In the world of rare diseases, patient testimonies about the extreme difficulties of receiving an accurate diagnosis for an illness are numerous. For instance, one woman, sick for most of her young life, was not properly diagnosed with idiopathic gastroparesis an ultra-rare disease that affects stomach motility and digestion until late in college after seeing numerous different specialists in multiple fields and undergoing a battery of testing.1 Another patient, now active in the rare disease advocacy community, went undiagnosed with familial partial lipodystrophy a disease that, among other things, causes selective fatty tissue loss for 37 years.2

Unfortunately, these stories are not unique. One survey indicated that it took on average 7.6 years to properly diagnose a rare disease patient in the United States.3 Another study indicated that a rare disease patient on average consulted eight different physicians before landing on an accurate diagnosis, with only 12.9 percent of respondents indicating that they had seen only one physician prior to diagnosis (23.5 percent of respondents had seen between six and 10 physicians).4 Frequently, rare disease patients exhibit similar symptoms as other, more common diseases, making diagnosis complicated and leaving patients confused and frustrated about a path forward. Further complicating the situation is that traditional treatments for more common illnesses that mimic rare disease symptoms, such as irritable bowel syndrome in the case of the aforementioned gastroparesis patient, may actually worsen a patient's condition.

As such, the misdiagnosis of rare diseases, in addition to being traumatic for patients and their families, can be extremely expensive. One study indicated that over a 10-year period, an undiagnosed rare disease patient cost over 100 percent more than the average patient. This was due in part to a significant increase in outpatient visits compared with the average patient. (The cost differential was heightened in pediatric patients.)5 Such data indicates that shortening the path to diagnosis for rare disease patients may lead not only to increase patient health but also to a significant reduction in overall long-term healthcare costs.

According to the National Institutes of Health (NIH), there may be upward of 7,000 rare diseases in the United States affecting as many as 30 million people, or nearly one-tenth of the U.S. population.6 Alarmingly, only 5 percent of identified rare diseases have an approved treatment. Despite this daunting figure, approximately 80 percent of rare diseases have genetic origins, a common factor that points to genetic (the testing of individual variants or individual/multiple genes and their effects on an individual) and genomic (the study through various methods of an individual's entire genome and its interaction with the environment) testing as logical tools for identifying and ultimately combating these illnesses.

Genetic Testing Becoming More Common

From concept to execution, the Human Genome Project at the NIH took approximately 15 years and involved the creation of the National Center for Human Genome Research (now the National Human Genome Research Institute, an official Institute at NIH), the collaboration of hundreds of national and international scientists, and an approximate, inflation-adjusted total investment of $5 billion.7,8Since that time, the cost of performing genetic and genomic testing has declined significantly, with a per-genome cost of slightly less than $1,000 in 2019 compared with per-genome costs of approximately $95 million and $30,000 in 2001 and 2010, respectively.9 This significant cost reduction, which has been associated with the development of next-generation sequencing platforms and leaps in computer hardware development, among other things, has opened the door for patients to more readily access these important resources.

Most tests fall into overall categories of DNA diagnostic testing that include single-gene tests, which can detect an abnormality in a gene associated with a particular genetic illness; whole exome sequencing, which sequences the protein-encoding regions of genes; or whole genome sequencing, which is the most rigorous in that it involves sequencing the individual's entire genome. Given the sheer number of rare diseases and the size of the human genome, it is not surprising that there are numerous genetic tests on the market today. One study indicated that there are approximately 75,000 genetic tests on the market, or 10 issued every day.10

However, insurance coverage for these technologies is minimal and inconsistent despite recent positive reception for the increased use of enhanced technologies for patient treatment through the Precision Medicine Initiative, the NIH's Cancer Moonshot and similar programs. One study indicated that coverage for multigene testing varied drastically by disease type and that tests for broad indications or a large range of genes (i.e., those tests that may be helpful in narrowing down disease possibilities in a diagnostic profile) are frequently not covered by insurers.11 It should be noted that some progress has been made on national coverage determinations for some more widely recognized testing technologies. For instance, next-generation sequencing, a revolutionary sequencing technology that sequences genetic material multiple times simultaneously against a reference genome, received a reissued national coverage determination under the Medicare program from the Centers for Medicare & Medicaid Services (CMS) in October 2019.12 However, while this decision was significant as a model for future coverage for genetic testing services, it was only a minor first step in that it was limited only to previously untested patients with ovarian or breast cancer who are Medicare eligible.13

The large and complicated landscape of genetic testing is partially responsible for the lack of insurance coverage for these technologies. For instance, there are only about 200 standardized Current Procedural Terminology (CPT) codes to identify various types of genetic tests to insurers, other physicians, hospitals and health systems, limiting the ability for payers to systematically cover these technologies. This is especially true when applying "medical necessity criteria," which requires a provider to submit accurate information showing that a treatment or test is medically necessary to treat or diagnose a specific illness in order for it to be reimbursed by a payer. Data have shown that a majority of spending in the past several years on genetic tests has gone to noninvasive prenatal tests, cancer screening tests and multiple-gene analyses.14 This is unsurprising given that some of these technologies target pre-identified, validated markers and that newer screening methods present fewer risks for patients than other, more traditional or invasive testing methods.15 For many conditions, however, showing the medical necessity of genetic testing is still a complicated and unpredictable process when a patient is in the middle or beginning of his or her diagnostic odyssey.

Thus, coverage of new genetic testing technologies continues to remain a major challenge for the medical community and a mystery for the tens of millions of U.S. patients with rare diseases. Although small-scale studies and other evidence show that the use of genetic testing as a means to more quickly and accurately diagnose patients can reduce overall health expenditures, policymakers still lack systematic data showing the effectiveness of genetic testing as a means of cutting overall health spending at a macro level.

Help on the Horizon?

Bills have been introduced as recently as the 116th Congress that would create demonstration projects to test coverage of genetic testing technologies for certain patients to help inform future expansions of genetic testing coverage. In addition, Reps. Diana DeGette (D-Colo.) and Fred Upton (R-Mich.), the original champions of the 21st Century Cures Act,16 recently issued a request for information to help inform a follow-on version of the landmark legislation dubbed "Cures 2.0."17 One of the main focuses of their inquiry is into "how Medicare coding, coverage, and payment could better support patients' access to innovative therapies." Expanded coverage to increase access to genetic testing technologies could certainly fit within this scope and would help supplement expanded access and coverage of other new and innovative healthcare technologies for rare disease patients.

Stakeholders across the rare disease landscape have also shared consistent concerns with the length of time between when a new or breakthrough medical technology is approved and when it receives coverage by insurers. Underutilized programs may help speed new technologies to the patients that need them by shortening the gap between approval and coverage. One such example is the U.S. Food and Drug Administration (FDA)-CMS parallel review program for medical devices, which was recently touted by U.S. Department of Health and Human Services (HHS) Deputy Secretary Eric Hargan at the recent FDA/CMS Summit18 and through which a next-generation sequencing test received a parallel approval and coverage determination in 2017.19 These efforts may help the scientific community and others assemble data about how greater access to these technologies positively affect patient care, provide information necessary for lawmakers to empower CMS, the FDA and others to work together on increasing coverage and access, as well as to create mechanisms to speed new technologies to patients in need.

In addition to testing expansion of coverage and access for genetic and genomic testing, further investments should be made into public-private partnerships and other information gathering networks that may centralize information from a diverse group of medical professionals to provide patients additional resources for rare disease diagnosis. For instance, the Undiagnosed Diseases Network, housed at the NIH, utilizes a dozen sites nationwide where teams of physicians assess rare disease patients and share data, including genetic testing data through a "sequencing core," to maximize the amount of national expertise available to pin down rare disease diagnoses that would be extremely difficult and expensive to receive if patients sought expertise individually.20 In addition to further investment in these resources, continued policy development and investment in the development of artificial intelligence technologies and diagnostic support software tools, which have shown promise in assisting physicians in the early detection of rare disease through symptom analysis,21 will provide additional means for patients to receive care more quickly through largely noninvasive means.

Finally, payers both public and private may lack expertise in understanding and evaluating genetic tests, especially for rare diseases. Insurers should prioritize hiring individuals to supplement their teams who have some form of advanced knowledge not only of rare diseases but also the nature of genetic testing technologies and how they are used to expedite disease diagnoses. This is especially true given the rapid development of new testing systems and the growing use of other diagnostic technologies promoted in part by provisions in the 21st Century Cures Act and other legislation.

While it typically refers to something that is uncommon, the term "rare" can also imply heightened value. Greater investment in improving the diagnostic odyssey for rare disease patients, including through greater coverage of new technologies, can only enhance the value and efficiency of the U.S. healthcare system for all patients not just the few.

Link:
Sequence of Events: Genetic Testing Offers Significant Promise, But Coverage and Access Limited - Lexology

Recommendation and review posted by Bethany Smith

This years Clinical Trials on Alzheimers Disease meeting began in mid-December with a bang and ended a few days later with hallway conversations laced with worry. The topic, in both cases, was aducanumab, an experimental drug for treating people with Alzheimers disease.

The meeting got off to celebratory start as a top Biogen scientist presented results showing that the highest dose of aducanumab may benefit people with mild cognitive impairment (MCI) and elevated amounts of a protein called amyloid in the brain. That presentation represented an about-face for the company, which had pulled the plug on two trials of the drug in March.

Yet even the most enthusiastic interpreters of the drugs effects on measures of cognition and function agreed that the benefit to patients was a mild slowing, not a halt, and it was certainly not a cure for Alzheimers disease.

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But we also learned that as aducanumab clears amyloid from the brain, it can cause both microscopic hemorrhages and swelling in the brain, particularly in individuals who have a heightened genetic risk of developing Alzheimers disease dementia.

With these facts in hand, aducanumab becomes a kind of thought experiment. What if we could treat mild cognitive impairment caused by Alzheimers disease with a somewhat effective but costly and risky drug? The answers are discomforting.

For much of the 20th century, America largely ignored dementia. It was widely believed that its most common cause was senility, an extreme stage of aging. That changed in April 1976 with a 1,200-word essay titled The Prevalence and Malignancy of Alzheimer Disease: A Major Killer in the Archives of Neurology. In it, neurologist Robert Katzman argued that older adults with disabling cognitive and behavioral problems did not have senility but had Alzheimers disease, a medical problem in need of the full force of American medicine to diagnose, treat, and ideally prevent.

Nearly half a century later, America hasnt come close to solving the problem of Alzheimers disease and other causes of dementia: We dont have effective treatments and we also dont have an effective physician workforce to prescribe and administer them.

Parsing out age-related cognitive complaints from mild cognitive impairment and explaining that diagnosis is a challenging task. There arent currently enough clinicians skilled to evaluate the millions of older adults with cognitive complaints, care for those with MCI and dementia, and prescribe a costly drug that slows but does not cure Alzheimers disease and poses risks to the very same brain they are trying to treat.

Imagine that the FDA approves aducanumab, or a drug like it. Individuals with mild memory problems who dont have MCI should be sent home with reassurance or with treatments for the problems causing their memory complaints, such as anxiety, too much alcohol, or poor sleep. Those with MCI thats about 15% of older Americans would be candidates for PET scans to measure the amount of amyloid in the brain.

