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LONG ISLAND CITY, N.Y., June 15, 2020 /PRNewswire/ --Loki Therapeutics, an immuno-oncology company developing next-generation cancer therapeutics that leverage childhood vaccination recall antigens, today announced the formation of its Scientific Advisory Board (SAB), and the appointment of several prominent members, including Eileen O'Reilly, MD, of Memorial Sloan Kettering Cancer Center; Alan Forsythe, Ph.D., of Forsythe and Bear, LLC, a biostatistical consulting firm; and John McAuliffe, MD, Ph.D., F.A.C.S., and Jennifer Chuy, MD, both of Montefiore Medical Center/Albert Einstein College of Medicine.

Drs. O'Reilly, Forsythe, McAuliffe and Chuy will work closely with Loki's Founder and CEO Chris Bradley and principal scientist Claudia Gravekamp, Ph.D., to advance the development of the company's AWAKE platform, a potentially groundbreaking approach to cancer treatment that activates and redirects pre-existing memory T cells created during childhood vaccination to target and eliminate cancer cells.

"We are pleased to announce the launch of our SAB with the expertise of these inaugural members," said CEO Chris Bradley. "The SAB will provide critical guidance to Loki as we advance towards Phase 1 studies designed to confirm the clinical potential of our lead drug candidate derived from our AWAKE technology platform. Our SAB's medical, clinical trial and regulatory expertise will be instrumental in guiding Loki as we develop therapies that harness the power of memory T cells created during childhood vaccination to generate a powerful and immediate immune response to solid tumors and metastases."

Unique among immunotherapy approaches, Loki's AWAKE platform utilizes non-pathogenic microbes (attenuated Listeria monocytogenes) for the precise delivery of childhood vaccine recall antigens to tumor microenvironments and into tumor cells. Loki's lead program AWAKE-LM-TT capitalizes on the childhood vaccination for tetanus toxoid (TT) to trigger an immune response to solid tumors presenting the tetanus antigens.

SAB MembersEileen O'Reilly, MD Dr. O'Reilly is Section Head for Hepatopancreaticobiliary/ Neuroendocrine Cancers, Gastrointestinal Oncology Service, Co-Director for Medical Initiatives at the David M. Rubenstein Center for Pancreatic Cancer at Memorial Sloan Kettering (MSK) and is an Attending Physician and Member at MSK and Professor of Medicine at Weill Cornell Medical College. Dr. O'Reilly holds the Winthrop Rockefeller Endowed Chair in Medical Oncology. She is the Principal Investigator of multiple trials in pancreas cancer and has authored/co-authored more than 275 articles, editorials and book chapters.

Dr. O'Reilly received her medical degree at Trinity College (Dublin University) in Ireland. She completed her residency training in Ireland and fellowship training at MSK. Dr. O'Reilly's research and clinical activities focus primarily on pancreatic and hepatobiliary malignancies, and her research directions include integration of molecular and genetic-based therapies for the treatment of pancreas cancer along with development of adjuvant and neoadjuvant therapies and identification of biomarkers for therapy selection.

Dr. O'Reilly teaches and mentors junior faculty, oncology fellows, residents and medical/other students and has numerous teaching and other awards. She also chairs several prestigious medical committees.

Alan Forsythe, Ph.D.Dr. Forsythe has extensive experience in drug development management and statistical consulting. He has served as Partner and President of Forsythe and Bear, LLC Biostatistical Consulting Firm since 2009 and as a member of the NIH/NCI Interoperability Workgroup in 2006. He is a fellow of the American Statistical Association.

Dr. Forsythe held roles of increasing responsibility from 1991-2005 at Amgen, Inc., including as Vice President of Corporate Biomedical Information. There, he held direct responsibility for the Biostatistics, Epidemiology and Health Economics departments, and was responsible for planning, organizing and monitoring the implementation of new ways to increase profitability by shortening the time to get products to market around the world while improving the quality of the material presented to regulatory bodies for approval. He also had global oversight of long-range development plans for current and emerging Amgen products with respect to biometrics, epidemiology and health economics, and reviewed and approved all protocols for clinical studies, as well as proposed clinical publications and presentations.

