Category Archives: Genetic Medicine

This clinical pilot will evaluate the effectiveness of Digbi Health's obesity management digital program personalized to each bariatric surgery patient, one-year post sleeve gastrectomy, based on their lifestyle, genetic and gut microbiome risks, in further reducing weight and maintaining weight loss. Obesity is a complex metabolic disease and an ongoing epidemic, with associated inflammatory, digestive, musculoskeletal, skin morbidities, as well as a risk factor for diabetes, cardiovascular disease, COVID-19, and reduced life expectancy, which currently affects 42 percent of the adult population in the United States. Bariatric surgery is the most effective long term intervention for morbid obesity, and successful bariatric surgery outcomes depend on lifelong changes in eating patterns and social support.

Research indicates individuals' genetic and gut microbiome makeups are intrinsically linked to their metabolism and following personalized nutrition recommendations and meal plans curated to an individual's genetic and gut microbiome markers may assist with further weight loss and ongoing weight maintenance post sleeve gastrectomy.

"WVU Bariatrics is excited to partner with Digbi Health to better understand how genomic, gut microbiome and metabolomic factors can contribute to successful weight loss following bariatric surgery," said Nova Szoka MD, FACS, FASMBS, Assistant Professor at J.W. Ruby Memorial Hospital, WVU Bariatrics Surgical Weight-Loss Center and principal investigator of the study.

"Digbi Health is the first company to operationalize a genetic and gut microbiome-based prescription-grade platform for doctors and payers to deliver weight loss, digestive health, and diabetes care programs at scale," said Ranjan Sinha, CEO, and founder of Digbi Health.

"Digbi is committed to empowering people suffering from obesity and chronic inflammatory lifestyle illnesses, struggling with ineffective one-size-fits-all diets, with personalized nutrition and lifestyle support that works for them. Through this collaboration with WVU Medical, we aim to deeper explore the critical importance of personalized nutrition and its direct impact on people suffering from obesity and associated illnesses," said Sinha.

More information about J.W. Ruby Memorial Hospital, WVU Medical, and Surgical Weight-Loss Center can be found here.

About Digbi HealthDigbi Healthis a first-of-its-kind precision digital therapeutics company that offers a prescription-grade digitally enabled personalized obesity and obesity-related gut, skin disorders, hypertension, and other cardiometabolic health management programs based on an individual's gut biome, genetic risks, blood markers, and lifestyle factors. Digbi Health and members of its physician network are committed to empowering people to take control of their own health and wellness. Digbi Health is prescribed by doctors, healthcare providers, and insurance companies.

SOURCE Digbi Health

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Digbi Health partners with West Virginia University Medicine and WVU Bariatrics Surgical Weight-Loss Program to improve postoperative weight loss...

Even the most promising drugs can hit a pothole in the road to U.S. Food and Drug Administration (FDA) approval, particularly when working in the complicated, evolving world of gene therapies.

After touting positive Phase III resultsat ASH (American Society of Hematologys annual meeting),uniQuregot slapped with anFDA clinical holdfor their hemophilia B treatment due to a patient developing liver cancer.

Theresearch team alerted the FDA of the development after a lesion was found during a routine one-yearfollow-up.A hepatocellular carcinoma (HCC) isexpectedto be confirmed with a full surgical resection.

However, the cancer may not be related to the gene therapy, as this particular patient was high risk for liver cancerwith a long history of hepatitis C, hepatitis B virus, findings of non-alcoholic fatty liver disease and advanced age.UniQurepointed out thatchronicinfections with hep B and C are associated with 80% of HCC cases.

Patient safety will always be our top priority, and we are working closely with the FDA and our advisors to conduct a thorough investigation into the cause of this event which we expect to be completed in early 2021, said RicardoDolmetsch, Ph.D., president of R&D atuniQure. We will investigate whether there is a relationship to treatment. At this time, we do not have adequate data to determine a possible causal relationship, especially in the context of the other known risk factors.

