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Only a Silent Mind Can Be a Healing Mind – SFGate

Posted: March 31, 2020 at 5:48 pm

By Deepak Chopra, MD

Crises call for action, and the COVID-19 crisis has triggered global action, much of it motivated by alarm, fear, and the dread of uncertainty. But what about the individual person who feels afraid and uncertain? Id like to propose an answer based on the silent mind. I realize that this approach might sound a bit alien and spiritual in the wrong way, but building castles in the air or retreating into yourself isnt what silent mind is about.

Silent mind is about reconnecting to your source. Everyone relies on the top layer of the mind, which is active, constantly thinking and feeling. But when these feelings get fixated on anxiety, alarm, dread, and uncertainty, the active mind cannot pull itself out of its own spiral. Mental activity becomes useless to heal itself, just as a runaway car cannot apply its own brakes.

What is needed is a reset. The reset isnt just mental. Your thoughts are received by every cell in your body, and in turn all kinds of processes are affectedthe immune response, hormonal cycles, sleep, and overall mind-body balance, or homeostasis. If the active mind becomes confused and chaotic, balance is disrupted everywhere. What to do?

Centuries ago, in every culture, a deeper level of mind was discovered, and the usual expression surrounding this level, which is silent, calm, and undisturbed, became religious, as in the Old Testament injunction, Be still and know that I am God. If we replace God with your source, the message comes through to modern ears: Be still and know that I am your source. The most direct result of heeding this message would be to meditate, because meditation gives direct access to silent mind.

But countless modern people have tried meditation, and they do not experience the kind of reset that is needed in a crisis. Partly this is due to lack of commitment; the average person has tried meditation and left it behind, or only meditates when a sort of psychological Band-Aid is needed. Let me look a bit deeper to show what has been missed, because silent mind is truly the only healer.

In medical school homeostasis is described as basically physical. If you go for a run, your heart rate, respiration, blood flow to muscles, digestive process, etc. are thrown out of balance, but once you stop running, homeostasis is restored. At the negative end of experience, if you experience a great shock, the fight-or-flight response throws you into extreme imbalance, but when the shock ends, balance is restored. Unfortunately, under a constant threat like COVID-19, the shock doesnt end. The usual stress response is designed to last no more than a few minutes. Extended to days and weeks, it turns on itself and begins to create damage.

The damage first appears psychologically. Under constant stress, people feel tired, grumpy, depressed, anxious, irritable, impatient, and so on. Keep up the pressure, and the next stage is fatigue, lethargy, dullness, and depression. If the stress still doesnt abate, physical symptoms start to develop, often beginning with insomnia as the result of hormonal interactions being thrown out of whack. There is a lot more to say about this, but the bottom line is that a holistic reset is needed.

Without noticing it, you have been holistically resetting yourself for your entire life. Homeostasis isnt just physical; it involves the whole person. The command center for resetting the whole person isnt found in our cells, not even our brain cells, and it isnt found in the active mind, which is just the top layer. The command center for holistic resetting is at the source. Be still and know that I am your source. The evidence for this has existed for decades. Meditation affects heart rate, respiration, brain activity, inflammation markers, and stress levels. Medical science studies each of these factors individually, but we shouldnt miss the forest for the trees. Everything comes back to the same source.

Your source is still and silent; you come closest to it in deep, dreamless sleep. But in a crisis, everything doesnt automatically go back into balance the way your heart rate will return to normal after you quit running. It turns out that there is useful silence and not-so-useful silence. As consciousness starts to move from its silent source, different paths open up, and the paths you have favored become your unique way of turning silence into something else.

Nobody handed you a users manual, but in broad terms, silent mind takes a path that is either/or. Let me map how these pathways diverge.

Fear or love

Separation or unity

Suffering or bliss

Renewal or habit

Self-esteem or selfdoubt

Security or insecurity

Comfort or stress

Acceptance or resistance

Awareness or unconsciousness

These choices arise from silence; they have the same source but travel in opposite directions. If a person is fully conscious or awake, the pathways are directed toward the desirable experiences of love, security, bliss, creativity, renewal, and so on. But as things stand, we are all entangled in a web of choices that are mixed. We suffer but also feel bliss; we love but also fear; we feel self-worth but also self-doubt.