Dr. Forsythe also previously served as Senior Vice President at Viratek, Inc., which is part of a $200 million pharmaceutical company, as Regional Director of Scientific Research at Southern California Kaiser Permanente Medical Group, as Adjunct Professor of Biomathematics at University of California at Los Angeles (UCLA) Medical School and as Adjunct Professor of Preventive Medicine at University of Southern California (USC). He has helped author more than 100 publications.

John McAuliffe, MD, Ph.D., F.A.C.S.Dr. McAuliffe is Associate Program Director General Surgery Residency, Attending Surgeon and Assistant Professor at Montefiore Medical Center and Site Director at Montefiore's Albert Einstein College of Medicine. Since joining the Montefiore team in 2016, his clinical focus has been on endocrine, neuroendocrine, and pancreaticobiliary benign and malignant diseases.

Dr. McAuliffe's research focuses on tumor biology, dissemination, and therapeutics in pancreatic adenocarcinoma via tumor-associated macrophages. He is the principal investigator on research projects focusing on surgical education quality improvement. His work has been published in a number of peer-reviewed journals and books and has been presented nationally. He is board certified in general surgery and complex general surgery oncology and is a member of numerous professional societies.

Dr. McAuliffe received his Ph.D. at the University of Texas Graduate School of Biomedical Sciences at Houston and his MD from the McGovern Medical School in Houston. Dr. McAuliffe began his postgraduate training with a year-long fellowship at the University of Texas MD Anderson Cancer Center, after which he trained at the University of Alabama-Birmingham Hospital. While there, he completed an internship and residency in General Surgery, becoming Administrative Chief Resident in his final year. He then completed a fellowship in Complex General Surgical Oncology at Memorial Sloan-Kettering Cancer Center.

Jennifer Chuy, MDDr. Chuy is an Attending Physician in hematology and oncology at Montefiore Medical Center and Assistant Professor of Medicine at Montefiore's Albert Einstein College of Medicine. She is a part of the division of gastrointestinal oncology and works closely with the multidisciplinary pancreatobiliary team. She has worked closely with scientists at the Albert Einstein School of Medicine on translational studies in pancreatic cancer funded by PANCAN. She is actively involved in the education of the medical oncology fellows.

She is board certified in internal medicine, hematology and oncology, and completed an internal medicine residency and a hematology/oncology fellowship at Albert Einstein College of Medicine after earning her medical degree at Northwestern University's Feinberg School of Medicine.

About Loki Therapeutics Loki Therapeutics is an immuno oncology company that is developing a new generation of cancer therapeutics that efficiently deliver childhood recall antigens to tumor cells with genetically attenuated bacterial vectors. Loki's AWAKE technology platform offers a potentially groundbreaking approach to cancer treatment by activating and redirecting pre-existing memory T cells created during childhood vaccination to target and eliminate cancer cells. Key to AWAKE is its utilization of attenuated Listeria Monocytogenes for the precise delivery of recall antigens to tumor environments and into the tumor cell. Loki's lead development program AWAKE-LM-TT capitalizes on the childhood vaccination for tetanus toxoid (TT) to generate an immune response to solid tumors and metastases presenting the tetanus antigens. Loki is currently advancing AWAKE-LM-TT as a potential treatment for metastatic pancreatic cancer, as well as metastatic ovarian cancer. Loki is also pursuing additional development programs based on other childhood vaccines, including polio (AWAKE-LM-PV) and mumps. For more information on Loki Therapeutics please visit http://www.lokitx.com.

ContactsTiberend Strategic Advisors, Inc.Miriam Miller (Investors)mmiller@tiberend.com212-375-2694

Ingrid Mezo (Media)imezo@tiberend.com646-604-5150

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Loki Therapeutics Forms Scientific Advisory Board to Advance Development of "Childhood Vaccination"-Based Cancer Therapeutics - BioSpace

London: Genes may soon reveal who would develop severe COVID-19 symptoms, remain asymptomatic or only have a mild coronavirus attack, say researchers.

Data from popular home genetic-testing kits could help scientists shed light on why some people who catch coronavirus have no symptoms while others become very ill, according to the team from the University of Edinburgh in the UK.

Researchers are now asking people who have used DNA testing services to gain ancestry or health insights to join a study that aims to identify key genes involved in the body's response to the infection.

"Understanding the effect genes have on susceptibility to COVID-19 could aid efforts to tackle the pandemic, and help combat future disease outbreaks," said the researchers.

