StemCell Therapy

Joint research program between the Japan Agency for Medical Research and Development and the Canadian Institutes of Health Research focuses on advancing technologies in epigenomics, deepening McGills relationship with Japans RIKEN institute.

By Junji Nishihata

In recent years, the concept of trained immunity has emerged as a new area of interest. This refers to the bodys inherent ability to reprogram the innate immune cells to respond differently to subsequent infections. Where does this recall come from? And what are the epigenetic mechanisms that enable it to persist? Indeed, the pursuit of answering the questions raised by trained immunity have been hampered by a lack of useable tools to facilitate research.

The Faculty of Medicine and Health Sciences Professor David Langlais will be pursuing this study thanks to a grant from a new collaboration between the Canadian and Japanese research communities. The novel program is a partnership of the Japan Agency for Medical Research and Development (AMED) and the Canadian Institutes of Health Research (CIHR). Aimed at early-career researchers such as Langlais, the collaborative research to be supported by the AMED and CIHR builds upon a far-reaching cooperation between McGill and RIKEN that uses genomics and immunology to address major questions in biomedical research.

Japans prestigious RIKEN Institute is composed of over 3,000 scientists working in seven centres across Japan. Its past body of achievements includes two Nobel prize winners. For the current project, Langlais will be interfacing with the teams of Drs Jay Shin and Aki Minoda at RIKENs Center for Integrative Medical Sciences, based in Yokohama.

In 2018, the McGill-RIKEN partnership received support from Quebecs Fonds de Recherche (FRQ). A new 5-year McGill-RIKEN agreement, signed on July 15, 2020, covers the assignment of joint laboratory space for the collaboration at McGill and the RIKEN Yokohama site, exchanges of scientists and trainees, and continuation of a joint symposium that has been held annually since 2017. In July, the RIKEN International Affairs Section also made a 5-year award of 10 million JPY annually towards these joint activities. The collaboration with RIKEN is part of McGill's wider engagement with Japanese academic institutions, which includes the joint McGill-University of Kyoto PhD degree in genomic medicine established in 2018, which is also supported by the FRQ and is the first of its kind for the two Universities.

Professor Mark Lathrop, Director of McGill Genome Centre, said: "We are very pleased that the AMED and CIHR will support this joint research project between McGill and RIKEN. The way the teams will be working closely together to bring their complementary expertise at the leading-edge of genomics and immunology to bear on very important biomedical questions is exemplary of the research that the McGill-RIKEN collaboration is intended to promote."

The $300,000 grant (awarded by CIHR over three years; AMED will grant 30 million JPY over the same time frame to the Japanese researchers) will enable three separate aspects to the study of trained immunity. First, it will permit the study of the phenomenon at the biological level, to better understand how chromatin conformation inherits the ability to recall pathogens from the past and eliminate newcomers. But to do so at the level of the individual cell will require the development of a new suite of tools which is the second aim of the project, and one of the main remits of the RIKEN side of the collaboration.

Specifically, the project seeks to develop bulk and single-cell Single-Molecule chromatin capture techniques that will reveal how the individual cells can be reprogrammed for the purposes resisting infections.

The third objective of the project will be to develop the necessary algorithms to interpret the resultant bioinformatics, a process that is sure to involve large amounts of data, in other words, Big Data. This portion of the study will be undertaken by Professor Hamed Najafabadi, in McGills Department of Human Genetics. A recipient of a Sloan Fellowship in 2018, Najafabadi brings considerable expertise in bioinformatics to the project.

Our teams are already fully integrated on this project, as well as on other collaborative research efforts, said Langlais referring to his collaborators in Japan. We will have exchanges with student internes attending our counterpart institutions, and we are expecting this work will result in a number of published papers, he explained, clearly enthused by the prospect. The expectation is that this will result in truly novel technologies that will advance the field of epigenomics overall.

In 2018, Langlais was among the McGill researchers figured prominently in Qubec Science magazines annual list of the top 10 scientific discoveries in Quebec. This joint research program announcement comes amidst deepening ties between Japan and Canada, as evidenced by Prime Minister Justin Trudeaus announcements in 2019, as well as parallel moves in Quebec.

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McGill team wins grant to study 'trained immunity' with Japanese researchers - McGill Newsroom

Naturopathicmedicine is a distinct primary health care system that blends modern scientificknowledge with traditional and natural forms of medicine. It is based on thehealing power of nature and it supports and stimulates the bodys ability toheal itself. Naturopathic medicine is the art and science of disease diagnosis,treatment and prevention using natural therapies including: botanical medicine,clinical nutrition, hydrotherapy, homeopathy, naturopathic manipulation,traditional Chinese medicine/acupuncture, lifestyle counselling and healthpromotion and disease prevention. Canadian Association of Naturopathic Doctors

Naturopathyis a cornucopia of almost every quackery you can think of. Be it homeopathy,traditional Chinese medicine, Ayurvedic medicine, applied kinesiology,anthroposophical medicine, reflexology, craniosacral therapy, Bowen Technique,and pretty much any other form of unscientific or prescientific medicine thatyou can imagine, its hard to think of a single form of pseudoscientificmedicine and quackery that naturopathy doesnt embrace or at least tolerate. Dr. DavidGorski

Naturopaths claim that they practice based on scientificprinciples. Yet examinations of naturopathic literature, practices andstatements suggest a more ambivalent attitude. NDhealthfacts.org neatlyillustrates the problem with naturopathy itself: Open antagonism toscience-based medicine, and the risk of harm from integrating these practicesinto the practice of medicine. Unfortunately, the trend towards integratingnaturopathy into medicine is both real and frightening. Because good medicineisnt based on invented facts and pre-scientific beliefs it must be groundedin science. And naturopathy, despite the claims, is anything but scientific. Scott Gavura (Science-Based Medicine)

Naturopathic training does not prepare them to be primary care physicians.Their profession is not science-based, does not have a science-based standardof care, and is largely a collection of pseudoscience and dangerous nonsenseloosely held together by a vague nature is always best philosophy.

This is one of those situations where most people will not believe that thesituation can be as bad as it really is. This is similar to when I describe topeople, who are hearing it for the first time, what homeopathy actually is.They usually dont believe it, because they cannot accept that something sononsensical can be so widespread and apparently accepted in our society. Thesame is true when I tell people about the core chiropractic philosophy of lifeenergy (at least for those chiropractors who have not rejected their roots), orabout what Scientologists actually believe.

One common reaction is the no true Scotsman logical fallacy. Defenderswill insist that what we are describing is the exception, and that a realnaturopath is not like that. Obviously there will be a range of practice(especially since there is no standard), but the pseudoscientific treatmentsthat make up naturopathy are not the exception. They are at the core of theireducation and their philosophy. Dr. Steven Novella

Naturopathic medicine is an eclectic assortment ofpseudoscientific, fanciful, and unethical practices. Implausible naturopathicclaims are still prevalent and are no more valid now than they were in 1968. Kimball C. Atwood

Naturopathic medical school is not a medical school inanything but the appropriation of the word medical. Naturopathy is not a branchof medicine. It is a combination of nutritional advice, home remedies anddiscredited treatments Naturopathic practices are unchanged by research andremain a large assortment of erroneous and potentially dangerous claims mixedwith a sprinkling of non-controversial dietary and lifestyle advice. The Massachusetts Medical Society

Naturopathy[1]is, and always has been, a declaration of pseudoscience and pseudomedicinemixed together with truism dressed-up in cheap makeup to appear legitimate,respectable, even advanced and modern, and real, as per the first statement atthe top in contrast to reliable and respected voices following it. Ignorance ina tutu is still ignorance.

Its notan alternative way of knowing, a different form of medicine, or a novel line ofthought. Its not cheaper than medicine because real medicine works on thecases needing it and, therefore, utilize the finances of patients properly,i.e., effectively.

Naturopathsare not doctors, medical doctors, or real MDs. By peddling nonsense assensible, they harm the public good and, thus, become a negative force insociety, as purveyors of illegitimate practice. Why deal a light critique toindividuals harming public in the most important areas of life, for example,medical care or health?

In turn,as self-proposed practitioners for the betterment of the health of the public,they detract attention and legitimacy away from real medical doctors, realmedicine, in addition to the finances of the public. If alternative medicinebecame effective, then it would become non-alternative medicine, also known asmedicine. So, whats the point of it, in the first place?

As notedin Freethought for the Small Towns: Case Study,Canadians and Others Convictions to DivineInterventionism in the Matters of the Origins and Evolution, Making a Buck as a Mountebank Astrologers, Mediums,and Psychics, The Message of William Marrion Branham: ResponsesCommentary, The Fantastic Capacity for Believing the Incredible,religious fundamentalism, pseudoscience, and pseudomedicine, play off oneanother, as gullibility in the pulpit informs gullibility in the wellnessmarketplace, and vice versa.

Oneignorance feeds into another. Whether in the local Township of Langley or inthe wider province of British Columbia, even in small towns including FortLangley, this is the nature of the pseudoscience and pseudomedicine landscape.Bad people, even thinking themselves good, bilk the public earning good money,even bad money or minimum wage income.