A crisis throws us into deeper confusion as it entangles us in too many wrong responses. Healing consists of allowing the silence to go in the right pathways. In every spiritual or wisdom tradition, pure consciousness unfolds, if let alone, in the direction of love, creativity, renewal, and evolution. There is no injunction that says, Be still and lets see what happens or Be still and who knows how that will work out for you?Instead, the mindbody balance we all have relied upon since infancy is directed positively. Health and wholeness are the norm; creativity and renewal are the norm.

This is why I believe that the COVID-19 crisis can lead to healing, because without a doubt everyone feels the need for a rest. Follow this need toward your source, and it will be fulfilled. This is a time when the rest brings into play the infinite power of consciousness. All we have to do is align ourselves with that power at the level of silent mind.

DEEPAK CHOPRA MD, FACP, founder of The Chopra Foundation, a non-profit entity for research on well-being and humanitarianism, and Chopra Global, a modern-day health company at the intersection of science and spirituality, is a world-renowned pioneer in integrative medicine and personal transformation. Chopra is a Clinical Professor of Family Medicine and Public Health at the University of California, San Diego and serves as a senior scientist with Gallup Organization. He is the author of over 89 books translated into over forty-three languages, including numerous New York Times bestsellers. His 90th book and national bestseller, Metahuman: Unleashing Your Infinite Potential (Harmony Books), unlocks the secrets to moving beyond our present limitations to access a field of infinite possibilities. TIME magazine has described Dr. Chopra as one of the top 100 heroes and icons of the century.

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Only a Silent Mind Can Be a Healing Mind - SFGate

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LIVE UPDATES: Wisconsin will still hold April 7 election, Cuomo hits back at Trump – Business Insider India

Posted: March 31, 2020 at 5:48 pm

NY Gov. Andrew Cuomo called for unity in his Monday press briefing after President Donald Trump went after him earlier in the morning in a phone interview on "Fox & Friends."

Weighing in on Cuomo's boosted approval rating 87%, according to a new Siena poll Trump said, "one of the reasons he's successful is because we've helped make him successful."

On Sunday, Trump also accused New York hospitals of hoarding ventilators and other medical equipment.

"I'm not going to engage in politics, not because I'm unwilling to tangle, but because I think it's inappropriate, and I think it's counter-productive, and I think it's anti-American," Cuomo said when asked about Trump's comments.

"Forget the politics," Cuomo said. "We have a national crisis. We are at war. There is no politics. There is red, white, and blue."

Trump and Cuomo have clashed over the federal government's inability to get the New York up to 30,000 ventilators and more broadly over the severity of the pandemic with Trump more impatient to end social distancing and "re-open the economy."

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LIVE UPDATES: Wisconsin will still hold April 7 election, Cuomo hits back at Trump - Business Insider India

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A Baby’s Milestones and the Next Steps for Novartis in Gene Therapy – Xconomy

Posted: March 31, 2020 at 5:47 pm


Cameron Hewitts family celebrates the anniversary of her life not once, but twice yearly: Besides her September birthday, they commemorate a day in Marchthe anniversary of two-year-old Cameron receiving the gene therapy that gave her a new shot at life.

Cameron was born with spinal muscular atrophy (SMA), a genetic disorder that leads to the progressive loss of muscle function. In its most common and severe form, the disease leads to death within one or two years. The Hewitt family, which lives in Apex, NC, credits Zolgensma, a gene therapy approved by the FDA last year, as the reason Cameron lived to her second birthday and continues to improve today.

FDA approval for the intravenously administered treatment covers patients with SMA type 1 who, like Cameron when she enrolled in a clinical trial in 2018, are younger than 2. Zolgensmas maker, Novartis (NYSE: NVS), wants approval to treat more types of SMA in patients of all ages. For that, more data are needed. Novartis is continuing to generate supporting data from additional clinical trials. Meanwhile, its subsidiary AveXis, which developed the treatment, is working to boost its capacity to produce Zolgensma and other gene therapies in its pipeline that are now being prepared for tests in humans.