More than 30 million people worldwide have used genetic testing services. Researchers are now urging them to share their DNA data to help speed up discoveries that could help fight the virus.

"Some people suffer no ill effects from coronavirus infection, yet others require intensive care. We need to identify the genes causing this susceptibility, so we can understand the biology of the virus and hence develop better drugs to fight it," said Jim Wilson, Professor of Human Genetics at the University of Edinburgh.

By providing gene data, volunteers will help the team avoid the costly, time-consuming task of collecting the hundreds of thousands of DNA samples that would otherwise be needed to map the genes involved.

The team aims to identify genes that influence the risk of developing COVID-19 and those that affect disease severity, by comparing volunteers' symptoms -- or lack of them -- with their DNA.

Researchers also aim to analyse the long-term health consequences of infection and self-isolation.

The study is supported by the Medical Research Council, Biotechnology and Biological Science Research Council, Health Data Research UK and Wellcome Trust.

"To identify the genes that explain why some people get very sick from coronavirus and others don't, we need the solidarity of a large proportion of people from different countries who can share their DNA testing results with us. In this case, size really matters," said Albert Tenesa, Professor of Quantitative Genetics at the University of Edinburgh.

Link:
Researchers to identify genes that put some at severe corona risk - ETHealthworld.com

The HIV virus can take refuge in the brain even when treated with antiretroviral therapies, only to later infect other organs in the body if that treatment is stopped, a new study in mice and human tissue suggests.

Untreated HIV, the virus that causes AIDS, cripples the immune system and leaves the body vulnerable to life-threatening illness. Combination antiretroviral therapy, or cART, can significantly lower concentrations of the virus in the body, to the point that the pathogen can become undetectable, symptoms largely disappear and the treated person is no longer infectious to others. But cART must be taken daily, and if treatment pauses, the virus may reemerge from hidden sanctuaries in the body.

The new study, published June 11 in the journal PLOS Pathogens, suggests that one of these hideouts is in brain cells called astrocytes. Astrocytes constitute roughly 60% of the total cells in the human brain, according to the report, and in an infected person, the study authors estimate that between 1% and 3% of these cells could harbor HIV.

Related: Going viral: 6 new findings about viruses

"Even 1% could be significant as a reservoir, as a sanctuary site, for the virus," said study author Lena Al-Harthi, a professor and chair in the Department of Microbial Pathogens and Immunity at Rush University Medical Center in Chicago. "If we're going to try to find an HIV cure, you can't neglect the role of the brain as a reservoir."

Al-Harthi and her colleagues drew their conclusions from a mouse model of HIV injected with human cells, as well as examinations of postmortem human brain tissue. While both experiments provide insight into the role of astrocytes in HIV infection, more work must be done to nail down exactly how the virus takes hold in human patients, an expert told Live Science.

"Animal models can tell us quite a bit. They're not humans, but they can inform us quite a bit," said Dr. Lishomwa Ndhlovu, a professor of immunology in medicine at Weill Cornell Medicine, who was not involved in the study. If astrocytes can act as a reservoir for HIV in human infection, and that virus can exit the brain and trigger infection elsewhere, as the mouse study indicates, "we do need to figure out how to eliminate the virus from these compartments" to devise a successful cure, he said.

Astrocytes, named for their star-like shape, come in a variety of subtypes and play critical roles in the central nervous system, according to BrainFacts.org, a public information initiative run in part by the Society for Neuroscience. The cells help deliver nutrients to neurons, or the brain cells that transmit electrical signals, and they can spur or subdue inflammatory reactions in the brain. Astrocytes also shape and maintain the wiring of the central nervous system and fortify the blood-brain barrier, a border of tissue separating circulating blood from brain cells.

Scientists knew that the HIV virus infiltrates the brain during infection, as infected people can develop dementia and other cognitive deficits.

"The role of astrocytes in HIV infection has always been controversial," Al-Harthi told Live Science. Previous studies suggested that the star-shaped cells can become infected with HIV, but much of the research used cells in petri dishes, which may not replicate infection processes in a living animal, Al-Harthi wrote in a 2018 report published in The Journal of NeuroVirology. A few studies have utilized live animals but used "traditional" methods, such as tagging viral proteins or genetic material with fluorescent compounds, to scan for the virus that may not be sensitive enough to accurately detect the low levels of HIV present in astrocytes. No study attempted to address whether, once infected, astrocytes could somehow release HIV to organs beyond the brain.