The hold is not expected to affect the pivotal trial results as the dosing of the patients is completed.

While several treatments areavailable for hemophilia B, doctors and patients are holding out hope for a cure that will stop the disease at its source. Thats thepotential that genetic medicine has held for this area.

UniQuresdrug, AMT-061, substantially increased production of the blood-clotting protein factor IX, thefactor missing forhemophiliaBpatients,innearly all trial participants with moderate to severe disease.

Drug developers have run into a challenge with gene therapies in patients with high levels of neutralizing antibodies. Those antibodies can disarma gene therapyby attacking its viral vector delivery systembefore it ever has the chance to help thepatient.Many gene therapy trials wont enroll patients who test positive for neutralizing antibodies.

UniQuresgene therapy is delivered by an AAV5 vehicle, whichis believed to be harder to impair than other vectors.The company enrolled 23 patients with neutralizing antibodiespresent.Only one patient did not respondto the therapy. That was one who had a large amount of theantibodies- encouraging results that at least some with neutralizing antibodies could go on the therapy.

All patients in our hemophilia B gene therapy program, including the 54 patients in HOPE-B, will continue to be monitored by their care teams while we gather additional information as rapidly as possible, noted CEO Matt Kapusta. We do not anticipate any impact to our regulatory submission timeline for the hemophilia B program as a result of this clinical hold.

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Hope on Hold: Promising Hemophilia B Therapy Paused by FDA - BioSpace

MORGANTOWN, W.Va. (WV News) Dr. Mary Louise Russell is a member of the team of experts at the WVU Medicine Childrens that specializes in pediatric physical medicine and rehabilitation.

She and her colleagues treat children who have movement issues due to a wide range of causes, Russell said.

Sometimes the children only have movement problems, and sometimes the movement problems are part of a more complex picture, she said. For example, children with Down syndrome can have movement problems, and they might be behind on learning to walk. But, they might have other problems, too, like developmental delay and even some other medical difficulties, like heart defects or GI problems.

She sees children of all ages, from just nine days old to beyond age 18, Russell said.

For new patients, well go all the way to 18 years. Although, for some who have had pediatric onset of chronic conditions, well see them into at least young adulthood, say mid-20s, she said. That would usually be a person with cerebral palsy.

Cerebral palsy is among the most common diagnosis she sees, Russell said.

Cerebral palsy is a problem with how the brain works that effects how somebody moves. It can be confined to the movement control part of the brain, or there may be other problems, like seizures or developmental delay, she said.

There are a variety of treatments for patients with cerebral palsy, Russell said.

The first treatment to be used is physical and occupational therapy, she said. In the United States, there is a service called Birth to Three or Early Intervention that is a federal government-supported service administered by states for kids to get in-home therapy services.

Other treatments for cerebral palsy include braces, walkers, crutches, or wheelchairs, Russell said.

Medications can be administered to reduce tone spasticity, and orthopaedic or neurosurgery can help to reduce muscle spasticity, she said. There is a variety of possible treatments that cover a range of issues.

The COVID-19 pandemic has forced her to rely on remote visits with some patients, which isnt quite the same as seeing them in person, Russell said.

I think its better than nothing, but for what I do say for a patient with cerebral palsy I really like to be able to feel how much resistance they give me when trying to stretch their arms or their legs, she said. Thats kind of a hard thing to do from a screen.

She looks forward to being able to see all of her patients in-person again in the near future, Russell said.

A lot of families right now are reluctant to bring their children for in-person visits, and I can understand. Many of these kids are medically fragile or chronically ill, she said. We just need to wait a little while longer for their in-person visits.

Russell, who has more than 30 years of medical experience, said she has seen significant leaps in what genetics can reveal about a patients condition.

What has been the biggest help to us has been advances in genetics, she said. I can remember when we would just diagnose them and say They have low muscle tone. Now, were able to identify genetic syndromes and where parts of a chromosome may be missing or duplicated.

When she was first starting out information was much more limited, Russell said.

Having genetic information helps you with set reasonable goals, she said. You may not be able to fix the underlying problem, but you know what to expect and to plan for.

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WVU Medicine Children's Dr. Mary Louise Russell brings 30-plus years experience in the treatment of children with movement issues - WV News

The mRNA vaccine pioneers are a married couple, Ugur Sahin and his wife Ozlem Tureci of Germany, who, after examining the viruss sequence last year, realized that their scientific team could come up with a vaccine to prevent its spread. They had spent years studying mRNA and successfully applying it to halt cancers and other maladies. They yielded results within weeks.

Fortunately, they were entrepreneurs as well as research scientists and turned their successful company, BioNTech, on a dime to execute their plans. A collaboration with Pfizer followed. In Massachusetts, Modernas science team also swung into action, using their expertise in mRNA biotech to rapidly develop another vaccine, which has proven 95 per cent effective in trials.

Both companies had already built platforms to create vaccines for any infectious disease by simply inserting the correct mRNA sequence to match the disease.

Now that the roll-out is underway, and a new mutation has come to light, the question is: will the pandemic end? The answer is yes and then some. There are five other vaccines undergoing final trials, which may advance the field more.

Most importantly, the world now has an mRNA template that can fast-track immunizations against viruses like COVID-19 and that will also be utilized to vaccinate against rare diseases, cancers and other major killers such as HIV, TB, malaria and rabies.

None of this is written in stone, but science marches on. So the good news is that the bad news of 2020 will come to an end, and a new era has begun that will transform health care for generations.

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Diane Francis: The development that will transform health care - Financial Post

Researchersarepoised to makeunprecedentedbreakthroughsinhuman health thanks toadvancesin biomedical and computational sciencesthathave drivencritical tools and technologiessuch as genetic engineering,synthetic biology, andartificial intelligence.

Thats the messageDr. VictorDzau, president oftheU.S.National Academy ofMedicine, delivered to Medicine by Designs fifthannual symposium on Dec. 7 and 8.

Thevirtual event, whichattracted more than 500 registrants from across North America, focused on the theme of better science throughconvergence theintegration of approaches from engineering, science, medicine and other fields to expand knowledge and spark innovation.

I think for younger people, there is really not a more exciting time, in my opinion, to do research than now, because we can really see that some of the initial concepts that people have about health and medicinecan be realizedand truly transform the way we do health andmedicine.

In his talk, Dzau focused on the National Academy of MedicinesHealthy Longevity Global Challenge,an international competition that aims to catalyze transformative ideas and breakthroughs that will extend human healthand lifespan.

That program is one of the inspirations for Medicine by DesignsGrand Questions Program, which seeks to fund bold research that promises dramatically better health outcomes by changing the future of regenerative medicine.

Through our Grand Questions Program, we are thinking about what comes next and how to overcome fundamental problems in regenerative medicine,saidMichael Sefton, executive director of Medicine by Design andUniversity Professorin the department of chemical engineering and applied chemistry and theInstitute of Biomedical Engineeringat the University of Toronto.

We have a broad definition of regenerativemedicine, andpreventing degeneration can be as important as the next cell therapy.

Sefton pointed out that the symposium theme of better science through convergencefocusedon a key aspect of Medicine by Design:That we combine campus and hospital investigators, transformative science and translational elements, junior and senior investigators, and local and international collaborators, to address fundamental problems in regenerative medicine.

Thesymposium also featured a talk byRobert Langer, David H. KochInstitute Professorin the department of chemical engineeringat the Massachusetts Institute of Technology. The most highly cited engineer in history, he spoke about lessons helearnedfromhisscientific and business successes and how he decidedto be his own champion after facing criticism for his novel ideas early in his career.

If you try to do things whether its convergence, or things that a lot of people disagree with you have tohang in there, Langer said.Having good intellectual property has been key toraising the funds to do these things, and medicine is an incredibly expensive thing.

And finally, you really need teams that are super driven, and I think these startup companies have been a wonderful way to do this.

The symposium was organized around four sessions: translation, inflammation, biomaterials andimmunoengineering.Invited speakers from across North AmericaincludedKim Warren(AVROBIO),Kenneth Walsh(University of Virginia),Sarah Heilshorn(Stanford University)andMegan Levings(University of British Columbia).

All speakers fromU of T and its partnerhospitals were lead investigators on Medicine by Designs multi-disciplinary, multi-institution team projects. They included:John Dick,Clinton RobbinsandShaf Keshavjee(University Health Network (UHN));Molly Shoichet(department of chemical engineering and applied chemistry and Institute of Biomedical Engineering);Juan CarlosZiga-Pflcker(Sunnybrook Health Sciences Centre);andAndras Nagy(Sinai Health System).

Ted Sargent, vice-president of research and innovation, and strategic initiatives,and a University Professor in the Edward S. Rogers Sr. department of electrical and computer engineering,opened the symposium by congratulatingMedicine by Design on its successful mid-term review, which was conducted in early 2020 by a panel of international experts and theCanada First Research Excellence Fund(CFREF), which funds Medicine by Design.

Medicine by Design has amplified existing areas ofexcellenceatU of Tandour partner hospitals (Toronto Academic Health Sciences Network),and pushed the boundaries of regenerative medicine to tackle cell-based therapies, strategies for endogenous repair and the use of a stem cell lens to target the triggers of disease,Sargent said. In fact, Medicine by Design is such a compelling collaborative, cross-disciplinary initiative that itis a template fora new class of initiatives at the University ofToronto theInstitutional Strategic Initiativesportfolio whosepurpose is to mobilize ambitious,groundbreaking, collaborative, multi-institutional research networks that tackleimportantresearch problems, buildmajorexternal partnershipsboth with industry and emerging companies as well as with global academic peers;and foster societal impact.

They support the pursuit of grand challenges and bold ideas across disciplinary boundaries,further elevate U of Ts profile in high priority research areas of strategic importance,and enable us to realize transformational impacts on issues of major societal import.

The symposium also offered an opportunity for almost 45trainees to present their research during a poster session.KerstinKaufmann, a post-doctoral fellow in the laboratory ofJohn Dick(Princess Margaret Cancer Centre,UHN), won first place.JonathanLabriola, apost-doctoral fellowinSachdev Sidhuslab(Donnelly Centre for Cellular and Biomolecular Research, U of T), placed second, whileSabihaHacibekiroglu, a post-doctoral fellow in the lab ofAndras Nagy(Lunenfeld-Tanenbaum Research Institute, UHN)placed third.The awards were sponsored by STEMCELL Technologies.

YasamanAghazadeh,a post-doctoral fellow in the labsofCristina Nostro(McEwen Stem Cell Institute, UHN)andSara Nunes Vasconcelos(Toronto General Hospital Research Institute,UHN),won theCCRMTranslation Awardfor the poster with the greatest translational potential.AndAi Tian, a post-doctoral fellow fromJulien Muffatslab (The Hospital for Sick Children), won thePeoples Choice Award, a new award this year that wasdetermined byvotingby symposium attendeesand sponsored byBlueRockTherapeutics.

Funded by a $114-million grant from CFREF, Medicine by Design brings together more than 145principal investigators at the University of Toronto and its affiliated hospitals to work at the convergence of engineering,medicineand science. It builds on decades of made-in-Canada excellence in regenerative medicine dating back to the discovery of stem cells in the early 1960s by Toronto researchers James Till andErnest McCulloch.

Regenerative medicine uses stem cells to replace diseased tissues and organs, creating therapies in which cells are the biological product. Regenerative medicine can also mean triggering stem cells that are already present in the human body to repair damaged tissues or to modulate immune responses. Increasingly, regenerative medicine researchers are using a stem cell lens to identify critical interactions or defects that prepare the ground for disease, paving the way for new approaches to preventing disease before it starts.

(Photo of Robert Langer by Jason Alden)

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Medicine by Design symposium highlights importance of convergence in regenerative medicine and human health - News@UofT

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The coronavirus pandemic crisis shows no signs of abating, even with a vaccine coming on to the markets. Were still facing severe social lockdown policies, with a number of states (such as California, Minnesota, and Michigan) forcing even harsher restrictions on this round than previously.Its a heavy blow for the leisure industry that is still reeling from one of the most difficult years in memory. The difficulties faced by restaurants are getting more press, but for the cruise industry, corona has been a perfect storm.Prior to the pandemic, the cruise industry which had been doing $150 billion worth of business annually was expected to carry 32 million passengers in 2020. Thats all gone now. During the summer, the industry reeled when over 3,000 COVID cases were linked to 123 separate cruise ships, and resulted in 34 deaths. After such a difficult year, its useful to step back and take a snapshot of the industrys condition. JPMorgan analyst Brandt Montour has done just that, in a comprehensive review of the cruise industry generally and three cruise line giants in particular."We believe cruise shares can continue to grind higher in the near term, driven overwhelmingly by the broader vaccine backdrop/progress. Looking out further, operators will face plenty of headwinds when restarting/ramping operations in 2Q3Q21, but significant sequential improvement of revenues/cash flows over that period will likely dominate the narrative, and we believe investors will continue to look through short-term setbacks to a 2022 characterized by fully ramped capacity, near-full occupancies, and so far manageable pricing pressure," Montour opined.Against this backdrop, Montour has picked out two stocks that are worth the risk, and one that investors should avoid for now. Using TipRanks Stock Comparison tool, we lined up the three alongside each other to get the lowdown on what the near-term holds for these cruise line players.Royal Caribbean (RCL)The second-largest cruise line, Royal Caribbean, remains a top pick for Montour and his firm. The company has put its resources into facing and meeting the pandemics challenges, shoring up liquidity and both streamlining and modernizing the fleet.Maintaining liquidity has been the most pressing issue. While the company has resumed some cruising, and has even taken delivery of a new ship, the Silver Moon, most operations remain suspended. For Q3, the company reported adjusted earnings of -$5.62, below consensus of -$5.17. Management estimates the cash burn to be between $250 million and $290 million monthly. To combat that, RCL reported having $3.7 billion in liquidity at the end of September. That included $3 billion in cash on hand along with $700 million available through a credit facility. Total liquidity at the end of Q3 was down more than 9% from the end of Q2. Since the third quarter ended, RCL has added over $1 billion to its cash position, through an issue of $500 million senior notes and a sale of stock, putting an additional 8.33 million shares on the market at $60 each.In his note on Royal Caribbean, Montour writes, [We] are most constructive on OW-rated RCL, which we believe has the most compelling set of demand drivers... its extensive investments in premium priced new hardware, as well as consumer data, all set RCL up well to outgrow the industry in revenue metrics, margins, and ROIC over the longer term.Montour backs his Overweight (i.e. Buy) rating with a $91 price target. This figure represents a 30% upside potential for 2021. (To watch Montours track record, click here)Is the rest of the Street in agreement? As it turns out, the analyst consensus is more of a mixed bag. 4 Buy ratings and 6 Holds give RCL a Moderate Buy status. Meanwhile, the stock is selling for $69.58 per share, slightly above the $68.22 average price target. (See RCL stock analysis on TipRanks)Norwegian Cruise Line (NCLH)With a market cap of $7.45 billion and a fleet of 28 ships, Norwegian Cruise Line found its relatively smaller size as an