Theseindividuals and, more fundamentally, fraudulent practices, should be combatteddirectly, even at the legislative level as they have been enforced in countrieslike the United States largely through legislative efforts. Why such a directedeffort at legislation rather than randomized double-blind trials? Let me knowhow those homeopathic studies turn out.

InBritish Columbia, widely, when you do a search, you can find more than 100places, so associations, colleges, clinics, centres, integrative clinics, medicalcentres, practitioners, and so on. All devoted to a pseudoscientific practice withinone province. All either harming the bank accounts through fraudulentpractices, or, potentially, harming the public.

Personally,they should not be able to operate in British Columbia generally, or in theTownship of Langley in particular. Its easily viewable as a wide range ofpseudomedicine postulated as real medicine while without proper medical credentials,only fake qualifications, as in real to the fake medicine while faketo the real medicine.

Theresa large number of practitioners and clinics of naturopathy, includingassociations, colleges, and institutes, such as the College Of NaturopathicPhysicians Of British Columbia and the BC Naturopathic Association/BCNA.

Its a literal zoo with the number of them. In a general search of the Canadianprovince of British Columbia, one set includes Dr. Janine Mackenzie ND, AbbyNaturopathic Clinic: Dr. Cristina Coloma ND, Horizons Holistic Health Clinic,Edgemont Naturopathic Clinic, Boucher Naturopathic Medical Clinic, Dr. AggieMatusik, Integrative Naturopatic Medical Centre, Dr. Marisa Marciano, ND, Dr.Melanie DesChatelets ND, Vitalia Naturopathic Doctors Vancouver, Dr. Grodski White Rock Naturopathic, Dr. Lindsey Jesswein, ND, Noble Naturopathic, LocalHealth Integrative Clinic, Dr. Carlson-Rink C., Dr. Andrea Gansner NaturopathicPhysician, Dr. Lorne Swetlikoff, BSc.,, ND, Polo Health + Longevity Centre, ANew Leaf Naturopathic Clinic, Dr. E. DSouza-Carey, ND Family Health Clinic.

Another,second set includes Family Health Clinic: Naturopathic Medicine and MidwiferyCare, Integrated Health Clinic, Dr. Jiwani, Naturopathic Physician SurreyClinic (Not Vancouver) Autoimmune Weight Loss, Dr Andrew Eberding NaturopathicDoctor, Boucher Institute of Naturopathic Medicine, Meditrine NaturopathicClinic, Vancouver Naturopathic Clinic, Selkirk Naturopathic Clinic, Cross RoadsNaturopathic clinic, OZONE THERAPY BC: Dr. Walter Fernyhough, Dr. Allana PoloN.D Polo Health + Longevity Centre, Pangaea Clinic of Naturopathic MedicineInc, Dr Eric Chan, Dr Tawnya Ward, Dr. Rory Gibbons, Naturopathic Physician,Dr. Caroline Coombs Naturopathci Doctor, Dr. Brian Gluvic, KitsilanoNaturpathic Clinic, Agency Health, and Richmond Alternative Medical Clinic.

There theres the third set with Arc Integrated Medicine Delta & Surrey Naturopathic Doctors, Dr. Kali MacIsaac, Naturopathic Doctor, Aspire Naturopathic Health Centre Naturopath North Vancouver Dr. Emily Habert, ND, Dr. Hal Brown, Red Cedar Health Ray Clinic, Lonsdale Naturopathic Clinic, Metrotown Naturopathic and Acupuncture, Yaletown Naturopathic Clinic, Flourish Naturopathic, Northshore Naturopathic Clinic, and Dr. Jonathon F. Berghamer.

Thefourth set includes Dr. Scarlet Cooper, ND., Dr. Terrie Van Alystyne,Naturopathic Physician Whistler, Butterfly Naturopathic, Dr. Jason Marr, ND:Naturopathic Doctor, Peninsula Naturopathic Clinic, Dr. Karen Fraser, YaletownIntegrative Clinic, Serenity Aberdour ND Horizon Naturopathic Inc, Dr.Tasneem Pirani-Sheriff, ND, Avisio Naturopathic Clinic & VitaminDispensary, Dr. Robyn Land, Naturopathic Physician, Springs Eternal NaturalHealth, Dr. Alaina Overton, Cornerstone Health Centre: Maryam Ferdosian, ND,Dr. Kim McQueen, BSc, ND, Dr. Safia Kassam, and Restorative Health.

Thefifth set of them include Dr. Esha Singh, ND, Dr. Bobby Parmar NaturopathicDoctor, Lansdowne Naturopathic Centre, West Kelowna Integrative Health Centre,Dr. Shalini Hitkari, ND, Dr. Jolene Kennett, Naturopathic Doctor, Dr. KarinaWickland, ND, Dr. Phoebe Chow Lumicel Health Clinic, Dr. Maltais Lise,Vitality Wellness Centre, Dr. Lisa Good, ND, Dr. Heidi Lescanec, ND, Dr. RodSantos, ND, Inc., West Vancouver Wellness Centre, Dr. Kully Sraw, NaturopathicPhysician, Juniper Family Health, Dr. Peter Liu, ND, Garibaldi Health Clinic,Dr. Kayla Springer, ND, and Dr. Donna Ogden, ND, MSc, Naturopathic Doctor.

Thesixth yes, theres more set includes Dr. Cortney Boer, ND, Burnaby HeightsIntegrative HealthCare Inc., Dr. Amelia Patillo, ND, Jamie Sculley, Dr. EwingRobert J., Central Park Naturopathic Clinic, Dr. Kira Frketich, Living WellnessCentre, Dr. Jennifer Brown, ND, Dr. Randi Brown Naturopathic Doctor, WestShore Family Naturopathic Ltd., Rejuv-Innate Naturopathic Clinic-Dr. JamieGallant, Dr. Tonia Winchester, Nanaimo Naturopathic Doctor TonicNaturopathic, NaturopathicVictoria.net, Fourth and Alma Naturopathic MedicalCentre, Cheam Wellness Group, Maureen Williams, Dr. Meghan Dougan, ND, Dr.Brittany Schamerhorn, ND, and Dr. Jenna Waddy.

Theseventh almost there set includes Inner Garden Health, Dr. Brit Watters,ND, Dr. Laruen Tomkins, ND, The Natural Path Clinic Inc., Elizabeth Miller, Dr.Jennifer Moss Naturopathic Physician, Dr. Penny Seth-Smith, Seeded Nutrition,Northern Centre for Integrative Medicine, Aqua Terra Health, Dr. Kelsea Parker,ND, Maple Ridge Naturopathic Clinic, Newleaf Total Wellness Centre, VitalityIntegrative Health, Dr. Orissa Forest, BSc, ND, Acacia Health Dockside, Dr.Megan Kimberley, Naturopath, Dr. Landon McLean Healthcare, Back to Our RootsIndigenous Medicine, and N.A. Hemorrhoids Centre.

Theeighth set is Legacies Health Centre, Kelowna Naturopathic Clinic, MarseillesRemedy Traditional Oil Blend, Lani NYkilchuk, ND, Dr. Heather van der Geest,ND, Hummingbird Naturopathic Clinic, Dr. Elli Reilander, ND, BodaHealth, TheNatural Family Health Clinic, Dr. Chelsea Gronick, Naturopathic Doctor, Dr.Carla Cashin, ND, Dr. Karen McGree, Saffron Pixie Yoga & Naturopathy, WildHeart Therapies and Farmacy, Dr. Andrea Whelan, Well+Able Integrated HealthLTD., Dr. Kim Hine, ND, Dr. Graham Kathy, Dr. Emily Freistatter, NaturopathicDoctor, Inner Garden Health.

The ninth set is Dr. Emily Pratt, BSc, ND, Inc., Life Integrative, Dr. Michael Tassone, ND, Harbour Health: Massage Therapy, Physiotherapy, Chiropractor, Naturopath, Broadway Wellness, Spokes Clinical Naturopathy, Dr. Fulton Lynne, Electra Health, Dr. Macdonald Deidre, Ray Lendvai Naturopathic Physicians, Dr. Maryam Ferdosian, ND, Yinstill Reproductive Wellness, Prajna Wellness, Fountain Wellness & Physiotherapy, Qi Integrated Health, Paradigm Naturopathic Medicine, Apex Chiropractic Coquitlam, Kamloops Naturopathic Clinic, Dr. Carmen Anne Luterbach, and Dr. Mar Christopher.

The final and tenth set is Dr. Lawrence Brkich, The Phoenix Centre, Cave Cure & Therapies, Twisted Oak Holistic Health, Coast Therapy Maple Ridge, Balance Natural Health Clinic, Dr. Theresa Camozzi, ND, BC Pulse Therapy, Naramata Lifestyle Wellness-Best Naturopathy, Meditation, Weight Management Centre Okanagan, Acubalance Wellness Centre, Ltd., Dr. Milanovich David, Catalyst Kinetics Group, and Dr. Kimberly Ostero, BSc., ND, and Kontinuum Naturopathic Medicine, Inc.

Theobvious benefit in these titles compared to the astrologers, mediums, andpsychics, is the appearance of professionalism, while, in a mysterious manner,acquiring an entire reputation based on a fallacious premise, pseudomedicine,in addition to a false title.