Al-Harthi and her team developed two new mouse models to address this crucial question.

Related: Top 10 mysterious diseases

First, the authors placed human fetal astrocytes, derived from extracted brain tissue, in petri dishes and infected those cells with HIV. They then injected the infected cells into the brains of lab mice, one set of newborn mice and one set of adult mice. They found that, in both sets of mice, the infected astrocytes passed on the virus to CD4 cells a type of immune cell that helps orchestrate the body's immune response and are specifically targeted by the HIV virus.

After picking up an infection from astrocytes, infected CD4 cells migrate out of the brain and into other tissues. When "the brain is already seeded, the virus can come out and reseed peripheral organs," Al-Harthi said.

In particular, the authors noted that the spleen and lymph nodes become infected as a result of this process. By blocking the movement of CD4 cells, the authors could cut this chain of viral transmission.

To ensure that the virus could infect astrocytes on its own, without their assistance, the authors also ran an experiment in which they injected healthy human astrocytes into mice and infected the animals with HIV afterward. In this scenario, some human astrocytes still became infected and released HIV into the rest of the body. Notably, the virus could still escape from the brains of mice given cART treatment, "albeit at low levels" compared to untreated mice. If the treatment was stopped, the virus from the brain triggered a full-blown infection.

To confirm aspects of their mouse experiments, the authors examined the donated brains of four HIV-infected individuals, all of whom received effective cART treatment. (The report did not specify how each donor died, but noted that the virus was effectively suppressed by cART at the time of death.) The team found that a small percentage of astrocytes contained HIV genetic material in their nuclei, indicating that the cells had been infected.

Many questions about astrocytes and HIV remain to be answered. For instance, certain subtypes of astrocytes may serve as reservoirs of HIV, while others don't, Al-Harthi said. And while the mouse experiments demonstrated that HIV can exit the brain, the postmortem tissue analysis could not confirm that the same occurs in humans.

"Animal models, none of them are perfect," so there may be differences in how infection unfolds in people, Al-Harthi said.

For example, during natural HIV infection, the virus can accumulate genetic mutations each time it replicates, and the genetic material required for infection can be lost in the process, Ndhlovu said. To fully understand the role of astrocytes in HIV, researchers will need to determine how much of the virus present in human astrocytes can actually trigger infection, he said.

Al-Harthi and her team began to address this question by examining postmortem brain tissue and analyzing what segments of HIV genetic material could be found within but further studies will need to confirm that the found virus is both able to infect cells and migrate to other organs in the body, Ndhlovu said. In addition, scientists will need to determine the exact route HIV takes out of the brain in order to infect other organs, as that information would also be crucial for developing treatments that target the brain and finding a successful cure, he added.

Originally published on Live Science.

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HIV may hide out in brain cells, ready to infect other organs - Live Science

Most people know that the novel coronavirus has killed older people in far higher numbers and disproportionatelyaffected people of color. But the gender gap in COVID-19 deaths is less well knownand understood.

It is being reported across China, Italy, Spain, Iran, and Germany that the number of men testing positive and dying from COVID-19 is double that of women, said Cummings School associate professorJanetrix Hellen Amuguni, VG11. In Italy, men have accounted for 71 percent of the deaths.

But why are men dying in higher numbers than womenand are both sexes being similarly affected?

This question requires an extensive analysis from both a gender and sex perspective, said Amuguni, an expert on the relationship between gender roles and infectious disease in global health.

It might be that the mens immune systems are built in a different way from womens, she said. Or it could be that gender roles linked to behaviors of men and womenaccess and control over resources and opportunities, power dynamics, and cultural norms that determine what men or women do dailyare playing an underlying role in determining who contracts this infection.

To study the many factors potentially at play, Amuguni has assembled a team of researchers from Cummings School and the Tufts School of Medicine. The scientists have been awarded $50,000 inCOVID-19 Rapid Response Seed Fundingfrom Tufts University and Tufts Medical Center to study sex differences and gender disparities in COVID-19.

Michael R. Jordan, A94, M98, an assistant professor at the School of Medicine, has cared for scores of people with COVID-19 as an attending physician in the Division of Geographic Medicine and Infectious Diseases at Tufts Medical Center.