SMA stems from a defect in survival motor neuron gene 1 (SMN1), which is responsible for producing a protein needed for the health and proper function of motor neurons. The disease leads to muscle weakness that worsens with age. That muscle weakness isnt necessarily apparent at birth. Cameron hit her initial development milestones, her father, Josh Hewitt, said at a launch event last month for AveXiss Durham, NC, facility. But at 4 months old, Josh and Kimberly Hewitt, her mother, noticed Cameron appeared weaker and had more difficulty moving. SMA patients have difficulty sitting up, rolling over, or even lifting their heads. The rare condition is found in about 1 in every 11,000 births, according to patient group Cure SMA.

Cameron is one of an estimated 378 North Carolinians living with the disease. She was 5 months old when her diagnosis was confirmed. The Hewitts then learned their infant daughter had two treatment options. The first was nusinersen (Spinraza), a drug developed by Biogen (NYSE: BIIB) and Ionis Pharmaceuticals (NASDAQ: IONS) that in 2016 became the first FDA-approved SMA treatment. That drug, given as an injection into the spinal fluid, was developed to boost production of the functional SMA protein by targeting the SMN2 gene. Patients need that injection every four months for the rest of their lives. Unlike Zolgensma, Spinraza is approved for all SMA types in patients of any age.

The second option was the experimental AveXis gene therapy. It uses a modified virus to deliver genetic instructions into a patients cells, giving them the capability to produce the needed muscle proteinpotentially for life. Though the AveXis treatment is meant to be given once, the long-term durability of its therapeutic effect is still unknown.

Novartis launched Zolgensma last June with a $2.1 million price tag before factoring in rebates or discounts. As a one-time treatment, the therapy is intended to avoid costly hospitalizations and other expenses that come with managing SMA. Its price falls on the upper end of the range the Institute for Clinical and Economic Review (ICER) calculates is cost effective for patients and the health system.

The watchdog group, a frequent critic of high drug prices, pointed to Zolgensma Phase 3 data (Camerons study) showing that 21 of the 22 patients who received the gene therapy were still alive. The lone patient death was deemed to be unrelated to the treatment. ICER says its price range assumes the US widely adopts newborn screening for SMA. In 2018, the US Department of Health and Human Services put SMA on the list of diseases it recommends states include in their newborn screening programs.

Since winning FDA approval, Zolgensma has made headway with insurers, gaining coverage for 97 percent of those who have private health insurance, Dave Lennon, AveXiss president, told Xconomy in an interview. For those covered by Medicaid, that figure is 50 percent.

No one jumps up and down and thanks of us for charging seven figures for a product, Lennon says. But at the same time, they understand the value proposition that it brings and they understand that the investment of $2 million now saves a lot of money down the road.

Last year, 200 insured patients were treated with Zolgensma, Novartis reported in its 2019 financial results. The gene therapy generated $361 million in sales that year. Those figures are expected to grow as newborn screening reveals more babies with SMA.


Zolgensma revenue will also grow if the therapy is approved for patients 2 and older who span a range of SMA types. Clinical trials are underway. But AveXis is also overcoming concerns about previous preclinical tests. Last August, the FDA reported that AveXis notified the agency that some of its submitted preclinical mouse data were manipulated. However, the regulator found no evidence that patient data were altered and said the gene therapy should remain on the market. AveXis fired those allegedly responsible for the data manipulation and integrated its quality controls with those of Novartis. Novartis CEO Vas Narasimhan said on conference call Monday that the FDA has closed its inquiry into this matter with no penalties imposed on the company.

But last October, Novartis announced a partial clinical hold on a Phase 1/2 study testing an intrathecal formulation of the gene therapyan injection into the spinal fluid. This study is intended to support approval of the treatment for patients with SMA type 2. Lennon says a review of data in monkeys found Next Page

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at

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A Baby's Milestones and the Next Steps for Novartis in Gene Therapy - Xconomy

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Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies – Yahoo Finance

Posted: March 31, 2020 at 5:47 pm

WALTHAM, Mass., March 31, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies and a mission to develop transformative medicines for devastating diseases, today announced it has closed an oversubscribed $60 million Series A financing. Seed venture investors F-Prime Capital and New Enterprise Associates (NEA) co-led the round alongside new investor Atlas Venture, with participation from seed investors Alexandria Venture Investments, Lonza and Partners Innovation Fund.