Itsless turtles, turtles, turtles, all the way down, and more falsehoods all theway down, and to the top. People with all the accoutrement of theprofessional and medical world while, in fact, lacking the substance, thecontent, and so mimicking, or parroting, the forms and stylings of them.

A shame, a scandal in the province, a waste of the publics dime, a tax on the wellbeing of the province as a whole because real medicine exists, and ignorance without proper medical bases, while idiotic in its proposition and imbibing by the general public. Everyones to blame here; while, some are more culpable than others.

Thisshows both a failure in critical thinking on the part of the public, individualsentering into the schools for training, and a firm action on the part of theproper authorities to regulate public health in such a manner as todelegitimize failed philosophies from the 1800s proposed as modern medicine.

Asstipulated, succinctly, by the skeptic Wiki, RationalWiki, the titles of ND inBritish Columbia naturopaths and naturopathic physicians, self-proclaimed, asin Naturopathic Doctor, does not mean a doctor, a physician, or a medicaldoctor.

Thesetitles, ND, remain false proclamations of credentials and qualifications, byand large, rejected by both mainstream medicine and mainstream science. Theseare a manner in which to attempt to co-opt the earned legitimate legacy ofmodern medical science and modern science, as per credentials, e.g., MD, withillegitimate pseudoscience and pseudomedicine.

In fact,the issue in North America is widespread, as stated by RationalWiki, in Alternative Medicine Education, thereare actually 7accreditedinstitutionsin North America that award this degree (as of 2012), 5 in theUnited States(Bastyr University, National College ofNatural Medicine, National University of Health Sciences,SouthwestCollege of Naturopathic Medicineand University of BridgeportCollege of Naturopathic Medicine) and 2 inCanada(Boucher Institute ofNaturopathic Medicine, and Canadian College of Naturopathic Medicine).Forthose who want a shorter route, it is also widely available fromdiploma mills.

Theseindividuals will use the title of Dr. If you dont believe me, then I wouldpropose looking at the ten sets above. How often does the use of the term Dr.get used in the public face of the institutions?

Next, wecan ask about the private face. How many? How often? It is probably more, andmore forcefully, because Dr., rightfully, earned the title because theeducation is more difficult and the positive effects on society far more great.

That which was known as health fraud in prior generations through consistent efforts continues to be regarded more as medicine rather than medicine.

It should be halted, deconstructed, and shown for its farcical foundations and direct, and indirect, harms on the public.

[1] Even Wikipedia, as a minor resource, itstates:

Naturopathyornaturopathicmedicineis a form ofalternativemedicinethat employs an array ofpseudoscientificpractices branded asnatural, non-invasive, or promotingself-healing. The ideology and methods of naturopathy are basedonvitalismandfolk medicine, rather thanevidence-basedmedicine(EBM).Naturopathic practitioners generallyrecommend against followingmodern medical practices, including but notlimited tomedical testing,drugs,vaccinations, andsurgery.Instead, naturopathic practicerelies on unscientific notions, often leading naturopaths to diagnoses andtreatments that have no factual merit.

Naturopathy is considered by themedical professionto be ineffective and harmful, raisingethicalissues about its practice.In addition to condemnations and criticism from the medical community, such as theAmerican Cancer Society,naturopaths have repeatedly been denounced as and accused of beingcharlatansand practicingquackery.

Assistant Editor, News Intervention,Human Rights Activist.

Scott Douglas Jacobsen is the Founder of In-Sight: Independent Interview-Based Journal and In-Sight Publishing. He focuses on North America for News Intervention. Jacobsen works for science and human rights, especially womens and childrens rights. He considers the modern scientific and technological world the foundation for the provision of the basics of human life throughout the world and advancement of human rights as the universal movement among peoples everywhere. You can contact Scott via email.

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Naturopathy - How Not to be a Doctor and Harm the Public Good - News Intervention

Newswise Individuals with high ADHD-traits that do not meet the criteria for a diagnosis are less able to perform tasks involving attentional regulation or emotional control after a sleepless night than individuals with low ADHD-traits, a new study from Karolinska Institutet published inBiological Psychiatry: Cognitive Neuroscience and Neuroimagingreports.

While it can cause multiple cognitive impairments, there is considerable individual variation in sensitivity to the effects of insomnia. The reason for this variability has been an unresolved research question for long. In the present study, KI researchers investigated how sleep deprivation affects our executive functions, which is to say the central cognitive processes that govern our thoughts and actions. They also wanted to ascertain if people with ADHD tendencies are more sensitive to insomnia, with more severe functional impairments as a result.

ADHD (attention deficit hyperactivity disorder) is characterized by inattention, impulsiveness and hyperactivity; however, the symptoms vary from person to person and often also include emotional instability.

"You could say that many people have some subclinical ADHD-like symptoms but a diagnosis is only made once the symptoms become so prominent that they interfere with our everyday lives," says Predrag Petrovic, consultant and associate professor in psychiatry at the Department of Clinical Neuroscience at Karolinska Institutet, Sweden, who led the study along with Tina Sundelin and John Axelsson, both researchers at Karolinska Institutet and the Stress Research Institute at Stockholm University.

The study included 180 healthy participants between the ages of 17 and 45 without an ADHD diagnosis. Tendencies towards inattentiveness and emotional instability were assessed on the Brown Attention Deficit Disorder (B-ADD) scale.

The participants were randomly assigned to two groups, one that was allowed to sleep normally and one that was deprived of sleep for one night. They were then instructed to perform a test that measures executive functions and emotional control the following day (a Stroop test with neutral and emotional faces).

The researchers found that the sleep-deprived group showed worse performance in the experimental tasks (including more cognitive response variability). Moreover, people with high ADHD-traits were more vulnerable to sleep deprivation and showed greater impairment than those with low ADHD-traits.

The effects were also related to the most prominent type of subclinical ADHD-like symptom, in that after being deprived of sleep, the participants who displayed more everyday problems with emotional instability had larger problems with the cognitive task involving emotional regulation, and those who had more everyday inattention symptoms had larger problems with the non-emotional cognitive task.

"One of the reasons why these results are important is that we know that young people are getting much less sleep than they did just ten years ago," explains Dr Petrovic. "If young people with high ADHD-traits regularly get too little sleep they will perform worse cognitively and, what's more, their symptoms might even end up at a clinically significant level."

###

The study was supported by grants from the Swedish Research Council, Forte (the Swedish Research Council for Health, Working Life and Welfare), Riksbankens Jubileumsfond, Karolinska Institutet, Region Stockholm, the Swedish Society of Medicine, the Sderstrm-Knigska Foundation and the Osher Centre for Integrative Medicine. The study is part of a doctoral project by Orestis Floros, who is also a psychiatrist specialising in ADHD.

Publication: "Vulnerability in executive functions to sleep deprivation is predicted by subclinical ADHD symptoms". Orestis Floros, John Axelsson, Rita Almeida, Lars Tigerstrm, Mats Lekander, Tina Sundelin, Predrag Petrovic.Biological Psychiatry: Cognitive Neuroscience and Neuroimaging, 17 December 2020, doi: 10.1016/j.bpsc.2020.09.019.

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Individuals with high ADHD-traits are more vulnerable to insomnia - Newswise

The latest episode of the Oncology Peer Review On-The-Go podcast discusses a review article on patient-reported outcomes of cancer-related pain.

In this episode of "Oncology Peer Review On-The-Go", CancerNetwork takes a look at an article publishing in early 2021 for the Journal ONCOLOGY, titled Patient-Reported Outcomes of Pain and Related Symptoms in Integrative Oncology Practice and Clinical Research: Evidence and Recommendations.

The piece summarizes current and validated patient-reported outcomes (PROs) specific to cancer-related pain. The piece also recommends commonly used PROs to be standardized to aid integrative oncology clinicians and researchers in both patient care and study design. For the first of 2 interviews regarding this piece, CancerNetwork spoke with 3 authors of the article, W. Iris Zhi, MD, PhD, and Ting Bao, MD, DABMA, MS, from the Memorial Sloan Kettering Cancer Center, and Xin Shelley Wang, MD, MPH, of the MD Anderson Cancer Center.

For the response perspective, CancerNetwork spoke with Linda Carlson, PhD, of the Cumming School of Medicine at the University of Calgary. Carlson expanded on the work done by Zhi and her colleagues, emphasizing the importance of understanding reliability and validity. Carlson focused on the importance of the research topic, and the impact properly validated PROs can have on patients for the cancer care team.

Dont forget to subscribe to the "Oncology Peer Review On-The-Go" podcast on Apple Podcasts, Spotify or anywhere podcasts are available.

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Oncology Peer Review On-The-Go: Recommending Patient-Reported Outcomes of Pain for Cancer Care - Cancer Network

The holiday season is in full swing and, even in these unusual times, it seems likely that the period between now and the new year will be filled with yummy, calorie-laden foods, such as cookies, pies and hot chocolate topped with whipped cream.

With so much temptation, some people might still be wondering how to control their weight. One popular weight loss method thats emerged during the past few years is a technique called intermittent fasting, in which people severely limit their calorie intake in some way. Though loyalists claim to drop pounds this way, experts say just how effective the method is remains unclear.

Its a way people think they can lose weight very quickly, says Kelly Gruber, a registered dietitian with Bridgeport Hospital. But theres not a lot of research out there.