Several studies suggest that men with COVID-19 do less well, and we know that many diseases can affect men and women differently, said Jordan. He noted that biological differences between men and women may affect how severe or deadly COVID-19 is for them.

Higher fatality rates were seen in men than women with severe acute respiratory syndrome (SARS) and Middle East respiratory syndrome (MERS), two other deadly infections caused by coronaviruses, said Tess Gannaway, V16, a Ph.D. candidate in infectious disease and global health at Cummings School and member of the Tufts research team.

One hypothesis about COVID-19s higher fatality rates in men is that the female hormone estrogen may be protective in some way, said Jordan. And some scientists suspect that male hormones called androgens, such as testosterone, may be to blame for mens poorer outcomes.

Genetic differences linked to the X chromosomeof which women inherit two versus mens onealso may lead to different immune responses or host environments for the infection in men and women, Jordan said.

Gannaway noted that a 2017 study of mice infected with the virus that causes SARS pointed to important sex differences. The male mice were more likely to die than female mice when infected with that coronavirus.

Higher amounts of virus accumulated in the lungs of the males. And the male mice had a different immunological response to the SARS virus that ended up making them more susceptible to the development of disease, said Gannaway.

Estrogen appeared to counter both these mechanisms in the female mice infected with SARS, she said. When scientists reduced or eliminated estrogen levels in female mice, for example, the female mice had mortality rates from SARS that were closer to the rates of the male mice.

With that in mind, its really important to characterize how the immune system is responding to COVID-19 in the two sexes, so you can target interventions more accurately for males and females, Gannaway explained. It may even help determine what we need to target within the male subset for better drug development and other new therapies.

To isolate any sex-based differences for SARS-CoV-2, the virus causing COVID-19, the Tufts researchers will conduct studies in mice at the Tufts New England Regional Biosafety Laboratory. The team also plans to draw on a new Tufts resourcethe Tufts Medical Center/Tufts University COVID-19 Biorepository and Comprehensive COVID-19 Database.

The biorepository is collecting blood and other samples from up to 400 hospitalized patients with COVID-19 who consent to have these used for research. Meanwhile, the database will collect anonymous data on all individuals testing for COVID-19 at Tufts Medical Center, regardless of their results.

Specimens for the biorepository are taken from patients at multiple time points, said Jordan, the director of the COVID-19 biorepository and database. So well be able to look at the viral loads in the nose and throat, as well as the evolution of antibodies over time in male and female human patients, and we can compare that with what we see in the mice.

The Tufts researchers also will investigate gender-based factors that may contribute to increased susceptibility and mortality in COVID-19.

Mens increased susceptibility and mortality may reflect other diseases or health conditions, said Jordan. Theres likely an interplay between the overall health and genetics. For example, men may be more likely than women to be overweight or to have high blood pressure.

Theres speculation that mens lifestyles might predispose them to develop COVID-19, explained Amuguni. For example, in China men are more likely to be smokers than women, and so have more compromised lungs. Theres also evidence that men take longer than women to seek necessary health care.

Men may tend to work in occupations that put them at higher risk, too, noted Marieke Rosenbaum, V14, MG14, VG14, another member of the Tufts research team. Perhaps men are more likely to greet someone by putting their hand on their shoulder or shaking hands, she said.

If we can identify what gender-specific behaviors make men at higher risk for COVID-19, we can identify what precautions might help curb transmission and what behavioral changes would reduce other gender-based impacts, said Rosenbaum.

Even though the early data suggests that men typically have a higher risk of dying from COVID-19 overall, the coronavirus has killed more women than men in Massachusetts. Scientists suspect this may be because women tend to live longer than men, and people living in long-term care facilities were the population hit hardest by COVID-19 in the state.

The pandemic may also be disproportionately harming women and other individuals in other ways, Amuguni said.

There are gender-related consequences as a result of confinement related to stay-at-home advisories and orders, and these would differ across the world, she explained. We need to consider simple questions such as, has the workload increased for men or women? What does this mean for decision making, access, control over resources, and power dynamics? And if there an increase in domestic violence, who is affected the most?

Gannaway noted that women may be at higher risk losing their jobs or of contracting COVID-19 as a result of the pandemic. For example, seventy percent of the care force working within the health-care sector are women, and these women have more exposure to the coronavirus and greater risk of getting the disease, she said.