To evaluate the millions of Americans who see a doctor because my memory isnt as good as it used to be, overworked and underskilled clinicians are likely to take shortcuts: Never mind diagnosing mild cognitive impairment. Just order the amyloid test. If its positive, prescribe the drug. Otherwise, dont prescribe it.

That approach will be costly. A PET scan for brain amyloid costs around $4,000. Less-costly spinal fluid tests could substitute, but few clinicians are skilled at performing them. Aducanumab, as a manufactured and injected monoclonal antibody, will be expensive. The risk of small swellings and bleeds in the brain would require MRIs to assess safety, which would increase the need for clinicians skilled in interpreting the scans and adjusting treatment plans.

A drug like aducanumab presents clinicians with other novel challenges. It is one of several drugs whose risks, and possibly its benefits as well, are associated with having the ApoE4 gene a gene known to increase an individuals lifetime risk of developing Alzheimers disease dementia. The decision to start the drug may well include ApoE testing so individuals can better understand their risks and possibly responses to the drug.

Genetic testing means that clinicians will have to practice genetic counseling at visits that may need to expand from the dyad of patient and caregiver to include an extended and worried family. A prescription for aducanumab would be startling news for a patients siblings, adult children, and grandchildren: You too may have the Alzheimers gene. You too may want to have an amyloid test.

A treatment that slows Alzheimers disease, that delays the onset of dementia, promises to reduce disability and preserve autonomy. The failure to properly prescribe it could, however, increase the spectacular tallies of the time and costs of caregiving that define much of the Alzheimers crisis.

Lets assume that additional studies show that aducanumab does indeed slow the progression of Alzheimers disease with benefits that exceed its risks. Some of those who take the drug will die of other causes, such as heart disease or cancer, before dementia takes hold. But others will, in time, experience more and more disabling cognitive impairments. As they do, theyll need care.

Some will be cared for in nursing homes or facilities devoted to dementia care. Most will be cared for at home. The Alzheimers Association estimates that in 2018, 16.3 million family members and friends provided 18.5 billion hours of unpaid care to people with Alzheimers and other dementias.

This care ought to include education and training for patients and caregivers. It should also include activity programs tailored to patients abilities and disabilities. These include memory cafs, where people come together not as patients but persons, and centers whose staff members are skilled at creating days that are safe, social, and engaging, with activities such as reminiscence, music, theater, art, and exercise.

Although these ought to be the standard of care, few of them are routinely available to caregivers and patients. Doctors dont typically prescribe them, and their costs are mostly paid out of pocket. A 2013 report estimated that these out-of-pocket costs, together with the time caregivers devote to care, make up as much as half of the diseases annual $200 billion-plus cost.

A disease-slowing treatment that reduces disability ought to reduce the time spent on caregiving. But it will not allow the U.S. to ignore its fractured and disorganized system of dementia care and how this nonsystem offloads much of the costs onto patients and families. Medicare, which was created in 1965, does not pay for long-term care. We must update this antiquated law and support long-term care.

The ability to control Alzheimers disease with a drug will also demand that we engage with difficult issues regarding life and death. Disease-slowing treatments for Alzheimers will challenge our criteria for access to hospice care, as well as to physician aid in dying. Individuals with a chronic and progressive disease like Alzheimers may, in time, decide they no longer want treatment. A robust ethic of respect for persons supports their right to stop treatment. It is entirely possible that some patients, as they decline, may decide: Enough. This disease has progressed. I want to stop treatment.

After that decision or if the drug doesnt work what kind of palliative care is available when death is not in six months away but may be six years away, or longer? Medicares hospice benefit is available only to individuals with six months or fewer to live.

Physician aid in dying, which is available to residents of nine states and the District of Columbia, is also not an option. Individuals who choose this route must have a prognosis of living six months or fewer, be able to decide to end their life, and be able to take the lethal dose of medication.

We ought to be deeply concerned that the limited access to care and its cost are not perverse incentives to seek aid in dying.

We should also expect that the more we control the natural history of Alzheimers disease, the more well begin to question when were dying of it and how we should die.

Katzman foreshadowed this in closing his 1976 essay: In focusing attention on the mortality associated with Alzheimer disease, our goal is not to prolong the lives of severely demented persons, but rather to call attention to a disease whose etiology must be determined, whose course must be aborted, and ultimately a disease to be prevented.

In 2012, the National Plan to Address Alzheimers Disease premiered a strategy to achieve Katzmans vision. Goal number one was that by 2025 we will prevent and effectively treat the disease. Research on aducanumab and other drugs in the pipeline that target amyloid and other causes of neurodegeneration is one route to achieving this. Equally important is disseminating strategies that promote brain health exercise, education, smoking cessation, and the like that have been decreasing the risk of developing dementia since the 1970s.

We do this research with hope that drug interventions will help address the economic and moral costs that have transformed Alzheimers from Katzmans common disease into a crisis. At the same time, we must be mindful that these interventions will present new economic and moral costs. If we fail to address them, the crisis will endure.

Jason Karlawish, M.D., is co-director of the Penn Memory Center and a site investigator for clinical trials sponsored jointly by the National Institute on Aging and Novartis (Generations program) and the NIA and Eli Lilly (the A4 Study). You can follow him on twitter @jasonkarlawish.

Excerpt from:
Aducanumab isn't the simple solution to the Alzheimer's crisis - STAT - STAT

Recommendation and review posted by Bethany Smith

SPRINGFIELD As people throughout Illinois prepare to ring in the New Year, the Senate Republican Caucus is continuing to highlight new laws that will take effect on Jan. 1.

The new laws kicking off 2020 cover everything from protecting children under DCFS care to safeguarding individuals privacy and personal information.

New DCFS requirements seek to better protect children

The Department of Children and Family Services spent much of 2019 under scrutiny because of its mishandling of the abuse of a 5-year-old child, who was later beaten to death by his parents.

This tragic event, along with other mishandled issues inside the department, led the General Assembly to take a more in-depth look into the agency and pass several new requirements to better protect the children under DCFS care.

Beginning Jan. 1, DCFS will have to meet the following requirements:

House Bill 831/PA 101-0043: DCFS must notify the Department of Public Health and the Department of Healthcare and Family Services of all reports involving children alleged to have been abused or neglected while hospitalized.

House Bill 3587/PA 101-0155: DCFS must establish and maintain a toll-free number to respond to requests from the public about its post-placement and post-adoption support services.

Senate Bill 1743/PA 101-0166: DCFS must develop and conduct a standardized survey to gather feedback from children who are aging out or have transitioned out of the foster care system, and place a locked suggestion box in each group home and shelter.

House Bill 1551/PA 101-0237: DCFS must comply with several new guidelines when a child in its custody is returned to their parents or guardian.

Senate Bill 1239/ PA 101-0583: DCFS must report alleged abuse or neglect of a child by a person who is not the childs parent, a member of the childs immediate family, a person responsible for the childs welfare, an individual residing in the same home of the child or a paramour of the childs parent to the appropriate local enforcement agency.

Baby changing stations required in all public buildings

Finding a location to change your little ones diaper will be a lot easier for parents starting in 2020. House Bill 3711 requires all public buildings with restrooms open to the public to have at least one baby diaper changing station accessible to both men and women. Additionally, signs must be posted near the entrance indicating the location of the diaper changing station.

New laws protect pets

Laws protecting pets are among those that will take effect Jan. 1.

House Bill 3390 requires pet boarding facilities that do not have 24/7 staffing to be equipped with a fire sprinkler system or a fire alarm monitoring system that triggers notification to local emergency responders. This new law is in response to a fire at a West Chicago kennel that killed several animals.

Also, at the start of the New Year, cat owners will be required to have their cats vaccinated for rabies. All cats four months or older, excluding feral cats, must receive a rabies vaccination and have a subsequent vaccination within a year of the first one occurring.

New laws ensure privacy, protect personal information

Protecting individuals personal information is the intent behind several new laws taking effect Jan. 1.

House Bill 2189 prohibits direct-to-consumer commercial genetic testing companies from sharing any genetic test information or other personally identifiable information about a consumer with any health or life insurance company without written consent from the consumer.

Under Senate Bill 1624, data breaches impacting more than 500 Illinois residents as a result of a single breach must be reported in the most expedient time possible to the Attorney General.

House Bill 2408 prohibits a person from posting private compromising images of another person online. It also provides for a process for a person to obtain a take-down order to have the images removed.

The full list of January 1 new laws

Theres more to know before 2020 kicks off! To view a full list of laws taking effect January 1, visit https://bit.ly/2EfHMVQ.

Warmest wishes to you and yours for a very Merry Christmas.

If you have any additional thoughts or ideas, please visit my website at http://www.senatorstewart.com and use the form to send me an e-mail.

Read this article:
From the Capitol: Heres part 2 of the new laws for Illinois - Freeport Journal-Standard

Recommendation and review posted by Bethany Smith

The 5,000-year-old skeleton, designated M53, with physical dysplasia. Image: S. E. Halcrow et al., 2019

The discovery of a 5,000-year-old skeleton of a young adult who had serious physical limitations suggests a form of social support existed in Neolithic China, but only to a point, as this individual may have met an untimely death.

The skeleton was found at the Guanjia site in Chinas Henan Province, and it dates back to the late Neolithic Yangshao cultural period (33002900 BCE). A recent analysis of this skeleton has revealed an array of physical and possibly cognitive health issues that would have required others to take care of this individual, according to new research published in the International Journal of Paleopathology.

Designated M53, this person had normal body proportions but was far shorter than typical, with a slender build. The researchers, a collaborative team from Queens College in New York, the University of Otago in New Zealand, and other institutions, were unable to determine the individuals sex, but the person died after reaching adulthood. M53 was buried according to normal Yangshao customs, but their hands were positioned behind their back, for reasons that arent clear. Analysis of the skeleton also revealed neck fractures, which likely contributed to M53's deathinjuries that were possibly inflicted intentionally.

Importantly, M53 had bones that were very thin and hollow, similar to severe osteoporosis, and they likely had very weak muscles, as evidenced by specific skeletal muscle markings. Taken together, this suggests M53 had physical dysplasia, leading to a kind of dwarfism, an underdeveloped body, and a feebleness. This individual wouldve been very physically limited and possibly even paralyzed. Whats more, if M53 was affected by severe hormonal problemswhich appears to be the casethey may have also experienced cognitive delays and mood disorders, according to the new research.

Ekaterina Pechenkina, a co-author of the study and an anthropologist at Queens College, said these anomalous traits may have been caused by a reduced function of the pituitary gland, a critical gland that regulates our growth and general well-being, she explained in an email to Gizmodo. Among other things, it produces the growth hormone. Indeed, her teams analysis of the skull revealed depressions in a bony structure that hosts the pituitary glanda sign that a tumor had caused the gland to stop working properly.

Pechenkina said all these skeletal manifestations suggest hypopituitarism as a diagnosis, a rare condition in which the pituitary gland cant produce sufficient hormones, leading to a host of problems, including muscle weakness. Consequently, this rare find is offering new insights into the cultural norms of the period and how individuals with noticeable physical differences or disabilities were treated.