advantage in this pandemic time. With a smaller and newer fleet, overhead costs, especially ship maintenance, were lower. These advantages dont mean that the company has avoided the storm. Earlier this month, Norwegian announced a prolongation of its suspension of voyages policy, covering all scheduled voyages from January 1, 2021 through February 28, 2021, plus selected voyages in March 2021. These cancellations come as Norwegians revenues are down in the third quarter, the top line was just $6.5 million, compared to $1.9 billion in the year-ago quarter. The company also reported a cash burn of $150 million per month.To combat the cash burn and minimal revenues, Norwegian, in November and December, took steps to improve liquidity. The company closed on $850 million in senior notes, at 5.875% and due in 2026, during November, and earlier this month closed an offering of common stock. The stock offering totaled 40 million shares at $20.80 per share. Together, the two offerings raised over $1.6 billion in new capital.On a more positive note, Norwegian is preparing for an eventual resumption of full services. The company announced, on Dec 7, a partnership with AtmosAir Solutions for the installation of air purification systems on all 28 vessels of its current fleet, using filtration technology known to defeat the coronavirus.JPMs Montour points out these advantages in his review of Norwegian, and sums up the bottom line: This coupled with a relatively newer, higher-end, brand/ship footprint would generally lead us to believe it was in a good position to outperform on pricing growth, though its demographics skewing to older age customers probably will remain a drag through 2021. Ultimately, NCLH is a high-quality asset within the broader cruise industry, with a higher beta to a cruise recovery, and it should see outperformance as the industry returns and investors look further out the risk spectrum.Montour gives the stock a $30 price target and an Overweight (i.e. Buy) rating. His target implies an upside of 27% on the one-year time frame.Norwegian is another cruise line with a Moderate Buy from the analyst consensus. This rating is based on 4 Buys, 4 Holds, and 1 Sell set in recent months. Like RCL above, the stock price here, $23.55, is currently higher than the average price target, $23.22. (See NCLH stock analysis on TipRanks)Carnival Corporation (CCL)Last up, Carnival, is the worlds largest cruise line, with a market cap of $23.25 billion, more than 100 ships across its brands, and over 700 destination ports. In normal times, this giant footprint gave the company an advantage; now, however, it has become an expensive liability. This is clear from the companys fiscal Q3 cash burn, which approached $770 million.Like the other big cruise companies, Carnival has extended its voyage cancellations, or, in the companys terms, the pause in operations. The Cunard line, one of Carnivals brands, has cancelled voyages on the Queen Mary 2 and the Queen Elizabeth through early June of next year. Carnival has also cancelled operations in February from the ports of Miami, Galveston, and Port Canaveral, and pushed back the inaugural voyage of the new ship Mardi Gras to the end of April 2021. These measures were taken in compliance with coronavirus restrictions.Carnivals shares and revenues are suffering deep losses this year. The stock is down 60% year-to-date, despite some recent price rallies since the end of October. Revenues fell to just $31 million in the fiscal third quarter, reported in September. Carnival reported a loss of nearly $3 billion in that quarter. The company did end the third quarter with over $8 billion in available cash, an impressive resource to face the difficult situation.This combination of strength and weakness led Montour to put a Neutral (i.e. Hold) rating on CCL shares. However, his $25 price target suggests a possible upside of 23%.In comments on Carnival, Montour wrote, [We] believe that some of the same relative net yield drags it saw in 2018-2019 due to its sheer size will likely become top of mind on the other side of this crisis However, given CCLs relative share discount, less pricing growth ahead of the crisis, and geographical diversification, we see it as the company with the least downside over the next few months and are not surprised by its recent outperformance. We believe this will reverse in the 2H21. Overall, Carnival has a Hold rating from the analyst consensus. This rating is based on 10 reviews, breaking down to 1 Buy, 8 Holds, and 1 Sell. The stock is selling for $20.28 and its $18.86 average price target implies a downside potential of ~7%. (See CCL stock analysis on TipRanks)To find good ideas for stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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