StemCell Therapy

Company to hold conference call for analysts and investors today at 18:00 CET

Amsterdam, The Netherlands, April 30, 2020 Kiadis Pharma N.V.( Kiadis, Kiadis Pharma or the Company) (Euronext Amsterdam and Brussels: KDS), a clinical-stage biopharmaceutical company, today announces its audited 2019 Annual Results for the year ended December 31, 2019, which have been prepared in accordance with International Financial Reporting Standards (IFRS) as adopted by the European Union.

Arthur Lahr, CEO of Kiadis commented, 2019 was a transformational year for Kiadis with the acquisition of CytoSen Therapeutics in the first half; the termination of the ATIR101 development program in the second half; and the restructuring and refocus of our organization solely on Kiadis natural killer (K-NK) cell therapies in the fourth quarter. We were faced with some difficult decisions during the year, but we were always guided by our core values of always doing what is right and putting our patients first. In the face of adversity, I am proud of the decisions that our team has made and believe that we have emerged in 2020 as a stronger organization.

Key Developments (including post reporting period)

Revenue & Other IncomeThe Group did not record revenue and/or other income in 2019 and 2018.

Operating Expenses

OPERATING RESULTSAs a result of the overall increase in total operating expenses, the Group's operating loss increased from EUR25.2 million in 2018 to EUR73.2 million in 2019.

NET FINANCIAL RESULT

NET RESULTAs a result of the above items, the loss for the year increased by EUR22.8 million to EUR52.6 million in 2019 versus a loss of EUR29.8 million in 2018. CASH FLOWSTotal cash and cash equivalents decreased by EUR30.8 million from EUR60.3 million at year-end 2018 to EUR29.5 million at the end of 2019. This decrease mainly results from the net operating cash outflow amounting to EUR48.3 million, capital expenses of EUR4.5 million and repayments of outstanding loans of EUR5.7 million, offset by the net proceeds of a share offering for a total amount of EUR25.3 million and cash balances of CytoSen for an amount of EUR3.1 million, which we acquired on June 5, 2019.

EQUITYThe Company's equity position amounted to EUR34.3 million at year-end 2019 versus EUR44.1 million at the end of 2018, a decrease of EUR9.8 million. The main drivers of this decrease are the loss for the year of EUR52.6 million offset by net proceeds of a share offering of EUR25.3 million in total and shares issued upon the acquisition of a business combination.Earnings per shareThe undiluted loss per share for 2019 increased to EUR 1.92 compared to EUR 1.46 in 2018.

Annual Report

The Annual Report 2019 is available on Kiadis Pharmas website.

Conference Call and PresentationTo participate in the conference call, please call one of the following numbers ten minutes prior to commencement of the call:Standard International: +44 (0) 2071 928338Netherlands, Amsterdam: +31 (0) 207956614UK, London: +44 (0) 8444819752US, New York: +16467413167US, toll free: 18778709135

Event Plus Passcode: 4968027

A live audio webcast of the call can be accessed from the Events and Presentations section of the Companys website, https://ir.kiadis.com/events-and-presentations or at https://edge.media-server.com/mmc/p/6ctgdx37.

For more information, please contact:

About Kiadis Pharmas K-NK-Cell Therapies

Kiadis Pharmas K-NK platform is designed to deliver potent NK cells to help each patient, without the need for genetic engineering. Kiadis Pharmas programs consist of off-the-shelf and haploidentical donor NK-cell therapy products for the treatment of liquid and solid tumors as adjunctive and stand-alone therapies.

The Companys PM21 particle technology enables improved ex vivo expansion and activation of cytotoxic NK cells supporting multiple high-dose infusions. Kiadis Pharmas proprietary off-the-shelf NK-cell platform is based on NK cells from unique universal donors and can make NK-cell therapy product rapidly and economically available for a broad patient population across a potentially wide range of indications.

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Kiadis Pharma is developing K-NK002, which is administered as an adjunctive immunotherapeutic on top of HSCT, and K-NK003 for the treatment of relapse/refractory acute myeloid leukemia. In addition, Kiadis Pharma has pre-clinical programs evaluating NK-cell therapy for the treatment of solid tumors.

About Kiadis PharmaFounded in 1997, Kiadis Pharma is building a fully integrated biopharmaceutical company committed to developing innovative therapies for patients with life-threatening diseases. With headquarters in Amsterdam, the Netherlands, and offices and activities across the United States, Kiadis Pharma is reimagining medicine by leveraging the natural strengths of humanity and our collective immune system to source the best cells for life. The Companys shares are listed on the Euronext Amsterdam and Brussels under the ticker KDS. Learn more at http://www.kiadis.com.Kiadis Pharma is listed on the regulated market of Euronext Amsterdam and Euronext Brussels since July 2, 2015, under the symbol KDS. Learn more at kiadis.com.

Forward Looking StatementsCertain statements, beliefs and opinions in this press release are forward-looking, which reflect Kiadis Pharmas or, as appropriate, Kiadis Pharmas officers current expectations and projections about future events. By their nature, forward-looking statements involve a number of known and unknown risks, uncertainties and assumptions that could cause actual results, performance, achievements or events to differ materially from those expressed, anticipated or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, regulation, competition and technology, can cause actual events, performance, achievements or results to differ significantly from any anticipated or implied development. Forward-looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, Kiadis Pharma expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or projections, or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither Kiadis Pharma nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the anticipated or implied developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Kiadis Pharma announces annual results for the year ended December 31, 2019 - Yahoo Finance

A study completed by the University Hospitals at Leicester showed that heart function in patients with type 2 diabetes (T2D) may be improved more effectively through exercise training than through a weight loss regimen.

The study was funded by the National Institute for Health Research (NIHR) and similarly conducted at the NIHR Leicester Biomedical Research Centre (BRC).

Heart failure is one of the most common complications in people with type 2 diabetes, and younger adults with type 2 diabetes already have changes in their heart structure and function that pose a risk of developing heart failure, Gaurav Gulsin, a BHF clinical research fellow at the University of Leicester, trainee heart physician, and a lead author of the study, said We wanted to confirm the abnormalities in the structure and function of the heart in this patient population using the latest scanning techniques, and explore whether it is possible to reverse these through exercise and/or weight loss.

The randomized study incorporated a total of 87 participants, between 18 and 65 years of age, with type 2 diabetes, who underwent echocardiography and a magnetic resonance imaging (MRI) scan in order to verify their early heart dysfunction, as well as exercise tests to analyze cardiovascular fitness. Patients were randomly selected into 1 of 3 groups routine care, supervised aerobic exercise training, or low-energy meal replacement program all of which lasted for 12 weeks; 76 participants remained on the program for the entire duration of the study.

Researchers from the University Hospitals of Leicester reported that those who followed the supervised exercise program demonstrated significantly improved heart function compared with the control group.

The results also suggested that while a low-energy diet did not alter heart function, the program did show favorable effects on heart structure, vascular function, and diabetes reversal in 83% of the patient group.

Limitations of the study included small population size and failure of nearly 1 in 5 patients in the exercise arm of the study to complete the program, effectively restricting the studys application in future clinical practice.

Senior study author Gerry McCann, NIHR research professor and professor of cardiac imaging at the University of Leicester and a consultant cardiologist at Leicesters Hospitals said, Through this research we have shown that lifestyle interventions in the form of regular exercise training may be important in limiting and even reversing the damage to heart structure and function seen in younger adults with type 2 diabetes. While losing weight has a beneficial effect on heart structure, our study shows that on its own it does not appear to improve heart function.

The findings have been published in the journal Diabetes Care.