We know that M53 was a tiny individual with very slender, fragile bones and underdeveloped muscles, who despite their limited physical abilities survived into adulthood, said Pechenkina.

Survival in this 5,000-year-old northern Chinese farming community wouldve required considerable physical strength, Pechenkina explained. Chopping wood to build fires during the cold winters wouldve been an essential skill, she said. To acquire food, people needed to turn soil, plant seeds, water crops, and so on.

I think that a slender and a fragile individual, such as M53, needed lots of support and help from their family members, said Pechenkina. It is unlikely that they were ostracized by their community, she said, as there was no evidence of abuse or signs of old fractures. And because M53 was buried in a (reasonably) typical way, they were regarded as one of their own, she said.

The exact model of care, in the words of the researchers, is unknown, but Sian Halcrow, a co-author of the study and a researcher at the University of Otago, said it very likely existed in some form.

Thats because the dysplasia likely had some associated health effects from an early age, and that would have meant M53 would have had extra care needs, said Halcrow in a press release. But they lived into adulthood so its likely they were the recipient of care from other members in their family or wider society.

That said, Pechenkina and her colleagues believe M53 likely died from the aforementioned neck fracture.

Their neck vertebrae appear to be fractured around the time of death, which makes it likely that eventually they were killed by their group members, either because they became to be seen as a burden or because their health deteriorated or, perhaps, because their caretakers became too old or died, said Pechenkina. For me, an interesting question is whether our M53 individual was recognized as different from an early childhood or only after a certain age when their short stature and fragility became apparent, she said, adding that, I suspect that up until a certain age they were likely cared for as a child.

The troublesome circumstances of M53s death notwithstanding, their survival into adulthood would have required lots of time, patience, and resourcesand also a certain degree of compassion and acceptance. Yes, the Neolithic was tough, but it was clearly far from barbaric.

No doubt, this discovery shows one way in which Neolithic culture was socially advanced, but we shouldnt be too surprised (evidence from earlier this year revealed that Neolithic European babies drank animal milk from sippy cups, as just one example). Evidence of social support goes back even further in timeas far back as the Neanderthals and other archaic humans, who cared for severely physically disabled members of their communities. Prosocial behaviors such as these, it might be argued, contributed greatly to the success of our species.

Originally posted here:
Rare Skeleton Points to Compassion, Care, and Tragedy in Prehistoric China - Gizmodo

Recommendation and review posted by Bethany Smith

For its promising investigational therapeutic approach to neurodegenerative diseases, including progressive multiple sclerosis (MS), BrainStorm Cell Therapeutics is theBuzz of BIO 2020 winnerin the Public Therapeutic Biotech category.

The Buzz of BIO contest identifies U.S. companies with groundbreaking, early-stage potential to improve lives. The event also is anopportunity to make investor connections that could take products to the next phase.

Ten biotechnology companies are nominated in each of the three categories of Buzz of BIO: Public Therapeutic Biotech, Private Therapeutic Biotech, and Diagnostics and Beyond. In the Public Therapeutic Biotech category that BrainStorm won, nominated companies must be actively developing a publicly traded human treatment intended for review by theU.S. Food and Drug Administration.

As a developer of autologous cellular therapies for debilitating neurodegenerative diseases, BrainStorm is testing its investigational therapy,NurOwn, in progressive MS patients, for whom treatment options are limited.

The therapy is based on patients own bone marrow-derived mesenchymal stem cells that are engineered to secrete growth factors. Such factors are thought to protect nerves from damage, promote the repair of myelin (the protective coat of neurons that is destroyed in MS), and ultimately slow or stabilize disease progression.

BrainStorms current open-label Phase 2 clinical study (NCT03799718) is enrolling up to 20 adults with either secondary progressive or primary progressive MS at three U.S. sites:theKeck School of Medicine of USC, the Stanford School of Medicine, and theCleveland Clinic. After undergoing a bone marrow aspiration to collect cells, each participant will receive three intrathecal (injected into the spinal cord) NurOwn cell transplants within 16 weeks, and will be tracked for at least another 12 weeks to assess safety and effectiveness. Contact information for the trial centers is available here.

Thanks to everyone who voted for BrainStorm during the Buzz of BIO competition,Chaim Lebovits, BrainStorm president and CEO, said in a press release.

As the winner of the contest, BrainStorm also was invited to givea presentation at theBio CEO & Investor Conference, to be held Feb. 1011 in New York City.

The entire management team at BrainStorm was very pleased with the results of this competition, and we look forward to presenting to an audience of accredited investors who may benefit from the companys story, said Lebovits. We thank the BIO[Biotechnology Innovation Organization] team for singling out BrainStorms NurOwn as a key technology with the potential to improve lives.

NurOwn cells also are being tested in a Phase 3 trial (NCT03280056) in patients with amyotrophic lateral sclerosis (ALS).

Total Posts: 1,053

Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

More:
BrainStorm Cell Therapeutics Wins 2020 'Buzz of BIO' Award - Multiple Sclerosis News Today

Recommendation and review posted by Bethany Smith

SEATTLE--(BUSINESS WIRE)--According to Coherent Market Insights, the global bone graft and substitutes market is estimated to be valued at US$ 3,046.7 million in 2019, and is expected to exhibit a CAGR of 5.5% during the forecast period (2019-2027).

Key Trends and Analysis of the Global Bone Graft and Substitutes Market:

Key players in the market are focusing on receiving product approvals for bone grafts and bone graft substitutes are expected to fuel growth of the global bone graft and substitutes market over the forecast period. For instance, in December 2016, CoreBone, an Israel-based company manufacturing bone grafts from corals received approval from European Medicines Agency (EMA) for use this product in Europe, which is expected to generate a significant revenue for the region through its sales.

Request your Sample PDF copy @ https://www.coherentmarketinsights.com/insight/request-sample/1050

Moreover, adoption of inorganic growth strategies by major key players is expected to boost the market growth over the forecast period. For instance, in February 2019, Orthofix Medical acquired Options Medical, LLC, a medical device distributor based in Florida, U.S. The acquisition aims to enhance the sales force. Moreover, in 2017, LifeNet Health acquired Austria-based tissue bank- AlloTiss Gemeinntzige Gewebebank GmbH. The acquisition enabled LifeNet Health to establish training and distribution center in Vienna, Austria to support the use of allograft in Europe.

Furthermore, rising road accidents and injuries is expected to propel the market growth over the forecast period. For instance, in 2018, as per the World Health Organization (WHO) estimation, around 1.35 million people are reported to die each year due to road traffic crashes, globally.

Among regions, Asia Pacific is expected to show significant growth in the global bone graft and substitutes market, owing to increasing road accidents in the region. For instance, according to the World Health Organization report in 2013, Asia Pacific region reported around 153,000 fatalities due to road accidents or around 400 deaths each day. It also stated that road traffic death rate in South-East Asia region was 17.0 per 100,000 population, compared to the global rate of 17.4, where there was considerable variation seen within the region ranging from 3.5 in the Maldives to 36.2 in Thailand in 2015.

Key Market Takeaways:

Buy this Report (Single User License) @ https://www.coherentmarketinsights.com/insight/buy-now/1050

Market Segmentation:

Read more:
Global Bone Graft and Substitutes Market to Surpass US$ 4673.5 Million by 2027 Coherent Market Insights - Business Wire

Recommendation and review posted by Bethany Smith

Friday, December 20, 2019 2:40 p.m. CST

By Michael Erman

NEW YORK (Reuters) - Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease.

The company said on Friday that it will be open to refining the process in the future, but it is not making any changes at this time. The program is for patients in countries where the medicine, called Zolgensma, is not yet approved for the rare genetic disorder, which can lead to death and profound physical disabilities.

At $2.1 million per patient, Zolgensma is the world's costliest single-dose treatment.

Novartis said the program will open for submission on Jan. 2 and the first allocation of drugs would begin in February. Novartis's AveXis unit, which developed the drug, will give out 50 doses of the treatment through June for babies under 2 years old, it said on Thursday, with up to 100 total doses to be distributed through 2020.

Patient advocacy group SMA Europe had a conference call with the company on Friday, according to Kacper Rucinski, a board member of the patient and research group who was on the call.

"There are a lot of ethical questions, a lot of design questions that need to be addresses. We will be trying to address them in January," Rucinski said. He said the program has no method of prioritizing who needs the treatment most, calling it a "Russian roulette."

The company said it developed the plan with the help of bioethicists with an eye toward fairness.

"This may feel like youre blindly passing it out, but it may be the best we can do," said Alan Regenberg, who is on the faculty at Johns Hopkins' Berman Institute of Bioethics and was not among the bioethicists Novartis consulted with on the decision. "It may be impossible to separate people on the basis of prognosis out of the pool of kids under 2," he said.

According to Rucinski, the parties will continue their discussion in January "to see what can be improved in the design" of the program.

Novartis said on Thursday that because of manufacturing constraints it is focused on providing treatment to countries where the medicine is approved or pending approval. It has one licensed U.S. facility, with two plants due to come on line in 2020.

Zolgensma, hit by turmoil including data manipulation allegations and suspension of a trial over safety concerns, is the second SMA treatment, after Biogen's Spinraza.

Not all of the SMA community are opposed to Novartis' program.

Rajdeep Patgiri moved from the United Kingdom to the United States in April so his daughter could receive Zolgensma. She has responded well to the treatment, and Patgiri worries that negative attention to the program could keep patients from receiving the drug.

"The best outcome for all patients would be if everybody could get the treatment. Given all the constraints, a lottery is probably the fairest way to determine who" receives the treatment, he said.

(Reporting by Michael Erman; Additional reporting by John Miller in Zurich; Editing by Leslie Adler)

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Novartis in talks with patients upset about lottery-like gene therapy giveaway - KFGO News

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Gene therapy is a way of delivering healthy genes or genetic material to cells in order to treat genetic disorders. The most common way to do this is using adeno-associated viruses (AAVs). The outer part of the virus, called the capsid, is generally retained, but the viral genes are replaced with the therapeutic genes. Attempts have been made to improve the capsid or shell of the virus, but usually fail. George Church and his team at Harvard Medical School with the original researchers at the Karolinska Institute and Lund University in Sweden, have developed a technique to modify the capsid. They have also launched a company, Dyno Therapeutics, to develop the approach.

The groups research, by senior author Tomas Bjrklund, with Lund, was published in PNAS, the Proceedings of the National Academy of Sciences of the United States of America.

The technique allows the researchers to engineer the virus shell to deliver the gene package to the exact cell type in the body they intend to treat. The process leverages computer simulations and modeling with gene and sequencing technology.

Thanks to this technology, we can study millions of new virus variants in cell culture and animal models simultaneously, Bjorklund said. From this, we can subsequently create a computer simulation that constructs the most suitable virus shell for the chosen applicationin this case, the dopamine-producing nerve cells for the treatment of Parkinsons disease.

The technique also dramatically decreases the need for laboratory animals. The millions of variations on the same therapy can be studied in the same individual.

The authors wrote, A challenge with the available synthetic viruses used for the treatment of genetic disorders is that they originate from wild-type viruses. These viruses benefit form infecting as many cells as possible in the body, while therapies should most often target a particular cell type, for example, dopamine neurons in the brain.