The proceeds will be used to advance the companys platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.

The company is led by recently appointed Chief Executive Officer Rick Modi, who has a proven track record of building value at companies including AveXis, InterMune, MedImmune and Centocor. Joining the board of directors as part of the financing are Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, NEA; and Robert Weisskoff, Ph.D., Partner, F-Prime Capital. Industry veteran and gene therapy leader Sean Nolan will chair the board.

At Affinia Therapeutics, were setting a new standard in gene therapies by leveraging our proprietary platform to methodically engineer novel AAV vectors and gene therapies that have remarkable targeting properties, said Modi, CEO of Affinia. We are pleased to partner with such a distinguished syndicate to advance our platform and investigational product candidates toward the clinic for patients in need.

The companys technology was licensed from Lonza and Massachusetts Eye and Ear. It was developed at the Grousbeck Gene Therapy Center and further advanced under a sponsored research agreement with Lonza led by Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School and a co-inventor of AAV9.

In addition to Vandenberghe, joining Affinia Therapeutics as scientific co-founders are Botond Roska, M.D., Ph.D., Director, Institute of Molecular and Clinical Ophthalmology Basel; Aaron Tward, M.D., Ph.D., Assistant Professor, University of California, San Francisco; and Eric Zinn, Ph.D. student, Mass. Eye and Ear and Harvard University. Together, these scientists have authored more than 200 papers and filed more than 20 patents in the field of gene therapy.

Affinia Therapeutics is bringing together complementary expertise allowing us to realize a rational design future for AAV vectors, promoters and other components of gene therapies. By leveraging synthetic and systems biology combined with high-throughput screening and tissue and single-cell resolution, we are aspiring to achieve much-needed improved pharmacological control of this novel modality in medicine, said Vandenberghe, Director, Grousbeck Gene Therapy Center at Mass. Eye and Ear.

The Series A financing comes after Vandenberghe and his team successfully developed AAVSmartLibraries comprising thousands of functional novel AAV vectors. Each vector is uniquely identified, and the libraries can be screened across species for parameters of high interest, including tissue tropism, manufacturing yield and pre-existing immunity. Observations arising from each library screen provide insights into the vectors structure-function, enabling the rational design of novel vectors and gene therapies with remarkably improved properties. Affinia Therapeutics has potentially the worlds largest library of patented functional AAV vectors.

Affinia Therapeutics methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialization of transformative medicines, said Mathers, General Partner at NEA. We are pleased to accelerate the impact of this exciting field.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet

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Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies - Yahoo Finance

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NeuBase Therapeutics Announces Positive, Preclinical Data Validating its Novel Genetic Therapy PATrOL Platform – GlobeNewswire

Posted: March 31, 2020 at 5:47 pm

Demonstrates broad biodistribution, including across the blood-brain barrier into the central nervous system, and into skeletal muscle, in non-human primates (NHPs) after systemic administration

Durable and therapeutically relevant drug concentrations achieved in NHPs after single intravenous dose

Potent cell-based activity and allele-specific enrichment in patient-derived cell lines

Platform validation data supports expansion of the therapeutic pipeline into new organ systems previously unreachable with first-generation antisense oligonucleotide technology

Management to hold a conference call today at 8 a.m. ET

PITTSBURGH, March 31, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq:NBSE) (NeuBase or the Company), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies to address genetic diseases, today announced positive preclinical data from its pharmacokinetics studies in non-human primates (NHPs) and in vitro pharmacodynamics data in patient-derived cell lines. NeuBase believes these data validate the key advantages of the proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform and support the Companys decision to advance the development of its Huntingtons disease (HD) and myotonic dystrophy type 1 (DM1) programs, as well as the potential expansion of its therapeutic pipeline into other indications.