Gender roles, distribution of labor, and resources historically have played an important role in the spread of other infectious diseases, as well as in their control and prevention, noted Amuguni.

Therefore, these issues need to be addressed to understand better the risks for COVID-19and to develop adequate prevention and control strategies, she said.

Amuguni pointed to Ebola as one example. When this deadly virus spill overs from animals to people, the first person infected is usually a male. Thats because the index case, as it is called, is often someone who has been going into animals habitatand in Africa, hunters are typically men.

However, Amuguni said that over the first forty-five days of the Ebola outbreak in Liberia, 75 percent of the people who died were women.

Ebola was being transmitted primarily among women because of their important role in their communities, she said. When someone was sick, women cared for that person. And when people died, women were the ones cleaning their bodies and cooking and serving food at their funerals.

Of course, like the sex differences, the possible gender-related risk factors for COVID-19 are just hypotheses until theyve been rigorously studied. Thats why the second component of the Tufts teams study will include interviews to gather more qualitative data on what gender-based factors may increase susceptibility or spread of the infection.

We need to really dig in and listen to the experiences of all these people to understand their situations and to look for trends, said Rosenbaum.

With the help of Tufts Medical Center, the researchers will administer online surveys and hold focus groups with people who have recovered from COVID-19.

For example, public-health policies and programs also may have different impacts on straight men and women, the lesbian, gay and bisexual community, and people who identify as non-binary, Jordan said. We want to explore if these affect individuals decision-making around health-seeking behavior or testing, or their access to testing and ability to isolate safely at home.

Its a different way to approach health care and medical research during an outbreak, said Amuguni. Most of the time we approach infectious diseases as a medical emergency and focus solely on treating people.

But we have to look at other social determinants to figure out what other factors are actually related to the pandemic, she added. What role did gender factors play to get us to this situationand what role can they play to help the response and get us out of this situation?

Genevieve Rajewski can be reached at genevieve.rajewski@tufts.edu.

Link:
Why Is There a COVID-19 Gender Gap? - Tufts Now

Like many states, the UK government has committed to supporting disruptive innovations.1 These are considered to hold greater potential for economic growth and development than incremental advances in established technologies. Within this broad strategy the bioeconomy, the area of industrial activity based on commercialising life sciences research is given a particular importance. The bioeconomy includes sectors like biofuels, agricultural biotechnology, and medical biotechnology.2 In the latter case, advances in medical biotechnologies hold promise for treating, and even curing, serious and chronic diseases as well as driving growth and prosperity. Regenerative medicine (RM), the biotechnology-based use of cells, tissues, and genes as medicinal products, is certainly disruptive in that they differ in important ways from traditional pharmaceuticals and medical devices.3

The UK has taken a number of policy measures to support the development of the RM industry. The Regenerative Medicine Platform funding schemes promote and co-ordinate academic translational research. The Catapult centres, including the Cell and Gene Therapy Catapult, the Medicines Discovery Catapult and the High Value Manufacturing Catapult, provide advice, facilities and infrastructure to support businesses, especially Small and Medium-sized Enterprises (SMEs); with potential to contribute to the RM value chain. The Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Office offers a RM advice service to help academic and commercial developers navigate the complex regulatory framework for biological therapies, while the recent Accelerated Access Review proposed a raft of measures to speed up the regulatory timeline for transformative new therapies more generally.4

However, it does not necessarily follow that all parts of the biomedical sector will be equally disrupted by any given RM technology, nor that all RM technologies will be disruptive in exactly the same way.5 The ESRC-funded Biomodifying Technologies project6 analysed three case studies of biotechnologies with disruptive potential: gene-editing which allows faster, more accurate genetic modification, induced pluripotent stem cell (iPSC) technology that allows an ordinary skin or blood cell to be turned into a stem cell capable of producing any tissue type in the human body, and 3D bioprinting which can produce three-dimensional structures made from living tissues.

Gene editing and iPSC are advances on earlier generations of genetic engineering and stem cell technologies. They align reasonably well with the existing skill sets, goals, equipment, and techniques of researchers working in both academic and commercial settings. They are not especially disruptive at the level of basic research. Bioprinting requires skills, tools and techniques from engineering, materials chemistry, computer-aided design, biology, and medicine. This has necessitated greater disruption in the form of organisational change, to create new research groups and foster collaborative learning across disciplines.