Current approaches to finding the most advantageous viruses for gene therapy use random screening, enrichment and, the authors say, serendipity. Their technique is dubbed BRAVE (barcoded rational AAV vector evolution). In BRAVE, each virus displays a peptide derived from a protein. That peptide as a known function on the AAV shell surface and what they call a unique molecular barcode in the packaged genome.

By sequencing the RNA-expressed barcodes, they can map the binding sequences from hundreds of proteins simultaneously. They liken the technique to accelerating evolution from millions of years to just weeks.

Bjorklund said The reason we can do this is that we study each generation of the virus in parallel with all the others in the same nerve cells. Unlike evolution, where only the best suited live on to the next generation, we can also learn what makes the virus work less well through this process. This is crucial when building computer models that interpret all the information.

The study showed the potential for using machine learning for AAV design, although the research fell short of actually designing an improved AAV that could be used in clinical testing. Thats where Dyno Therapeutics comes in, working to improve and develop the technique.

Luk Vandenberghe, director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear, told C&EN, Chemical & Engineering News, What theyve done here is truly a remarkable tour de force.

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Dyno Therapeutics Launches to Improve Viral Vectors for Gene Therapy - BioSpace

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Though the research was intended as a proof of concept, the experimental gene therapy slowed tumour growth and prolonged survival in mice with gliomas, which constitute about 80% of malignant brain tumours in humans.

The technique takes advantage of exosomes, fluid-filled sacs that cells release as a way to communicate with other cells.

The research was carried out by scientists at the Ohio State University and published in the journal Nature Biomedical Engineering.

While exosomes are gaining ground as biologically friendly carriers of therapeutic materials because there are a lot of them and they dont prompt an immune response the trick with gene therapy is finding a way to fit those comparatively large genetic instructions inside their tiny bodies on a scale that will have a therapeutic effect.

This new method relies on patented technology that prompts donated human cells such as adult stem cells to spit out millions of exosomes that, after being collected and purified, function as nanocarriers containing a drug.

When they are injected into the bloodstream, they know exactly where in the body to find their target even if its in the brain.

Senior study author L. James Lee, professor emeritus of chemical and biomolecular engineering at Ohio State University, said: Think of them like Christmas gifts: the gift is inside a wrapped container that is postage paid and ready to go. This is a Mother Nature-induced therapeutic nanoparticle.

In 2017, Lee and colleagues made waves with news of a regenerative medicine discovery called tissue nanotransfection (TNT). The technique uses a nanotechnology-based chip to deliver biological cargo directly into skin, an action that converts adult cells into any cell type of interest for treatment within a patients own body.

By looking further into the mechanism behind TNTs success, scientists in Lees lab discovered that exosomes were the secret to delivering regenerative goods to tissue far below the skins surface.

The scientists placed about one million donated cells on a nano-engineered silicon wafer and used an electrical stimulus to inject synthetic DNA into the donor cells. As a result of this DNA force-feeding, as Lee described it, the cells need to eject unwanted material as part of DNA transcribed messenger RNA and repair holes that have been poked in their membranes.

The electrical stimulation had a bonus effect of a thousand-fold increase of therapeutic genes in a large number of exosomes released by the cells, a sign that the technology is scalable to produce enough nanoparticles for use in humans.

Essential to any gene therapy is knowing what genes need to be delivered to fix a medical problem. For this work, the researchers chose to test the results on glioma brain tumours by delivering a gene called PTEN, a cancer-suppressor gene. Mutations of PTEN that turn off that suppression role can allow cancer cells to grow unchecked.

For Lee, founder of Ohio States Center for Affordable Nanoengineering of Polymeric Biomedical Devices, producing the gene is the easy part. The synthetic DNA force-fed to donor cells is copied into a new molecule consisting of messenger RNA, which contains the instructions needed to produce a specific protein. Each exosome bubble containing messenger RNA is transformed into a nanoparticle ready for transport, with no blood-brain barrier to worry about.

The testing in mice showed the labelled exosomes were far more likely to travel to the brain tumours and slow their growth compared to substances used as controls.

Because of exosomes safe access to the brain, Lee said, this drug-delivery system has promise for future applications in neurological diseases such as Alzheimers and Parkinsons disease.

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Mother Nature provides new gene therapy strategy to reverse disease - Health Europa

Recommendation and review posted by Bethany Smith

Inside each of our cells is a genetic hourglass. Every time our cells dividewhich they have to do to keep us alivetheir 23 pairs of chromosomes remain nearly identical. Except for one intentional change: After each division, a cells chromosomes get a little bit shorter.

Ten years ago, a group of scientists won the Nobel Prize in medicine for discovering these ever-shortening DNA sequences at the end of our chromosomes, called telomeres. After a cell has divided a certain number of timesknown as the Hayflick limitits telomeres are so short that the cell knows its time to peacefully shut itself down. When enough cells die off, organs wear out, and eventually, we die, too.

This discovery ushered in decades of aspirational research that set out to understand the role of telomeresand the protein that can rebuild them, called telomerasein aging. Perhaps, if scientists could figure out how to flip our biological hourglasses over, our cells could replicate for longer. Our organs would tire more slowly, and we could delay death.

The Nobel-winning research began way back in the 1970s with the work of biologist Elizabeth Blackburn. But even after four decades, its still not clear if telomeres can safely be manipulated to thwart aging.

That hasnt stopped some scientists from betting on artificially extending telomeres to support longevity: Just last week, Kansas-based biotech startup Libella Gene Therapeutics announced that it would begin early clinical trials testing out a gene therapy that could lengthen telomeres, according to OneZero.

That approach, which as of yet has only been tested in mice, is indicative of humans deep desire to roll back the clock. But the deeper scientists go into the field, the more complicated the story behind telomeres gets: Theres evidence that they may play an important role in other aspects of our health, and that cell division may not be the only reason they shrink over time. Before scientists can try to safely harness telomeres to improve our health, theyll have to answer these questions.

One anti-aging strategy that researchers have investigated involves telomerase, the telomere-building protein that Blackburns colleague Carol Greider discovered on Christmas Day in 1984.

Telomerase is an important tool for cells that divide frequentlylike blood cells, the lining of our digestive systems, or sperm and egg cells. These cells regenerate so often that they need an enzyme to regularly rebuild the caps on the end of their chromosomes.

All the other kinds of cells in our bodies shouldnt have telomerase. But if they did, theoretically, their telomeres would never shrink. They could keep dividing beyond their normal Hayflick limit.

Theres one big problem, though: Cells that have telomerase but arent supposed to often wind up to be cancerous.

In approximately 90% to 95% of cancers, during the process of oncogenesis, telomerase is reactivated, says Masood Shammas, a lead scientist at the Dana Farber Cancer Institute in Boston. As cancer cells spread, theyre able to build their telomeres back upallowing them to keep dividing and dividing and dividing.

This means that messing with telomerase to somehow extend lifeas Libella is attempting to, by injecting patients with a virus containing the gene that codes for telomeraseis risky business.

On the other hand, it also means that blocking telomerase could be a way to treat cancer. Shammas has worked on clinical trials that have tested telomerase-targeting drugs with a company called Geron. Although their original drug worked in mouse models, it failed in early-stage clinical trials for people, because it had some nasty side effects. As a result, scientists have had to put stopping telomerase on hold until they can figure out how to make it only work in cancer cells.

An alternative strategy focuses not on rebuilding telomeres, but slowing their shrinkage in the first place. Scientists are trying to understand what, in addition to normal cell division, causes telomeres to contract. Maybe limiting these activities could decelerate aging in a way that doesnt accidentally reactivate a cancer pathway.

The activities that can slow telomere degradation are still being researched. It seems, though, that theres a lot of daily living that may play a role in telomere length. Anything that damages DNA will damage telomeres, says Shammas.

Telomeres are particularly vulnerable because theyre more exposed on the ends of the chromosomes. Smoking, drinking, and eating red meats fried in oilswhich all produce molecules that can bind to and distort DNAmay harm your telomeres, too. They also happen to all be known carcinogens.

Of course, this doesnt mean their effects are felt immediately, or that these activities will definitely lead to telomere shortening or cancer. Its their cumulative effect over a lifetime, plus other factors that scientists havent nailed down yet, that we need to watch out for. And clinicians generally advise against these activities anyway.

Perhaps more surprisingly, a life-affirming action may also cause telomeres to shrink: Pregnancy.

Dan Eisenberg, a biological anthropologist at the University of Washington, has studied how telomeres behave over time for people who become pregnant. A large cohort study he and his team published last year looked at women in the Philippines. After controlling for age, they found that the more times someone had been pregnant, the shorter their telomeres were. Each pregnancy seemed to shorten a persons telomeres by the equivalent of as many as four years of life.

This could be because of how taxing pregnancy can be on the body. Developing a fetus takes about twice the energy a person normally uses. Theres less energy available to maintain and repair cells for the long-term, Eisenberg says.

While it seems counterintuitive that evolution would penalize a person for reproducing, it may be a necessary trade-off. Perhaps the benefit of spreading new genes into the world is worth the cost of slightly shorter telomeres, Eisenberg explained. After all, evolution doesnt affect the processes that happen to us after we after our reproductive years. Weve already achieved the goal of immortality by way of our progeny.

So, lifestyle modifications to prevent telomere shortening dont sound too appealing. And so far, the only activity that researchers have found that can naturally extend telomeres in the slightest may be exercise. The only thing that world show that can activate telomerase activity is regular exercise, says Shammas. But its still not clear why this is the case, and it certainly doesnt mean that hitting the gym can stave off all aging.

Which brings us back to the promises made by companies like Libella, the gene therapy outfit currently promoting a telomere therapy. With four decades of telomere research yet to produce better guidance than cut down on red meat and exercise more, its easy to appeal to the insecurities and fears of the aging population with less-than-fully-baked treatments.

As OneZero reported, Libellas study is slated to begin early next year in Colombia. Likely, its running there to skirt the US Food and Drug Administrations (FDA) requirement for an Institutional Review Board, which ensures the safety of clinical research participants. Generally, clinical trials overseen by the FDA have been preceded by trials in at least two animal species to show theyre safe and effective. So far, the studies that have backed Libellas gene therapy are based just in mice.

This study has caused a lot of experts to raise eyebrows, particularly when it comes to the ethical issue of asking participants to pay for a therapy with high risks. The company is charging $1 million for each of its five aging but otherwise healthy participants, as well as five participants who have Alzheimers disease and five who have a form of artery disease.

But the trial also raises the question of whether aging itself is a disease worth treating. With any disease, there has to be a disease-free state, says Suresh Rattan, biogerontologist at Aarhus University. In the case of a situation like aging whose main cause is life itself, when will we say that we have treated it? Evolution didnt design us to live forever.

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Science is still studying how telomeres are linked to longevity - Quartz

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Gene therapy is providing unprecedented hope for growing numbers of patients and families. This game changer in medicine restores vision in babies born with congenital blindness, reconstitutes defences against infection in inherited immunodeficiencies and offers the perspective of curing the devastating neuromuscular disease, spinal muscular atrophy.

Gene therapy is also removing the need for repeat blood transfusions in adolescents with the inherited blood disorder, beta-thalassemia. Meanwhile, in oncology, CAR-T therapies, involving genetic modifications of a patients own immune cells, are proving life-saving for children or adults with certain types of blood cancers.