Dr. George Church, professor of genetics at Harvard Medical School and member of the National Academy of Sciences, stated, Given the activity and broad biodistribution observed in these studies and the potential for easier target definition, I believe the PATrOL technology may have a potent impact on the future of drug development and treatment of genetic diseases.

Non-Human Primate Pharmacokinetic Study

Quantitative whole-body autoradiography was performed on NHPs.A PATrOL-enabled compound was radio-labeled, and theresulting material was injected into NHPs at 5 mg/kg via a bolus tail vein injection. At four hours, twelve hours, and seven days post-dosing, NHPs were sacrificed andsectioned into 40 m slices.Slices were exposed to autoradiography imaging plates alongside a dilution series of radioactive PNA in whole blood.Upon imaging, the dilution series enabled an analysis of the amount of compound in each of the tissues. In addition, prior to sacrifice, whole blood, urine, and feces were collected from the NHPs at specified timepoints.The major conclusions from this study include:

Compound crosses the blood-brain barrier and into the key deep brain structures, including the caudate, supporting a key capability for the development of the Companys lead program in HD; Delivery of the compound to skeletal muscle, the primary organ system that is affected in DM1;Because both HD and DM1 have manifestations outside of the primary affected organ, the broad biodistribution of the compounds may enable a potential whole-body therapeutic solution in both indications.

96% of administered compound remained in vivo after a one-week period (latest timepoint tested);Redistribution over one week after administration between organ systems enriches concentrations in key brain regions up to two-fold, including in those deep brain structures most relevant for HD;Retention of ~90% of compound concentrations achieved in skeletal muscle over the course of one-week post-single-dose administration; and

Patient-Derived Huntingtons Cell Line Pharmacodynamic Studies

Multiple Huntingtons disease candidate compounds were incubated with HD-derived cells and assayed for their toxicity and their ability to selectively knock down mutant huntingtin protein (mHTT) expression by engaging with the CAG repeat expansion in the huntingtin (HTT) gene transcript. Multi-well plates were seeded with cells and candidates were added to the culture at various concentrations.Cells were grown for three days and thereafter assayed for cell death.Cell pellets were also collected, lysed, and run on gradient SDS-PAGE gels.Following the transfer of the proteins to a membrane, the membrane was probed with anti-huntingtin and anti-beta-actin antibodies.Secondary antibodies were used to image the immunoblots.The beta-actin bands were used to normalize the amount of protein across the wells.The amounts of mutant and wild type huntingtin protein in treated cells were compared to untreated cells to determine the level of knockdown.The major conclusions from this study include:

In addition, PATrOL enabled compounds were generally well-tolerated in vivo after systemic administration, both after single dose administration in NHPs and multi dose administration in mice for over a month.

We believe the PATrOL platform has the potential to create drugs that are easy for patients to take at infrequent intervals after they have tested positive for a genetic disease but before symptoms emerge, said Dietrich Stephan, Ph.D., chief executive officer of NeuBase. We believe the best way to effectively manage degenerative genetic diseases is to get ahead of the disease process, and we believe that can only be achieved with early diagnosis coupled with well-tolerated, effective, and easily administered therapies.

Dr. Robert Friedlander, chief medical officer of NeuBase and member of the National Academy of Medicine, stated, An allele specific approach that can be systemically administered and cross the blood brain barrier would be an ideal drug profile for many untreatablegenetic diseases.I believe that NeuBase is moving towards realizing this goal.

The intersection of the NHP pharmacokinetic data and the in vitro patient-derived pharmacodynamic data provides a roadmap to create a pipeline of therapeutic candidates which can reach target tissues of interest after systemic administration and achieve the desired activity at that dose. NeuBase believes the data from these studies support the advancement of the Companys HD and DM1 programs into lead optimization and subsequent IND-enabling studies, as well as provide a roadmap for the future expansion of the Companys therapeutic pipeline into other indications, including oncology.

Dr. Sam Broder, former Director of the National Cancer Institute of the National Institutes of Health and member of the National Academy of Sciences, stated, I believe that the NeuBase strategy of targeting transcripts before they become dangerous mutant proteins has the potential to deliver a dramatic improvement in our collective capabilities to effectively treat a wide range of genetic diseases, including some of the most deadly cancers, by targeting driver mutations and accelerating immunotherapy capabilities.