For all three technologies, there are also well-established pathways to extract near-term value from basic research: peer-reviewed publications, patent applications, and the market for reagents, tools, and equipment. Each case demonstrates clear growth in the number of papers, patents, and reagent/equipment sales, although the rate of acceleration is greatest for CRISPR-based gene editing and slowest for bioprinting.

The pathways to realise longer-term, clinical, and economic value are less well established for RM. The healthcare sector is seen as particularly resistant to disruptive innovations, due to the lengthy regulatory process and powerful incumbent firms, which have historically been wary of investing in RM.7 The process of scaling laboratory protocols for cell or gene-based therapies into industrial procedures, taking products through clinical trials to establish safety and efficacy, and securing reimbursement, is every bit as experimental and involves as much learning by trial and error as exploratory laboratory research, but with much higher financial stakes. Interest from incumbents appears to be growing, as recent years have seen an increase in the number of cell or gene-based therapies reaching the market. However, there is no off the shelf manufacturing solution, as different RM products have different attributes: in the industry there is a popular idiom the product is the process. This means that the acceleration seen at the basic R&D stage does not unproblematically translate into speedy translation further down the pathway.

Rather, initial clinical applications of gene editing, iPSC and bioprinting are targeted at a more limited range of niche applications. The niches for each technology are shaped by a number of critical factors. Smaller tissues, such as the eye require fewer replacement cells or lower titres of gene editing vector, which are more manageable with current manufacturing capacity. The challenges of manufacturing at scale, combined with high anticipated costs, combine to make narrowly defined subsets of disease categories, with high unmet need, a preferred route for commercial development, especially where there is potential for a disruptive new product to demonstrate significant Quality of Life gains over the current standard of care.

Indications that draw on procedures, standards and requirements established for previous therapies are seen as less risky and thus promising clinical targets. Gene editing to treat thalassemia and other blood disorders builds on decades of clinical expertise with the bodys haematopoietic (blood-forming) system, gained by treating leukaemia patients. Even treatments that were not ultimately successful such as foetal stem cell transplants for Parkinsons disease (PD) can provide expertise with clinical trials and regulation to support a next-generation iPSC-based cell therapy for PD.

While the government has rightly been wary of picking winners, as particular niches for early clinical adoption of biomodifying technologies become apparent they may require specific, targeted support, to complement the broader support for the field already provided by polices described above. Innovations in related fields such as biomaterials and automation, potentially supported by the High Value Manufacturing Catapult, are likely to improve manufacturing capacity and speed over time. These innovations may be relatively incremental in the manufacturing phase but could have disruptive effects further down the value chain at the clinical delivery phase, as greater supply makes biomodifying RM therapies accessible to less tightly defined patient cohorts. The next policy challenge will be to provide targeted support for clinical delivery whilst avoiding lock-in to infrastructure or procedures that would inhibit the evolution of the field over time.

The research underpinning this piece was supported by the Economic and Social Research Council grant number ES/P002943/1 and the Leverhulme Trust grant number RPG-2017-330

References

1 Department for Business, Industry and Industrial strategy (2017) Industrial Strategy: building a Britain fit for the future. https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/730048/industrial-strategy-white-paper-web-ready-a4-version.pdf

2 Department for Business, Industry and Industrial strategy (2018) Bioeconomy strategy: 2018 to 2030. https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/761856/181205_BEIS_Growing_the_Bioeconomy__Web_SP_.pdf

3 Open Access Government (2019) The promises and challenges of biomodifying technologies for the UK https://www.openaccessgovernment.org/biomodifying-technologies/68041/

4 Accelerated Access Review (AAR). (2016). Final Report: Review of Innovative Medicines and Medical Technologies. London: The Crown.

5 Joyce Tait & David Wield (2019) Policy support for disruptive innovation in the life sciences, Technology Analysis & Strategic Management, DOI: 10.1080/09537325.2019.1631449

6 Open Access Government (2019) The promises and challenges of biomodifying technologies for the UK https://www.openaccessgovernment.org/biomodifying-technologies/68041/

7 Joyce Tait & David Wield (2019) Policy support for disruptive innovation in the life sciences, Technology Analysis & Strategic Management, DOI: 10.1080/09537325.2019.1631449

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Leader and Advocate for LGBTQ Family-Building Equality to Speak about Equitable Fertility and Family-building Benefits Trends and What it Means for Employers That are Behind the Curve

NORWALK, Conn. (PRWEB) June 15, 2020

RMA of Connecticut, a leading fertility practice whose exceptional quality and care helps couples and individuals grow their family, has announced its founder and medical director, Dr. Mark Leondires, a leader and advocate for LGBTQ+ family-building equality and the founder of Gay Parents To Be, an international program serving the LGBTQ+ community offered through RMA of Connecticut, will participate in the NASDAQ/Progyny sponsored webinar, Understanding LGBTQ+ Family Building + Benefit Trends.

The webinar, Understanding LGBTQ+ Family Building + Benefit Trends, is a featured Pride Month event, and will be held on Wednesday, June 17th from 1:00pm - 2:00pmEST. The panel will be moderated by NASDAQ's Head of Client Engagement, Josh Machiz and will feature two other distinguished panelists including Bonnie Sechrist, Director of Health Management Consulting at Willis Tower Watson and Lisa Greenbaum, Chief Client Officer at Progyny. The webinar will be open to the public in addition to NASDAQ's client network and can be accessed through Zoom. Topics for discussion will include LGBTQ family-building options (including surrogacy, adoption, and reciprocal IVF), equitable fertility and family-building benefits trends, and what it means for employers that are behind the curve in offering inclusive benefits.

"It's an honor to speak at the NASDAQ/Progyny-sponsored webinar, Understanding LGBTQ+ Family Building + Benefit Trends," said Dr. Mark Leondires, founder of RMA of Connecticut, Gay Parents To Be, and Gays with Kids. "While many companies are making major strides in offering inclusive fertility benefit coverage for all people, there is still work to be done. Speaking up for the rights of LGBTQ people who want to be parents and have equal access to family-building benefits, will hopefully and eventually lead to a world with family-building coverage for everyone."

"Now more than ever, family comes first," said Lisa Greenbaum, Chief Client Officer at Progyny, a leading fertility benefits management company in the US. "It is imperative that employers and physicians, like Dr. Leondires, work together to advocate for the LGBTQ+ community and ensure that all employees have equitable family building benefits."

In the race to attract and retain top talent, companies have begun offering inclusive benefits, a step in the right direction when it comes to equal benefits and fertility coverage for LGBTQ persons. Dr. Mark Leondires will speak about LGBTQ pathways to parenthood and the importance for more companies to offer equitable family-building benefits to all employees.

Gay Parents To Be was created and conceived by Dr. Mark Leondires. He has very personal knowledge of the LGBTQ family-building experience and is uniquely qualified as a gay father of two and a top reproductive endocrinologist to help couples through the process. Dr. Leondires spends his spare time advocating for large corporations to provide equitable family-building benefits for all employees.

For additional information and to sign up for the webinar, please visit, https://nasdaq.zoom.us/webinar/register/WN_Xr8jXS8ORaek5nSKZaXB_A

About Reproductive Medicine Associates of Connecticut (RMA of Connecticut)

RMA of Connecticut is a leader in fertility care, specializing in a range of infertility treatments. Our assisted reproductive technologies (ART) include intrauterine insemination (IUI), in-vitro fertilization (IVF) and pre-implantation genetic testing (PGT). RMA of Connecticut is Fairfield County's largest fertility clinic and egg donation center. Through RMA of Connecticut's Integrated Fertility and Wellness Center, we offer nutrition counseling, individual and couples psychological counseling, acupuncture and yoga, as well as financing and support services for our patients going through infertility treatment.

Our internationally recognized Gay Parents To Be program at RMA of Connecticut specializes in LGBTQ family building. For the last three years, RMA of Connecticut has been recognized as a Leader in Healthcare Equality by the Human Rights Campaign.

For the original version on PRWeb visit: https://www.prweb.com/releases/dr_mark_leondires_founder_and_medical_director_of_rma_of_connecticut_to_participate_in_nasdaq_progyny_sponsored_webinar_on_lgbtq_family_building_and_benefit_trends/prweb17178883.htm

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Dr. Mark Leondires, Founder and Medical Director of RMA of Connecticut, to Participate in NASDAQ/Progyny Sponsored Webinar on LGBTQ Family-Building...