healththat aims to support research into the mechanisms and treatment of idiopathic pulmonary fibrosis, a serious lung disease.

This is a major step forward in bioengineering lungs, Vunjak-Novakovic said. The creation of de-epithelialized whole lungs with functional vasculature may open new frontiers in lung bioengineering and regenerative medicine.

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Working Lung Model with Intact Vasculature Likely to Aid Research, Lung Transplants – Lung Disease News

Recommendation and review posted by Fredricko

Faculty Mentors Guide Aspiring Researchers from Mexico in Program – University of Texas at Dallas (press release)

Posted: September 7, 2017 at 2:46 pm

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Sept. 7, 2017

Fifteen undergraduates participated in a summer program at The University of Texas at Dallas that invites students from Mexico to explore STEM-related research careers in fields such as biology, geographic information systems, materials science, physics, computer science and bioengineering.

The UT Dallas-Mexico Summer Research Program is designed to equip students from participating Mexican universities with the skills and knowledge needed to pursue careers in these fields.

Through the program, students work with faculty mentors to identify and manage a research project, then analyze the data and present their findings. Students were competitively selected from more than 240 applicants.

UT Dallas-MexicoSummer Faculty Mentors

Dr. Zachary Campbell, Dr. Michael C. Biewer, Dr. Vibhav Gogate, Dr. Kelli Palmer, Dr. Murat Kantarcioglu, Dr. Fan Zhang, Dr. Nicholas Fey, Dr. Robert Gregg, Dr. Julia Chan, Dr. Vincent Ng, Dr. Ronald A. Smaldone, Dr. Xiaohu Guo, Dr. Julia Hsu, Dr. Dinesh K. Bhatia, Thomas Lambert

Dr. Juan Gonzlez, professor of biological sciences in the School of Natural Sciences and Mathematics and the programs academic director, said participants gain enhanced knowledge and experience with research careers.

One of our goals is to provide the students with experiences that will better inform them on the rewards of a future research career and inspire them to pursue a higher degree in one of the STEM fields, Gonzlez said. We also hope to enhance further the collaboration between the research communities of both Mexico and the U.S.

The program, which has existed for 13 years, was organized by the Office of Graduate Studies, the Provosts Office and the International Center, with the co-sponsorship of the School of Natural Sciences and Mathematics, the School of Arts and Humanities and the Office of Undergraduate Education. Since 2002, 139 undergraduate students have taken part in the UT Dallas program.

The program is made possible through a partnership with 100,000 Strong in the Americas and the U.S.-Mexico Bilateral Forum on Higher Education, Innovation, and Research.

Dr. Julia W.P. Hsu, Texas Instruments Distinguished Chair in Nanoelectronics and professor of materials science in the Erik Jonsson School of Engineering and Computer Science, described program participant Laura Yoselyn Quiroga as an enthusiastic and eager young scientist.

She is talented and ambitious, and has done a lot of reading. She is not afraid of getting her hands dirty and doing hard work. Her performance this summer strongly suggests that she will be successful in graduate school. As a mentor, it is very gratifying to see her flourish during the short time at UT Dallas, Hsu said.

Summer Research Participants

Adzuira Musule Palacios, Christopher Jquez Prado, Sandra Berenice Mendoza Peuuri,Zayd Alejandro Grajales Moreno,Juan Maldonado Juregui,Miriam Yamasaki Aguilar, Daniel Ayala Nio, Jonathan Martnez Garca, Jos Gonzlez Ayerdi, SwilmaLabastida, Laura Yoselyn Quiroga Lpez, Irving Osiel Castillo Rodriguez, Noor Beatriz Tuma Schmidt, Anaid Alethia Candido Lopez, Gerardo Ocampo Daz

Dr. Nicholas Fey, assistant professor of bioengineering and mechanical engineering, praised the creative ideas that participantSwilma Labastida brought to her summer research project.

Swilma operated with incredible maturity and independence for a researcher of her age. We are excited to submit the findings from her scientific studies for publication at an international conference and in a biomechanical engineering journal this fall. I hope she considers graduate studies in engineering and that she applies to UT Dallas, Fey said.

Computer science student Christopher Jquez Prado said he appreciated the collaborative interaction with his mentor, Dr. Murat Kantarcioglu.