Conference Call

NeuBase Therapeutics, Inc. will discuss these data and next steps for development during a webcasted conference call with slides today, March 31, 2020, at 8:00 a.m. ET. The live and archived webcast of this presentation can be accessed through the IR Calendar page on the Investors section of the Companys website, The dial-in details for the call are 877-451-6152 (domestic) or +1-201-389-0879 (international), and conference ID: 13701118. The archived webcasts will be available for approximately 30 days following the presentation date.

About NeuBase Therapeutics

NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform is designed to permit the rapid development of targeted drugs, thereby potentially increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements include, among other things, statements regarding the Companys goals and plans and the Companys pharmacokinetics and pharmacodynamics studies. These forward-looking statements are distinguished by use of words such as will, would, anticipate, expect, believe, designed, plan, or intend, the negative of these terms, and similar references to future periods. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those described in the risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Companys current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Companys plans to develop and commercialize its product candidates; the Companys plans to commence clinical trials in Huntingtons disease and myotonic dystrophy type 1 and to potentially expand the pipeline into other indications; the utility of the preclinical data generated in existing studies performed by the Company in determining the results of potential future clinical trials and of the potential benefits of the PATrOL platform technology; the timing of initiation of the Companys planned clinical trials; the timing of the availability of data from the Companys clinical trials; the timing of any planned investigational new drug application or new drug application; the Companys plans to research, develop and commercialize its current and potential future product candidates; the clinical utility, potential benefits and market acceptance of the Companys current and potential future product candidates; the Companys commercialization, marketing and manufacturing capabilities and strategy; the Companys ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCDaniel@lifesciadvisors.comOP: (617) 535-7746

NeuBase Media Contact:Travis Kruse, Ph.D.Russo Partners, LLCtravis.kruse@russopartnersllc.comOP: (212) 845-4272

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NeuBase Therapeutics Announces Positive, Preclinical Data Validating its Novel Genetic Therapy PATrOL Platform - GlobeNewswire

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REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV Technology Platform for the Treatment of Rare Metabolic Disorder – Herald-Mail…

Posted: March 31, 2020 at 5:47 pm

ROCKVILLE, Md. and NOVATO, Calif., March 31, 2020 /PRNewswire/ --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, and Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced a new exclusive, worldwide license agreement, extending the companies' existing gene therapy partnership.

Under the terms of the agreement, REGENXBIO has granted Ultragenyx an exclusive, worldwide license, with rights to sublicense, to REGENXBIO's NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder. In return for these rights, REGENXBIO will receive an upfront payment of $7 million, ongoing fees, milestone payments, and royalties on net sales of products incorporating the licensed intellectual property.

"We are pleased to expand our long-standing relationship with Ultragenyx through this new license agreement, which will enable Ultragenyx to apply our proprietary NAV Vectors to the development of a new gene therapy for rare diseases," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Today's announcement provides further validation of the breadth and depth of our intellectual property portfolio, and we remain committed to working closely with leading companies to enable the development of important gene therapy programs."

"This latest license agreement reinforces Ultragenyx's commitment to developing gene therapies for multiple rare diseases and adds a new indication to our relationship with REGENXBIO," said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. "

Ultragenyx is a longstanding licensee partner of REGENXBIO. In addition to today's agreement, Ultragenyx has in-licensed NAV Vectors for the development of gene therapies to treat Ornithine Transcarbamylase (OTC) Deficiency, Glycogen Storage Disease Type Ia (GSDIa), CDKL5 Deficiency, Hemophilia A, and Wilson Disease.


REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.


Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the Company's website at

REGENXBIO Forward-Looking Statements

This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements,whether as a result of new information, future events or otherwise.

Ultragenyx Forward-Looking Statements

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.


Tricia TruehartInvestor Relations and Corporate

Investors:Heather Savelle,

Media:David Rosen,

Ultragenyx Contacts:

Investors & MediaDanielle

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REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV Technology Platform for the Treatment of Rare Metabolic Disorder - Herald-Mail...

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