In fact, its hard to even accurately define intermittent fasting, because there are multiple techniques, experts say. Some intermittent fasters eat normally for five days of the week, then stringently restrict their calories (to anywhere from 400 to 600 calories a day, depending on the dieter) for two days of the week.

Other fasters have a window of time (usually about eight hours) in which to eat normally, then refrain from food for the remainder of the day. Other methods can include an every-other-day method, in which fasters eat normally one day, then consume only a few calories the next.

The studies are a little bit all over the place because theres no standard definition of what intermittent fasting means, says Ellen Liskov, outpatient nutrition specialist at Yale New Haven Hospital. Ive had clients tell me theyre interested in intermittent fasting, but that means different things to different people.

She and other experts, Gruber included, say another complicating factor is that there isnt a ton of research on intermittent fasting, and whats there isnt necessarily conclusive.

For instance, a study published in September in the Journal of the American Medical Assocation looked at 116 participants, some of whom were only allowed to eat between noon and 8 p.m., and some of whom observed a more typical meal plan, of three consistent meals with snacks throughout the day. The study showed that both groups lost weight and the researchers ultimately concluded that time-restricted eating, in the absence of other interventions, is not more effective in weight loss than eating throughout the day.

Other research, most of it in animals, shows evidence of weight loss, and some improvement in blood sugar levels, cholesterol levels and blood pressure. A paper published by Harvard Medical School in 2018 mentioned this, and added that research in humans has shown the method to be fairly safe and effective in humans, but perhaps not more so than other diet plans.

Many dietitians seem mixed on intermittent fasting. It can be difficult, because its not something people are going to stay with long term and it doesnt really teach you how to to eat correctly, Gruber says. It can lead to a calorie deficit over the course of a week, but, over the long-term, is it really a healthy eating pattern?

There are potential drawbacks to intermittent fasting, says Sunida Infahsaeng, a registered dietitian, and director of clinical nutrition at St. Vincents Medical Center, in Bridgeport. If its not done under the care of a physician, it can lead to low blood sugar, low blood pressure, dehydration, headaches and other issues. Infahsaeng says another potential issue is that people can end up overeating on their normal eating days, resulting in no weight loss, or even weight gain.

And there are some groups of people who should stay away from intermittent fasting entirely, such as pregnant women, children, the elderly and those with diabetes.

But, for other groups there are possible benefits of this way of eating, Infahseng says. For one thing, it can make people more aware of what they consume during their eating windows, which could lead to healthier habits.

Dr. Joseph Feuerstein, director of integrative medicine at Stamford Hospital, says he sees one big benefit to restricting eating to one window of time during the day. It stops you eating at night, which is a major weight gain factor, he says.

Ultimately, experts say, if people are really interested in intermittent fasting, they should consult a medical professional first. If someone tells me Im dead set on intermittent fasting Im doing this, then I will work with them to do it safely, Gruber says.

acuda@ctpost.com; Twitter: @AmandaCuda

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'Is it really a healthy eating pattern?': Experts mixed on intermittent fasting - CT Insider

Having recently discussed coffins in this column, and what with it being a rather unusual year in so many ways, we should perhaps discuss how to engineer not ending up in one this Christmas.

It wouldnt really be Christmas without snow, which means that in the UK, its almost never Christmas. There has only been snow on the ground at over 40 per cent of UK weather stations on Christmas Day four times in the 51 years leading up to 2015. However, this might be a blessing should you sit down this Christmas with a copy of Surveillance for injuries: cluster of finger amputations from snow-blowers, which rather alarmingly suggests that on Christmas Eve, 1982, 15 Denver residents had a finger amputated following a snow blower accident.

My advice is never clear blocked snow from your snow-blower with your hands. Now should you be living in Australia, you probably think this doesnt apply to you, and youd be right. Yet there is a large Christmas spike in A&E admissions in your country and New Zealand from jet ski and boat propeller accidents, not to mention barbeque injuries something we dont have to bother with too much in the UK.

Even when staying indoors, Christmas can be dangerous. Thankfully these days, we rarely illuminate highly flammable Christmas trees with candles although the Swiss, preferring this traditional light, clock up around one serious burns case a year from tree candles. More worrying nowadays is the quality of electric lighting on trees; nearly half of Christmas tree fires in the USA between 2013 and 2017 were due to failed electrics. More bizarrely, one-fifth were started deliberately! Its not just fires.

Putting up the lights is just as dangerous as switching them on. A Canadian study showed that those injured in this way spend an average of 15 days in hospital, and 5 per cent of those admissions sadly die. Its also worth checking the wiring before plugging the lights in, as 1 in 40 of us have suffered an electric shock from them. Thats not including the 26 people who died between 1997 and 2010 in the UK when they foolishly tried to water their real trees with the lights still switched on. I hardly need to mention to any parents reading the dangers of smaller, more excitable folk swallowing or inhaling the tiny bulbs. While Im on the subject, they should avoid holly, mistletoe, Christmas cherry and Christmas rose, all of which are very poisonous to children.

Other decorations are no safer. According to the Royal Society for the Prevention of Accidents, 1 in 50 people have fallen out of the loft just retrieving the decorations in the first place and US fire trucks respond to an average of 780 fires a year caused by decorations excluding those lethal Christmas trees being left too close to a heat source.

Christmas lunch is also a minefield. One in five of us cuts ourselves just preparing the vegetables, and the Food Standards Agency estimates around 11 million of us defrost our turkeys in an unsafe place, risking campylobacter and salmonella among other potentially deadly infections. Even if we survive the main course, 600,000 of us have burnt ourselves roasting chestnuts on an open fire. I blame Nat King Cole.

Perhaps we should all go out instead? Certainly not! In an Advances in Integrative Medicine article based on an analysis of hospital records for injuries connected to Christmas products and activities between 2007 and 2016, in the US there were 240,626 toboggan-related injuries over the holiday period. Nor do we have to injure ourselves. A rather depressing article Assault-related facial injuries during the season of goodwill, which set out to see if the Season of Goodwill over the 12 days of Christmas resulted in a drop in deliberate facial injuries, found exactly the reverse. Christmas is, in fact, the best time to get punched in the face.

If all this has made you decide to stay in and pour yourself a large drink, you shouldnt do that either. The authors of Santa baby, hurry [extra carefully] down the chimney tonight Prevalence of Christmas-related injuries 2007-2016 in the United States note that:

The exuberance those with high levels of Christmas spirit (and potentially high levels of ingested spirits) possess may lead them to become overconfident in their abilities to perform physical tasks, or practice potentially dangerous activities safely. Overconfidence is a known risk factor for injuries in motor vehicle accidents and workplace accidents, and as such is likely to also be a factor in Christmas-related injuries.

Interestingly, these overconfidence injuries occur almost exclusively in men.

Surely were finally safe to go and see Santa Claus himself? Again, this is risky. In the US, 31 children a year are seen in Emergency Rooms having either fallen off Santas lap or tripped having run away from him in fear.

Happy Christmas!

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The eccentric engineer: 'tis the season to be jolly... and safe - E&T Magazine

Eat This, Not That!

For months, Dr. Anthony Fauci, the nation's top infectious disease expert and the director of the National Institute of Allergy and Infectious Diseases, said the coronavirus vaccines would be so safe, he'd take one. And in fact others have been looking to him to see if it's safe before taking theirs. "People like Anthony Fauci, who I know, and I've worked with, I trust completely," former President Barack Obama said. "So if Anthony Fauci tells me this vaccine is safe, and can vaccinate, you know, immunize you from getting Covid, absolutely I'm going to take it."Today, Fauci took it. Read on to see what he saidand to ensure your health and the health of others, don't miss these Sure Signs You've Already Had Coronavirus.Fauci Feels "Extreme Confidence" in the VaccineBefore getting the first dose of the Moderna shot, Dr. Fauci said "it's important for two reasons. One is that I'm an attending physician here on the staff at the National Institutes of Health Clinical Center. And so I do see patients, but as important or more important is as a symbol to the rest of the country, that I feel extreme confidence in the safety and the efficacy of this vaccine. And I want to encourage everyone who has the opportunity to get vaccinated so that we could have a veil of protection over this country that would end this pandemic."When You'll Get Your Own VaccineWith two vaccinesone from Pfizer-BioNTech and one from Modernadistribution is expected to "speed up," say experts. "We know we have the vaccine available to get to 20 million people by the end of December and then a total of 50 million by the end of January," Alex Azar, the United States Secretary of Health and Human Services, told the Today Show.Fauci has previously said he's hopeful that "by the time you get into the middle, towards the end of the first quarter of 2021, you will have accounted for and vaccinated those who are in the higher priority groups." Then, "I would project by the time you get to April, it will be 'open season,' in the sense of anyone, even the non-high priority groups could get vaccinated," Fauci said.His next few months will be devoted to convincing you to get yours; the vaccine needs to be taken by enough people so we can develop herd immunity. "Why do I say that it is the only way that we're going to end this epidemic?" he asked earlier this month. "If we get, I would say a range from 70 to 85% of the population vaccinated, we will create an umbrella or a blanket as it were of herd immunity over the population to the point that the virus is not going to have any place to go in us. In other words, we could essentially end this outbreak in this country, as we know it, and put it in the rear view mirror.""I think now that we have in our hands vaccines that are 94 to 95% effective," he said, "so we need to make sure that we make that efficacy turn into effect."RELATED: If You Feel This, You May Have Already Had COVID, Says Dr. FauciHow to Survive This PandemicAs for yourself, follow Fauci's fundamentals and help end this surge, no matter where you livewear a face mask, social distance, avoid large crowds, don't go indoors with people you're not sheltering with (especially in bars), practice good hand hygiene, get vaccinated when it becomes available to you, and to protect your life and the lives of others, don't visit any of these 35 Places You're Most Likely to Catch COVID.