All these revolutionary treatments are now approved by regulatory agencies in Europe or the US. Unfortunately, they carry astronomical price tags which prevent their effective delivery to patients. As one case in point, Bluebird Bios Zynteglo for treating beta-thalassemia, has a list price of 1.57 million.

Can high prices be justified?

Gene therapy manufacturers defend their prices by pointing to high development and manufacturing costs, small markets, and unique therapeutic effectiveness as compared to the current standard of care. However, R&D costs are kept secret, and higher numbers of patients eligible for a given therapy do not translate into lower prices.

Indeed, several arguments the manufacturers put forward are dubious or even far-fetched. As of today, claims that a single administration of a gene therapy product will ensure a lifelong cure are simply not supported by the scientific evidence.

Likewise, value-based pricing is often misconceived. As stated by the US Institute for Clinical and Economic Review in its 2017 white paper on gene therapy, the established value of a treatment reflects the maximum price society might be prepared to pay for it - but should not dictate the price that is actually paid. In an ideal world, actual prices should provide market-consistent returns for shareholders and sufficient incentive to innovate.

The EU, a pioneer in gene therapy

European scientists, institutions and charities have been central to the development of gene therapy. The world's first successful clinical trial was reported in 2000 by Alain Fischer and his team at Necker Hospital in Paris, while the first authorisation of a gene therapy product in a regulated market was granted by the European Medicines Agency in 2012.

According to the Cordis database of EU-supported research, 86 gene therapy projects for rare diseases had funding from the European Commission during the FP7 (2007-2013) and Horizon 2020 (2014-2020) research programmes. One can estimate that overall more than 1 billion has been invested in this area by the EU Commission, member states and not-for-profit organisations.

To ensure European patients benefit from these achievements and investments, it is essential to ensure reasonable pricing of gene therapies. Laudable efforts are currently being made by the World Health Organization to increase transparency, and by some member states to join forces in negotiating prices, but such initiatives are unlikely to solve the current crisis as they do not address its root, namely that the sole objective of most gene therapy companies is to maximise the return on investment and shareholder value.

A way forward: enforcing the corporate social responsibility of gene therapy manufacturers

As I recently argued with Alain Fischer and the economist Mathias Dewatripont in the journal Nature Medicine (November 25, 2019), now is the time to reflect on how to enforce the corporate social responsibility of gene therapy companies.

Among the measures we would like to see considered are the insertion of clauses into technology transfer agreements made between academic organisations receiving grants from the European Commission and for-profit companies to make reasonable pricing compulsory.

We also propose to make reimbursement of gene therapies by EU healthcare payers conditional on the companies which are commercialising these products being certified for their corporate social responsibility. This is in line with several commitments made recently by pharma companies. For example, in August 2019, the CEOs of US-based pharma companies signed the Business Roundtable Statement, affirming their commitment to generate value for all their stakeholders not just their shareholders.

Also in August, Novartis announced it had joined the Value Balancing Alliance, a body whose goal is to increase transparency around business decisions, work with external bodies to develop accounting frameworks, and shift priority from profit maximisation to optimising value creation.

Earlier this year, the pharmaceutical company Chiesi was certified as a Benefit Corporation, meaning its legally defined goals include positive social impact in addition to profit.

Of course, the effective implementation of such commitments and their translation into reasonable pricing policies will require both incentives and regulatory controls. The starting point should be a renewed multi-stakeholder conversation with industry, investors, regulators, payers and, of course, patients.

Professor Michel Goldman is Co-director of the I3h Institute at the Universit Libre de Bruxelles and former Executive Director of the EU Innovative Medicines Initiative.

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Viewpoint: EU should take a lead in enforcing the corporate social responsibility of gene therapy manufacturers - Science Business

Recommendation and review posted by Bethany Smith

RESEARCH TRIANGLE PARK AskBio, a gene therapy company based in RTP that recently raised $225 million from investors, will pay up to $240 million in upfront and milestone payments for a license to use gene therapy technology from Massachusetts-based Selecta Biosciences as part of a regime to treat Pompe disease.

The license is for ImmTOR, what Selecta describes as a immune tolerance platform. The technology is addressing barriers to repeat administration of gene therapies.

Pompe disease affects between 5,000 and 10,000 people a year, affectingventilator, cardiac and skeletal muscles and can cause motor neuron dysfunction, with effects on cognition, hearing, speech and fine motor skills, AskBio says.

There is a demonstrated unmet medical need for better treatment approaches for Pompe disease, and this collaboration will enable us to effectively advance our Pompe program with the added benefit of Selectas ImmTOR technology, said Sheila Mikhail, CEO and co-founder of AskBio, in a statement. The opportunity to re-treat patients holds significant promise, and we are pleased to be able to leverage our relationship with Selecta and apply the ImmTOR technology to potentially overcome the challenges associated with re-administering systemic AAV gene therapies.

The companies initially announced a partnership in August.

AskBio gets $235 million in gene therapy support

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Triangle-based AskBio to pay up to $240M for rights to use gene therapy technology - WRAL Tech Wire

Recommendation and review posted by Bethany Smith

XconomyBoston

Ring Therapeutics, a Flagship Pioneering spinout, launched Thursday with ambitious plans to expand the universe of vectors available for gene therapy delivery.

Gene therapy, treatments intended to treat disease by inserting a gene instead of using drugs or surgery, has had a banner year, with the second ever such therapy approved this year in the US.

Ring want to use itsresearch into viruses that exist in the human body without apparent negative effects to provide more and better options to fuel the rise of gene therapy treatments.

For the past two years, Flagship Pioneering partner and Rings founding CEO Avak Kahvejian says the company has been exploring the human commensal viromebasically, a group of viruses that exist within humans without negative effectsfor its potential to address limitations of the vectors currently used.

The sector relies heavily on adeno-associated viruses (AAVs), which naturally infect humans but arent known to cause disease, to deliver the DNA. Previous exposure, however, can spark an immune response.

A lot of the workhouses in gene therapy have either been pathogenic viruses or viruses that have been taken from other species or viruses that are highly immunogenic, or all of the above, Kahvejian tells Xconomy. That leads to a certain number of limitations, despite the successes and advances weve made to date.

A number of issues stymie widespread use of AAVs, Kahvejian says, including the fact that 10 percent to 20 percent of people have at one time or another been infected with such a virus, thereby building up an immune response to it. Another concern is where such gene therapies end up, because viruses tend to gravitate toward certain types of tissues, and to go elsewhere, require special tweaking.

The Cambridge, MA-based startup believes the viruses it has found are unlikely to cause an immune response or prove pathogenic, given their ubiquity in the body.

Like extrachromosomal DNAa new discovery at least one company is exploring for its potential as a target in cancer treatmentsthe viral sequencing Ring is studying are circular pieces of DNA that exist outside the 23 chromosomes of the human genome.

Ring says it has found thousands of these viruses that coexist with our immune system. It aims to use those to develop vectors that can facilitate gene replacement throughout the bodymultiple times, if necessary. While gene therapy is thought of as a one-time fix, cell turnover means whatever the fix engendered by the inserted gene could falter over time, necessitating a re-up.

Kahvejian wouldnt share a timeline for Rings plan to develop re-dosable, tissue-targeted treatments.

Were looking at the unique features and activities of these viruses in different tissues to establish the various vectors were going to pursue, he said.

Flagship, which pursues scientific questions in-house and builds and funds companies around the answershas put $50 million toward Ring, which has about 30 employees.

Rings president is Rahul Singhvi, an operating partner at Flagship. Most recently he was chief operating officer of Takedas global vaccine business unit. Its head of R&D is Roger Hajjar, who has led gene therapy trials in patients with heart failure.

Ring is the second startup Flagship has spun out this month. Cellarity launched last week.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options - Xconomy

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By Dan Ross

Earlier this month, researchers out of the University of Pennsylvania published a breakthrough study which identified several biomarkers associated with shockwave therapy. This study is the first of its kind to identify a possible biological signal revealing shockwave usagean important development for a therapy that leaves no visible trace, but one that has historically generated welfare concerns due to its analgesic effects.

Because its not a drugits applied to the surface of the skinits just not an easy thing to detect, said lead researcher Mary Robinson, assistant professor of veterinary pharmacology and director of the Equine Pharmacology Laboratory at the University of Pennsylvania School of Veterinary Medicine, in a press release. After a lot of trial and error, our study was able to measure changes in levels of five inflammatory factors, some of which we could detect up to three weeks after the shockwave therapy.

The study was conducted on ostensibly healthy horses. Robinson told the TDN that she and her colleagues will continue to research these biomarkers on other populations of racehorses, including injured horses, injured horses that have received shockwave therapy, and healthy horses in training. For this next step, researchers will dip into the universitys large BioBank library of biological samples.

Robinson was also a co-author on a paper published in November proving proof of principle, she said, that they were able to detect experimental gene therapy which had been administered via intra-articular injection. Gene doping and its performance enhancing potential is an emerging problem in horse racing. Broadly speaking, the term refers to the twin practices of gene editingchanging animal DNA before theyre bornand gene therapy, which refers to the implantation of a piece of DNA into an adult animal to prevent or treat a disease or disorder.

In the following Q&A, Robinson discusses shockwave usage, the implications of her shockwave biomarker study, and her ongoing work in gene doping.

TDN: Can you discuss how the shockwave study was conducted?

MR: The study that was just published is looking at the effect of giving a single dose of shockwave to the front of the cannon bone to 11 healthy horses. Whats really novel about this study is we could find changes in the blood that were reflective of these horses receiving shockwave therapy. The biobank is for the next stepsthis initial study was more a proof of concept study.

Essentially, what we did, a week before shockwave was given, we took samples each day. After we gave the dose of shockwave, we then collected samples after that dose for up to three weeks. And we were able to see these changes occurring very rapidly, within 24 hours after the shockwave dose, and some of them lasted for up to three weeks.

What that tells me is we probably will need to follow horses out over time. And so, for each individual animal, in order to make these biomarkers make sense, were going to need to know what their normal profile looks like, and then follow that out over time.

TDN: How did you end up narrowing in on those 10 select biomarkers (of which five indicated shockwave usage)?

MR: That was more a resource limitation than anything else. Its very difficult to do these types of studies on horses, and that maybe speaks to some of the challenges we face in identifying biomarkers. One of the difficult things is there just arent the resources out there.

One of the ways we narrowed in on those 10 was to see which pro-inflammatory and anti-inflammatory proteins were similar between horses and humans, so that we might be able to use the resources available for humans to try to measure these things in horses. We spent quite a lot of time validating the methods that were used to make sure they would work for horses. And we chose pro and anti-inflammatory factors because we know shockwave causes a very acute inflammatory response. It increases the blood flow to the area.

TDN: Whats next in your research?

MR: As you mention, these were healthy horsesthey did not have an injury. And were looking at anti-inflammatory factors which you would maybe expect to be affected by an injury. We need to be able to look at what the effect of an injury is going to do to these factors, and then we need to look at what the effect of giving shockwave on top of that injury is going to do to those factors.