I initially expected some sort of boss-employee scenario, but to my surprise and enjoyment we’ve been working together in a cooperative way, Prado said.

Francisco de la Torre, Consul General of Mexico in Dallas, said partnerships like the summer research program benefits both Mexico and the U.S.

This academic cooperation, where UT Dallas excels as a leader in Texas, is helping expand opportunities for educational exchanges, scientific research partnerships and cross-border innovation so we can continue building bridges for mutual economic prosperity and sustainable social development, de la Torre said.

Media Contact: Robin Russell, UT Dallas, (972) 883-4431, [emailprotected]or the Office of Media Relations, UT Dallas, (972) 883-2155, [emailprotected].

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Faculty Mentors Guide Aspiring Researchers from Mexico in Program – University of Texas at Dallas (press release)

Recommendation and review posted by Guinevere Smith

Differentiating Back Pain from Kidney Pain | Massage …

Posted: September 7, 2017 at 1:44 am

Massage therapists are often in a difficult position; western medical diagnosis is beyond their scope of practice, yet awareness and recognition of medical disorders is crucial to their clients well-being and safety. Mistaking kidney inflammation for a muscular strain can result in inappropriate therapy. Learn how to detect kidney inflammation masquerading as back pain, and what to do about it.

Sign up for our free Natural Wellness newsletter! Gain lifestyle suggestions for supporting a healthy heart and stress management, tips and advice on exercising and maintaining a healthy weight, how to beat fatigue, ways to decrease joint pain, as well as learn valuable facts about kidney health.

One of the primary reasons for client visits to clinically oriented massage practices is back pain. Recognizing the signs of kidney inflammation enables therapists to differentiate it from a muscular strain/sprain.

Often mistaken to be tense or strained back muscles, kidney inflammations most prominent symptom can be back pain. Three kidney inflammatory conditions that can cause back pain are:

When a client complains of back pain, from the lower-thoracic to the mid-lumbar regions, it is important to rule out kidney inflammation. When the kidneys are not functioning optimally, the bodys ability to manage fluid balance is impaired, often resulting in edema. Systemic circulatory massage is inappropriate for these individuals, as it would push more fluid through an already overburdened system. Additionally, if the kidneys are inflamed, they are more susceptible to injury from vigorous massage. The kidneys are vulnerable because they are only partially protected by the rib cage. The right kidneys location underneath the liver results in its position being slightly lower than the left kidney.

A solid, anatomical understanding of each kidneys location will contribute to accurate differentiation. The superior border of the kidney reaches the level of the 12th thoracic vertebrae. The inferior border lies just above the horizontal plane of the umbilicus, typically level with the 3rd lumbar vertebrae. The inferior border is one finger breadth superior to the iliac crest. The center of the kidney, where the ureter is attached, is level with the intervertebral disc between the first and second lumbar vertebrae. Therefore, pain originating in the kidney is typically felt in the upper lumbar region and can radiate to the upper right or left quadrant of the abdomen.

A thorough client history and intake is an essential component of safe practice, especially when evaluating back pain. To help the bodyworker identify kidney involvement, the three kidney inflammatory conditions previously listed are explained below.

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Differentiating Back Pain from Kidney Pain | Massage …

Recommendation and review posted by Fredricko

Anatomy Review: Slow and Fast Twitch Muscle Fibers …

Posted: September 7, 2017 at 1:44 am

Without properly functioning muscles the human body would be unable to move. Muscles help to propel us through space, pump our blood, expand and contract our lungs and move nutrients through our body. One aspect of our skeletal muscles is responsible for athletic stamina. Learn about fast and slow twitch muscles, how they can determine whether you would be a better sprinter or marathoner and the role of massage therapy.

There is no getting around it; all body functions that involve movement require muscle activity. It may be as obvious as when we walk, rise from sitting or toss a ball. It may be movement we take for granted such as our heartbeat or in the iris of the eye. Muscles control the movement of food through the digestive system and enable us to breath. Some muscles are used occasionally; some are in constant use, remaining contracted to help the body maintain posture in defiance of gravity. By their very movement and resulting friction, muscle fibers provide the heat that maintains a consistent body temperature as well as assist in the pumping of blood and lymph in and out of cells.