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Carey Mulligan and Emerald Fennell on why 'Promising Young Woman' is the ultimate #MeToo revenge film - Yahoo Canada Shine On

Eat This, Not That!

For months, Dr. Anthony Fauci, the nation's top infectious disease expert and the director of the National Institute of Allergy and Infectious Diseases, said the coronavirus vaccines would be so safe, he'd take one. And in fact others have been looking to him to see if it's safe before taking theirs. "People like Anthony Fauci, who I know, and I've worked with, I trust completely," former President Barack Obama said. "So if Anthony Fauci tells me this vaccine is safe, and can vaccinate, you know, immunize you from getting Covid, absolutely I'm going to take it."Today, Fauci took it. Read on to see what he saidand to ensure your health and the health of others, don't miss these Sure Signs You've Already Had Coronavirus.Fauci Feels "Extreme Confidence" in the VaccineBefore getting the first dose of the Moderna shot, Dr. Fauci said "it's important for two reasons. One is that I'm an attending physician here on the staff at the National Institutes of Health Clinical Center. And so I do see patients, but as important or more important is as a symbol to the rest of the country, that I feel extreme confidence in the safety and the efficacy of this vaccine. And I want to encourage everyone who has the opportunity to get vaccinated so that we could have a veil of protection over this country that would end this pandemic."When You'll Get Your Own VaccineWith two vaccinesone from Pfizer-BioNTech and one from Modernadistribution is expected to "speed up," say experts. "We know we have the vaccine available to get to 20 million people by the end of December and then a total of 50 million by the end of January," Alex Azar, the United States Secretary of Health and Human Services, told the Today Show.Fauci has previously said he's hopeful that "by the time you get into the middle, towards the end of the first quarter of 2021, you will have accounted for and vaccinated those who are in the higher priority groups." Then, "I would project by the time you get to April, it will be 'open season,' in the sense of anyone, even the non-high priority groups could get vaccinated," Fauci said.His next few months will be devoted to convincing you to get yours; the vaccine needs to be taken by enough people so we can develop herd immunity. "Why do I say that it is the only way that we're going to end this epidemic?" he asked earlier this month. "If we get, I would say a range from 70 to 85% of the population vaccinated, we will create an umbrella or a blanket as it were of herd immunity over the population to the point that the virus is not going to have any place to go in us. In other words, we could essentially end this outbreak in this country, as we know it, and put it in the rear view mirror.""I think now that we have in our hands vaccines that are 94 to 95% effective," he said, "so we need to make sure that we make that efficacy turn into effect."RELATED: If You Feel This, You May Have Already Had COVID, Says Dr. FauciHow to Survive This PandemicAs for yourself, follow Fauci's fundamentals and help end this surge, no matter where you livewear a face mask, social distance, avoid large crowds, don't go indoors with people you're not sheltering with (especially in bars), practice good hand hygiene, get vaccinated when it becomes available to you, and to protect your life and the lives of others, don't visit any of these 35 Places You're Most Likely to Catch COVID.

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Keyontae Johnson will be released from hospital Tuesday - Yahoo Canada Shine On

Both studies were conducted as pilot studies in a pre-post observational design. Details are already reported elsewhere [13]. The primary outcome for both studies was the proportion of days with reaching the fasting goal of 15h out of the total number of days recorded per participant in the diary. According to the study protocol, secondary outcomes were, among others, changes in sleep quality and duration, and HRQoL between baseline and follow-up.

Participants at the Ulm University were recruited with the support of the occupational health management and by flyers. Exclusion criteria were pre-existing metabolic conditions. Patients at the GPs office were informed about the study by flyers in the waiting room or were invited by the doctor during a consultation. Exclusion criteria were insulin dependent diabetes or any other disease for which fasting is contraindicated [3]. Finally, 63 participants at the Ulm University and 40 participants at the GPs office were included in the studies.

Participants in both studies were asked to limit their daily food intake to 89h and subsequently extend their nightly fasting period to 1516h. The duration of the intervention was 3 months. At baseline, participants had an introductory conversation with the principal investigator or the physician to clarify possible questions and problems in advance, and were given an information brochure. In addition, all participants were offered to contact the respective study centre at any time if they had questions or problems.

Baseline assessment comprised a questionnaire to collect data on lifestyle, health behaviour and HRQoL (EQ-5D VAS) [14], and anthropometric measurements of waist, height, and weight (for details see [13]). All participants were given a diary to record the times of their first and their last meal, and the quality and duration of their sleep. The latter was assessed on a visual analogue scale ranging from 0 (worst possible sleep quality) to 100 (best possible sleep quality). The waist-to-height ratio (WHtR) was calculated by the division of waist by height in centimetre, abdominal obesity was then defined as WHtR 0.5, as recommended by the literature [15]. Body weight in kilogram was divided by height in meters squared to determine body mass index (BMI), and subsequently categorized into overweight ( 25) and obesity ( 30).

After 3 months, follow-up measurements were performed in the same manner, with some additional items in the questionnaire regarding the individual experience and attitudes towards TRE.

Baseline characteristics are reported descriptively for each study group and for both groups combined. Differences between groups were tested by applying t-test, Welchs t-test or Mann Whitney U test according to distribution and heterogeneity in variance for continuous data, and Fishers exact test for categorical data.

Follow-up data, and computational differences between baseline and follow-up data, presented as the respective , were treated the same way. Pre and post comparisons for both groups taken together were determined by the Wilcoxon signed-rank test for related samples.

For each participant, mean values and standard deviations were calculated for the data from the diaries. Time of first meal and time of last meal were utilized to determine the duration of the fasting and the eating phase. For all days recorded, the percentage of days with fasting target reached was calculated. Differences between groups were tested as described above.

To assess differences between sleep duration and quality at the beginning and at the end of the TRE intervention period, mean values were calculated for the first 10% and the last 10% of data (or days), respectively. Subsequently the differences between the first and the last 10% of the data were calculated as the respective . They are reported together with the average number of days recorded per group and for the whole group.

Pearsons correlation coefficient was applied to test bivariate correlations between continuous variables.

Linear regression analyses were conducted for the pre-post differences in HRQoL and the differences in sleep quality between the first 10% and the last 10% of days recorded. Potential factors that might correlate with the HRQoL or sleep quality were identified and, together with variables that differed at baseline between both groups, tested in a stepwise backward elimination. Sex, age, baseline values of HRQoL, the sleep quality and sleep duration on the first 10% of reported days, mean duration of fasting, percentage of fasting target reached, and finally group membership were considered as potential associated factors. Anthropometric measures represented both, potential associated factors and differences between groups at baseline. Therefore, weight, waist circumference, BMI, WHtR, overweight, obesity, abdominal obesity as well as the respective between pre and post measures of the continuous variables were considered in the regression analysis. All assumptions of linear regression (linear relationship, multivariate normality, multicollinearity, auto-correlation, homoscedasticity) were examined.

The significance level for two-sided tests was set at =0.05. All statistical analyses were carried out by using the statistical software packages IBM SPSS Statistics for Windows, Version 25.0. (IBM Corp., Armonk, NY, USA).

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Associations of time-restricted eating with health-related quality of life and sleep in adults: a secondary analysis of two pre-post pilot studies -...

Eat This, Not That!

For months, Dr. Anthony Fauci, the nation's top infectious disease expert and the director of the National Institute of Allergy and Infectious Diseases, said the coronavirus vaccines would be so safe, he'd take one. And in fact others have been looking to him to see if it's safe before taking theirs. "People like Anthony Fauci, who I know, and I've worked with, I trust completely," former President Barack Obama said. "So if Anthony Fauci tells me this vaccine is safe, and can vaccinate, you know, immunize you from getting Covid, absolutely I'm going to take it."Today, Fauci took it. Read on to see what he saidand to ensure your health and the health of others, don't miss these Sure Signs You've Already Had Coronavirus.Fauci Feels "Extreme Confidence" in the VaccineBefore getting the first dose of the Moderna shot, Dr. Fauci said "it's important for two reasons. One is that I'm an attending physician here on the staff at the National Institutes of Health Clinical Center. And so I do see patients, but as important or more important is as a symbol to the rest of the country, that I feel extreme confidence in the safety and the efficacy of this vaccine. And I want to encourage everyone who has the opportunity to get vaccinated so that we could have a veil of protection over this country that would end this pandemic."When You'll Get Your Own VaccineWith two vaccinesone from Pfizer-BioNTech and one from Modernadistribution is expected to "speed up," say experts. "We know we have the vaccine available to get to 20 million people by the end of December and then a total of 50 million by the end of January," Alex Azar, the United States Secretary of Health and Human Services, told the Today Show.Fauci has previously said he's hopeful that "by the time you get into the middle, towards the end of the first quarter of 2021, you will have accounted for and vaccinated those who are in the higher priority groups." Then, "I would project by the time you get to April, it will be 'open season,' in the sense of anyone, even the non-high priority groups could get vaccinated," Fauci said.His next few months will be devoted to convincing you to get yours; the vaccine needs to be taken by enough people so we can develop herd immunity. "Why do I say that it is the only way that we're going to end this epidemic?" he asked earlier this month. "If we get, I would say a range from 70 to 85% of the population vaccinated, we will create an umbrella or a blanket as it were of herd immunity over the population to the point that the virus is not going to have any place to go in us. In other words, we could essentially end this outbreak in this country, as we know it, and put it in the rear view mirror.""I think now that we have in our hands vaccines that are 94 to 95% effective," he said, "so we need to make sure that we make that efficacy turn into effect."RELATED: If You Feel This, You May Have Already Had COVID, Says Dr. FauciHow to Survive This PandemicAs for yourself, follow Fauci's fundamentals and help end this surge, no matter where you livewear a face mask, social distance, avoid large crowds, don't go indoors with people you're not sheltering with (especially in bars), practice good hand hygiene, get vaccinated when it becomes available to you, and to protect your life and the lives of others, don't visit any of these 35 Places You're Most Likely to Catch COVID.