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Study: Exercise More Effective Than Weight Loss for Improving Heart Function in Diabetes - Drug Topics

An estimated 425 million people globally have diabetes, accounting for 12% of the world's health expenditure. Living with diabetes is no easy feat, for people with type one diabetes missing a meal or going for an unplanned walk could dangerously reduce their blood glucose level, and severe low blood sugar at night may mean that regaining consciousness is impossible - a daunting thought for people to have to face when they live alone

In recent years technological developments have progressed and an enhanced algorithm has been created which is available in the US and expected in Europe within months, and it allows the pump to act as an automated delivery system as it guides insulin dosing in response to changes in blood glucose levels. This development is hugely beneficial as type one diabetics can now wear two small interconnected electronic devices that have significantly reduced the risk of falling blood sugar levels at night. A continuous glucose monitor (CGM) can check their blood sugar levels in real-time and communicate with a programmable insulin pump that delivers tiny amounts of insulin into the tissue under their skin. The technology has continued to improve its ease of use; users now spend less time inputting details of what they are to eat and they are less vulnerable to the effects of stress, hormones, and alcohol on their blood sugar level. Yet the system isnt perfect - many users still report frequently waking up in the middle of the night from the CGM warning them of high blood sugar levels or that the pump needs to be topped up with insulin.

These kinds of systems help to regulate sugar levels, avoid highs and lows and make the users' lifestyle significantly easier by reducing the need for constant monitoring and top-ups of insulin. Technologies like this are provided by devices from companies such as Medtronic and Diabeloop, and by CamDiabs CamAPS FX an Android phone app designed to work with commercially available insulin pumps and CGMs.

As with all medical technological developments, each new generation of devices has to go through a lengthy process of regulatory approval, which has consequently driven some people with diabetes to build their own DIY systems, using algorithms to control insulin dosing based on data from their CGMs. These DIY systems are able to automate the majority of the calculations that each individual would normally do whilst measuring their blood sugar levels, to make predictions and adjust insulin dosing, not only does the use of an automated system reduce the adjustments that people have to make themselves and the mental strain that comes with living with diabetes but also minimizes the chances of wrong predictions or insulin dosing due to human error.

Despite the growth of automated systems for diabetes, widespread adoption of these systems seems to have plateaued, one reason being the expenses rising up to 3,000-4,500 a year taking acquisition, maintenance and the need to replace pumps every few years into account. Recently, more research has been done into creating a more cost-effective method for creating an automated diabetes kit. An example of this is the smartpen, which are handheld devices for injecting insulin record details about the time and quantity of a patients dose and transmitting them to a mobile phone app via Bluetooth, used in combination with a CGM, the app can them advise people with diabetes on the amount of insulin they should take, costing under 1000 a year.

But pumps, CGMs and pens involve repeated skin piercing. The answer to these issues seems to lie with the use of electrocardiogram signals to track glucose levels and by applying artificial intelligence algorithms to a heartbeat scan, machines using ECG are able to detect low glucose with accuracy to match a CGM monitor, whilst being non-invasive and environmentally friendly due to the minimal waste produced. Trials with diabetic patients are to begin soon and wearable ECG-based monitors could be on the market within a few years with huge potential to still grow and develop in order to ease human experience, possibly by being incorporated into smart fabrics, so there is no need to wear separate devices.

Predictive algorithms, artificial intelligence and machine learning are beginning to tackle the issues that diabetics face in their day to day lives, and the future prospects for a completely automated system are promising, potentially providing a risk-free future for people living with the disease.

Read more:
How Close Are We To Creating A Fully Automated Diabetes Kit? - AI Daily

Breaking News

R. Kelly has no shame ... he's making yet another plea to the judge to let him out of jail, but this time he claims he has proof he's vulnerable to COVID-19.

The disgraced singer's lawyer filed docs, obtained by TMZ, asking the judge to release his client pending his trial and claiming the Bureau of Prisons failed to disclose Kelly's medical tests from March showing he is "likely diabetic."

In docs, Kelly's lawyer also claims the singer has high cholesterol and high blood pressure ... further putting him in a vulnerable position if he contracts the deadly coronavirus while penned up at MCC Chicago.

The judge has not yet made a decision on whether to release Kelly. TMZ broke the story ... he swung and missed TWICE last month. During Kelly's first attempt, the judge denied him saying, among other things, the singer is a flight risk. Not long after, his team made a second attempt, but again, the judge said no dice.

The judge had previously expressed concern Kelly might intimidate witnesses if he's allowed out of jail. Kelly addresses that in the new docs, referring to one of his former girlfriends who used to visit him regularly in jail and correspond with him. He says during all those visits he was well aware she is one the Jane Doe victims in his case, and still never attempted to obstruct justice.

He doesn't name her, but our Kelly sources tell us he's talking about Azriel Clary, who's now working with the prosecution. Kelly says if he had tried to intimidate her, she would have reported him to prosecutors by now.

Normally, they say the third time's a charm, but R. Kelly's a different story ... for so many reasons.

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R. Kelly Says He's Likely Diabetic and Takes Third Shot at Jail Release - TMZ

When Ming L. Wang developed a new way to test glucose levels in saliva to monitor diabetes, he wasnt thinking of making COVID-19 tests.

But after he saw the lack of testing options in the U.S. as COVID-19 cases skyrocketed, Wang decided to turn his testing device into something people could use to track the spread of SARS-CoV-2, the coronavirus that causes the illness.

The current tests sometimes take too long, says Wang, distinguished professor of civil and environmental engineering at Northeastern. You need to take a few days, and sometimes do the tests many times to minimize false negatives.

Ming Wang is a distinguished professor of civil and environmental engineering at Northeastern. Photo courtesy Ming Wang

For nearly a decade, Wang has been perfecting his glucose testing device, which can perform quick and easy tests from saliva samples. That test kit uses a disposable chip equipped with sensors to detect glucose moleculesno finger pricks, doctors, or pain involved. It was patented in 2018 and is now being tested in preclinical trials, Wang says.

Now, Wang is reconfiguring the devices biosensor to test for SARS-Cov2 molecules in the saliva of people who carry the coronavirus and give an accurate diagnosis within three minutes of testing.

Wang says that device is intended to work even in the early stages of infection, before the onset of COVID-19 symptoms. And it is designed to detect antibodies in an effort to help determine whether someone who has recovered from the disease might be protected from it in the future.

Thats very important, Wang says. You need to know who has had it to test someones immunity after contracting the virus.

Testing during the early stages of infection is critical, Wang says, because reports have shown that people can shed the coronavirus before showing some of the most common symptoms of COVID-19, even without ever displaying them.

Wangs tests are intended to be accurate enough to be used in healthcare facilities, but also practical enough to be used at home and without the need for healthcare personnel.

As scientists around the world speed up their research to learn more about the unknowns behind the coronavirusabout immunity and transmission, for exampleWang says rapid home tests can help people answer one of the most important questions of the COVID-19 pandemic: How do I know if Ive got the virus?

Recent estimates in the U.S. suggest that the coronavirus has been spreading far more widely and efficiently than health officials thought, suggesting it moves silently amongst people who havent yet developed symptoms. Those estimates underscore the importance of having a quick and accurate tool that people can use at home, Wang says.

The biosensor within the device is designed to use gold nanoparticles to read tiny signals produced as the coronavirus interacts with key protein molecules to hijack human cells and replicate into millions more of itself. The sensor then reads the signals released by those interactions to detect the coronavirus.

Wang says he is focusing on sampling saliva because the method produces fast results without being invasive.

The most common tests for COVID-19 first insert a cotton swab into a persons throat, lungs, or nose to sample the genetic material of the coronavirus, and then rely on a technique called polymerase chain reaction, which involves making millions of copies of the genetic material to determine the presence of the coronavirus. The entire process can take several hours or days to complete.

And while new methods have been recently developed to help healthcare personnel test for the coronavirus using saliva, the key is in using a simple electronic device that relies on the chemistry within saliva to test in minutes and without specialized biotechnicians, Wang says.

You could use it for port entry facilities, you could use it for physician offices, urgent care centers, nursing facilities, he says. It takes about two-three minutes to complete, and about $10 per test.

Wangs coronavirus testing kits will take at least 6 months to develop. Still, he is hopeful they will come out during a time when there is an urgent need for new technology that can serve as the basis for future tests to help prevent other global health emergencies such as COVID-19or recurring ones.

We could be expecting the second or third run of COVID-19 to come next winter in 2021, maybe up to 2022 or beyond, Wang says. Once this platform is set and we can use it, we can redesign the sensor for other viral infections.

For media inquiries, please contact Jessica Hair at j.hair@northeastern.edu or 617-373-5718.

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Can a new tool for diabetes patients solve the problem of COVID-19 testing? - News@Northeastern

"Coronavirus stocks," the stocks of companies that make products and provide services that are experiencing strong demand stemming from the COVID-19 pandemic, have been getting a ton of attention. (Indeed, like many financial writers, I recently wrote an article on the topic, outlining8 top coronavirus stocks.)

However, some stocks that don't fall into this category have also been strong performers in recent months, as well as over the long term. For instance, you'd have more than doubled your money had you invested in these "non-coronavirus stocks" a year ago: diabetes specialists Insulet and DexCom and real estate investment trust Safehold.

Image source: Getty Images.

Company

Market Cap

Forward P/E

Projected Annualized5-Year EPS Growth*

YTD 2020 Return (Loss)

1-Year Return

10-Year Return

S&P 500

Data sources: Yahoo! Finance and YCharts. Data as of April 30, 2020. P/E = price-to-earnings ratio. EPS = earnings per share. YTD = year to date. *Wall Street's consensus estimate.

If you're looking for a growth market in which to invest, I'd suggest the diabetes space. Unfortunately, the incidence of the disease (both type 1 and type 2) has been rising around the world. Indeed, many healthcare experts consider diabetes an epidemic in the United States and many other countries.

Two top names in the diabetes space are medical-device makers Insulet and DexCom. Insulet makes the leading tubeless insulin pump, the Omnipod, and DexCom produces acontinuous glucose monitoring (CGM) system, the G6.

In fact, these two companies are partners. In the second half of this year,Insulet plans to launch its Horizon automated insulin delivery system, which uses aDexCom CGM. Moreover, the Horizon system's insulin pump will be controlled by a smartphone.

On Tuesday, DexCom reported its first-quarter 2020 results, which crushed Wall Street's expectations. Sales rose 44% year over year to $405.1 million, sailing by the$357.6 million analyst consensus estimate. Adjusted earnings per share (EPS) landed at $0.44, compared to the year-ago period's loss of $0.05 per share. This result demolished the consensus estimate of $0.14.

Insulet is scheduled to report first-quarter results on Thursday, May 7, after market close.

Image source: Getty Images.

Safehold is a real estate investment trust (REIT) that specializes in commercial property (but no retail) ground leases in the 25 largest markets in the U.S.It buysthe land underlying commercial real estate projects, which it leases back to the owners of the structures on the land. The company uses the rental income to grow the business and pay shareholders a modest dividend, currently yielding 1.2%.

Safehold was founded in 2016 andheld itsinitial public offeringin 2017, so it's relatively small for a REIT and doesn't have much of a track record.That said, it seems worth watching.

Like most companies, Safehold expects to have some near-term pain due to the pandemic. By the end of the first quarter, deals were largely on hold across the commercial real estate market. However, management also believes the company should bounce back quickly once the dust settles a bit. Here's what CEO Jay Sugarman had to say during the April 23 first-quarter earnings call:

We continue to be engaged in conversations with customers on a number of fronts, and would expect our better price, more efficientcapital to be in demand once there's more clarity about the future. We expect deals put on hold may very well come back, and thatexisting customers will find the opportunities to deploy capital and seek our help in capitalizing those opportunities. ...

As for the existing portfolio, all of our ground leases paid [their rent] in April.

In the first quarter, Safehold's revenue soared 84% year over year to $40.2 million. Net income jumped 56% to $17.4 million, which translated to earnings per share coming in flat with the year-ago period at $0.36. (The number of shares outstanding increased, which is why EPS was only flat while net income rose significantly.)

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3 "Non-Coronavirus Stocks" That Would Have Doubled Your Money Over the Last Year - Motley Fool

F. Hoffmann-La Roche Ltd

Global Type 2 Diabetes Treatment Market: Competitive Landscape

This section of the report lists various major manufacturers in the market. The competitive analysis helps the reader understand the strategies and collaborations that players focus on in order to survive in the market. The reader can identify the players fingerprints by knowing the companys total sales, the companys total price, and its production by company over the 2020-2026 forecast period.

Global Type 2 Diabetes Treatment Market: Regional Analysis

The report provides a thorough assessment of the growth and other aspects of the Type 2 Diabetes Treatment market in key regions, including the United States, Canada, Italy, Russia, China, Japan, Germany, and the United Kingdom United Kingdom, South Korea, France, Taiwan, Southeast Asia, Mexico, India and Brazil, etc. The main regions covered by the report are North America, Europe, the Asia-Pacific region and Latin America.

The Type 2 Diabetes Treatment market report was prepared after various factors determining regional growth, such as the economic, environmental, technological, social and political status of the region concerned, were observed and examined. The analysts examined sales, production, and manufacturer data for each region. This section analyzes sales and volume by region for the forecast period from 2020 to 2026. These analyzes help the reader understand the potential value of investments in a particular country / region.

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Key Benefits for Stakeholders:

The report provides an in-depth analysis of the size of the Type 2 Diabetes Treatment world market, as well as recent trends and future estimates, in order to clarify the upcoming investment pockets.

The report provides data on key growth drivers, constraints and opportunities, as well as their impact assessment on the size of the Type 2 Diabetes Treatment market.

Porters 5 Strength Rating shows how effective buyers and suppliers are in the industry.

The quantitative analysis of the Type 2 Diabetes Treatment world industry from 2020 to 2026 is provided to determine the potential of the Type 2 Diabetes Treatment market.

This Type 2 Diabetes Treatment Market Report Answers To Your Following Questions:

Who are the main global players in this Type 2 Diabetes Treatment market? What is the profile of your company, its product information, its contact details?

What was the status of the global market? What was the capacity, the production value, the cost and the profit of the market?

What are the forecasts of the global industry taking into account the capacity, the production and the value of production? How high is the cost and profit estimate? What will be the market share, supply, and consumption? What about imports and export?

What is market chain analysis by upstream raw materials and downstream industry?

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Tags: Type 2 Diabetes Treatment Market Size, Type 2 Diabetes Treatment Market Trends, Type 2 Diabetes Treatment Market Growth, Type 2 Diabetes Treatment Market Forecast, Type 2 Diabetes Treatment Market Analysis

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Type 2 Diabetes Treatment Market Growth Opportunities, Challenges, Key Companies, Drivers and Forecast to 2026 Cole Reports - Cole of Duty

The team that developed Zolgensma is back for round two. A group of former AveXis executives and investors unveiled a new gene therapy company, and theyre wasting no time. With 15 programs, $30 million in seed funding and an unrivaled partnership with UT Southwestern Medical Center, Taysha Gene Therapies plans to be in the clinic by the end of the year.