The human body has three types of muscle skeletal, cardiac and smooth. Skeletal muscle is responsible for posture and movement of bones. It also guards the entrances and exits of the digestive, respiratory and urinary tracts. Cardiac muscle is what the heart is made of. Smooth muscle is found in the gut, around the bronchi, within the urinary tract, the reproductive organs and in the walls of the blood vessels. Skeletal muscle moves in response to nerve impulses. Cardiac and smooth muscle fibers respond to changes in local environment, hormone fluctuation, pH balance, ions and temperature among other factors.

Skeletal muscles connect bone to bone with tendonous attachments. When they contract, the bones generally pull closer together or flex. When they are at rest, the bones are further apart or extended. More complicated movements, such as circumduction, supination, pronation or rotation are a combination of flexion and extension, a blending of muscular contraction and relaxation.

Normally the muscles act together in a coordinated manner, producing smooth, efficient movements. Some movements are under conscious control, especially when first learning specific tasks such as writing or riding a bicycle, other less so like the blinking of eyes or scratching an itch. With disorders such as Parkinsons, the signals from the nervous system to the muscles are confused, producing antagonist and agonistic movements at the same time, resulting in either oscillatory movement (tremors) or persistent spasm (rigidity).

There are two basic categories of skeletal muscle fast twitch (also referred to as fast glycolic or Type IIB) and slow twitch (slow oxidative or Type I). Within the fast twitch there is a second category sometimes referred to as intermediate, Type IIA, or fast oxidative fibers. Each has their own set of characteristics and purpose. The percentage of fast, slow and intermediate twitch muscle fibers varies from person to person. The proportion is determined by genetics but can change with physical conditioning. Certain hormones, such as human growth hormone (HGH), testosterone and thyroid hormones can also stimulate the metabolism and size of muscle fibers.

Fast Twitch (Type IIB):

Slow Twitch (Type I):

Intermediate (Type IIA):

The physiology of fast or slow twitch muscles is most often utilized in sports. Athletes who run provide a good example. Those who spring short distances generally will have a higher percentage of fast twitch muscle fibers, while those who run in marathons have more slow twitch fibers.

Another example often used is the dark and light meat of poultry. The breast muscles or white meat of a free-range chicken consist of fast twitch muscle fibers needed for brief burst of flight, while the red or dark meat in their thighs and legs are used for walking and standing.

The type of skeletal muscle fiber is largely determined by heredity. Massage cant change that. What massage can do is help to bring oxygen and nutrients to muscle cells, which in turn can help them to work more efficiently and heal quicker.

Individuals who are highly athletic or participate in competitive sports have a tendency to test their bodies to maximum endurance and, at the very least, experience sore muscles. They often get injured in their pursuit. Some injuries are serious others are minor, involving microscopic tears in muscle tissue. When administering massage, care needs to be taken to customize the session to the athlete as well as the sport. Sports massage techniques, most of which are derived from traditional Swedish massage, have been found to be the most effective in promoting healing and maintaining optimal fitness.

To maximize the effectiveness of your massage, make sure to ask your clients about any physical training they participate in, whether it be recreationally or professionally. Knowing this may help you determine just which massage techniques will be best for them.

Advanced Anatomy & PhysiologyCommon Sports InjuriesSports MassageSwedish Massage for ProfessionalsUnderstanding Sports Massage

British Broadcasting Company. Muscles Fast and slow twitch. BBS Science & Nature. Available from Internet; accessed 25 March 2010.

Haycock, Bryan. Fiber Types, Training, and Hypertrophy. Think Muscle. Available from Internet; accessed 25 March 2010.

ICBS, Inc. Sports Massage. Available from Internet; accessed 25 March 2010.

Premkumar, Kalyani. (2004). The Massage Connection: Anatomy and Physiology, 2nd ed. Baltimore: Lippincott Williams & Wilkins.

Robson, David. How Do Fast-Twitch & Slow-Twitch Muscle Fibers Influence Athletic Performance?. Available from Internet; accessed 25 March 2010.

Wikimedia Foundation, Inc. Muscle. Wikipedia. Available from Internet; accessed 25 March 2010.

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Anatomy Review: Slow and Fast Twitch Muscle Fibers …

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