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Robert Lewandowski is the best football player of 2020 - Yahoo Canada Shine On

The argument for continued investment

C> is a high potential and maturing sector, and is an already crowded environment, playing host to numerous start-ups and now, through M&A, recognised big pharma firms. Much like the rush to find a COVID-19 vaccine that dominates headlines worldwide, not every company involved will be able to succeed.

But finnCaps finnLife watch list of 50 leading AIM-listed biotech companies demonstrates that there is room for numerous companies to contribute to, and profit from, C>. Examining three entirely different approaches to CAR-T therapy, it is possible to see just how much space there is for this exciting sector, therefore displaying the case for continued investment.

Innovative CAR-T therapy demonstrates the depth of C> potential

CAR-T therapy in its existing form is a relatively new and specialised approach at treating cancer. It takes T cells from a patients bloodstream and genetically modifies them in a laboratory. These T cells are then injected back into the bloodstream with the aim of targeting and killing cancer cells.

While it has been shown to be an effective treatment, there are risks and side effects. One is the two-step autologous process (the slow time it takes for cell expansion sometimes as long as two weeks) while another is cytokine release syndrome (CRS), which occurs when cytokine molecules are inadvertently released, but too quickly to target just the tumours and instead target healthy cells.

The next generation of CAR-T treatments shows that there is space for a multitude of start-ups to be active in the C> space as they all help find varied solutions to these problems without negating the effectiveness of CAR-T.

One example is Horizon Delivery, a company that is developing its CYAD-02 project, which will help transport T cells more effectively to the tumour via the use of SMARTvector products.

The product underwent its first phase 1 trial test in January 2020 with a patient who was suffering from acute myeloid leukaemia. Horizon Delivery is also an industry leader in CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) screenings, meaning they can identify key genes or genetic sequences that draw out specific functions of a cell type from thousands of potential variants.

In a cancer context, this means they can route out and exclusively eliminate problematic cells that may have shown signs theyd resist a future cancer treatment.

Another example is Maxcyte, a global cell- based therapies and life sciences company that is developing its CARMA process, where a patients peripheral blood mononuclear cells (PBMCs) are removed and modified. The modified cells can then be used to target an array of different cancers.

Currently the company is conducting a phase 1 trial for advanced ovarian cancer in a dose escalation trial that will treat four separate cohorts the fourth of which was administered in March 2020.

Another example which shows the versatility of new CAR-T innovation is provided by Oxford Biomedica, a gene and cell therapy company specialising in the development of gene-based medicines.

Rather than a contained project or platform, its contribution to CAR-T is through a contract manufacturing development organisation. Collaborating with pharma companies, Oxford Biomedica uses its infrastructure to produce other companies licensed products, including Novartis Kymriah treatment (alongside other undisclosed CAR-T-related products).

With fast-moving innovation finally allowing multiple C> treatments to gain regulatory approval, along with a huge pipeline of upcoming therapies and an influx of funding and M&A activity, investing in C> no longer entails taking a bet on potential the future is finally here.

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After years of potential, cell and gene therapy is ready for the pharmaceutical mainstream - PMLiVE

Neurogene has raised $115 million in a second-round financing that will accelerate its plans to start clinical trials of a range of gene therapies for inherited neurological diseases, initially focusing on a form of Batten disease.

Batten disease is a group of disorders caused by a deficiency in proteins that allow fatty substances build up in nervous tissue, causing seizures, visual impairment, mobility loss and early death.

The New York biotechfounded by former Wall Street analyst Rachel McMinn, Ph.D., two years agosaid some of the cash will be used to advance its lead programs targeting Batten disease caused by CLN5 and CLN7 mutationstwo rare and rapidly progressive subtypes that occur in later childhood.

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It will also fund development of candidates for lysosomal storage disorders Charcot-Marie-Tooth disease (CMT) type 4J, caused by changes in the FIG4 gene, and aspartylglucosaminuria (AGU), which arises when the AGA gene is mutated.

The funding will also go toward developing its gene therapy platform and scaling up its manufacturing capacity. It adds to the $68.5 million the biotech raised in its series A round last year.

The AGU and CMT4J programs had been scheduled to reach the clinic this year, but Neurogene has been making quicker progress with the Batten disease drive.

Furthest along right now is its CLN5 candidate, which should start clinical trials next year. Batten disease is an autosomal recessive disorder, which means it only develops if a person inherits two copies of a faulty gene for their parents.

Neurogene intends to treat it by using an adeno-associated virus vector to deliver a replacement CLN5 gene, restoring the activity of the protein it codes for. While the function of that protein isnt well understood, the hope is that restoring its activity will slow down or halt the progression of the disease.

Neurogene isnt the only group eyeing the potential for gene therapies to make a difference to the lives of patients with Batten disease.

Abeona Therapeutics has been working in this area for a few years, and earlier this year licensed its ABO-202 candidate for CLN1 diseasealso known as infantile Batten diseaseto Taysha Gene Therapies for $7 million upfront and up to $56 million in milestones. It is also developing ABO-201 for CLN3 disease.

Last year, Amicus Therapeutics presented the first results with its CLN6 gene therapyacquired as part of its $100 million takeover of Celenex in 2018that seem to indicate a slowing of neurological decline.

Meanwhile, researchers at Cornell University have carried out a phase 1 gene therapy trial in late- infantile (CLN2) disease with similar positive results, and have also started a phase 1/2 study in this group.

Gene therapy has generated tremendous hope for the many families and patients with severe genetic disorders, McMinn said.

We believe our focus on improved product design, innovative technology, cutting-edge vector manufacturing and premier analytics will help fulfill the potential of genetic treatments.

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Neurogene adds another $115M to the pot for trials for its gene therapies - FierceBiotech

Tada Images/Shutterstock

Thermo Fisher Scientific has been as busy as Santas elves this December. The company is significantly expanding its footprint with new facilities in the United States and in Europe.

This week, the company announced it started construction of a new cGMP plasmid DNA manufacturing facility inCarlsbad, Calif.This expansion builds on the company's continued investment in cell and gene therapy services. The site will expand the company's clinical and commercial capabilities for cGMP plasmid DNA used as a critical raw material to develop and manufacture cell and gene-based therapies including life-saving cancer treatments as well as mRNA vaccines. In addition, the site will have the capability to produce large-scale plasmid DNA as a primary drug substance for DNA therapies.

The California site will span 67,000 square feet and is expected to be completed in the first half of 2021. The facility will feature advanced technologies, including single use equipment with up to 1,000L scale, digital connectivity and data visibility to enable operational efficiencies and operator training, the company said. The new commercial facility will add approximately 150 jobs over the next 12 months.

The race to develop new transformative cell and gene therapies and vaccines is outpacing supply of commercial-quality plasmid DNA that can be produced at scale, Mike Shafer, senior vice president and president of pharma services at ThermoFisher said in a statement. Our new state-of-the art site will not only tackle the supply bottleneck for our customers, but also uniquely positions us to deliver robust, end-to-end cell and gene therapy capabilities.

Shafer said Thermo Fisher is making strategic investments in capacity, technology and expertise across its global network so the company can accelerate innovation and enhance productivity for its customers.

The California site isnt the only expansion the company is undertaking. To meet accelerating demand for robust clinical supply chain services throughout Europe, Thermo Fisher Scientific has expanded its pharma services footprint with two new facilities inRheinfeldenand Weil am Rhein, Germany. The new sites will boost clinical supply chain continuity and specialized cold chain and cryogenic expertise across Europeand globally.

TheRheinfeldensite opens in late December 2020 and the Weil am Rhein site will open in January 2021.InRheinfelden, the new 86,000-square-foot/8,000-square-meter facility significantly increases the company's footprint for secondary packaging, storage, logistics and distribution of clinical supplies to investigator sites across Europe. In Weil am Rhein, the new 9,600-square-foot/890-square-metercryocenterprovides specialized ultra-low-temperature, cryogenic storage and cold chain expertise for clinical supply chain needs for cell and gene-based therapies, including COVID-19 vaccine candidates.