These horses were healthy, but they were not in any type of exercise regimen, so we also need to assess the effect of exercise on these factorsits well documented that [biomarker] IL-6 that we identified is affected by exercise. So, well need to make sure that we take that into account.

TDN: When will you start?

MR: Weve already started. Weve been working on collecting these BioBank samples for several years now. It was just in the last year and a half that weve been able to quadruple the number of samples that weve collected because of the funding from the Horse Breeders Association. Within the next year, were going to be starting to make some comparisons using the markers that weve now identified.

TDN: What can you tell us about the BioBank?

MR: The funding for the BioBank is from the Pennsylvania Horse Breeders Association. I was able to start the bank using some internal funds from the [University of Pennsylvania School of Veterinary Medicine] New Bolton Center, and then the PHBA very generously donated money for us to expand the bank, which allowed us to hire an additional person, and get some more freezers, so that we can collect samples from horses at the racetrack and in training at various training facilities.

TDN: The BioBank primarily contains Thoroughbred samples, right?

MR: Yes, although were actually going to be meeting with some other groups from the Standardbred industry to see if they would be interested in supporting it as well.

TDN: How soon before you possibly narrow in on certain shockwave biomarkers that can be used for regulatory purposes?

MR: I dont know that well ever be able to conclusively prove that shockwave had been administered. I think what we hope to do is identify instead biomarkers that suggest that theres something concerning going on in an individual horse. Whether those relate specifically to shockwave or whether those relate to a horse that has some underlying injurythat somebodys trying to cover up or somebody doesnt even know existsthats what this biological passporthopes to do.

TDN: What are your general thoughts about shockwave usage for horses in training?

MR: Currently in Pennsylvania, we dont allow it on the backside of any of the tracks, and that really is because its not something that we can detect currently. We do not want to have it risked being used too close to a race, and thats a problem with shockwave: it has a very strong analgesic effect, just as effective as the nerve block when its used on an injury. The key is, it can be used as a therapeutic, but you have to absolutely rest the horse. You cannot run the horse shortly after its received shockwave.

TDN: Do you agree with the Association of Racing Commissioners Internationals model rule requiring a 10-day stand-down, given what we currently know about the analgesic effect?

MR: I do. But theres only one study thats definitively shown that theres an analgesic affect. I think that they stopped around two days in that study, and its not really clear whether that effect would have lasted longer or not. Theres potential that it could have an analgesic effect for a longer period of time.

TDN: What are the implications of gene doping for horse racing?

MR: Its another new way to treat disease. Instead of having to give multiple doses, the whole point of gene therapy is you can give a single dose and have a more permanent treatment. So, when you think of it in that regard, the potential for using it illicitly to try to enhance performance is huge, because a single dose might be able to create a lasting effect. So, I do think its a serious concern. Its been on the WADA [World Anti-Doping Agency] anti-doping list since 2003. So, our human colleagues have certainly been concerned about it for quite some time.

TDN: Do things that go on in human athletics often makes their way into horse racing?

MR: We certainly have seen that pattern, although the reverse is possible. Some people may feel more comfortable experimenting with horses than with humans. Of course, cost always becomes an issue. But its possible there are people with the means who are willing to try and win at all costs. So, thats what were trying to prevent.

TDN: How big an issue is it currently for horse racing?

MR: Its really hard to know at this point. There certainly has been suspicion and discussion and talk about it. I have to say I was pretty skeptical that this was a problem until more recently. Now that the therapies are being approved in humans, theyve accelerated the ability for people to actually develop these therapies, so, I think its something we need to address as soon as possible as an industry.

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Q&A: UPenn's Mary Robinson Talks Shockwave and Gene Doping - Thoroughbred Daily News

Recommendation and review posted by Bethany Smith

DetailsCategory: More NewsPublished on Friday, 20 December 2019 13:54Hits: 103

Selecta eligible to receive upfront and milestone payments of over $240 million

AskBios AAV gene therapy combined with ImmTOR could prevent the formation of neutralizing antibodies and potentially enable re-treatment of patients with Pompe disease

WATERTOWN, MA and RESEARCH TRIANGLE PARK, NC, USA I December 19, 2019 ISelecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), today announced that the companies have entered into a license agreement. Under the terms of the agreement, AskBio has exercised its option to exclusively license rights to develop and commercialize Selectas immune tolerance platform, ImmTOR, for use in adeno-associated virus (AAV) gene therapy for the treatment of Pompe disease. When used in combination with AAV gene therapy vectors, Selectas ImmTOR has been shown to inhibit the immune response to the vector (Nature Communications, October 2018).

Selecta and AskBio previously announced a strategic partnership in August 2019 to jointly develop, manufacture, and commercialize targeted therapeutics for next-generation AAV gene therapies in areas of high medical need. Under the terms of this new license agreement, Selecta is eligible to receive upfront and milestone payments of over $240 million plus royalties on product sales.

We are pleased to advance our ImmTOR platform in a new partnership with AskBio for Pompe disease, a serious and progressively debilitating disease, said Carsten Brunn, Ph.D., President and Chief Executive Officer of Selecta. We are excited to collaborate with AskBio to potentially enable the retreatment of AAV gene therapies.

There is a demonstrated unmet medical need for better treatment approaches for Pompe disease, and this collaboration will enable us to effectively advance our Pompe program with the added benefit of Selectas ImmTOR technology, said Sheila Mikhail, CEO and co-founder of AskBio. The opportunity to re-treat patients holds significant promise, and we are pleased to be able to leverage our relationship with Selecta and apply the ImmTOR technology to potentially overcome the challenges associated with re-administering systemic AAV gene therapies.

Pompe disease is a rare, progressive, debilitating condition that affects 5,000 to 10,000 people worldwide. It impacts ventilator, cardiac and skeletal muscles and can cause motor neuron dysfunction, with effects on cognition, hearing, speech and fine motor skills. Pompe disease can manifest any time between infancy and late adulthood and may have differing symptoms and severity, depending on when it develops. There is a substantial unmet medical need for more efficient and effective treatments for Pompe disease. Currently, the only approved treatment is ERT with recombinant human GAA (rhGAA), a chronically administered therapy requiring increasing doses, which can elevate both costs and treatment burden.

AboutSelecta Biosciences, Inc. Selecta Biosciences, Inc.is a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance technology (ImmTOR) platform.Selectaplans to combine ImmTOR with a range of biologic therapies for rare and serious diseases that require new treatment options due to high immunogenicity. The companys current proprietary pipeline includes ImmTOR-powered therapeutic enzyme and gene therapy product candidates. SEL-212, the companys lead product candidate, is being developed to treat chronic refractory gout patients and resolve their debilitating symptoms, including flares and gouty arthritis. Selectas proprietary gene therapy product candidates are in preclinical development for certain rare inborn errors of metabolism and incorporate ImmTOR with the goal of addressing barriers to repeat administration.Selectais based inWatertown, Massachusetts. For more information, please visithttp://selectabio.com.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy platform company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10 and an extensive AAV capsid library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors and self-complementary DNA. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit https://www.askbio.com/.

About Pompe DiseasePompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

SOURCE: AskBio

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AskBio Licenses Rights to Selecta Biosciences' ImmTOR Immune Tolerance Platform for the Treatment of Pompe Disease | More News | News Channels -...

Recommendation and review posted by Bethany Smith

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Obamacare wasn't just about coverage. The health law created the Center for Medicare and Medicaid Innovation, fueling the move to value-based care, or paying doctors and hospitals for making patients healthy, rather than for each visit or surgery. While many of center's experiments focus on the Medicare health program for the elderly, private insurers are taking similar approaches across the US.

Andy Slavitt, the acting Centers for Medicare and Medicaid Services administrator from 2015 to 2017, said the health law's creation of the innovation center, together with the private experimentation, was creating rapid changes in US healthcare.

"In effect, you're saying to the healthcare system, instead of thinking about how to make revenue, we're going to give you a fixed revenue amount, and you think about the things you can control for," Slavitt said. Slavitt is now a founder of the healthcare research group United States of Care and a general partner at the venture-capital firm Town Hall Ventures.

And while Obamacare helped fuel the move to value, the Trump administration has continued to push the healthcare system in the same direction.

"There's been bipartisan support and, and I would say, work throughout the entire system around value-based care," said Seema Verma, the current Centers for Medicare and Medicaid Services administrator. "There's a lot of hope around and consensus around value-based care."

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Biggest healthcare and biotech developments of the decade - Business Insider Nordic

Recommendation and review posted by Bethany Smith

In a recent study published by Prophecy Market Insights, titled, Global Gene Therapy for Rare Disease Market Research Report, analysts offers an in-depth analysis of global Gene Therapy for Rare Disease market. The study analyses the various aspect of the market by studying its historic and forecast data. The research report provides Porters five force model, SWOT analysis, and PESTEL analysis of the Gene Therapy for Rare Disease market. The different areas covered in the report are Gene Therapy for Rare Disease market size, drivers and restrains, segment analysis, geographic outlook, major manufacturers in the market, and competitive landscape.

Key Players of Gene Therapy for Rare Disease Market:

Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., UniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and Biomarin Pharmaceutical Inc.

Download Sample Copy of This Report @ https://prophecymarketinsights.com/market_insight/Insight/request-sample/397

The research report, Gene Therapy for Rare Disease Market presents an unbiased approach at understanding the market trends and dynamics. Analysts have studied the historical data pertaining to the market and compared it to the current market trends to paint an object picture of the markets trajectory. The report includes SWOT analysis and Porters five forces analysis to give the readers an in-depth assessment of the various factors likely to drive and restrain the overall market.

Market Segmentation:

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Table of Contents

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the global Gene Therapy for Rare Disease market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the global Gene Therapy for Rare Disease market is analyzed, taking into consideration price, revenue, sales, and market share by company, market concentration rate, competitive situations and trends, expansion, merger and acquisition, and market shares of top 5 and 10 companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the global Gene Therapy for Rare Disease market as well as some useful information on their business. It talks about the gross margin, price, revenue, products and their specifications, applications, competitors, manufacturing base, and the main business of players operating in the global Gene Therapy for Rare Disease market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Gene Therapy for Rare Disease market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This part of the research study shows how different application segments contribute to the global Gene Therapy for Rare Disease market.

Market Forecast: Here, the report offers complete forecast of the global Gene Therapy for Rare Disease market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Upstream Raw Materials: The report provides analysis of key raw materials used in the global Gene Therapy for Rare Disease market, manufacturing cost structure, and the industrial chain.

Marketing Strategy Analysis and Distributors: This section offers analysis of marketing channel development trends, indirect marketing, and direct marketing followed by a broad discussion on distributors and downstream customers in the global Gene Therapy for Rare Disease market.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

Appendix: Here, we have provided a disclaimer, our data sources, data triangulation, market breakdown, research programs and design, and our research approach.

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Gene Therapy for Rare Disease Market to Witness Increased Incremental Dollar Opportunity During the Forecast Period 2020 2030 - Bulletin Line

Recommendation and review posted by Bethany Smith

() CEO Doug Doerfler tells Proactive it has appointed a new executive vice president as it continues with a Phase I trial of its MCY-M11 cancer drug. Doerfler says Shruti Abbato will serve as executive VP of business development for its CARMA cellular therapies, which is aiming to provide faster treatment than existing cancer therapies.

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Recommendation and review posted by Bethany Smith

A leading research firm, Zion Market Research added a latest industry report on "Global Gene Therapy Market" consisting of 110+ pages during the forecast period and Gene Therapy Market report offers a comprehensive research updates and information related to market growth, demand, opportunities in the global Gene Therapy Market.

According to the report the Future of Gene Therapy Market Reviewed in a New Research Study 2018-2026

The Gene Therapy Market report provides in-depth analysis and insights into developments impacting businesses and enterprises on global and regional level. The report covers the global Gene Therapy Market performance in terms of revenue contribution from various segments and includes a detailed analysis of key trends, drivers, restraints, and opportunities influencing revenue growth of the global consumer electronics market.This report studies the global Gene Therapy Market size, industry status and forecast, competition landscape and growth opportunity. This research report categorizes the global Gene Therapy Market by companies, region, type and end-use industry.

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The Gene Therapy Market report mainly includes the major company profiles with their annual sales & revenue, business strategies, company major products, profits, industry growth parameters, industry contribution on global and regional level.This report covers the global Gene Therapy Market performance in terms of value and volume contribution. This section also includes major company analysis of key trends, drivers, restraints, challenges, and opportunities, which are influencing the global Gene Therapy Market. Impact analysis of key growth drivers and restraints, based on the weighted average model, is included in this report to better equip clients with crystal clear decision-making insights.

The Gene Therapy Market research report mainly segmented into types, applications and regions.The market overview section highlights the Gene Therapy Market definition, taxonomy, and an overview of the parent market across the globe and region wise.To provide better understanding of the global Gene Therapy Market, the report includes in-depth analysis of drivers, restraints, and trends in all major regions namely, Asia Pacific, North America, Europe, Latin America and the Middle East & Africa, which influence the current market scenario and future status of the global Gene Therapy Market over the forecast period.

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The Gene Therapy Market report provides company market size, share analysis in order to give a broader overview of the key players in the market. Additionally, the report also includes key strategic developments of the market including acquisitions & mergers, new product launch, agreements, partnerships, collaborations & joint ventures, research & development, product and regional expansion of major participants involved in the market on the global and regional basis.

Major Company Profiles Covered in This Report:

UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

Some of the major objectives of this report:

1) To provide detailed analysis of the market structure along with forecast of the various segments and sub-segments of the global Gene Therapy Market.

2. To provide insights about factors affecting the market growth. To analyze the Gene Therapy Market based on various factors- price analysis, supply chain analysis, porter five force analysis etc.

3. To provide historical and forecast revenue of the Gene Therapy Market segments and sub-segments with respect to four main geographies and their countries- North America, Europe, Asia, and Rest of the World.

4. Country level analysis of the market with respect to the current market size and future prospective.

5. To provide country level analysis of the market for segment by application, product type and sub-segments.

6. To provide strategic profiling of key players in the market, comprehensively analyzing their core competencies, and drawing a competitive landscape for the market.

7. Track and analyze competitive developments such as joint ventures, strategic alliances, mergers and acquisitions, new product developments, and research and developments in the global Gene Therapy Market.

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Future of Gene Therapy Market Reviewed in a New Research Study 2018-2026 - Industry News Ledger

Recommendation and review posted by Bethany Smith

Transparency Market Research (TMR) has published a new report titled, Viral Vector & Plasmid DNA Manufacturing Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20192027. According to the report, theglobal viral vector & plasmid DNA manufacturing marketis expected to exceed a value of US$ 400 Mn by the end of 2019. The global market is anticipated to surpass US$ 2 Bn by 2027 and expand at a high double digit CAGR from 2019 to 2027. Rise in prevalence of cancer, genetic disorders, and increase in number of clinical studies are expected to augment the global market from 2019 to 2027. The viral vector & plasmid DNA manufacturing market is projected to expand owing to an increase in the awareness regarding viral vector based treatments and technological advancements in developing countries.

Increasing prevalence of cancer, genetic diseases, and infectious diseases

According to Cancer Research UK, there were 17 million new cases of cancer in 2018 and four most common types of cancer worldwide were breast, lung, bowel, and prostate cancers, which account for approximately 43% of all new cases. According to WHO estimates, currently, 10,000 of human diseases are known to be monogenic, caused by modifications in a single gene in human DNA. The global prevalence of all single gene diseases at birth is approximately 10/1000. In Canada, it is estimated that monogenic diseases may account for approximately 40% of the work of hospital-based pediatric practice. Increased prevalence of such disorders demands improved treatments, which in turn is anticipated to propel the viral vector & plasmid DNA manufacturing market during the forecast period.

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Increase in awareness regarding gene therapy

Awareness regarding gene therapy is increasing worldwide, as compared to the last few decades. According to an article published by Human Gene Therapy, the acceptance of gene therapy for severe disorders, such as Alzheimer Disease, is high as compared to less severe disease, such as attention deficit hyperactivity disorder. Furthermore, acceptability of gene therapy is increasing, and there is a strong need to provide the public and patients with up-to-date information. Moreover, opportunities to engage in the discourse about areas of research in gene therapy is a priority. This is estimated to propel the viral vector & plasmid DNA manufacturing market during the forecast period.

Cancer segment dominates the global market due to large number of clinical trials ongoing worldwide

In terms of disease, the cancer segment dominated the global viral vector & plasmid DNA manufacturing market, followed by genetic disorders. The segment accounted for a prominent market share, due to availability of approved viral vector-based cancer drugs and several ongoing clinical trials for the treatment of a variety of cancers. This creates lucrative opportunity for entry into the viral vector & plasmid DNA manufacturing market.

Plasmid DNA segment dominates the global market due to wide use in the manufacturing of viral vectors as well as DNA based vaccines

In terms of type, plasmid DNA is a highly attractive segment of the global viral vector & plasmid DNA manufacturing market, followed by the adeno-associated virus (AAV) segment. This is attributable to the extensive utilization of plasmid DNA as raw material in the manufacturing of various viral vectors.Furthermore, several studies have emphasized the benefits of DNA- based vaccines over conventional vaccines. The adeno-associated virus (AAV) segment is anticipated to expand at a considerable CAGR during the forecast period, due to several advantages offered by AAV over other vectors, making them the vector of choice for various clinical trials.

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North America dominates the global market owing to high acceptance of viral vector based treatments in the region

North America dominates the global viral vector & plasmid DNA manufacturing market due to a large patient pool, technological advancements, and high acceptance of advanced treatments in the region. The region is estimated to maintain its dominance during the forecast period. Moreover, rising healthcare expenditure, availability of approved gene therapy treatments, and increasing investments are key factors that are anticipated to boost the viral vector & plasmid DNA manufacturing market in the next few years. The viral vector & plasmid DNA manufacturing market in Asia Pacific is projected to expand at a notable CAGR due to increasing awareness regarding viral vector based products in developing countries and rising research initiatives in countries such as Japan and China.

Investments by key players is driving the globalviral vector & plasmid DNA manufacturing market

Major players operating in the viral vector & plasmid DNA manufacturing market include CobraBiologics, Novasep Inc., Cell and Gene Therapy Catapult, Kaneka Eurogentec S.A., FUJIFILM Diosynth Biotechnologies Inc., Spark Therapeutics, Inc. Merck KGaA, uniQure N.V., and Lonza.

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Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through adhoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving methodologies and leveraging existing data repositories.

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Viral Vector and Plasmid DNA Manufacturing Market is Estimated to Expand at a Robust CAGR by 2027 - Testifyandrecap

Recommendation and review posted by Bethany Smith

Takeda is spending big to add a new piece to their oncology R&D puzzle.

This morning the global pharma company picked up an alliance with Turnstone Biologics, which has been building a new viral immunotherapy platform to complement its work on oncolytics, partnered with AbbVie for the past 2 years.

Dubbed the vaccinia virus platform out of a lab in Ottawa, R&D chief Mike Burgess describes it as a highly selective virus as a consequence of engineering, exquisitely selective for cancer cells in contrast to normal cells. And it can be used to deliver a payload of transgenes for Flt3 ligand, anti-CTLA-4 antibody, and IL-12 cytokine that replicate in cells.

Takeda is offering up a smorgasbord of cash to close the deal, with $120 million flowing to Turnstone for the upfront, near-term milestones and an upcoming equity investment which goes a long way to funding its next stage of development. Theres also $900 million more in longer-range milestones on the table.

Turnstone has been low key for the last few years, since AbbVie stepped up with an option deal on their oncolytics work, part of a wave of development work aimed at going Amgens Imlygic one better. Turnstone CEO Sammy Farah tells me the pact is still in place something AbbVie confirmed for me as well but has no interest in getting into the details of whats been going on there.

But hes a lot more voluble about the vaccinia platform.

Drawn from the lab of John Bell and his colleagues at The Ottawa Hospital Research Institute and the University of Ottawa, Turnstone turned up at AACR a little more than a year ago to offer preclinical mouse data to back up the potential in using it to fight cancer.

The reason why its so exciting, it offers a multi-pronged attack on cancer, says the CEO. Single modalities dont cut it anymore, but a combination combined with the therapeutic properties of theirs itself can be cutting edge in new therapies.

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Takeda puts $120M in near-term cash on the table to complete a new oncology platform deal - Endpoints News

Recommendation and review posted by Bethany Smith

The global Acromegaly Treatment market is driven by the growing prevalence of the genetic disease, changing lifestyle. Also, factors such as rising incidence of hormonal diseases, such as hypopituitarism and endocrine diseases, and high demand for the advanced treatment is expected to increase the demand for Acromegaly treatment market.

Factors, such as unavailability of precise treatment and high cost of the surgery can restrain the market growth.

Some of the key players operating in this market include Pfizer Inc., Chiasma Inc., Novartis AG, Ipsen Biopharmaceuticals Inc., Wockhardt Ltd., Troikaa Pharmaceuticals Limited, GlaxoSmithKline plc, Aegis Therapeutics LLC, Crinetics Pharmaceuticals Inc, Daewoong Pharmaceutical Co Ltd, Peptron Inc, among others.

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Increasing government support, favorable government insurance policies and schemes for the patients and rapid developments in technology will offer lucrative opportunities.

Based on Application, the Acromegaly Treatment market is segmented into Hospitals, Clinics, and others.

Based on Disease Types, the Acromegaly Treatment market is segmented into Ectopic Acromegaly, Pseudo Acromegaly.

Regionally, North America was the largest revenue generator in the Acromegaly Treatment market in 2017, because of high investments in research and development activities to investigate the applications of Acromegaly Treatment market.

Key Benefits of the Report:

* Global, Regional, Country, Application, and Disease Types Market Size and Forecast from 2014-2025

* Detailed market dynamics, industry outlook with market specific PESTLE, Value Chain, Supply Chain, and SWOT Analysis to better understand the market and build strategies

* Identification of key companies that can influence this market on a global and regional scale

* Expert interviews and their insights on market shift, current and future outlook and factors impacting vendors short term and long term strategies

* Detailed insights on emerging regions, Application& Disease Types, and competitive landscape with qualitative and quantitative information and facts.

Global Acromegaly Treatment Industry 2019 Market Research Report is spread across 121 pages and provides exclusive vital statistics, data, information, trends and competitive landscape details in this niche sector.

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Target Audience:

* Acromegaly Treatment providers

* Traders, Importer and Exporter

* Raw material suppliers and distributors

* Research and consulting firms

* Government and research organizations

* Associations and industry bodies.

Research Methodology

The Market is derived through extensive use of secondary, primary, in-house research followed by expert validation and third party perspective like analyst report of investment banks. The secondary research forms the base of our study where we conducted extensive data mining, referring to verified data sources such as government and regulatory published materials, technical journals, trade magazines, and paid data sources.

For forecasting, regional demand & supply factor, investment, Market dynamics including technical scenario, consumer behavior, and end use industry trends and dynamics , capacity Production, spending were taken into consideration.

We have assigned weights to these parameters and quantified their Market impacts using the weighted average analysis to derive the expected Market growth rate.

The Market estimates and forecasts have been verified through exhaustive primary research with the Key Industry Participants (KIPs) which typically include:

* Original Manufacturer,

* Application Supplier,

* Distributors,

* Government Body & Associations, and

* Research Institute.

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Table Of Content

1 Executive Summary

2 Methodology And Market Scope

3 Acromegaly Treatment Market Industry Outlook

4 Acromegaly Treatment Market Type Outlook

5 Acromegaly Treatment Market Application Outlook

6 Acromegaly Treatment Market Regional Outlook

7 Competitive Landscape

End Of The Report

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Recommendation and review posted by Bethany Smith

Gurugram/New Delhi: In a ground-breaking procedure, Haematologists and Bone Marrow Transplant specialists successfully treated Anurag Mishra, a 47-year-old man from New Delhi, suffering from Multiple Sclerosis (MS) from the past seven years.

Multiple sclerosis (MS) is a life-long condition, known to reduce life-expectancy. MS affects the brain and spinal cord that leads to serious disabilities.

The most common symptoms of MS include loss of sensation and balance, restricted arm or leg movement and vision loss in one or both the eyes.

Mishra, who was bedridden earlier, is back to his normal routine life, was diagnosed with MS an autoimmune neurodegenerative disease, where the body's own defence system starts attacking its nervous system, without any specific reason

Dr Rahul Bhargava, Director, Department of Clinical Hematology & Bone Marrow Transplant, Fortis Hospital in Gurugram with his team performed autologous bone marrow transplant where they used Mishra's stem cells for transplant, thereby reducing the chances of rejection and infections.

"In an autologous BMT procedure, the healthy stem cells from the patient are taken out and preserved. Chemotherapy is then administered to reset the body's immunity and then the stem cells are injected back to rescue the person from the side effects of chemotherapy," said Bhargava.

After the surgery, the patient is kept under isolation for a few months to ensure he/she does not contract any infection. In this case, when Mr Anurag approached us, he was entirely dependent on others for his basic needs. But within six months after the treatment, he is back on his legs and is carrying on with his normal life," Bhargava added.

According to the patient, the attacks are sudden and may affect any part of your body, limiting your abilities.

"Extreme pain and disabilities this disease gave, made me very scary and depressing. I think I am very lucky to get to know about Dr Rahul Bhargava and team, who cured me miraculously," Mishra said.

"Too much delay in the procedure can considerably affect the clinical outcomes. In the case of Mr Anurag, recovery is 90 per cent, which means he received the treatment within recovery time-frame," Dr Bhargava said.

See more here:
Delhi: 47-year-old successfully treated with bone marrow transplant - ETHealthworld.com

Recommendation and review posted by Bethany Smith

Bipartisan legislation provides record funding for lifesaving cellular transplants and eliminates a Medicare payment barrier for seniors.

Washington, DC, December 20, 2019 --(PR.com)-- National Marrow Donor Program (NMDP)/Be The Match applauds Congress for passing bipartisan legislation that provides record levels of funding to increase access for patients to cellular transplants, which can be the only curative treatments for blood cancers such as leukemia or lymphoma and other blood diseases. The bill also increases access to these same therapies for senior citizens by fixing a Medicare reimbursement issue that can be a barrier for them to these life-saving procedures.

Increasing funding levels for these programs and bringing Medicare payment policies for these procedures up to date represents major victories for the 1.3 million Americans fighting blood cancers, said Brian Lindberg, Chief Legal Officer and General Counsel of NMDP/Be The Match. By increasing funding for life-saving cellular transplants and removing Medicare barriers that inhibit access to care, Congress has given hope to patients in need of these curative treatments.

We are honored to have broad support from members in the House and Senate who stand with us and our mission to find matched donors for every patient in need of these cellular therapies, Lindberg added. Increasing patient access to life-saving bone marrow and cord blood transplant is NMDP/Be The Matchs top priority.

The program works closely with organizations throughout the nation to recruit volunteer donors for the registry and with public and private insurers to ensure that all patients have equal access to treatment.

The $30 million included in the final legislation for the C.W. Bill Young Cell Transplantation Program, an increase of $5.4 million over last year, and the $17.3 million for the National Cord Blood Inventory, an increase of $1.0 million, will help reduce barriers to transplant by:

Advancing new and innovative methods of providing the best possible transplant to every patient in need, regardless of socioeconomic status, age, ethnic ancestry, or any other individually defining characteristic; Continuing to simplify processes and systems to reduce time to transplant, providing the patient and their physician the therapy the patient needs exactly when he/she needs it; and Protecting access to transplant by allowing NMDP to pursue our vision of achieving equal outcomes for all.

In the case of older Americans, inadequate Medicare transplant reimbursement, primarily for donor-related costs, poses a significant barrier to patient access.

Unlike Medicare payment policies for the acquisition of solid organs for transplant, Medicare does not provide separate payments for the cost of acquiring the cells for transplant (which can include the cost of identifying genetically matched donors, collecting the cells, and transporting them to the transplant hospital). As a result, hospitals take substantial financial losses on these life-saving procedures, which often require a 20-to-30-day hospital stay on average, because the reimbursement rate does not come close to covering the true costs of treatment.

NMDP/Be The Match looks forward to working closely with the Centers for Medicare & Medicaid Services (CMS), which operates the Medicare program, to ensure that this critical payment reform is implemented as quickly as possible, so that Medicare beneficiaries are not at risk of being denied the bone marrow, peripheral blood stem cell, or cord blood transplant they need to survive.

About National Marrow Donor Program/Be The MatchFor people with life-threatening blood cancers such as leukemia and lymphoma, a cure exists. National Marrow Donor Program(NMDP)/Be The Match connects patients with their donor match for a life-saving marrow or umbilical cord blood transplant and works to identify and eliminate financial and other barriers faced by these patients. NMDP also provides patients and their families one-on-one support, education, and guidance before, during and after transplant.

Contact Information:National Marrow Donor ProgramEllen Almond(703) 548-0019Contact via Emailhttps://bethematch.org/

Read the full story here: https://www.pr.com/press-release/802090

Press Release Distributed by PR.com

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National Marrow Donor Program/Be The Match Applauds Congress for Its Support of Patients with Blood Cancers and Other Diseases - Benzinga

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Last Christmas a student from London received what he says was the best Christmas present ever the chance to save a strangers life.

Will Briant, 23, from Kennington, was found to be the best match for a patient with blood cancer in desperate need of a stem cell transplant. Will recently received a letter informing him that his anonymous recipients transplant had been a success and that he is now doing well.

Will initially joined the Anthony Nolan stem cell register in 2014. His girlfriend, who volunteered with Edinburg Universitys Blood, Bone Marrow and Transplant Society, which is part of blood cancer charity Anthony Nolans student volunteer network called Marrow, suggested that he sign up.

Will said: My girlfriend, Libby, told me this amazing statistic that a quarter of all stem cell donors sign up through Marrow at university, so I couldnt not join.

If it wasnt for Marrow and for Libby, I wouldnt have become a donor and given someone hope of a second chance of life just before Christmas.

After Will joined the Anthony Nolan register the charity confirmed his tissue type. Every time the charity was informed that someone needed a transplant it compared the patients tissue type to Wills and over 750,000 others on the register, as well as registers across the world.

In December last year, Will received an email from Anthony Nolan, informing him that he had come up as a potential match for a blood cancer patient in desperate need of a stem cell transplant. Will then went to his GP for blood tests, to confirm that he was in fact the best possible match.

Will said: Just a week before Christmas, I got the best Christmas present ever. I was told that I was the best match for the patient, and I would be donating early in the new year!

I was so excited. When you sign up you know that its such a tiny chance that youll be found as the best match for someone, so to actually be chosen felt really exciting. Also, because it was just before Christmas, it felt quite exciting to know that the patient would find out that they had a match just in time for Christmas!

On average, people who join the stem cell register have around a 1 in 800 chance of being asked to donate in the next five years, but for men aged 16-30, its 1 in 200. This is why Anthony Nolan need more young men to join the register.

At the beginning of this year, having spent Christmas at home with his family, Will donated his stem cells at The London Clinic.

Will said: For four days before the donation I had a course of G-CSF injections to increase the number of stem cells I was producing. This caused mild flu-like symptoms, I just felt a bit tired and achy really. The whole way through, I kept thinking about the recipient, and how, in this context, I was absolutely delighted to have mild flu-like symptoms! It was quite strange to be doing it for real, after talking to so many potential donors when I volunteered with Marrow at university!

Libby, the same girlfriend who had suggested Will consider signing up to the register four years earlier, accompanied him to his donation.

Will said: I sat in a hospital bed for four hours and was so pampered by the staff there! There was a huge choice of different lunches, endless coffees and I got to watch programmes on my iPad.

Following his donation Will then went back to his studies and his job, barely giving a second thought to what hed just done. However, this all changed when a month after the donation he received a letter of thanks from the recipient of his stem cells.

Will said: It was honestly the best letter Ive ever received. It was especially powerful because it really hit home, that not only had I given him a second chance of life, but also, I had given his wife, his children, his grandchildren and his friends more precious time with him.

Patients and recipients must remain anonymous for two years following a transplant, but they are able to communicate via anonymous letters and cards. After the two-year period, if both parties agree, they are allowed to meet.

Just recently, Will also received a letter from the hospital at which his recipient received their stem cell transplant to say that the donation had been successful and even though recovery can be a long process, he is currently recovering well. Will is hoping that they will both exchange Christmas cards this year.

Anthony Nolan is the charity that finds matching stem cell donors for people with blood cancer and blood disorders and gives them a second chance of life. It costs 40 for Anthony Nolan to add each new donor to the register, so the charity needs financial support to help it continue to give patients, their family and their friends hope.

Terence Lovell, Director of Engagement at Anthony Nolan told Charity Today: Our amazing stem cell donors, like Will, continue to enable many patients with blood cancer to spend Christmas with their loved ones, who wouldnt be here without their act of kindness.

Anyone wanting to support our work can visit our website and make a donation, which will help give someone like Wills recipient, a second chance of life in the future. Without your support, there is no cure.

Anthony Nolan also carries out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery. Find out more about Anthony Nolan this Christmas by visitinghttps://www.anthonynolan.org/

Read more:
'Last Christmas...' London student saved a stranger's life, this year he is alive and celebrating his gift of life - Charity Today News

Recommendation and review posted by Bethany Smith