Its working on adeno-associated vector (AAV) gene therapies for monogenic diseasesthat is, diseases caused by a defect in a single geneof the central nervous system. Tayshas lead program targets GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord, but the companys portfolio includes conditions that are more prevalent.

The company plans to start clinical trials for three more programs by the end of 2021: treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and a SLC6A1 genetic epilepsy, which is similar to Dravet syndrome, Taysha CEO and co-founder R. A. Session II told FierceBiotech.

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And thats not allTaysha has the option to pick up four more prospects from UT Southwestern.

The reason why we can tackle 15 programs with the option to four additional programs is were able to focus on what we do best and were able to allow our collaborators to focus on what they do best, Session said.

Under their partnership, UT Southwestern is working on discovery and preclinical work all the way through IND-enabling studies. It has its own GMP viral manufacturing site to support that work, as well as clinical development, which Taysha will pick up. The company will also take care of regulatory strategy, commercial manufacturing and commercialization. Its a skill set that Tayshas management team honed at AveXis as itdeveloped the gene therapy that eventually became Novartis spinal muscular atrophy treatment Zolgensma.

We essentially flew the plane and built it at the same time when we were developing AveXis We have people with the experience of being able to develop, manufacture and commercialize a gene therapy program and were marrying that with a best-in-class academic research institution, Session said.

RELATED: FDA lets Novartis off the hook in Zolgensma data manipulation

Dividing the labor creates this engine for innovation that allows the partners to advance many programs in parallel, Session added. With about 50 people in its gene therapy unit, UT Southwestern can develop a capsidthe protein shell of a virusfor treatment delivery or get a candidate into animal models much more quickly than a biotech going it alone, he said. The same goes for late-stage development and commercialization on Tayshas side.

For its initial programs, Taysha is focusing on AAV gene therapies because the team knows they work.

AAV9 is the best way we have to treat monogenic CNS disease. If its not broke, dont fix it, Session said. We know how to effectively dose AAV9; we know its safe, effective and efficient. And we know its scalable. These are problems we had to solve at our previous company.

But, moving forward, Taysha and UT Southwestern are working on new technologies, including an AAV delivery platform that would allow for the redosing of gene therapies as well as an AAV capsid platform aimed at improving target delivery.

The rest is here:
Taysha Gene Therapies hits the ground running with $30M, 15 programs - FierceBiotech

R.A. Session II, president, CEO, and founder of Taysha Gene Therapies [Background image: Olena Yepifanova via iStock]

Dallas-based biotech Taysha Gene Therapies has emerged out of stealth with a $30 million seed round and a new strategic partnership with UT Southwestern. The company aims to end severe and life-threatening diseases of the central nervous system caused by variation in a single gene, also known as monogenic CNS diseases.

Hitting the ground running, the company launches with a pipeline of 15 adeno-associated virus gene therapy programs, along with options to four others.

Taysha plans human testing this year and expects to file four Initial New Drug applications in 2021. In addition, the biotech is developing an improved treatment delivery platform that uses machine learning, DNA shuffling, and something it calls directed evolution.

The gene therapy startup closed its round of seed financing in early March just as the Dow dropped a couple of thousand points, according to a report in EndpointsNews.

If you had to ask me, was I worried? Absolutely. I think I wouldnt be human if I wasnt, RA Session II, president, CEO, and founder of Taysha, told the healthcare publication.

Session had a lot of certainty to balance out all the uncertainty of launching a new biotech during a pandemic, noted Endpoints Amber Tong.

The experience of a proven management team, including some who previously led the development and commercialization of the first FDA-approved gene therapy for CNS disease, Zolgensma, is part of the equation.

The startup intends to combine the speed, scale, and expertise of its partners UT Southwestern Gene Therapy Program with the experience of the Taysha team.

Session says the launch reunites former investors and executives from AveXis. Tayshas funding round was co-led by PBM Capital, the first institutional investor in AveXis, and Nolan Capital, the investment fund of former AveXis CEO Sean Nolan.

Tayshas Board of Directors played a key role in the formation of the company, it said. The board is comprised of Sean Nolan, Paul Manning of PBM Capital, Claire Aldridge, Ph.D., of UT Southwestern, and RA Session II, President, CEO, and Founder of Taysha.

Nolan, who serves as Chairman of the Board, sees promise in Tayshas approach for patients suffering from many devastating CNS diseases.

This is an exciting time for gene therapy, he said.

The strategic partnership with UT Southwestern could help Taysha to rapidly and efficiently translate novel AAV gene therapies from bench to bedside, the company said.

UTSW is home to some of the brightest minds in gene therapy, Sessions says. By joining forces with UT Southwestern, Taysha can advance its potential cures with both speed and scale.

The collaboration is ultimately creating an engine for new cures, said Claire Aldridge, Ph.D., a Taysha board member and associate vice president of commercialization and business development at UT Southwestern Medical Center.

Together, Taysha and UTSW will merge cutting-edge translational research, hands-on clinical care, and expertiseboth regulatory and commercial, she said.

Aldridge has already witnessed how quickly and efficiently the partners can leverage their collective expertise and resources. That means getting new gene therapies to the patients who so desperately need them, she said.

The UT Southwestern Gene Therapy Program, led by Steven Gray, Ph.D., Director of the Viral Vector Core and Assistant Professor in the Department of Pediatrics, and Berge Minassian, M.D., Division Chief of Child Neurology, has the capacity to support Tayshas wide range of preclinical and clinical development programs, the company said.

Under the partnership, UT Southwestern, which has developed a state-of-the-art viral vector manufacturing facility, will run discovery and preclinical research, as well as lead studies, provide manufacturing, and execute natural history studies.

For its part, Taysha will lead all clinical development, regulatory strategy, commercial manufacturing, and commercialization activities, according to the company.

A joint steering committee of key leadership members from Taysha and UT Southwestern will govern the collaboration.

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Read the original here:
Dallas Gene Therapy Startup Launches with $30M Seed Round and a UT Southwestern Partnership - dallasinnovates.com

Biotech executive, Julia Berretta, Ph.D., is named Chief Executive Officer

Genespire, a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, announced today the successful close of a 16M Series A financing from Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan. The company also announced the appointment of Julia Berretta, Ph.D., as Chief Executive Officer and member of the Board of Directors. Graziano Seghezzi, Managing Partner at Sofinnova Partners, and Lucia Faccio, Ph.D., Partner at Sofinnova Partners, will also join the Board.

Genespire was founded in March 2020 as a spin-off of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), one of the worlds leading cell and gene therapy research institutes spearheaded by gene therapy pioneer Prof. Luigi Naldini. The Company was co-founded by Fondazione Telethon and the San Raffaele Hospital, along with Prof. Naldini and Dr. Alessio Cantore.

The funds will be used to advance Genespires leading-edge platform technologies towards the development of novel gene therapies in two main areas: primary immunodeficiencies and metabolic genetic diseases.

"Our mission has always been to develop breakthrough solutions for genetic diseases," said Prof. Naldini, Genespires co-founder and Director of SR-Tiget. "This financing enables the company to translate our innovative science and early stage programs into clinical development. The appointment of Dr. Berretta as CEO is a major reinforcement of our team."

Sofinnova Partners Dr. Faccio added, "Genespire is an exciting investment with all the key ingredients for success: Outstanding scientists that developed the first ex-vivo gene therapy to market, experienced executives brought in through Sofinnova Partners network and game changing technologies that have the potential to impact the lives of patients with genetic diseases."

"I am thrilled to be joining Genespire and such exceptional scientific founders," said Dr. Berretta. "Genespire was born of decades of experience in the gene therapy field, and is optimally positioned to advance transformative therapies for patients affected by severe inherited diseases."

Dr. Berretta was part of the Executive Committee of Cellectis S.A., a Nasdaq-listed clinical stage gene editing company developing CAR-T cell therapies for cancer, where she led business development as well as strategic planning. She is also an independent Board member of Treefrog Therapeutics, an innovative stem cell company.

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. http://www.genespire.com

About Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public companies. It has backed nearly 500 companies over more than 48 years, creating market leaders around the globe. Today, Sofinnova Partners has over 2 billion under management.

For more information, please visit: http://www.sofinnovapartners.com

About Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit: http://www.telethon.it

Story continues

About Ospedale San Raffaele

Ospedale San Raffaele (OSR) is a clinical-research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. OSR is part of Gruppo San Donato, the leading hospital group in Italy. The hospital is a multi-specialty center with over 60 clinical specialties; it is accredited by the Italian National Health System to provide care to both public and private, national and international patients. Research at OSR focuses on integrating basic, translational and clinical activities to provide the most advanced care to our patients. The institute is recognized as a global authority in molecular medicine and gene therapy, and is at the forefront of research in many other fields. Ospedale San Raffaele is a first-class institute which treats many diseases and stands out for the deep interaction between clinical and scientific area. This makes the transfer of scientific results from the laboratories to the patients bed easier. Its mission is to improve knowledge of diseases, identify new therapies and encourage young scientists and doctor to grow professionally. For more information, please visit: http://www.hsr.it

About the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200429005417/en/

Contacts

Julia BerrettaCEO, Genespire S.r.linfo@genespire.com +39 02 83991300

Bommy LeeHead of Communications, Sofinnova Partnersblee@sofinnovapartners.com +33 (0) 6 47 71 38 11

North AmericaRooneyPartners LLCKate Barrettekbarrette@rooneyco.com +1 212 223 0561

FranceStrategiesImage (S&I)Anne Reinanne.rein@strategiesimage.com +33 6 03 35 92 05

Originally posted here:
Genespire Secures 16 Million Series A Financing from Sofinnova Partners to Advance Transformative Gene Therapies - Yahoo Finance

The Paris-based BrainVectis, a biotech developing gene therapies for Huntingtons disease and Alzheimers disease, has been acquired by the US clinical-stage gene therapy company AskBio.

AskBios gene therapy experience and manufacturing capacity will help to accelerate BrainVectis lead candidate for Huntingtons into phase I testing. In return, AskBio gets to expand its list of target indications.

AskBio recognized the scientific merit of the work at BrainVectis as an opportunity to strengthen our central nervous system clinical pipeline, Robin Fastenau, VP of Communications for AskBio, told me. No financial details about the acquisition were disclosed.

BrainVectis lead candidate focuses on increasing the expression of a protein called CYP46A1. This enzyme is key for turning excess cholesterol into a derivative that can be cleared from the brain into the blood. It is also reduced in Huntingtons and Alzheimers patients, allowing toxic levels of cholesterol to build up in the brain. By increasing the levels of this enzyme, BrainVectis aims to restore normal cholesterol metabolism and improve the clinical outcome.

So far, BrainVectis lead gene therapy candidate has shown proof-of-concept in animal models of Huntingtons. It also received orphan drug designation from the European Commission last year.

According to Nathalie Cartier-Lacave, CEO and Founder of BrainVectis, AskBio offers a strong cell-line manufacturing process. In particular, it can manufacture a range of viral vectors, including BrainVectis vector of choice: the adeno-associated virus.

This powerful adeno-associated virus technology and Askbios expertise in clinical applications will allow us to rapidly go to clinical application in patients, Cartier-Lacave told me.

There are currently no approved treatments able to slow down the progress of Huntingtons and Alzheimers. Combined with aging populations in developed countries, these debilitating diseases are creating a healthcare challenge. Many companies are trying and sometimes failing to develop drugs able to stop the progression of Alzheimers, for example, as such a drug could make a huge impact on society.

According to Cartier-Lacave, Huntingtons disease is the first target for the company going forward. The disease is caused by a mutation in a gene called HTT that is important for the function of nerve cells. Its a very severe disease for which we think the treatment may not only decrease the toxic mutated protein, but also preserve neurons from death, she added.

Gene therapies are becoming ever more popular in the biotech industry for their potential to tackle previously incurable conditions. BrainVectis is one of several companies aiming gene therapies at the brain; another is the French company Lysogene. However, there are major challenges with developing gene therapies for the brain, for example, getting the therapy past the blood-brain barrier and into the brain tissue.

AskBio might be up to the challenge, as it has taken several gene therapies to the clinic. Its proprietary treatment for the neuromuscular indication Pompe disease is currently in phase I/II, and it has licensed its technology to several big pharmaceutical companies. Those currently in clinical development include treatments for Duchenne muscular dystrophy (Pfizer), hemophilia (Takeda), and spinal muscular atrophy (AveXis), which was approved by the FDA last year, and is awaiting marketing approval by the EMA.

Image from Shutterstock

More:
AskBio Takes Over French Gene Therapy Company to Treat Alzheimers and... - Labiotech.eu

Hours after Gilead announced that an NIH trial testing their antiviral drug remdesivir in Covid-19 patients had succeeded, NIAID director Anthony Fauci sat on a couch in the Oval Office and gave the world the top-line readout.

The drug induced a 31% improvement on the primary endpoint of time to recovery: 11 days in the drug arm compared to 15 days in the placebo arm, he said, adding that patients taking the drug appeared less likely to die, with an 8% mortality rate in the drug arm compared to 11% in patients given the placebo.

The mortality data were not yet statistically significant, he cautioned but were trending in the right direction. Fauci, surrounded by President Trump, Vice President Mike Pence and several other advisors, said the news was a very optimistic sign in the hunt for treatments to fight the virus.

Although a 31% improvement doesnt seem like a knockout 100%, it is a very important proof of concept, he said. Because what it has proven, is that a drug has blocked this virus.

Fauci said more details would come and that the study would be submitted to a peer-reviewed journal. Trump, who deferred to Fauci in giving the readout, echoed Faucis commentary.

Its a beginning, that means you build on it, Trump said. But its a very positive event.

Shortly after the briefing, the New York Times reported that the FDA was preparing to issue an emergency use authorization for the drugs use in Covid-19. In an email to Endpoints News, the FDA did not confirm or deny the Times report, but a spokesperson said the agency has been engaged in sustained and ongoing discussions with Gilead Sciences regarding making remdesivir available to patients as quickly as possible, as appropriate.

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In the heart of Texas, the AveXis crew gathers for another go at gene therapy with $30M and a powerhouse academic pact to start - Endpoints News

CDMO Fujifilm Diosynth Biotechnologies (FDB) says partnering with OXGENE could reduce the lead time of its customers gene therapy projects by up to 25%.

The technology, licensed from UK-based OXGENE for an undisclosed fee, consists of Helper, Rep/Cap and Gene of Interest plasmids, used in combination with a clonal suspension a HEK293 cell line.

The AAV system is expected to reduce the length of the supply chain gene therapy customers, according to contract development and manufacturing organization (CDMO) FDB, with the standard lead-time from the start of process development to the first GMP manufacture potentially reduced by three to six months.

Image: iStock/PashaIgnatov

FDB will manufacture and stock a supply of Helper and Rep /Cap plasmids for clients engaged in process development and GMP manufacture of gene therapy programs. Gene of Interest (GOI) plasmid manufacture will be performed in-house using FDBs existing microbial capabilities and facilities at its site in College Station, Texas with cGMP manufacture planned to commence in Q4 2020.

The site is subject to numerous investments by the CDMO, the latest a $35 million expansion adding cell culture and high throughput manufacturing suites.

OXGENEs AAV system is superior to off-the-shelf plasmid systems for AAV titers, said Andy Topping, chief scientific officer at FDB, adding the agreement gives the CDMO plasmid to drug product capability for AAV systems and allows clients to avoid delays associated with GMP production of plasmids.

UK-based OXGENE was founded as a plasmid catalog business eight years ago. Sophie Lutter, scientific marketing and communications manager, told this publication how the firms technology works at the Phacilitate conference in January.

We start with custom plasmid design and engineering we have plasmid sets optimized for AAV and lentiviral production and then we pair that with our GMP-banked clonal suspension HEK293 cell lines and engineered derivatives, she explained. We take them through to process development, where we can support scales of up to 10 L.

With downstream purification as part of its platform, the company offers a full viral-vector package. This leaves the customer not only with the final viral vector, but also [with] the processes and protocols to take that through to GMP manufacture.

Read more:
Fujifilm licenses AAV tech to speed gene therapies - Bioprocess Insider - BioProcess Insider

Global CNS Gene Therapy Market Analysis

Persistence Market Research, in a recently published market study, offers valuable insights related to the overall dynamics of the CNS Gene Therapy market in the current scenario. Further, the report assesses the future prospects of the CNS Gene Therapy by analyzing the various market elements including the current trends, opportunities, restraints, and market drivers. The COVID-19 analysis section within the report offers timely insights regarding the impact of the global pandemic on the market. The presented study also offers data regarding the business and supply chain continuity strategies that are likely to assist stakeholders in the long-run.

As per the report, the CNS Gene Therapy market is set to grow at a CAGR of ~XX% over the forecast period (2019-2029) and exceed a value of ~US$ XX by the end of 2029. Some of the leading factors that are expected to drive the growth of the market include, focus towards research and development, innovations, and evolving consumer preferences among others.

Request Sample Report @ https://www.persistencemarketresearch.co/samples/27514

Regional Outlook

The report scrutinizes the prospects of the CNS Gene Therapy market in different geographical regions. The scope of innovation, consumer behavior, and regulatory framework of each region is thoroughly analyzed in the presented study.

Distribution-Supply Channel Assessment

The report provides a thorough analysis of the different distribution channels adopted by market players in the global CNS Gene Therapy market along with the market attractiveness analysis of each distribution channel. The impact of the COVID-19 pandemic on the different distribution channels is enclosed in the report.

Product Adoption Analysis

key players and product offerings

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The report aims to address the following pressing questions related to the CNS Gene Therapy market:

Key Takeaways from the CNS Gene Therapy Market Report

For any queries get in touch with Industry Expert @ https://www.persistencemarketresearch.co/ask-an-expert/27514

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CNS Gene Therapy Market to Slip Due to Delays in Production Amidst Coronavirus Outbreak Cole Reports - Cole of Duty

BOSTON and LONDON, May 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will host a conference call and live webcast on Thursday, May 7, 2020, at 8:00 a.m. ET to report its first quarter 2020 financial results and other business updates.

A live webcast will be available under "News & Events" in the Investors & Media section of the company's website at orchard-tx.com. The conference call can be accessed by dialing (866) 987-6504 (U.S. domestic) or +1 (602) 563-8620 (international) and referring to conference ID 8348144. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Contacts

Investors

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

Excerpt from:
Orchard Therapeutics to Webcast Conference Call of First Quarter 2020 Financial Results - GlobeNewswire

Documenting the Industry Development of Gene Therapy Market concentrating on the industry that holds a massive market share 2020 both concerning volume and value With top countries data, Manufacturers, Suppliers, In-depth research on market dynamics, export research report and forecast to 2029

As per the report, the Gene Therapy Market is anticipated to gain substantial returns while registering a profitable annual growth rate during the predicted time period.The global gene therapy market research report takes a chapter-wise approach in explaining the dynamics and trends in the gene therapy industry.The report also provides the industry growth with CAGR in the forecast to 2029.

A deep analysis of microeconomic and macroeconomic factors affecting the growth of the market are also discussed in this report. The report includes information related to On-going demand and supply forecast. It gives a wide stage offering numerous open doors for different businesses, firms, associations, and start-ups and also contains authenticate estimations to grow universally by contending among themselves and giving better and agreeable administrations to the clients. In-depth future innovations of gene therapy Market with SWOT analysis on the basis Of type, application, region to understand the Strength, Weaknesses, Opportunities, and threats in front of the businesses.

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***[Note: Our Complimentary Sample Report Accommodate a Brief Introduction To The Synopsis, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology are also Included]

An Evaluation of the Gene Therapy Market:

The report is a detailed competitive outlook including the Gene Therapy Market updates, future growth, business prospects, forthcoming developments and future investments by forecast to 2029. The region-wise analysis of gene therapy market is done in the report that covers revenue, volume, size, value, and such valuable data. The report mentions a brief overview of the manufacturer base of this industry, which is comprised of companies such as- Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc, Advantagene.

Segmentation Overview:

Product Type Segmentation :

Ex vivo, In Vivo

Application Segmentation :

Cancer, Monogenic, Infectious disease, Cardiovascular disease

To know more about how the report uncovers exhaustive insights |Enquire Here: https://market.us/report/gene-therapy-market/#inquiry

Key Highlights of the Gene Therapy Market:

The fundamental details related to Gene Therapy industry like the product definition, product segmentation, price, a variety of statements, demand and supply statistics are covered in this article.

The comprehensive study of gene therapy market based on development opportunities, growth restraining factors and the probability of investment will anticipate the market growth.

The study of emerging Gene Therapy market segments and the existing market segments will help the readers in preparing the marketing strategies.

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The market review for the global market is done in context to region, share, and size.

The important tactics of top players in the market.

Other points comprised in the Gene Therapy report are driving factors, limiting factors, new upcoming opportunities, encountered challenges, technological advancements, flourishing segments, and major trends of the market.

Check Table of Contents of This Report @https://market.us/report/gene-therapy-market//#toc

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Gene Therapy Market | on(impact of COVID-19) 2020-2029 Analysis on Growth, Future Demand 2020 - Jewish Life News

You can't lump all clinical-stage biotechs together. TakeCara Therapeutics (NASDAQ:CARA) andSangamo Therapeutics (NASDAQ:SGMO), for example. The two biotechs are about as different as night and day. One thing that both Cara and Sangamo have in common, though, are promising pipeline candidates.

Investors have placed a greater value on Sangamo's pipeline based on the company's higher market cap. But which of these biotech stocks is the better pick for long-term investors? Here's how Cara and Sangamo stack up against each other.

Image source: Getty Images.

All of Cara Therapeutics' hopes ride on one drug -- Korsuva. But that one drug has multiple formulations and multiple opportunities.

The most promising of these opportunities is for Korsuva injection in treating chronic kidney disease-associated pruritis (CKD-aP) in patients on hemodialysis. The itching (pruritis) experienced by many patients with CKD is very problematic. So far, there haven't been any approved treatments for CKD-aP.

That could soon change. Last week, Cara and its partner, Vifor Fresenius Medical Care Renal Pharma, announced positive results from a global late-stage study of Korsuva injection in treating CKD-aP in patients on hemodialysis. These results reinforced positive results reported last year from a U.S. study. The two companies plan to file for U.S. regulatory approval for Korsuva injection in the second half of 2020, followed closely by filing for European approval of the drug.

Cara anticipates completing an interim statistical analysis within the next couple of months for a phase 2 study evaluating an oral version of Korsuva in treating pruritis in patients with atopic dermatitis. The biotech is also conducting another phase 2 study of oral Korsuva in treatingpatients with pruritus and hepatic impairment due to primary biliary cholangitis (PBC).

Oral Korsuva should advance to a late-stage study in treating CKD-aP in patients who aren't on hemodialysis later this year. Cara reported results from a phase 2 study in December with the drug meeting its primary endpoint but failing to meet two secondary endpoints.

Korsuva injection could achieve peak annual sales topping $500 million. An oral version of the drug could boost that number considerably. With Cara Therapeutics' market cap currently below $750 million, the stock should have plenty of room to run if Korsuva wins regulatory approvals and reaches its commercial potential.

Unlike Cara Therapeutics, Sangamo isn't hanging its hat on only one candidate. The biotech's pipeline includes five different therapies in clinical testing. However, none of these programs are yet in late-stage testing.

Sangamo specializes in gene editing using a technique known as zinc finger nuclease (ZFN) technology and in developing gene therapies. The company's approach has captured attention from several big drugmakers.

Pfizeris partnering with Sangamo on developing SB-525, a gene therapy targeting hemophilia A. Sanofiteamed up with Sangamo on two gene-edited cell therapies in clinical studies. ST-400 targets treatment of rare blood disease beta-thalassemia, while BIVV-003 targets treatment of sickle cell disease.

Sangamo also has two wholly owned programs in phase 1/2 studies. Gene therapy ST-920 targets Fabry disease. ZFN gene-editing therapy SB-913 targets rare genetic diseasemucopolysaccharidosis type II (MPS II).

In addition to its clinical programs, Sangamo has 11 therapies in preclinical testing. Gilead Sciencesis collaborating with Sangamo on two of these preclinical programs. Pfizer and Japanese drugmaker Takeda have teamed up with the biotech on one preclinical program each.

In February, Sangamo announced a dealwith Biogen to develop gene therapies targeting neurological diseases. Three of Sangamo's preclinical programs are included in this agreement.

Sangamo's market cap stands at a little under $1 billion. While the company has a long way to go before any of its candidates could potentially win approval, it also has multiple shots on goal. Successes for only one or two of its experimental therapies would enable this small biotech to become much larger.

I think that both of these stocks could be big winners over the long run. However, my view is that Cara Therapeutics offers a better risk-reward proposition.

Although it's not a slam dunk that Cara wins FDA and EU approvals for Korvuva injection, I think the odds appear to be pretty good. Assuming those approvals are secured, Cara should be set to begin pulling in some solid revenue by late 2021.

Having said that, it wouldn't surprise me if Sangamo achieves greater success by the end of this decade. I think that investors should keep the biotech on their radar screens.

Visit link:
Better Buy: Cara Therapeutics vs. Sangamo Therapeutics - Motley Fool

CAMBRIDGE, Mass., May 1, 2020 /PRNewswire/ --Obsidian Therapeutics, a biotechnology company pioneering controllable cell and gene therapies, today announced it will share the architecture and components of its Safe Workplace Function Tool (SWFT) Productivity Solution. The SWFT Solution was designed in response to the COVID19 pandemic to support a safe work environment while maintaining productivity in the lab.

The SWFT Solution is a web-based application built and integrated into Microsoft 365 that allows scientists to view and schedule lab-based activities including by date, time and lab location. SWFT promotes collaboration and coordination between teams by predicting scheduling and occupancy conflicts, which allows team members to adjust their schedules to promote social distancing in the lab and office.

"The SWFT Solution has enabled Obsidian to continue to generate critical data across our cytoDRiVE development programs, while maximizing the safety of our staff," stated Catherine Stehman-Breen, M.D., Chief Research and Development Officer at Obsidian. "We have already shared this technology with large pharmaceutical and small biotechnology companies who are interested in our SWFT Solution to get their labs back up and running. We believe that it is more important than ever before to leverage one another's expertise in order to overcome challenges as we work tirelessly to deliver meaningful outcomes to patients in need."

To create Obsidian's bespoke application, (1) a capacity analysis was conducted, taking into consideration lab space and occupancy levels, and (2) in collaboration with scientists, workflow recommendations were implemented to determine an appropriate shift schedule. These steps facilitated the development of a solution that enabled scientists to plan their experiments with specific lab locations and shifts, as well as be alerted to and prevent any over-capacity issues. Obsidian implemented four three-hour lab blocks, with thirty-minutes of cleaning time between blocks, over a seven-day work week, to ensure that coronavirus-related safety recommendations were met.

Celeste Richardson, Ph.D., Vice President of Cell Therapy of Obsidian, stated, "We have a commitment to our employees to keep the health and safety of our employees top of mind while they work to bring innovative therapies to patients. The development of the SWFT Solution perfectly demonstrates Obsidian's culture of teamwork, determination and innovation."

Obsidian's IT Partner, TRNDigital, is continuing to iterate the tool to ensure it continues to meet scientists' needs and can be made available to others. The solution has been expanded to include density planning in the Obsidian offices. In addition, the SWFT Solution is scalable and flexible to other laboratory setups.

The SWFT solution was developed in-house by Henry Rogalin, Data Scientist, under the leadership of Nic Betts, Head of IT and Facilities, and in collaboration with a safety and facilities capacity team led by Jillian Giguere, Senior Manager of Laboratory Operations, Facilities, and EHS. For more information on SWFT, submit this inquiry form. Informational sessions and training will be hosted as requested.

About Obsidian TherapeuticsObsidian Therapeutics is a biotechnology company pioneering controllable cell and gene therapies to deliver transformative outcomes for patients with intractable diseases. Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. The cytoDRiVE platform can be applied to design controllable intracellular, membrane and secreted proteins for cell and gene therapies as well as other applications. The Company's initial applications focus on developing novel cell therapies for the treatment of cancer. Obsidian is headquartered in Cambridge, Mass. For more information, please visit http://www.obsidiantx.com.

Media Contact:Maggie BellerRusso Partners, LLCMaggie.beller@russopartnersllc.com646-942-5631

View original content to download multimedia:http://www.prnewswire.com/news-releases/obsidian-therapeutics-develops-and-shares-safe-workplace-productivity-solution-in-response-to-covid19-301050820.html

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Obsidian Therapeutics Develops and Shares Safe Workplace Productivity Solution in Response to COVID19 - P&T Community

The research report on Gene Therapy Market provides comprehensive analysis on market status and development pattern, including types, applications, rising technology and region. Gene Therapy Market report covers the present and past market scenarios, market development patterns, and is likely to proceed with a continuing development over the forecast period. The report covers all information on the global and regional markets including historic and future trends for market demand, size, trading, supply, competitors, and prices as well as global predominant vendors information.

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This market research report on the Gene Therapy Market is an all-inclusive study of the business sectors up-to-date outlines, industry enhancement drivers, and manacles. It provides market projections for the coming years. It contains an analysis of late augmentations in innovation, Porters five force model analysis and progressive profiles of hand-picked industry competitors. The report additionally formulates a survey of minor and full-scale factors charging for the new applicants in the market and the ones as of now in the market along with a systematic value chain exploration.

An outline of the manufacturers active within the Gene Therapy Market, consisting of

Sibiono GeneTech,Advantagene,Spark Therapeutics,Shanghai Sunway Biotech Co. LtdBluebird Bio,UniQure NVAvalanche Bio,Celladon,Sangamo,Dimension Therapeutics

The Gene Therapy Market Segmentation by Type:

Viral vectorNon-viral vector

The Gene Therapy Market Segmentation by Application:

Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Market Segment by Regions, regional analysis covers

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The competitive landscape of the Gene Therapy Market is discussed in the report, including the market share and new orders market share by company. The report profiles some of the leading players in the global market for the purpose of an in-depth study of the challenges faced by the industry as well as the growth opportunities in the market. The report also discusses the strategies implemented by the key companies to maintain their hold on the industry. The business overview and financial overview of each of the companies have been analyzed.

This report provide wide-ranging analysis of the impact of these advancements on the markets future growth, wide-ranging analysis of these extensions on the markets future growth. The research report studies the market in a detailed manner by explaining the key facets of the market that are foreseeable to have a countable stimulus on its developing extrapolations over the forecast period.

Key questions answered in this research report:

Table of Contents:

Global Gene Therapy Market Research Report

Chapter 1 Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

.CONTINUED FOR TOC

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Global Gene Therapy Market Size, Share, Growth, Revenue, Global Industry Analysis and Future Demand And Forecast To 2020-2026 Cole Reports - Cole of...

What are exosomes?

Exosomes are a class of cell-derived extracellular vesicles of endosomal origin, and are typically 30-150 nm in diameter the smallest type of extracellular vesicle.1 Enveloped by a lipid bilayer, exosomes are released into the extracellular environment containing a complex cargo of contents derived from the original cell, including proteins, lipids, mRNA, miRNA and DNA.2 Exosomes are defined by how they are formed through the fusion and exocytosis of multivesicular bodies into the extracellular space.

Multivesicular bodies* are unique organelles in the endocytic pathway that function as intermediates between early and late endosomes.3 The main function of multivesicular bodies is to separate components that will be recycled elsewhere from those that will be degraded by lysosomes.4 The vesicles that accumulate within multivesicular bodies are categorized as intraluminal vesicles while inside the cytoplasm and exosomes when released from the cell.

*Confusingly, there is inconsistency in the literature; while some sources differentiate multivesicular bodies from late endosomes, others use the two interchangeably.

Exosomes are of general interest for their role in cell biology, and for their potential therapeutic and diagnostic applications. It was originally thought that exosomes were simply cellular waste products, however their function is now known to extend beyond waste removal. Exosomes represent a novel mode of cell communication and contribute to a spectrum of biological processes in health and disease.2One of the main mechanisms by which exosomes are thought to exert their effects is via the transfer of exosome-associated RNA to recipient cells, where they influence protein machinery. There is growing evidence to support this, such as the identification of intact and functional exosomal RNA in recipient cells and certain RNA-binding proteins have been identified as likely players in the transfer of RNA to target cells.5,6 MicroRNAs and long noncoding RNAs are shuttled by exosomes and alter gene expression while proteins (e.g. heat shock proteins, cytoskeletal proteins, adhesion molecules, membrane transporter and fusion proteins) can directly affect target cells.7,8Exosomes have been described as messengers of both health and disease. While they are essential for normal physiological conditions, they also act to potentiate cellular stress and damage under disease states.2

Multivesicular bodies are a specialized subset of endosomes that contain membrane-bound intraluminal vesicles. Intraluminal vesicles are essentially the precursors of exosomes, and form by budding into the lumen of the multivesicular body. Most intraluminal vesicles fuse with lysosomes for subsequent degradation, while others are released into the extracellular space.9,10 The intraluminal vesicles that are secreted into the extracellular space become exosomes. This release occurs when the multivesicular body fuses with the plasma membrane.

The formation and degradation of exosomes.

This is an active area of research and it is not yet known how exosome release is regulated. However, recent advances in imaging protocols may allow exosome release events to be visualized at high spatiotemporal resolution.11

Exosomes have been implicated in a diverse range of conditions including neurodegenerative diseases, cancer, liver disease and heart failure. Like other microvesicles, the function of exosomes likely depends on the cargo they carry, which is dependent on the cell type in which they were produced.12 Researchers have studied exosomes in disease through a range of approaches, including:

In cancer, exosomes have multiple roles in metastatic spread, drug resistance and angiogenesis. Specifically, exosomes can alter the extracellular matrix to create space for migrating tumor cells.13,14 Several studies also indicate that exosomes can increase the migration, invasion and secretion of cancer cells by influencing genes involved with tumor suppression and extracellular matrix degradation.15,16Through general cell crosstalk, exosomal miRNA and lncRNA affect the progression of lung diseases including chronic obstructive pulmonary disease (COPD), asthma, tuberculosis and interstitial lung diseases. Stressors such as oxidant exposure can influence the secretion and cargo of exosomes, which in turn affect inflammatory reactions.17 Altered exosomal profiles in diseased states also imply a role for exosomes in many other conditions such as in neurodegenerative diseases and mental disorders.18,19Cells exposed to bacteria release exosomes which act like decoys to toxins, suggesting a protective effect during infection.20 In neuronal circuit development, and in many other systems, exosomal signaling is likely to be a sum of overlapping and sometimes opposing signaling networks.21

Exosomes can function as potential biomarkers, as their contents are molecular signatures of their originating cells. Due to the lipid bilayer, exosomal contents are relatively stable and protected against external proteases and other enzymes, making them attractive diagnostic tools. There are increasing reports that profiles of exosomal miRNA and lncRNA differ in patients with certain pathologies, compared with those of healthy people.17 Consequently, exosome-based diagnostic tests are being pursued for the early detection of cancer, diabetes and other diseases.22,23Many exosomal proteins, nucleic acids and lipids are being explored as potential clinically relevant biomarkers.24 Phosphorylation proteins are promising biomarkers that can be separated from exosomal samples even after five years in the freezer25, while exosomal microRNA also appears to be highly stable.26 Exosomes are also highly accessible as they are present in a wide array of biofluids (including blood, urine, saliva, tears, ascites, semen, colostrum, breast milk, amniotic fluid and cerebrospinal fluid), creating many opportunities for liquid biopsies.

Exosomes are being pursued for use in an array of potential therapeutic applications. While externally modified vesicles suffer from toxicity and rapid clearance, membranes of naturally occurring vesicles are better tolerated, offering low immunogenicity and a high resilience in extracellular fluid.27 These naturally-equipped nanovesicles could be therapeutically targeted or engineered as drug delivery systems.

Exosomes bear surface molecules that allow them to be targeted to recipient cells, where they deliver their payload. This could be used to target them to diseased tissues or organs.27 Exosomes may cross the blood-brain barrier, at least under certain conditions28 and could be used to deliver an array of therapies including small molecules, RNA therapies, proteins, viral gene therapy and CRISPR gene-editing.

Different approaches to creating drug-loaded exosomes include27:

Exosomes hold huge potential as a way to complement chimeric antigen receptor T (CAR-T) cells in attacking cancer cells. CAR exosomes, which are released from CAR-T cells, carry CAR on their surface and express a high level of cytotoxic molecules and inhibit tumor growth.29 Cancer cell-derived exosomes carrying associated antigens have also been shown to recruit an antitumor immune response.30

The purification of exosomes is a key challenge in the development of translational tools. Exosomes must be differentiated from other distinct populations of extracellular vesicles, such as microvesicles (which shed from the plasma membrane, also referred to as ectosomes or shedding vesicles) and apoptotic bodies.31 Although ultracentrifugation is regarded as the gold standard for exosome isolation, it has many disadvantages and alternative methods for exosome isolation are currently being sought. Exosome isolation is an active area of research (see Table 1) and many research groups are seeking ways to overcome the disadvantages listed below, while navigating the relevant regulatory hurdles along the way.

Produces a low yield and low purity of the isolated exosomes as other types of extracellular vesicles have similar sedimentation properties.

Low efficiency as it is labor-intensive, time-consuming and requires a large amount of sample. specialized equipment. High centrifugal force can damage exosome integrity

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Exosomes: Definition, Function and Use in Therapy - Technology Networks