These facilities combined with our established regulatory expertise will give customers the continuity and in-region capabilities to support clinical trials across multiple therapy areas, Shafer said.

In addition to the construction of new facilities, earlier this month, Thermo Fisher announced the expansion of several of its existing sites that will increase the companys capabilities for sterile drug product development and commercial manufacturing of critical medicines, therapies and vaccines.

Thermo Fisher is expanding its facilities in Greenville, N.C., Swindon, England and two sites in Italy, Ferentino and Monza. Not only will the expansion of these facilities boost commercial production lines and support capabilities for aseptic liquid and lyophilized vial filling, but the projects will also add approximately 1,000 new jobs. The expansions are expected to be completed over the next two years, Thermo Fisher said in its announcement.

In addition to expansions inNorth AmericaandEurope, the company recently announced significant projects inAsia-Pacific, including a new sterile manufacturing facility inSingaporeand a new integrated biologics and sterile drug development and manufacturing site inHangzhou, China.

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Thermo Fisher Invests in Gene Therapy Future with Huge Expansions - BioSpace

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 22 December 2020 18:50Hits: 116

TSHA-101 to be first bicistronic vector evaluated in human clinical trials; TSHA-101 designed to deliver both HEXA and HEXB transgenes within a single AAV9 vector construct

TSHA-101 CTA is the second clinical trial clearance received, in addition to TSHA-118s open investigational new drug application for CLN1

Interim data from Phase 1/2 trial anticipated in 2021

DALLAS, TX, USA I December 21, 2020 I Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that Queens University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health Canada for its investigator-sponsored Phase 1/2 trial exploring TSHA-101, Tayshas investigational AAV9-based gene therapy, for the treatment of infantile GM2 gangliosidosis.

TSHA-101 will be the first bicistronic vector to enter a first-in-human clinical study, which is a significant milestone for Taysha and for the field of gene therapy, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. GM2 is a devastating lysosomal storage disease with no approved treatments and todays CTA approval marks a formative moment for children suffering from this rapidly progressive and fatal disease.

The trial will be a single arm, open-label Phase 1/2 trial evaluating the use of TSHA-101 for the treatment of infants with GM2. The study will be sponsored by Queens University and led by Jagdeep S. Walia, MBBS, FRCPC, FCCMG, Clinical Geneticist and Associate Professor Head, Division of Medical Genetics (Department of Pediatrics) at Queens, and Director of Research (Department of Pediatrics), at the Kingston Health Sciences Centre.

Preclinical evidence to date supports our belief that TSHA-101, when given intrathecally as a bicistronic transgene packaged into a single AAV9 vector, has the potential to address the lysosomal enzyme deficiency, to change the disease trajectory and to improve patient survival, said Dr. Jagdeep S. Walia. We are pleased to have the support of Health Canada as we continue to advance TSHA-101.

Todays CTA approval is a culmination of our teams and Dr. Walias tireless efforts and a momentous occasion for children affected by GM2 along with their parents and caregivers, said RA Session II, Founder, President and CEO of Taysha. We are grateful to our partners at Queens University for their work to advance this gene therapy into the clinic.

About GM2 Gangliosidosis

GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the -hexosaminidase A enzyme. These genetic defects result in progressive dysfunction of the central nervous system. There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.

About TSHA-101

TSHA-101 is an investigational gene therapy administered intrathecally for the treatment of infantile GM2 gangliosidosis. The gene therapy is designed to deliver two genes HEXA and HEXB driven by a single promoter within the same AAV9 construct, also known as a bicistronic vector. This approach allows the simultaneous expression of a 1:1 ratio of the two subunits of protein required to generate a functional enzyme. It is the first and only bicistronic vector currently in clinical development and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA).

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

SOURCE: Taysha Gene Therapy

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Taysha Gene Therapies Announces Queen's University's Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of...

Eli Lilly has stepped into the gene therapy space after announcing a deal to acquire Prevail Therapeutics, a company focused on developing adeno-associated virus (AAV)-based gene therapies for neurodegenerative diseases.

Lilly will acquire Prevail for $22.50 per share in cash, plus one $4 contingent value right dependent on the first regulatory approval of a product from Prevails pipeline.

This reflects a potential consideration of up to $26.50 per share in cash for a total consideration of approximately $1.04bn.

For Lilly, the acquisition will extend its focus into developing gene therapies, establishing an in-house gene therapy programme anchored by Prevails current portfolio and AAV-based technology.

Prevails pipeline spans clinical-stage and preclinical neuroscience assets, including lead gene therapies PR001 for patients with Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) and PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

The companys preclinical pipeline also includes PR004, a potential gene therapy for patients with specific synucleinopathies, as well as candidates for Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders.

"The acquisition of Prevail will bring critical technology and highly skilled teams to complement our existing expertise at Lilly, as we build a new gene therapy programme anchored by well-researched assets, said Mark Mintun, vice president of pain and neurodegeneration research at Lilly.

We look forward to completing the proposed acquisition and working with Prevail to advance their ground-breaking work through clinical development, he added.

For Prevail to achieve the full value of the contingent CVR payment, the first regulatory approval arising from its current gene therapy pipeline must happen by 31 December 2024.

Failing regulatory approval by this date, Lilly said in a statement that the value of the CVR will decrease by approximately 8.3 cents per month until the expiration date 1 December 2028.

Within Prevails clinical pipeline, PR001 has already scored a US Food and Drug Administration (FDA) fast-track designation for the treatment of PD-GBA patients and nGD.

It has also been granted an FDA orphan drug designation for the treatment of Gaucher disease, and rare paediatric disease designation for the treatment of nGD.

Prevails PR006 gene therapy also has an FDA and European Commission orphan designation for the treatment of FTD, with the FDA also handing it a fast-track designation for FTD-GRN.

In November, Lilly signed a deal with Precision BioSciences focused on genome editing research, with an initial focus on developing in vivo therapies for Duchenne muscular dystrophy and two other undisclosed gene targets.

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Lilly picks up gene therapy programme in $1bn Prevail Therapeutics acquisition deal - PMLiVE

When former analyst Rachel McMinn started Neurogene from her apartment around three years ago, she would joke that theyd get office space as soon as her living room table was no longer big enough to hold company meetings.

We lasted about a year before my living room couldnt take it anymore, she said.

With several gene therapies for Batten disease and other lysosomal storage disorders in the preclinical and discovery stage, Neurogene is now bound for the clinic. And on Wednesday, they announced a $115 million Series B to get them there.

Gene therapy has generated so much enthusiasm for patients and families with these devastating disorders, but theres still a lot of science and innovation left on the table, McMinn said.

The CEO said Neurogene will split the Series B funds into four buckets, the first of which is advancing multiple gene therapy programs into the clinic. She anticipates filing the first IND in 2021 for CLN5, a rapidly progressive subtype of Batten disease caused by a variant in the CLN5 gene.

The second so-called bucket for the Series B funds will be expanding the companys portfolio, followed by another bucket for augmenting our resources for our novel technology platform, the CEO said. Then comes manufacturing.

Weve got the ability to make virus in-house, and the money from the financing will allow us to take that vector to the next stage and make GMP quality vector for use in dosing and clinical trials, McMinn said.

Because Neurogene manufactures products in-house, the biotech has gotten around the massive gene therapy manufacturing bottleneck, which has Big Pharma and big biotech spending billions on retrofitted plants and gene therapy factories.

The concept of gene therapy is simple: A viral particle is used to deliver a healthy copy of a gene to a patient with a dysfunctional gene. In the case of Neurogenes CLN5 candidate, viral vectors shuttle a payload into the body designed to make the CLN5 gene.

Over the next year, key milestones will be filing our first IND, completing the refurbishment of our GMP manufacturing facility, (and) advancing our programs towards the clinic, McMinn said. After CLN5, the goal is to file one to two INDs a year, she added.

The CEO previously served as an analyst at Piper Jaffray, Cowen and Bank of America Merrill Lynch, and as chief business and strategy officer at Intercept. During her time as an analyst, McMinn said most people would stay away from investing in neurology companies because drugs inevitably fail.

Theres really been nothing, very little innovation in devastating neurological disorders, for quite a long time, she said, adding that she was inspired to jump into R&D by an older brother who is neurologically impaired.

Neurogene attracted a slate of new and old investors, including EcoR1 Capital which led the round, and Redmile Group, Samsara BioCapital, Cormorant Asset Management, BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, Alexandria Venture Investments, and an undisclosed leading healthcare investment fund.

For me, I really want to make a difference, McMinn said, adding later, Im personally driven by developing something that is life-altering for people that really have no other option.

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After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News

Taysha Gene Therapies went public with a bang earlier this year with a $157 million IPO just five months after its Series A financing. Now, with the help of the city of Durham, North Carolina, Taysha is plotting an expansive development and manufacturing facility to ramp up supplies for the clinic and potentially the commercial market.

The new 187,000-square-foot cGMP plant will create roughly 200 jobs for Taysha Gene Therapies, which formed in late 2019 with the sole focus of developing therapeutics for monogenic diseases using an adeno-associate virus methodology. The announcement continues a rapid ascent for Taysha, which launched Series A financing just in April, and a mere five months later had its own Nasdaq ticker, offering a $157 million IPO and pricing shares at $20 apiece.

The facility is the result of a public-private partnership between Taysha, the city of Durham and the state of North Carolina. Taysha will invest $75 million into the facility, with additional state and local incentives totaling another $9.4 million. All told, the 200 jobs will come to fruition over a two-and-a-half-year period across all functions, including gene therapy development, analytics, manufacturing and quality control testing.

Tayshas facility is expected to be fully operational by 2023 and will add 2,000 liters of capacity supporting all aspects of scalable gene therapy manufacturing. The company said in a news release it will now be able to meet the clinical and commercial demands within the field when combined with its existing collaborations with the University of Texas Southwestern Medical Center and CDMO Catalent.

With our outstanding team of experts leading the charge, we expect this facility will serve as a center of excellence for gene therapy development, from preclinical studies through commercialization, and will further our leadership position in gene therapy as well as support our next phase of growth, RA Session II, Tayshas president, founder and CEO, said in a press release.

The new facility will serve as an integral part of rapid expansion for the Dallas-based biotech, which said it expects to have four open Investigational New Drug applications in 2021. Tayshas most advanced of those is a drug being developed for the treatment of GM2 gangliosidosis, a family of neurodegenerative disorders that includes Tay-Sachs disease and Sandhoff disease.

The other three such programs are looking to treat Rett Syndrome, epilepsy and SURF1 deficiencies.

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Taysha Gene Therapies looks to build on rapid IPO with $85M dev-manufacturing plant in North Carolina - Endpoints News

DUBLIN, Dec. 17, 2020 /PRNewswire/ -- The "Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global gene therapy market market.

Major players in the gene therapy market are Novartis AG, Bluebird Bio, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, Voyager Therapeutics, Applied Genetic Technologies Corporation, UniQure N.V., Celgene Corporation, Cellectis S.A. and Sangamo Therapeutics.

The global gene therapy market is expected to decline from $3.22 billion in 2019 to $3.18 billion in 2020 at a compound annual growth rate (CAGR) of -1.30%. The decline is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The market is then expected to recover and reach $6.84 billion in 2023 at a CAGR of 29.09%.

The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs. Gene therapy is used to replace faulty genes or add new genes to cure disease or improve the body's ability to fight disease. Only goods and services traded between entities or sold to end consumers are included.

North America was the largest region in the gene therapy market in 2019.

The gene therapy market covered in this report is segmented by gene type into antigen; cytokine; suicide gene; others. It is also segmented by vector into viral vector; non-viral vector; others, by application into oncological disorders; rare diseases; cardiovascular diseases; neurological disorders; infectious diseases; others, and by end users into hospitals; homecare; specialty clinics; others.

In December 2019, Roche, a Switzerland-based company, completed its acquisition of Spark Therapeutics for $4.3 billion. With this deal, Roche is expected to strengthen its presence in the gene therapy segment, support transformational therapies and increase its product portfolio. Spark Therapeutics is a US-based company involved in gene therapy.

The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for cancer and other diseases has influenced the health conditions of the population and has led to a low average life expectancy.

Luxturna, a one-time treatment for acquired retinal eye disease, costs $850,000 in the US and 613,410 in the UK, despite a markdown that is applied through Britain's National Health Service. Zolgensma, for spinal muscular atrophy, is valued at $2.1 million in the US and Zynteglo, which focuses on a rare genetic blood disorder, costs $1.78 million, thus restraining the growth of the market.

The use of machine learning and artificial intelligence is gradually gaining popularity in the gene therapy market. Artificial intelligence (AI) is the simulation of human intelligence in machines, which are programmed to display their natural intelligence. Machine learning is a part of AI.

Machine learning and AI help companies in the gene therapy market to conduct a detailed analysis of all relevant data, provide insights between tumor and immune cell interactions, and offer a more accurate evaluation of tissue samples often conflicted between different evaluators. For instance, since January 2020, GlaxoSmithKline, a pharmaceutical company, has been investing in AI to optimize gene therapy and develop off-the-shelf solutions for patients. It is also expected to reduce turnaround time and also the cost of gene therapies.

Key Topics Covered:

1. Executive Summary

2. Gene Therapy Market Characteristics

3. Gene Therapy Market Size And Growth 3.1. Global Gene Therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Gene Therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Gene Therapy Market Segmentation 4.1. Global Gene Therapy Market, Segmentation By Gene Type, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Gene Therapy Market, Segmentation By Vector, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.3. Global Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.4. Global Gene Therapy Market, Segmentation By End Users, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Gene Therapy Market Regional And Country Analysis 5.1. Global Gene Therapy Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Gene Therapy Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/fltbmv

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,...

NEW YORK and CLEVELAND, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that abstracts detailing new interim results from its ABO-102 Phase 1/2 Transpher A study for MPS IIIA and ABO-101 Phase 1/2 Transpher B study for MPS IIIB have been accepted for platform oral presentations during the late-breaking abstract session at the 17th Annual WORLDSymposium being held February 8-12, 2021.

Children born with MPS IIIA and MPS IIIB experience progressive neurodevelopmental decline and loss of motor function that is life-threatening, said Michael Amoroso, Chief Operating Officer of Abeona. We are excited to share new analyses from the Transpher A study that will add to the understanding of the potential for ABO-102 to help preserve neurocognitive development in patients with MPS IIIA when they are treated at a young age, and new results from the Transpher B study that will provide insights into ABO-101s biologic effect in patients with MPS IIIB.

Presentation Details

Title: Updated Results of Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)Abstract Number: 390Presenter: Kevin Flanigan, M.D., Center for Gene Therapy at Nationwide Childrens HospitalDate/Time: Friday, February 12, 2021, time to be determined

Title: Updated Results of Transpher B, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB)Abstract Number: 407Presenter: Maria Jose de Castro, M.D., Hospital Clnico Universitario Santiago de CompostelaDate/Time: Friday, February 12, 2021, time to be determined

About the Annual WORLDSymposium The WORLDSymposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. For additional information on the 17th Annual WORLDSymposium, please visit https://worldsymposia.org/.

About the Transpher A Study The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About the Transpher B Study The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-101 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About ABO-102 ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

About ABO-101 ABO-101 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

About Sanfilippo Syndrome Type A (MPS IIIA) Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Sanfilippo syndrome type B (MPS IIIB) Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeonas novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeonas fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit http://www.abeonatherapeutics.com.

Forward-Looking StatementsThis press release contains certain statements that are forward-looking 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, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Companys most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

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Abeona Therapeutics Announces Acceptance of Late-Breaker Abstracts Highlighting New Clinical Data for Novel AAV-based Gene Therapies in MPS IIIA and...

First gene therapy to receivefull EU marketing authorization for eligible MLD patients

One-time treatment with Libmeldy has been shown to preserve motor and cognitive function

Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele

BOSTON and LONDON and MILAN, Italy, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in theARSAgene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them, and most pass away before adolescence. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

Todays EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Libmeldy is Orchards first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.

With Libmeldy, a patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a self-inactivating (SIN) lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying disease with a single treatment.

The EC approval of Libmeldy comes more than a decade after the first patient was treated in clinical trials performed at our Institute, and ushers in a remarkable and long-awaited shift in the treatment landscape for eligible MLD patients, said Luigi Naldini, M.D, Ph.D., director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. Our team at SR-Tiget has been instrumental in advancing the discovery and early-stage research of this potentially transformative therapy to clinical trials in support of its registration through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele, and we are extremely proud of this achievement and what it means for patients and the field of HSC gene therapy.

MLD is a heart-breaking disease that causes immeasurable suffering and robs children of the chance of life, said Georgina Morton, chairperson of ArchAngel MLD Trust. As a community, we have been desperate for a treatment for young MLD patients, and we are incredibly excited to now have such a ground-breaking option approved in the EU.

The marketing authorization for Libmeldy is valid in all 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. Orchard is currently undertaking EU launch preparations related to commercial drug manufacturing, treatment site qualification and market access.

Data Supporting the Clinical and Safety Profile of Libmeldy

The marketing authorization for Libmeldy is supported by clinical studies in both pre- and early- symptomatic, early-onset MLD patients performed at the SR-Tiget. Early-onset MLD encompasses the disease variants often referred to as late infantile (LI) and early juvenile (EJ). Clinical efficacy was based on the integrated data analysis from 29 patients with early-onset MLD who were treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Clinical safety was evaluated in 35 patients with MLD (the 29 patients from the integrated efficacy analysis as well as six additional patients treated with the cryopreserved formulation of Libmeldy). Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in five out of 35 patients. Antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For further details, please see the Summary of Product Characteristics (SmPC).

About MLD and Libmeldy

MLD is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union for the treatment of MLD in eligible early-onset patients. In the U.S., OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) for any use. Libmeldy was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

About Fondazione Telethon, Ospedale San Raffaele and the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele, a clinical-research-university hospital established in 1971 to provide international-level specialized care for the most complex and difficult health conditions, and Fondazione Telethon, an Italian biomedical charity born in 1990 and focused on rare genetic diseases. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute hasgiven a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

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Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation:: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

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Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire