Archive for May, 2020

This comprehensive Artificial Intelligence in Life Extension Market research report includes a brief on these trends that can help the businesses operating in the industry to understand the market and strategize for their business expansion accordingly. The research report analyzes the market size, industry share, growth, key segments, CAGR and key drivers.

New vendors in the market are facing tough competition from established international vendors as they struggle with technological innovations, reliability and quality issues. The report will answer questions about the current market developments and the scope of competition, opportunity cost and more.

Market Summary:

The Artificial Intelligence in Life Extension market is a comprehensive report which offers a meticulous overview of the market share, size, trends, demand, product analysis, application analysis, regional outlook, competitive strategies, forecasts, and strategies impacting the Artificial Intelligence in Life Extension Industry. The report includes a detailed analysis of the market competitive landscape, with the help of detailed business profiles, SWOT analysis, project feasibility analysis, and several other details about the key companies operating in the market.

This report studies the Artificial Intelligence in Life Extension market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Artificial Intelligence in Life Extension market by product type and applications/end industries.

Company Coverage (Sales Revenue, Price, Gross Margin, Main Products, etc.):

Google,, Facebook, IBM, Intel, Microsoft, Gero, Deep Genomics, IBM Medical Sieve, Google DeepMind (DM) Health, Babylon Health (iOS, Android), Turbine.ai, and Insilico Medicine

The final report will add the analysis of the Impact of Covid-19 in this report Artificial Intelligence in Life Extension industry.

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Artificial Intelligence in Life Extension in its database, which provides an expert and in-depth analysis of key business trends and future market development prospects, key drivers and restraints, profiles of major market players, segmentation and forecasting. A Artificial Intelligence in Life Extension Market provides an extensive view of size; trends and shape have been developed in this report to identify factors that will exhibit a significant impact in boosting the sales of Artificial Intelligence in Life Extension Market in the near future

Scope and Segmentation of the Report

The segment analysis is one of the significant sections of this report. Our expert analyst has categorized the market into product type, application/end-user, and geography. All the segments are analyzed based on their market share, growth rate, and growth potential. In the geographical classification, the report highlights the regional markets having high growth potential. This thorough evaluation of the segments would help the players to focus on revenue-generating areas of the Vertical Farming market.

Regional Analysis

Our analysts are experts in covering all types of geographical markets from developing to mature ones. You can expect a comprehensive research analysis of key regional and country-level markets such as Europe, North America, South America, Asia-Pacific, and the Middle East & Africa. With accurate statistical patterns and regional classification, our domain experts provide you one of the most detailed and easily understandable regional analyses of the Artificial Intelligence in Life Extension market.

Competitive Landscape:

The research report also studied the key players operating in the Artificial Intelligence in Life Extension market. It has evaluated and explained the research & development stages of these companies, their financial performances, and their expansion plans for the coming years. Moreover, the research report also includes the list of planned initiatives that clearly explain the accomplishments of the companies in the recent past.

Research Methodology

The research methodology of the market is based on both primary as well as secondary research data sources. It compels different factors affecting the Artificial Intelligence in Life Extension industry such as historical data and market trends, different policies of the government, market environment, market risk factors, market restraints, technological advancements, forthcoming innovations, and obstacles in the industry.

Table Of Content

1 Report Overview

2 Global Growth Trends

3 Market Share by Key Players

4 Breakdown Data by Type and Application

5 North America

6 Europe

7 China

8 Japan

9 Southeast Asia

10 India

11 Central & South America

12 International Players Profiles

13 Market Forecast 2019-2026

14 Analysts Viewpoints/Conclusions

15 Appendix

Moreover, the research report assessed market key features, consisting of revenue, capacity utilization rate, price, gross, growth rate, consumption, production, export, supply, cost, market size & share, industry demand, export & import analysis, and CAGR.

Artificial Intelligence in Life Extension Market Key players influencing the market are profiled in the study along with their SWOT analysis and market strategies. The report also focuses on leading industry players with information such as company profiles, products and services offered financial information of last 3 years, key development in past five years.

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Artificial Intelligence in Life Extension Market Countries Analysis Report 2020 by Industry Size, Share, Growth Rate and Revenue - Latest Herald

New York, May 05, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Food Additives Market 2020-2024" - https://www.reportlinker.com/p03571280/?utm_source=GNW Our reports on food additives market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the rising demand for processed food, increasing complexities in the food supply chain and growing demand for home food preservation. In addition, rising demand for processed food is anticipated to boost the growth of the market as well. The food additives market analysis includes application segment, product segment, and geographic landscapes

The food additives market is segmented as below: By Application Bakery and confectionery Beverages Convenience food Snacks Others

By Product Flavors and enhancers Acidulants Colorants Sweeteners Others

By Geographic Landscapes North America APAC Europe South America MEA

This study identifies the increasing adoption of organic and natural ingredients as one of the prime reasons driving the food additives market growth during the next few years. Also, rising trend of non-GMO ingredients and shelf life extension of food products will lead to sizable demand in the market. The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our food additives market covers the following areas: Food additives market sizing Food additives market forecast Food additives market industry analysis"

Read the full report: https://www.reportlinker.com/p03571280/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Global Food Additives Market is expected to grow by $ 8.86 bn during 2020-2024 progressing at a CAGR of 4% during the forecast period -...

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This paper presents a fatigue-prediction methodology designed to extend the life of unbonded flexible risers and improve the accuracy of floating production, storage, and offloading (FPSO) vessel response analysis. The methodology combines measured-motion-response, maritime-environment, and process data to improve traditional time-domain dynamic analysis models, along with machine-learning (ML) techniques to develop a heading model for the FPSO.

Life extension of unbonded flexible risers is a challenge because of uncertainties associated with key parameters driving deterioration, such as how the riser was designed, manufactured, and installed and how it is operated. Fatigue service life is calculated during the design phase using models based on field-specific environmental data derived for the area and loads for the required design conditions per project specifications and international standards.

Using real measured data from the riser motions and from environmental sources can reduce uncertainties significantly, improving life-extension analysis for fatigue and other failure drivers. These data, when combined with high-end engineering assessments and considerations on data reliability, provide better insight and contribute to continued operations with acceptable risk for the risers.

The complete paper presents a methodology that uses measured environmental data and FPSO and riser response data in an ML environment to build more-realistic riser-response and fatigue-prediction models. A case example is presented for the risers suspended from the FPSO Fluminense producing from the Bijupir and Salema fields offshore Brazil. Because FPSO heading is important for vessel dynamics, especially roll, and the vessel dynamics are a key factor in the riser dynamics at this field, initial focus was on predicting vessel heading relative to swell. The heading model developed by ML showed good agreement and was used as a key tool in a traditional fatigue analysis. This analysis was based on historical sea states from the last 2 years. The results show that the fatigue analysis from the design phase is conservative and lifetime extension is achievable.

Because the fully instrumented measurement campaign ended after 4 months, the work focused on using all captured data to provide improved insight and develop both traditional simulation and ML models. For future fatigue predictions based on the developed fatigue counter, the objective is to maintain accuracy with less instrumentation.

In the present phase, FPSO and riser-response data from the 4-month campaign have been used to establish a correlation between riser behavior, environmental data, and FPSO heading and motion. Calibration of a traditional numerical model is performed using measurement data along with a direct waves-to-fatigue prediction based on modern ML techniques.

The ongoing work illustrates how real environmental data and response monitoring can reduce uncertainties and improve and extend flexible-riser life.

The improved armor-wire-fatigue methodology developed in this project uses accurate motion-response sensors directly installed on the riser and interfacing FPSO structures. The response data are combined with measured environmental data to build more-realistic fatigue models. The objective is to develop a model that represents the real-life responses of the FPSO in various swell- and wind-driven sea states, defining with high accuracy the long-term characteristics of the riser system.

Access to high-quality environmental data for tail-end production and life-extension projects of older assets may be a challenge. Efficient use of existing environmental data improves understanding of environmental loadings. Wind, wave, and surface-current data from different sources are assessed and verified for quality and potential weaknesses under varying conditions.

Actual operational data such as topside and subsea pressures and temperatures from the production information system are used instead of design limits to increase accuracy of fatigue development. Integrating the operators process-instrumentation network and riser-status databases enables use of actual exposure conditions. Measurement data, component data, and new insight from the improved analytical models are visualized by live contextualized dashboards that provide all stakeholders with the same easy-to-access, relevant, updated, and consistent information.

The complete paper discusses environmental data and motion monitoring. Because vessel roll is a key factor for the riser bending near the hang-off where the bend stiffener is located, models need a good representation of the wind, current, swell, and vessel heading to perform reliable fatigue analysis. When high-quality field measurement data are available at good resolution, better understanding of the dynamics and model improvement is achievable within a reasonable time. However, for periods without access to environmental and motion response data, good analytical models for both vessel response and for global and local riser analysis are necessary.

Swell is important for total wave conditions in Brazilian waters and is important for roll motion, especially for free-weathervaning FPSOs. In sea states dominated by swell, good insight in the heading of the FPSO relative to the incoming swell is important. During sea states with high wind, the FPSO will align with the wind direction and may expose the vessel to beam sea swell, possibly giving higher roll motions.

A key activity in this project was to develop a heading model for the FPSO, predicting the weathervaning response from input of wind, waves, and surface current. The authors preferred approach was to use field measurement data and ML. The more data are obtained, the more the model improves. Thus far, the model is trained on average from 3-hour sea states, so, for transient conditions, inaccuracies are inevitable. The model is trained as more field measurement data become available, and significant improvements are seen from the initial models to the current model. Several different ML approaches were tested to find the best possible approach for the data set of 4 months with 3-hour statistical data, giving approximately 600 samples after 3-hour periods with nearly no motions removed.

The ML processes used for developing the heading model were also used for the prediction of FPSO and riser-motion responses. The objective was to obtain a sufficiently accurate model enabling reliable prediction of dynamic responses in time periods where no response measurements are available.

The complete paper outlines the model-training process. The ML models proved to be on the same level or better than a time-simulation model in predicting motion responses when both models were fed with similar input from the environmental conditions.

As Fig. 1 illustrates, compared with the original design analysis, the accuracies of ML predictions based on real environmental data and models trained on measured field data enable a step toward reliable dynamics in flexible-riser fatigue.

Developing a reliable online fatigue counter for the flexible-riser armor wires began with validating the available environmental data for the FPSO site. A site with a comprehensive measurement program for waves, current, and wind with good time resolution would have been preferable. However, in this project, a robust and accurate alternative was found.

This approach will be relevant for the majority of brownfield assets. Newer installations in deeper waters likely will have more onsite measurements to support integrity-management analytics.

When combining quality weather data and observations; field measurements of FPSO, turret, and riser motion; and normalized fatigue/damage curves derived from validated models, the live fatigue counter can be established. The fatigue counter will account automatically for bore pressures different from planned operational pressure, as well as environmental conditions experienced in the field. Depending upon conservatism built into the design phase, possible life extension may be achieved and documented. By forecasting planned changes to operational conditions or possible changes in annulus environments, future scenarios may be exploited efficiently by assuming that wave, wind, and current in the coming years, on average, will be similar to those of recent years.

The complete paper includes a detailed discussion of the criteria and process used to develop the fatigue counter.

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Fatigue Prediction for Extended Riser Life and Improved Vessel-Response Analysis - Journal of Petroleum Technology

Across the globe there has been a notable uptick in consumer interest in the provenance and standards of food products, while a surge in demand for fresh produce and packaged goods means shelf life extension and stability are paramount concerns for food manufacturers.

Thats why we will be shining the spotlight on both issues in an exclusive webinar on May 12, where Dr Lan Ban, Research & Development Director at Kemin Food Technologies, will be presenting expert insights.

According to the company: Food safety is every brands number one priority while keeping product fresh for longer allows business to optimise its returns.

However, meeting consumers demands for safe and high-quality food requires extensive understanding of the chemical and biological pathways that affect products.

Thats why in this presentation, Kemin will assess how minor, yet hugely important, ingredients such as antimicrobials play a pivotal role in delaying microbial growth and preventing foodborne illness, while simultaneously ensuring brand longevity and loyalty.

Key takeaways will include an understanding of common food safety and shelf life challenges, and listeners will be equipped with toolkit for the best use of antimicrobials to solve them and boost business outcomes at the same time.

Register for the webinar here.

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Shelf-life and food safety: Hear exclusive expert insights on two of the biggest industry challenges right now - FoodNavigator-Asia.com

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Understanding and optimizing how vapour permeability so dramatically effects the shelf-life of products in MAP packaging materials is hard. There are two approaches the head in the sand approach or practical testing. Versaperm has introduced a MAP testing service to measure the critical vapour permeability for packaging and materials based around its new high-speed testing equipment which can often turn around results within 24 hours, instead of the weeks taken by conventional testing techniques.

Vapour permeability testing has led to a four to eightfold shelf-life extension for food some products. Getting it wrong can actually reduce shelf life and quality.

Versaperm can test and measure vapour permeability not just for clients material samples but also for their packed products in their MAP packaging for virtually any type of package or configuration. Permeability can not only be measured not just for common water vapour problems but for oxygen, carbon dioxide, ethylene, and all other MAP gases.

It is important to have the ability to measure both material samples and finished products as manufacturing, sealing and packaging processes can substantially change a packages permeability.

Results are accurate in the parts per million or parts per billion ranges depending on the material and vapour. Where desired Versaperm can also control environmental conditions such as temperature and pressure, even across diurnal and seasonal cycles.

As well as its sample testing Versaperm develops and manufactures its own ranges of vapour testing equipment which are used by companies, universities, and national standards laboratories around the world.

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The Head in the Sand Approach to MAP And a product's shelf life extension - International Supermarket News

As parties to merger or acquisition agreements carefully review their agreements to see what, if any, impacts the COVID-19 pandemic may have, the recent decision from the U.S. District Court for the Southern District of New York inNewmont Mining Corp. v. AngloGold Ashanti Ltd.[i]provides meaningful guidance for the interpretation of Material Adverse Effect (MAE) provisions in agreements governed by New York law. On March 18, 2020, inNewmont Mining, the Southern District of New York granted summary judgment for the seller, AngloGold Ashanti, Ltd. on the buyers, Newmont Mining Corporations, claims for damages arising from the alleged breach of an MAE provision. The decision inNewmont Mining, while decided under New York law, reinforces the established principle under Delaware law that buyers face an uphill battle when trying to establish an MAE.

An MAE provision in a merger or acquisition agreement typically includes three parts: 1) the definition of an MAE; 2) exclusion from that definition; and 3) exceptions to the exclusions. This article will discuss each part in turn, and discuss theNewmont Miningcourts reasoning for granting summary judgment to the seller relating to each part. Before diving into the details, however, it is worth keeping a few general principles about the interpretation of MAE provisions in mind.

GENERAL PRINCIPALS

When determining whether there is a breach of an MAE clause courts take a seller-friendly perspective that requires a buyer to make a strong showing to invoke its rights under MAE provisions.[ii] Thus, the Delaware Chancery Court has quipped, [a] contractual material adverse effect . . . is like a . . . tornadofrequently alleged but rarely shown to exist.[iii]

Courts consider the MAE provision as a backstop protecting the [buyer] from the occurrence of unknown events that substantially threaten the overall earnings potential of the target [company] in a durationally-significant manner.[iv] As a result, courts consider whether the alleged material adverse change was within the contemplation of the parties at the time they executed the agreement, whether it was within the control of the parties, and the magnitude of the impact on the relevant partys business.[v] MAE provisions are read in the larger context in which the parties were transacting.[vi] Thus, as theNewmont Miningcourt noted, a short-term hiccup in earnings should not suffice; rather the Material Adverse Effect should be material when viewed from the longer-term perspective of a reasonable acquiror.[vii]

Lastly, while the law in New York and Delaware regarding the interpretation of MAE provisions is largely consistent, the burden of proof required differs. Specifically, under Delaware law, a buyer must prove an MAE with clear and convincing evidence, whereas, in New York, the standard is a preponderance of the evidence.[viii]InNewmont Mining, this distinction, along with factual differences, led the court to distinguishAkorn, Inc. v. Fresenius Kabi AG[ix] the 2018 Delaware Chancery Court decision that made headlines because it found the existence of an MAE sufficient to terminate a merger agreement.[x]

THE DEFINITION OF AN MAE

Agreements typically define an MAE as any event, development or condition occurring that has had, or would be reasonably expected to have, a material adverse effect on the business, financial condition or results of operations of the company and its subsidiariestaken as a whole. For example, inNewmont Mining, where the buyer acquired a gold mine (the Mine) that contained a high grade mill (the Mill) that the seller was building as part of its Mine Life Extension project (MLE2), the MAE provision in the stock purchase agreement (SPA) defined a Company Material Adverse Effect, in relevant part, as any change, effect, event, occurrence, circumstance or state of facts that . . . (ii) is or would reasonably be expected to be materially adverse to the business, results of operations, condition (financial or otherwise) of . . .the Mine.[xi]

Importantly, the MAE provision referenced the Mine as a whole, not the Mill. This was of critical importance inNewmont Mining, where the only MAEs alleged by the buyer resulted from alleged problems with the Mill.[xii] As the court explained: the construction of the Mill was not the only component of the MLE2 project Moreover, Newmont bought the entire Mine, not just the one Mill, and was aware that the Mill was still being commissioned at the time the transaction closed.[xiii]Thus, the court reasoned:

If Newmont wanted the Company MAE definition to include materially adverse effects measured in terms of the Mill, it should have bargained for such a definition. Notwithstanding Newmonts assertion that the Mill was a hugely important piece of the [M]ine, and that it was the centerpiece and jewel of the transaction under the plain language of the SPA, Newmont must demonstrate that its Alleged MAEs had a materially adverse effect on the Mineas a whole.[xiv]

It thus concluded that Newmont had not established an MAE occurred because, [w]hile the percentage of gold expected to be recovered from Mill processing is not insignificant, and there may well be circumstances where an event involving the Mill does in fact constitute a materially adverse effect to the Mine as a whole, such circumstances are not present here.[xv]

Additionally, theNewmont Miningcourt highlighted that the buyers post-acquisition valuation of the Mine as a whole showed that no MAE had occurred. Specifically, the buyers post-acquisition valuation models showed that the Mine had increased in value after the transaction closed.[xvi] Thus, the court concluded that even if the Mill was underperforming, the buyers own analyses showed that the Mine as a whole did not suffer an MAE.[xvii]

EXCLUSIONS TO THE DEFINITION OF AN MAE

Second, MAE clauses usually exclude specified events, such as weather events, terrorism or military actions, general economic downturns, conditions existing generally within the companys industry, and other broad categories of market or credit conditions. InNewmont Mining,the SPA expressly listed certain exception to the definition of a Company MAE, including, among others, changes or circumstances relating to (a) the industries in which [the sellers] operate, (b) general economic effects or conditions affecting the Unites States or anywhere else in the world, (c) foreign exchange, equity or debt market conditions, (d) acts of God (including earthquakes, storms, fires, floods and natural catastrophes, and (e) the failure to meet any projections or forecasts (it being understood that that [sic] the facts or causes underlying or contributing to such failure may be considered in determining whether a Company Material Adverse Effect has occurred unless otherwise excluded pursuant to any of the other clauses of this definition).[xviii]

InNewmont Mining, the court found that the exclusions of projections and forecast was dispositive. The buyers alleged MAEs were tied to the Mills alleged inability to achieve two forecasted metrics, a design throughput of 250 short tons per hour, and a gold recovery rate of 76.5%. The court rejected the buyers argument that its alleged MAEs were premised on the underlying causes of the missed projections and not the projections themselves. The court so held because the buyers interpretation would transform an otherwise narrow carve-out into an exception that swallows the rule, as it would allow virtually any set of circumstances to constitute a Company MAE as long as it could identify a purported underlying cause for a failure to meet projections or forecasts.[xix] Thus, the court determined that the parties intent in including the parenthetical excepting underlying causes from the exclusion for projections was to clarify that, where an underlying cause or set of facts fits within the Company MAE definition but also results in a failure to meet a projection, that underlying cause or set of facts may still be asserted as a Company MAE even though it contributed to a missed projection.[xx] Thus, for example, if the Mill was destroyed, that event would not be excluded from the definition of an MAE simply because it also caused the Mill to not achieve certain projections.

EXCEPTIONS TO THE EXCLUSIONS

MAE provisions typically state that, with respect to some or all of the specified exclusions, they will not be excluded to the extent that they have disproportionately adversely affected the company and its subsidiaries (taken as a whole) as compared to others in the same industry. The exception to the specified exclusions was not an issue in theNewmont Miningdecision; however, such exceptions may become a focus of litigation in this post-COVID world. For example only, we expect that notwithstanding the severe economic impact that COVID-related disruptions have had on all businesses, buyers may attempt to find footing arguing that the impact on their recently-acquired or to-be acquired business was disproportionate or otherwise unique. The adjudication of such arguments will be fact intensive and require expert analysis.

CONCLUSION

COVID-19 will bring MAE provisions into the spotlight in M&A litigation as parties evaluate the impact to their soon-to-be or recently-acquired business. TheNewmont Miningdecision provides helpful guidance with respect to the requirement that alleged MAEs impact the acquisition target as a whole, and the interpretation of exclusions regarding the projections and forecasts that are typically included in MAE provisions.

[i]Newmont Mining Corp. v. AngloGold Ashanti Ltd., et al.,Opinion and Order, No. 1:17-cv-08065 (S.D.N.Y. Mar. 18, 2020), ECF No. 143 (Newmont Mining).

[ii]In re Ibp S'holders Litig. v. Tyson Foods, 789 A.2d 14, 144-45 (Del. Ch. 2001) (Tyson Foods).

[iii]Chyronhego Corp. v. Wight, No. 2017-0548-SG, 2018 Del. Ch. LEXIS 258, at *22 (Del. Ch. July 31, 2018)

[iv]Tyson Foods, 789 A.2d at 144.

[v]Newmont Miningat 34.

[vi]Tyson Foods, 789 A.2d at 141-42, 144.

[vii]Newmont Miningat 35 (quotingTyson Foods,789 A.2d at 68).

[viii]Tyson Foods, 789 A.2d at 94.

[ix]Akorn, Inc. v. Fresenius Kabi AG, et al.,Memorandum Opinion, No. 2018-0300-JTL (Del. Ch. Oct. 1, 2018) (Akorn).

[x]Newmont Miningat 46 n.30.

[xi]Id.at 2-3, 32-33 (emphasis added).

[xii]Id.at 39.

[xiii]Id.

[xiv]Id.at 39-40 (emphasis in original).

[xv]Id. at 40.

[xvi]Id.at 51-52.

[xvii]Id.

[xviii]Newmont Mining Corp. v. AngloGold Ashanti Ltd., et al.,The AGA Defendants Local Rule 56.1 Statement in Support of Their Motion for Summary Judgment at 242,supranote i,ECF No. 110.

[xix]Newmont Miningat 37.

[xx]Id.

1994-2020 Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C. All Rights Reserved.

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SDNY Ruling in Newmont Mining Lawsuit - The National Law Review

MIKE TYSON revealed how he has started training again having not thrown a punch for a staggering 15 years - and is being aided by stem-cell research therapy.

The Baddest Man on the Planet, who hung up his gloves in 2005 following defeat by Kevin McBride, wouldn't elaborate on why he was having the treatment but added that it was 'really wild what scientists can do'.

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Tyson was having an Instagram chat with basketball legend Shaquille O'Neal when he revealed that he had been training for the previous three days after 15 years away - and his new health regime.

Iron Mike said: "You know what I had done? I had stem-cell research therapy.

"I feel like a different person but I can't comprehend why I feel this way. It's really wild what scientists can do."

Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition that usually takes the form of a bone marrow transplantation.

He did not reveal what the exact condition was that was being treated.

Despite letting the gloves gather dust in the corner, it didn't take long for Iron Mike to show off that lethal punching power.

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The 53-year-old delighted fans in lockdown by uploading a viral video of him laying into a punchbag with his trademark speed and power.

Tyson's weight ballooned after retiring following battles with drug addiction and depression.

But he has since partnered with a new trainer, MMA coach Rafael Cordeiro, to kick-start his training after sensationally revealing his 15-year break.

During their chat, basketball legend O'Neal revealed how he ached for three days after playing with his sons.

Tyson responded: "That's just because you haven't done it for a while.

WHAT IS STEM CELL TREATMENT USED FOR?

Stem cell transplants are carried out when bone marrow is damaged or isnt able to produce healthy blood cells.

It can also be used to replace damaged blood cells as the result of intensive cancer treatment.

Here are conditions that stem cell transplants can be used to treat:

"If you continue to do it consistently you'll be back to normal.

"It's just like me, I haven't boxed or hit the bag for 15 years - it has been three days so far and I feel incredible."

Tyson, who has a 50-6 record, is reportedly gearing up for a sensational return amid plans to compete in exhibition bouts for charity later this year.

He told rapper T.I. last month: "I've been hitting the mitts for the last week.

"That's been tough, my body is really jacked up and really sore from hitting the mitts.

MIKE DROPTyson shows off body transformation with 53-year-old preparing for boxing return

NO GLOVE LOSTJoshua tells Froch to 'shut up, blud' as war of words continues

HOLY MOLYHolyfield hints at comeback aged 57 after Tyson announces plans to return to ring

DO OR TYMike Tyson's trainer 'saw life flash before his eyes' in scary training clip

IT WILL ALL END IN PIERSPiers Morgan and Eddie Hearn in Twitter spat over coronavirus

HOME FIGHTERDubois enjoys lockdown sparring with siblings after buying family pad

SHOWDOWNHall vs Bjornsson: How giants compare as Beast threatens deadlift record holder

WHATSAPP-ENING?Joshua sent Froch four now-deleted messages after his P4P rant

SUMMER LOVIN'Boxing set for JULY return as chiefs outline safety plans for fan-free events

"I've been working out, I've been trying to get in the ring, I think I'm going to box some exhibitions and get in shape.

"I want to go to the gym and get in shape to be able to box three or four-round exhibitions for some charities and stuff.

"Some charity exhibitions, make some money, help some homeless and drug-affected motherf****er like me."

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Mike Tyson, 53, reveals he had stem cell research therapy and hadnt hit bags for 15 YEARS before returning t - The Sun

A recent market study on the global Hematopoietic Stem Cell Transplantation (HSCT) market reveals that the global Hematopoietic Stem Cell Transplantation (HSCT) market is expected to reach a value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% during the forecast period (2019-2029). The impact of the COVID-19 pandemic on the global Hematopoietic Stem Cell Transplantation (HSCT) market is discussed in the presented study.

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The following doubts are addressed in the market report:

Key Highlights of the Hematopoietic Stem Cell Transplantation (HSCT) Market Report

The presented report segregates the Hematopoietic Stem Cell Transplantation (HSCT) market into different segments to ensure the readers gain a complete understanding of the different aspects of the Hematopoietic Stem Cell Transplantation (HSCT) market.

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Segmentation of the Hematopoietic Stem Cell Transplantation (HSCT) market

Competitive Outlook

This section of the report throws light on the recent mergers, collaborations, partnerships, and research and development activities within the Hematopoietic Stem Cell Transplantation (HSCT) market on a global scale. Further, a detailed assessment of the pricing, marketing, and product development strategies adopted by leading market players is included in the Hematopoietic Stem Cell Transplantation (HSCT) market report.

Companies Mentioned in the Report

The report profiles key manufacturers in the hematopoietic stem cell transplantation (HSCT) Market based on various attributes such as company details, SWOT analysis, strategic overview, financials, and business overview. Major players profiled in this report include Regen Biopharma, Inc., Escape Therapeutics, Inc., Lonza Group Ltd., and Pluristem Therapeutics Inc.

The global hematopoietic stem cell transplantation (HSCT) Market has been segmented as follows:

By Transplant Type

By Disease Indication

By Application

By Region

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Global trade impact of the Coronavirus Hematopoietic Stem Cell Transplantation (HSCT) Market Shares, Strategies and Forecast Worldwide, 2019-2027 -...

SAN CARLOS, Calif., May 05, 2020 (GLOBE NEWSWIRE) -- BioCardia, Inc.[Nasdaq: BCDA], a leader in the development of comprehensive solutions for cardiovascular regenerative therapies, today announced data from a recent animal study performed by the Company that demonstrate meaningful improvements in heart function for subjects treated with its allogenic (from another donor, or off the shelf) neurokinin 1 receptor positive mesenchymal stem cell (NK1R+ MSC) program for heart failure, known as CardiALLO. In addition, the Company is planning further exploration and discussion with the U.S. Food and Drug Administration (FDA) on the use of its allogenic cells for COVID-19 related Acute Respiratory Distress Syndrome (ARDS).

In the 26 animals treated with both low dose and high dose NK1R+ MSC, echocardiographic measures of cardiac ejection fraction, fractional shortening and cardiac outflow were meaningfully improved, with all three measures being statistically significant for both dosage levels over control animals.

The CardiALLO cell therapy is being developed initially to treat heart failure patients whose cells do not qualify for its lead autologous cell therapy, CardiAMP (BCDA-01).

BioCardia Chief Scientific Officer Ian McNiece, PhD, said, In light of these positive data on our allogenic NK1R+ MSC therapy, we expect to meet our internal timeline to complete our submission to the FDA for our first indication for CardiALLO, and potentially receive IND acceptance by the end of the second quarter. The MSCs that were studied are subtypes of MSC that we have delivered previously in our co-sponsored trials, which we believe have enhanced potency over MSC generated from unselected bone marrow cells. We look forward to seeing additional data from this animal study that are currently being analyzed, including histology and pathology of the heart and lungs.

COVID-19 Induced Acute Respiratory Distress Syndrome (ARDS) Exploration

The Company also intends to submit an IND for the use of its NK1R+ MSC delivered via intravenous (IV) infusion for Acute Respiratory Distress Syndrome (ARDS) caused by COVID-19.

Based on preliminary clinical reports on COVID-19, respiratory failure complicated by ARDs is the leading cause of death for COVID-19 patients.1 ARDS is a type of respiratory failure characterized by the rapid onset of widespread inflammation in the lungs.

The anti-inflammatory effects of MSC have been well-documented and MSC have been shown to reduce inflammation and injury in models of lung disease.2 The specific MSCs used in BioCardias allogenic cell therapy are expanded from cells selected for the presence of the NK1 receptor, which is known to bind to substance P, an important neuropeptide associated with inflammation throughout the body and a primary mediator of inflammation in the airways.3,4

Our NK1R+ allogenic MSC may have more potential than other MSC approaches being advanced today due to their interaction with Substance P, said BioCardia CEO Peter Altman, PhD. This COVID-19 related work will be the Companys first clinical investigation outside of the cardiac space and our first exploring therapy for the lung. A recent patent publication (US 2020/0101113 A1) shows that BioCardia has long intended for these remarkable reparative cells to be targeted for respiratory disorders, in addition to cardiovascular disease. Addressing inflammation in the lungs is an important contribution we may be able to make using our NK1R+ allogenic MSC therapy.

The Companys allogenic cells are expected to be manufactured at BioCardias clinical stage cell manufacturing facility in San Carlos, California.

About BioCardiaBioCardia, Inc., headquartered in San Carlos, California, is developing regenerative biologic therapies to treat cardiovascular disease. CardiAMP and CardiALLO cell therapies are the Companys biotherapeutic product candidates in clinical development. The Company's approved products include the Helix transendocardial delivery system and its steerable guide and sheath catheter portfolio. BioCardia also partners with other biotherapeutic companies to provide its Helix System and clinical support to their programs studying therapies for the treatment of heart failure, chronic myocardial ischemia and acute myocardial infarction.

Forward Looking Statements This press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include, among other things, references to the development of NK1R+ cells for the treatment of heart failure and ARDS secondary to COVID-19, potential FDA IND acceptances, and potential FDA filings, statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations. Such risks and uncertainties include, among others, the inherent uncertainties associated with developing new products or technologies. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.

We may use terms such as believes, estimates, anticipates, expects, plans, intends, may, could, might, will, should, approximately or other words that convey the uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results may differ materially from the forward-looking statements contained in this press release. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate. Additional factors that could materially affect actual results can be found in BioCardias Form 10-K filed with the Securities and Exchange Commission on April 9, 2020, under the caption titled Risk Factors. BioCardia expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.

Media Contact: Michelle McAdam, Chronic Communications, Inc.michelle@chronic-comm.com(310) 902-1274

Investor Contact: David McClung, Chief Financial Officerinvestors@BioCardia.com(650) 226-0120

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BioCardia Announces Positive Preclinical Results Supporting Investigational New Drug Application for Anti-Inflammatory Cell Therapy in Heart Failure -...

People with cancer who contract the new coronavirus appear to have a greater risk for severe COVID-19 illness and death, but this may depend on their cancer stage and the type of treatment they are receiving, according to recent research. In fact, those with early-stage cancer may fare as well as people who have not had cancer.

Researchers from some of the earliest and hardest hit epicenters of the COVID-19 pandemic described outcomes among cancer patients with the coronavirus (officially known as SARS-CoV-2) during a special session the American Association for Cancer Research (AACR) virtual annual meeting last week. Soon after the conference, another group of researchers published an analysis of mortality among cancer patients in New York City.

Early reports from China, where the pandemic originated in late December, showed that older people, those with compromised immune systems and those with underlying health conditions are more susceptible to severe COVID-19. One study saw a death rate of 6% for people with cancermore than twice as high as the overall estimated COVID-19 mortality rate in China, but lower than the rates seen in people with diabetes (7%) or cardiovascular disease (11%).

Chemotherapy medications and some targeted therapies for cancer can cause neutropenia, a temporary depletion of immune system white blood cells that fight infection. People who receive bone marrow stem cell transplants or CAR-T therapy or for blood cancers typically receive strong chemotherapy to kill off existing blood cells and make room for the new ones. Conversely, immunotherapies such as checkpoint inhibitors and CAR-T therapy unleash natural or engineered T cells to fight cancer, which in some cases can trigger an excessive immune response that leads to harmful inflammation.

Two reports at the AACR meeting provided updates from China. Li Zhang, MD, PhD, of Tongji Medical College described outcomes among 28 cancer patients with COVID-19 in Wuhan, the initial epicenter of the pandemic.

Seven had lung cancer and the remainder had 13 other cancer types. Just over a third had Stage IV, or metastatic, cancer. Nearly 30% acquired the coronavirus at medical facilities. About half had severe disease, 10 patients required mechanical ventilators and eight diedmostly from acute respiratory distress syndromegiving a mortality rate of 29%.

Although three quarters had ever undergone surgery, radiation or chemotherapy, a majority had not received treatment recently. Only one person received radiation, three received chemotherapy, two received targeted therapy and one received immunotherapy within two weeks prior to their COVID-19 diagnosis. Recent cancer treatment was associated with a fourfold increased risk of severe outcomes. However, the single patient treated with a checkpoint inhibitor (for liver cancer) had mild COVID-19 and a short hospital stay.

Similarly, as part of his discussion of immunotherapy for cancer in the COVID-19 era, Paolo Ascierto, MD, of the National Tumor Institute in Naples, noted that just two out of 400 patients on immunotherapy at his institute tested positive for the coronavirus, they were asymptomatic and they recovered quickly, leading him to speculate that immunotherapy might somehow be protective against COVID-19.

Hongbing Cai, MD, of Zhongnan Hospital of Wuhan University, presented data on 105 cancer patients and 536 age-matched people without cancer at 14 hospitals in Hubei province who developed COVID-19. Results were also published in Cancer Discovery. Twenty-two had lung cancer, 13 had gastrointestinal cancers, 11 each had breast cancer and thyroid cancer, nine had blood cancers such as leukemia or lymphomawhich affect white blood cells that carry out immune responsesand six each had cervical and esophageal cancer.

In general, patients with cancer deteriorated more rapidly than those without cancer, Cais team reported. Cancer patients with COVID-19 were nearly three times more likely to have severe or critical illness (34%), be admitted to an intensive care unit ICU (19%) or be put on a ventilator (10%). Whats more, people with cancer were about twice as likely to die as COVID-19 patients without cancer (11% versus 5%, respectively).

People with blood cancers or lung cancer, as well as those with metastatic cancer, had a higher risk of severe events. Two thirds of the blood cancer patients and half of the lung cancer patients had such events. Among the lung cancer patients, 18% were put on ventilators and 18% died. In contrast, no one with breast, thyroid or cervical cancer required ventilators or died.

In particular, those with blood cancersmore than half of whom had severe immune suppressionhad about a 10-fold higher risk of severe events or death. Two thirds had severe symptoms, 22% were put on ventilators and 33% died. These patients all had a rapidly deteriorated clinical course once infected with COVID-19, the researchers wrote.

People with metastatic cancer had about a six-fold higher risk of severe events or death. But people whose cancer had not yet spread were not significantly more likely to have severe events or die than COVID-19 patients without cancer. People currently on cancer treatment and those with a history of cancer who had completed treatment were both at higher risk.

People who underwent surgery within the previous 40 days had higher rates of severe events, ICU admission, ventilator use and death, but this was not the case for those who received only radiation. In this study, unlike Zhangs and Asciertos, people treated with immunotherapy did not fare so well. Four of the six patients who recently received checkpoint inhibitors had critical symptoms and two died.

Based on our analysis, COVID-19 patients with cancer tend to have more severe outcomes when compared to the non-cancer population, the researchers wrote. Although COVID-19 is reported to have a relatively low death rate of 2% to 3% in the general population, patients with cancer and COVID-19 not only have a nearly three-fold increase in the death rate than that of COVID-19 patients without cancer, but also tend to have much higher severity of their illness.

In a related study, Marina Chiara Garassino, MD, of Fondazione IRCCS National Tumor Institute in Milan, presented the first data from the international TERAVOLT registry, which is collecting data about COVID-19 among people with lung cancer and other thoracic malignancies. She noted that TERAVOLT was registering around 70 new cases per week from around the world per week.

This population may be especially vulnerable to COVID-19 due to older age, lung damage, smoking and underlying health conditions, Garassino said. Whats more, the symptoms of COVID-19 overlap with lung cancer, making diagnosis very challenging.

Garassino described results from the first 200 cancer patients with COVID-19 in more than 20 countries. Non-small-cell lung cancer was the most common type, and nearly three quarters had metastatic disease. About 20% received only targeted therapy, 33% received chemotherapy alone and 23% received immunotherapy alone.

A majority (76%) were hospitalized, but most were not offered intensive care for COVID-19; just 9% were admitted to an ICU and 3% were put on ventilators. More than a third (35%) died, mostly due to COVID-19 rather than cancer. Specific types of cancer treatment were not significantly associated with an increased risk of death.

But not all studies have seen worse COVID-19 outcomes among people with cancer. Fabrice Barlesi, MD, PhD, and colleagues looked at 137 COVID-19 patients with cancer at Gustave Roussy, a cancer center near Paris. They had a variety of cancer types, with blood cancers and breast cancer being most common. Nearly 60% had active advanced disease while 40% were in remission or being treated with potentially curative therapy.

Within this group, 25% had worsening COVID-19 after admission, 11% were admitted to the intensive care unit (ICU) and 15% died. Again, people with blood cancers were more likely to have worse outcomes. Treatment with chemotherapy within the past three monthsbut not targeted therapy or immunotherapydoubled the likelihood of worsening disease. But this only applied to people with active or metastatic cancer, not those who had localized disease or were in remission.

The 15% death rate among people with cancer at Gustave Roussy was lower than the 18% rate for all COVID-19 patients in Paris and in France, Barlesi said. His team concluded that both incidence and outcomes of COVID-19 among cancer patients seem to be comparable to the population as a whole. However, people with blood cancers, those treated with chemotherapy and frail patients are at greater risk.

Discussing how to manage cancer patients during the COVID-19 pandemic, Cai recommended self-protective isolation, strict infection control in hospitals and shifting some medical services online.

With regard to cancer treatment, she said, clinicians need to develop individualized plans based on a patients tumor type and stage of disease. She added that postponing surgery, if appropriate, should be considered in areas with current outbreaks. Radiation therapy, she said, could go ahead according to existing treatment plans with intensive protection and surveillance. Whether people with early-stage cancer need to postpone their treatment remains an unanswered question, she said.

Click hereto read the abstracts from the AACR COVID-19 and cancer session.Learn about What People With Cancer Need to Know About the New Coronavirus.

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Cancer Patients With COVID-19 May Have Higher Risk of Severe Illness and Death - Cancer Health Treatment News

The first-in-human, universal chimeric receptor antigen (CAR) T-cell (CAR-T) therapy GC027 was tolerable and resulted in antileukemic responses among patients with relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), according to results from a phase 1 trial presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020.1

Theuniversal CAR T cells target CD7, which, according to Xinxin Wang, PhD, ofGracell Biotechnologies Co, Ltd, in China, and lead author and presenter of thestudy, is a good target for T-ALL because it is expressed by more than 95% ofT-ALL patients.

GC027 isallogeneic, which may prevent the development of graft-versus-host disease. Theproduct is introduced using lentivirus for rapid elimination of T-ALL cells. Preclinicalstudies showed efficacy in a T-ALL xenograft model, and this prospective studyevaluated the safety and efficacy in humans.

Thesingle-arm, open-label study treated 5 adult patients with relapsed/refractoryCD7-positive T-ALL with a single infusion of 1 of 3 different dose levels ofG027: 0.6 x 107/kg, 3 x 107/kg, and 1.5 x 107/kg.Lymphodepletion therapy was administered prior to the G027 infusion. Theprimary endpoint was safety and the secondary endpoints included objectiveresponse rate (ORR) within 3 months after G027 infusion.

Patientswith extramedullary or central nervous system disease were excluded. Atbaseline, the median age was 24 (range, 19-38). Patients were heavilypretreated, with 5 median number of prior therapies (range, 1-9). Two patientshad high-risk disease and the median bone marrow tumor burden was a median of38.2% of blasts. None of the patients had undergone a prior allogeneic hematopoieticstem cell transplant.

Allpatients developed cytokine release syndrome (CRS), 4 of which were grade 3 and1 was grade 4. All cases were manageable and resolved with treatment andsupportive care. None of the patients developed neurotoxicity.

The completeremission (CR)/CR with incomplete hematologic recovery was 100%. By day 28, 4patients achieved a CR with negative for minimal residual disease (MRD) and 3of these patients remained MRD negative up to day 161. One patient achieved CRbut was MRD positive, and relapsed by day 29.

Peak CART-cell expansion in peripheral blood occurred between week 1 and 2.

As the first-in-human, universal CAR T-cell therapy for adult relapsed/refractory T-ALL, Dr Wang said, GC027 has demonstrated superior clinical efficacy and induced deep response in patients with acceptable safety profile. She added that trial enrollment is ongoing.

Reference

Wang X, Li S, Gao L, et al. Clinical safety and efficacy study of TruUCAR GC027: The first-in-human, universal CAR-T therapy for adult relapsed/refractory T-cell acute lymphoblastic leukemia (r/r T-ALL). Presented at: American Association for Cancer Research (AACR) Virtual Annual Meeting I; April 27-28, 2020. Abstract CT052.

This article originally appeared on Cancer Therapy Advisor

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First-in-Human Universal CAR-T Therapy Found Active in Relapsed/Refractory T-ALL - Oncology Nurse Advisor

A novel,bispecific CD19/CD22 chimeric antigen receptor T-cell (CAR-T) therapy wastolerable and resulted in responses among patients with acute lymphoblasticleukemia (ALL), according to results from a phase 1 trial presented at the AmericanAssociation for Cancer Research (AACR) Virtual Annual Meeting I 2020.

Thenovel CAR-T therapy was developed with the hypothesis that dual antigen-targetingstrategies may prevent antigen negative escape, Haneen Shalabi, DO, of theNational Cancer Institute and lead author and presenter of the study, said.

Thephase 1, dose-escalation study treated 13 young patients with ALL with theCD19/CD22 CAR-T therapy at 3 different dose levels, including 3 x 105,1 x 106, and 3 x 106. The bispecific construct containedFMC63 (CD19 scFv) linked with m971 (CD22 scFv) and a 4-1 BB costimulatorydomain.

Patientsunderwent lymphodepletion with fludarabine plus cyclophosphamide prior to theirCAR-T infusion. The primary endpoints were safety and toxicity, and thesecondary endpoints were efficacy, chimeric antigen receptor (CAR) expansion,and CAR persistence.

Atbaseline, the median age was 19.6 (range, 5.4-28.5). Patients had receivedprevious treatments, including hematopoietic stem cell transplant (54%),CD19-targeted therapy (69%), prior CD19 CAR T cell therapy (38.4%),blinatumomab (61.5%), CD22-targeted therapy (38.4%), inotuzumab (30.7%), andCD22 CAR-T therapy (15.4%). Extramedullary disease was present in 46.2% ofpatients.

CAR Tcells were well tolerated and toxicities were reversible in all patients, DrShalabi said.

Cytokinerelease syndrome (CRS) developed in 46% of patients, 15.4% of which was grade 3or higher. Both patients who developed grade 3 or higher CRS had received the 1x 106 dose level of the CD19/CD22 CAR-T product and both requiredtreatment with tocilizumab. One patient developed neurotoxicity, and hadreceived the 3 x 106 dose level.

Of the12 patients evaluable for efficacy, a complete response (CR) was achieved by42% (5) of patients, including all patients who received the 1 x 106 or 3 x 106 dose levelsof the CD19/CD22 CAR-T therapy. There were 2 nonresponders.

Two patientswho received 1 x 106 CAR-T and all patients who received the 3 x 106dose level were negative for minimal residual disease (MRD), with the remainingCRs demonstrating bone marrow clearance. Four of the 5 patients who were MRDnegative were also naive to CAR-T therapy.

Of the 5patients who achieved a CR, 2 relapsed with CD19-positive/CD22-positive diseaseand 3 remained in remission at a median 7 months after CAR T cell infusion.

Severalpatients, however, who were MRD negative in the bone marrow did not achieve CRin their extramedullary disease. Dr Shalabi said that these discrepant resultsbetween marrow and extramedullary disease suggests potentially limited CAR-Ttrafficking to sites of extramedullary disease. She suggested that treatmentat higher dose levels may be needed to overcome this limitation.

CAR T-cellexpansion occurred in all patents who responded, with a median peak inperipheral blood of 7%. At day 28, there were 1.3% CAR T cells in the bonemarrow. The persistence of the CAR T cells in peripheral blood was a median of45.6 days, as measured by flow cytometry.

Dr Shalabi concluded that this early experience with bispecific CD19/CD22 CAR T cells demonstrates clinical activity with reversible CRS and limited neurotoxicity. She noted that future studies will explore a 1 x 107 dose level, intensification of lymphodepletion prior to CAR-T infusion, and consideration of the potential role of immune checkpoint inhibitors to augment CAR-T in extramedullary disease.

Read more of Cancer Therapy Advisors coverage of AACR 2020 meeting by visiting the conference page.

References

Shalabi H, Yates B, Shahani S, et al. Safety and efficacy of CD19/CD22 CAR T cells in children and young adults with relapsed/refractory ALL. Presented at: American Association for Cancer Research (AACR) Virtual Annual Meeting I 2020; April 27-28, 2020. Abstract CT051.

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Novel Bispecific CD19/CD22 CAR-T Therapy Deemed Tolerable in Relapsed/Refractory ALL - Cancer Therapy Advisor

As the world continues to battle the coronavirus pandemic, scientists are looking towards gene therapy to find ways to develop vaccines for the Covid-19 virus. Gene therapy itself was developed based on how viruses work.

When a virus attacks a host, it introduces its genetic material into the host cell as part of its replication cycle. The genetic material serves as an instruction manual on how to produce more copies of the virus, hijacking the host body's normal production machinery to serve the needs of the virus. The host cells then produce additional copies of the virus, leading to more host cells being infected.

Like animals, humans have found a way to domesticate viruses as well, i.e., direct the virus's function to achieve favorable results, which is prominent in gene therapy. Such viruses which physically insert their genes into the host's genome could instead be used to carry "good" genes into a human cell. Scientists would first remove the genes in the virus that cause diseases, and replace those genes with genes encoding the desired effect.

All of this sounds quite sci-fi but it has been done numerous times in the past. Peter Kolchinsky, a virologist and a biotechnology investor, compiled how different viruses have been used for gene therapy in the past.

Kolchinsky tweeted, "SARS2 is a scary menace, but did you know that we've domesticated viruses? Like wolves vs dogs, we've tamed them, including some deadly ones, to perform many useful functions (and may help us stop SARS2)."

The human immunodeficiency virus (HIV) has killed millions of people. It works by disabling the host body's immune system until it can't defend the person against common, normally mild pathogens. Kolchinsky explained that HIV's special trick is to integrate its genome into that of the host body's cells.

This feature of HIV is used for gene therapy, as explained before, by replacing a chunk of the virus's genome with the hemoglobin gene to insert it into bone marrow stem cells of patients with sickle cell anemia, whose hemoglobin genes are malfunctioning.

Kolchinsky also tweeted, "Adenoviruses typically cause mild infections, including common colds. These, too, we are trying to use for gene therapies, particularly when we just want to temporarily make a protein in cells. One company is developing such an adenovirus gene therapy for heart disease to induce growth of new blood vessels when old ones are clogged. Another is using this virus to make oral vaccines that would otherwise require injection (eg flu vaccine pill). When we use a virus to deliver code for making something in cells, we call that a virus vector."

There is now a wealth of clinical experience with numerous vector types that include primarily vaccinia, measles, vesicular stomatitis virus (VSV), polio, reovirus, adenovirus, lentivirus, -retrovirus, adeno-associated virus (AAV) and herpes simplex virus (HSV).

However, as with all other procedures, viral vector-gene therapy has associated risks. Viruses can usually infect more than one type of cell, so, when viralvectorsare used to carrygenesinto the body, they might infect healthy cells as well as cancer cells.

Another danger is that the new gene might be inserted in the wrong location in the DNA, possibly causing harmful mutations to the DNA or even cancer. Moreover, when viruses are used to deliver DNA to cells inside the patient's body, there is a slight chance that this DNA could unintentionally be introduced into the patients reproductive cells. If this happens, it could produce changes that may be passed on if a patient has children after treatment.

One study to help find a vaccine for Covid-19 aims to use the principles behind gene therapy to get the vaccine ready. The researchers' method uses a harmless virus as a vector to bring DNA into the patient's cells. The DNA should then instruct the cells to make a coronavirus protein that would stimulate the immune system to fight off future infections.

While a mass-produced vaccine may still take a while, this study is one of at least 90 vaccine projects around the world trying to find a cure for Covid-19. However, some experts are worried that a vaccine may never be available. According to our previous report, Dr David Nabarro, a professor of global health at Imperial College London, who also serves as a special envoy to the WHO on Covid-19, said, "There are some viruses that we still do not have vaccines against."

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Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough - MEAWW

Veteran Bollywood actor Rishi Kapoor, who was battling leukemia for the last two years, died at Mumbais Sir HN Reliance Foundation Hospital on Thursday (April 30) morning. He was 67. Rishi Kapoor got treated for leukemia in the US for a year and had returned to India in September 2019.

Leukemia is a blood cancer caused by a rise in the number of white blood cells in your body. Those white blood cells crowd out the red blood cells and platelets that your body needs to be healthy. The extra white blood cells dont work right. A cancer of blood-forming tissues, hindering the body's ability to fight infection.

Dr Rahul Bhargava, Director, Haematology, Haemato Oncology and Bone Marrow Transplant, Fortis Memorial Research Institute, Gurugram says:

Leukemia, often fatal, is a blood cancer. As with cancer, it is associated with an exponential rise in the number of white blood cells. Symptoms begin to manifest slowly and can often be mistaken for something else sweating uncontrollably, shaking, fever, vomiting. It is caused by huge was is reduced focus on ones own health.

Intake of processed food, soda, artificial and synthetic sweets and preservatives, pesticide use in farms, lack of physical exercise, excessive tobacco and drug consumption, excessive alcohol intake, caffeine consumption are the main culprits causing cancer. Family history also plays an important role in a leukemia diagnosis. Only through screening can it be identified and if caught early managed. One needs to be mindful and ensure that they are employing good lifestyle habits.

This means eating green leafy vegetables and fruits, going for a 30 minutes walk every day or engaging in any other form of exercise for 30 minutes a day, drinking adequate amount of water to keep flushing out the toxins, controlling or completely eliminating our use of tobacco and other unnatural substances. Processed food and sugars should be consumed in control, they are the single largest cause of mutation to cells.

Leukemia symptoms vary, depending on the type of leukemia. Common leukemia signs and symptoms include:

Leukemia can also cause symptoms in organs that have been infiltrated or affected by the cancer cells. For example, if the cancer spreads to the central nervous system, it can cause headaches, nausea and vomiting, confusion, loss of muscle control, and seizures.

Leukemia can also spread to other parts of your body, including:

The major types of leukemia are:

Blood has three types of cells: white blood cells that fight infection, red blood cells that carry oxygen, and platelets that help blood clot. Every day, your bone marrow makes billions of new blood cells, and most of them are red cells. When you have leukemia, your body makes more white cells than it needs. These leukemia cells cant fight infection the way normal white blood cells do. And because there are so many of them, they start to affect the way your organs work. Over time, you may not have enough red blood cells to supply oxygen, enough platelets to clot your blood, or enough normal white blood cells to fight infection.

Leukemia is usually treated by a hematologist-oncologist. These are doctors who specialize in blood disorders and cancer. The treatment depends on the type and stage of the cancer. Some forms of leukemia grow slowly and don't need immediate treatment. However, treatment for leukemia usually involves one or more of the following:

Source: Information has been taken from various health website

Read more| National Honesty Day 2020: Date, history, significance and quotes on the value of honesty

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What Is Leukemia? Here's all you need to know - India Today

Global Cell Therapy Technologies Market was valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

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The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

The report study has analyzed revenue impact of covid-19 pandemic on the sales revenue of market leaders, market followers and disrupters in the report and same is reflected in our analysis.

Global Cell Therapy Technologies Market: Overview

Cell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

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Global Cell Therapy Technologies Market: Drivers and Restraints

The growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation Analysis

On the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional Analysis

North America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South AmericaKey players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company GE Healthcare Lonza Merck KGaA MiltenyiBiotec STEMCELL Technologies, Inc. Terumo BCT, Inc. Thermo Fisher Scientific, Inc. Sartorius AG

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Therapy Technologies Market Competition, by Players

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

Chapter Seven: Asia-Pacific Cell Therapy Technologies Revenue by Countries

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Therapy Technologies by Countries

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

Chapter Twelve: Global Cell Therapy Technologies Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Cell Therapy Technologies Market Report at: https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/

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Global Cell Therapy Technologies Market : Industry Analysis and Forecast (2019-2026) - MR Invasion

Javier Saade serves on several boards, is venture partner at Fenway Summer and is a senior advisor at FS Vector, Fenway Summers advisory affiliate. Previously, he was associate administrator and chief of investment and innovation at SBA.

The last two decades have ushered in significant change and transformation. I believe the 2020s will be dispositive in redefining the pillars of our economy, and COVID-19 magnifies this greatly. As of this writing there are 3,611,394 confirmed cases, and the U.S. accounts for 33% of those. We are now dealing with a 4.8% Q1 GDP contraction and expectations for Q2s shrinking runs into the 25% range, more than 30 million unemployed and a $7 trillion federal intervention in a span of six weeks.

Eric Schmidt recently predicted that the coronavirus pandemic is strengthening big tech. It is hard to disagree with him; it almost feels obvious. Big tech and other digital companies are net beneficiaries of new habits and behaviors. Some of this shift will be permanent, and well-capitalized tech companies are likely to expand their power by grabbing talent and buying companies for their IP then dissolving them.

With power comes political backlash and public wariness. One flavor of that counter pressure is already in full effect. Sen. Elizabeth Warren and Rep. Alexandria Ocasio-Cortez have proposed new legislation that seeks to curtail acquisition activity via the Pandemic Anti-Monopoly Act. Ill reserve judgment on their effort, but the theme is familiar: the strong get stronger and the weak get weaker, which further widens gaps and calcifies disparity.

The COVID-19 shock is highlighting a chasm that has evolved over decades. The digital divide, lack of capital access, sporadic paths to education and microscopic levels of wealth accumulation in communities of color and the implicit/explicit bias against non-coastal elites are some contributing factors.

During the 2008 crisis, the combined value of the five biggest companies ExxonMobil, General Electric, Microsoft, AT&T and Procter & Gamble was $1.6 trillion. Microsoft is worth almost that today all by itself. No need to talk about FAANG, because since the pandemics economic halt, Peloton downloads went up five-fold in a month, Zoom grew to 200 million users from 10 million in December and Instacart users grew six times in that period.

Roelof Botha of Sequoia Capital was recently quoted as saying, Like the killing off of the dinosaurs, this reorders who gets to survive in the new era. It is the shock that accelerates the future that Silicon Valley has been building. It is hard to argue with his views.

To be clear, I am a beneficiary of and a big believer in technology. Throughout my career I have managed it, invested in it and made policy on it. For example, one of the multi-billion-dollar programs I oversaw, the Small Business Innovation Research (SBIR) program, has invested more than $50 billion in tens of thousands of startups, which have collectively issued 70,000 patents and raised hundreds of billions of capital and 700 of them have gone public, including tech titans such as Qualcomm, Biogen and Symantec.

My point: I think about technology a lot, and, lately, about its repercussions. There is a massive shift afoot where more power and influence will be consolidated by these remarkable companies and their technology. Besides the economic consequences of the strong crushing the weak, there are serious ethical issues to consider as a society. Chamath Palihapitiya has been pretty vocal about the moral hazard of what is essentially a massive transfer of wealth and income. On one side you have mismanaged and/or myopic corporations and on the other, the counterparty is the American people and the money we need to print to bankroll the lifeline. I am not talking about Main Street here, by the way.

It is not hard to imagine a world in which tech alone reigns supreme. The ethical dilemmas of this are vast. A recent documentary, Do You Trust this Computer, put a spotlight on a frantic Elon Musk ringing the alarm bell on machines potential to destroy humanity. Stephen Hawking argued that while artificial intelligence could provide society with outsized benefits, it also has the potential to spiral out of control and end the human race. Bill Gates has been less fatalistic, but is also in the camp of those concerned with synthetic intelligence. In an interesting parallel, Bill has for years been very vocal on the risks pandemics pose and our lack of preparedness for them indeed.

These three men have had a big impact on the world with and because of technology. Their deep concern is rooted in the fact that once the genie is out of the bottle, it will make and grant wishes to itself without regard to humanity. But, is this doomsday thinking? I dont know. What I do know is that I am not alone thinking about this. With COVID-19 as a backdrop, many people are.

Algorithmic sophistication and computer horsepower continue to evolve by leaps and bounds, and serious capital continues to be invested on these fronts. The number of transistors per chip has increased from thousands in the 1950s to over four billion today. A one-atom transistor is the physical boundary of Moores Law. Increasing the amount of information conveyed per unit, say with quantum computing, is the most realistic possibility of extending Moores Law, and with it the march toward intelligent machines and a tech first world. The march has been accelerated, even if peripherally, by the pandemic.

While the promise of technology-driven progress is massive, there are some serious societal costs to exponential discovery and unleashed capability acceleration. Dartmouths Dr. James Moor, a notable thinker at the intersection of ethics and technology, believes that the use and development of technologies are most important when technologies have transformative effects on societies. He stipulates that as the impact of technology grows, the volume and complexity of ethical issues surrounding it increases. This is not only because more people are touched by these innovations, they are. It is because transformative technology increases pathways of action that outstrip governance systems and ethical constructs to tame it.

So what? The twists and turns of technology application lead to consequences, sometimes unknowable and for that reason we should be increasingly vigilant. Did Zuckerberg ever imagine that his invention would have been so central to the outcome of the 2016 election? Unknowable consequences, exhibit one. Interconnected systems touch every aspect of society, from digital terrorism to bioengineering to brain hacking and neural cryonics to swarm warfare, digital assets, intelligent weapons, trillions of IoT connected devices the list goes on.

As a society, we should be open to innovation and the benefits it ushers in. At the same time, we must also remain committed to sustainable tech development and a deployment mechanism that does not fail to shine a light on human dignity, economic inequality and broad inclusiveness. These seem like esoteric issues, but they are not, and they are being put to the test by COVID-19.

A fresh example of this thematic happened recently: Tim Bray, a VP and engineer at Amazons AWS, resigned because of the companys treatment of employees, and was quoted as saying, in part, Amazon treats the humans in the warehouses as fungible units of pick-and-pack potential. Only thats not just Amazon, its how 21st-century capitalism is done If we dont like certain things Amazon is doing, we need to put legal guardrails in place to stop those things.

Eliminating human agency has been at the core of innovation during the last four decades. Less human intervention in a call center, a hedge fund trading desk, a factory, a checkout line or a motor vehicle seems fine but in cases of greater importance, humans should remain more active or we will, at best, make ourselves irrelevant. In the past, labor displacement has been temporary, but it seems to me that the next wave is likely to be different in terms of the permanence of labor allocation, and big tech getting bigger will likely hasten this.

Innovative capability has been at the center of progress and living standard improvements since we harnessed fire. The worlds technology portfolio is an exciting one, but potentially terrifying to those who could be more hampered by it, such as the front-line workers on Main Street shouldering the health and economic brunt of the coronavirus.

Years ago, Peter Drucker pointed out that technology has transformed from servant to master throughout our history. Regarding the assembly line, he noted that it does not use the strengths of the human being but, instead, subordinated human strengths to the requirements of the machine.

In my opinion, Druckers quote is at the very core of our point in time, happening on a scale and speed that is hard to fathom and changing the digital divide amongst us into a digital canyon between us and technology.

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Technology and ethics in the coronavirus economy - TechCrunch

In August 1989, scientists made a blockbuster discovery: They pinpointed the faulty gene that causes cystic fibrosis, a cruel lung disease that killed many of its victims before they reached adulthood.

The human genome was uncharted territory, and the gene hunt had become an all-out international race, with laboratories in three countries searching for the root of the disease.

That fall, biologist James Wilson stood before an audience of researchers, physicians and cystic fibrosis patients and their families and described gene therapy, a way to replace the faulty gene with a good copy. Wilson had intended his talk to be technical and prophetic, but he was overwhelmed by the surging thrill in the room that science was about to save peoples lives.

It was one of the most amazing experiences that Ive ever had, Wilson said, adding, The expectations were through the roof.

The importance of the cystic fibrosis gene discovery went far beyond a single illness. It helped build the case for the $3 billion project to sequence the entire human genome, which would alter understanding of human biology and shed light on rare and common diseases.

But the story of cystic fibrosis has been illustrative in a way that no one could have anticipated back then. In the early days of human genetics, the path seemed straightforward: Find the gene, fix the gene and repeat for other diseases. The cystic fibrosis journey, from an exuberant moment of insight to a major success, would take 30 years of persistent, methodical work: a feat of science, business, fundraising and patience that has become a model for other diseases.

I specifically remember sitting with my doctor in the exam room, having the conversation that the gene was discovered, said Josh Taylor, 48, of Virginia Beach, who has cystic fibrosis. And him telling me the cure is just he literally said, In 5 to 10 years, were going to beat this.

It was not until late 2019 that another breakthrough fulfilled many of the hopes of 1989. Now, Taylor has what he has been waiting for all these decades a new drug, Trikafta, that is effective for 90 percent of patients. Doctors marvel at what they think will be possible if it is given at an early age: a full life span.

Cystic fibrosis developed when a child had the bad luck to inherit two faulty genes, one from each parent. Back then, there was no test to detect whether a parent carried a defective gene because no one even knew what the gene was.

As scientists developed new tools to probe human genetics, cystic fibrosis quickly became one of the top targets. It is the most common inherited disease among Caucasians, afflicting 30,000 Americans, and its motivated patient group spurred the work forward with funding.

All these human disease genes were floating around. We knew they were inherited, but we knew very little. We didnt know what the genes were, or where they were located, said Robert Nussbaum, a medical geneticist who was hunting genes for other diseases.

Francis Collins, now director of the National Institutes of Health and then a scientist at the University of Michigan working on cystic fibrosis, was photographed for the universitys graduates magazine sitting in a haystack holding a needle, to convey the magnitude of the technical challenge.

Almost everybody knew some family where it had happened, and it was heartbreaking to see what these kids go through, Collins said.

Robert Beall, then an executive vice president at the Cystic Fibrosis Foundation, which was funding the work, was also the most impatient human being I ever met to his credit, Collins said.

Collins partnered with biologist Lap-Chee Tsui, in Toronto holding joint lab meetings at a midway point on the long drive, in London, Ontario.

After years of work, Tsuis lab had narrowed the search to ever smaller stretches of DNA, pioneering new techniques in the search for the gene. Collins had invented a method to speed up the process called chromosome jumping, which allowed scientists to leap over sections of DNA something he compares to leaping from one street corner to the next to initiate searches. Jack Riordan, another scientist in Toronto, discovered a bit of DNA that looked like it might be a part of the gene, providing an essential lead.

In May, a scientist in Tsuis lab found a tantalizing clue three missing letters of DNA in a patient with cystic fibrosis. The team would need to confirm that this genetic mutation was the cause of the disease. Collins and Tsui were at a scientific conference at New Haven, Conn., a month later when they got more evidence.

One rainy night after the days program was over, the pair raced to Tsuis room, where he had installed a portable fax machine to receive updates from the lab. Among the papers that had spilled onto the floor was a table showing those three letters of DNA missing in multiple patients with cystic fibrosis, while they were present in healthy people.

Lap-Chee was a little more skeptical, Ive got to see more data, Collins recalled. I bought it, that was it. I wanted to scream and jump up and down.

The news report triggered frantic preparations to present the findings officially, and the work was published in Science magazine that September in three papers.

Collins would testify before Congress that it was necessary to fund the human genome project because the flat-out effort to find the cystic fibrosis gene simply would not be scalable in trying to understand thousands of other diseases.

Gene therapy, the thinking went, would soon cure cystic fibrosis, marking a turning point in the treatment of genetic diseases. The idea was relatively straightforward: Use a virus to ferry a good, functioning copy of the gene into patients lung cells.

But human biology turned out to have all sorts of ways of resisting an easy fix, and it quickly became clear that gene therapy would not be simple in real lungs.

Then the entire gene therapy field screeched halted in 1999 with the death of Jesse Gelsinger, a teenager with a metabolic disorder who died after being treated for the disorder in one of Wilsons gene therapy trials.

As the hope for a high-profile gene therapy success crashed, research continued on the basic, less glamorous work to untangle what went wrong with the cystic fibrosis gene. That understanding made it possible to develop ways to screen chemicals, to see if any showed promise as a drug.

Beall and Preston Campbell of the Cystic Fibrosis Foundation visited Aurora Biosciences, a San Diego biotech company that used robotics to massively speed up such testing.

Bob and I were like kids in a candy shop, Campbell recalled. After a small initial investment, the foundation stunned the nonprofit world in 2000 by awarding the company $40 million, a new kind of venture philanthropy arrangement in which if the company was successful, the nonprofit group would receive a share of the royalties.

A Massachusetts company, Vertex Pharmaceuticals, acquired Aurora in 2001, and although the cystic fibrosis work continued, it was considered a long shot, called the fantasy project internally, recalled Fred Van Goor, a scientist who joined the company around that time and became the biology lead for the cystic fibrosis program.

The scientific problem was huge: The most common gene mutation in cystic fibrosis created a protein that couldnt do its essential job in the cell. The protein didnt fold correctly, which interfered with its ability to reach the surface of the cell. And it didnt function well once there, where it was supposed to work as a gate. That meant theyd need multiple drugs to help patients one to get the protein to the right spot, the other one to open the gate.

Vertexs first drug candidate was focused on just one of the problems getting the gate to work better. Alone, it would help only about 4% of patients, whose disease was caused by a rare mutation. That drug, Kalydeco, was approved in 2012, but it remained unclear whether a drug could be made that would work for a larger group of patients.

Then, Vertexs main product a hepatitis C drug was eclipsed by a better treatment from a competitor, and the future of the company and its cystic fibrosis research was cast in doubt.

It obviously created an incredible crisis here at Vertex, said Jeff Leiden, chief executive of the company.

Vertexs board decided to bet on cystic fibrosis, and in 2015, a two-drug combination called Orkambi, was approved for a larger group of cystic fibrosis patients. Excitement about the drugs began to yield to a societal debate about their high prices; Orkambis launch price was $259,000 a year.

Meanwhile, the company would need to develop a third drug to treat more patients.

Drug trials are blinded so that neither the patients nor the scientists know which people are receiving the drug and which are receiving a placebo. When Trikafta, the triple drug combination that would ultimately be approved, was unblinded from one trial in October 2018, researchers finally saw the slide showing how the drug affected lung function.

There was a stunned silence in the room for a full minute. The drug worked.

Ten percent of cystic fibrosis patients, or about 3,000 people in the United States, are still waiting for a therapy that works for them.

Stacy Carmona, who was born just three years before the gene was discovered, is one of them.

Im so excited for the community. Im so excited for the CF friends I have who so desperately need the drug. There are so many people hanging on by a thread, waiting for this, Carmona said. The flip side of that is you cant help but wonder when is it going to be my turn?

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A cystic brosis success story -- over 30 years | Health - The Union Leader

In 2014, a sardonic tweet about the differences between mens and womens deodorant descriptors went viral. It read: Women's deodorant scents: rose, cotton, spring, meadow. Mens: WINTER ICE, SHARKNADO, GLACIER PUNCH, ANTIFREEZE, GUN. The tweet, which was shared more than 24,000 times, inspired scores of similar, fabricated product names poking fun at the gendered way in which skincare and grooming products are typically marketed to men and women.

Satire aside, anyone browsing the toiletries aisle cant help but be aware of the invisible barrierand, more often than not, the clearly marked signsseparating the droves of baby-pink, pastel and powder-blue womens skincare products from the mostly monochromatic mens grooming choices, reassuring prospective purchasers that they are, in fact, suitable for men. Aside from the potentially harmful psychological effects of gender-specific marketing, the very real pink tax fosters another type of inequityin 2015, a study commissioned by the New York City Department of Consumer Affairs uncovered that, on average, personal-care products marketed to women will cost up to 13 per cent more than similar products targeted at men.

As we move towards more inclusive definitions of gender, is gendered skincare simply an antiquated (and sexist) marketing ploy or does it truly cater to the distinct needs of male and female skin compositions?

Its no secret that industry insiders have known for a while now that gender in consumption is often nothing more than a social construct. Were conditioned from a young age to classify certain colours, toys and products as feminine, while we gradually learn to think of others as specifically masculine. Just think of the toys you giddily unwrapped before tucking into your Happy Meal as a childdolls for the girls, cars for the boys.

This phenomenon continues to inform our thinking well into adulthood, with men tending to be more concerned with maintaining a masculine identity and choosing to buy products that align themselves with that image; its even been shown that men purchase more in the presence of a strong-looking man. Such intelligence is invaluable for marketers determined to bolster their bottom line.

Marketing strategies and social conditioning notwithstanding, is mens skin really different enough to warrant an entirely separate skincare regimen? While its true that mens skin is between 20 and 25 per cent thicker than womens (as it contains more collagen and elastin) and it tends to produce more oil, the American Academy of Dermatology assures us that the basics of our daily skincare regime should be the same, regardless of gender. Instead of thinking of skin as either male or female, the most important factors to look out for when putting together an effective skincare routine are skin type (normal, sensitive, oily, dry or combination) and areas of concernthink anti-ageing, blemishes, dark spots, and so on.

With consumers everywhere increasingly casting a critical eye on traditional beauty norms, the past few years have witnessed a sharp rise in gender-neutral skincare. Well-established brands and products that have long positioned themselves as neutral go-tos such as Australian heavyweight Aesop and ubiquitous unisex fragrances, including CK One, are now being joined by up-and-coming newcomers aiming to disrupt the gender-specific market.

Panacea, the K-Beauty-inspired skincare line, continues its hot streak in 2020. The brainchild of Korean-American co-founder Terry Lee, Panacea was conceived with the idea of introducing a gender-agnostic approach to skincare, while reducing the typical (and time-consuming) 10-step, K-Beauty approach to a mere three steps. The newest addition to the ever-expanding roster of universal pampering products is American Eagles in-house wellness and skincare line, Mood. This non-gendered range of hemp-derived CBD products includes everything from face oil and bath bombs to pillow mists.

Also looking to break down gender-specific beauty is Alll, which provides a personalised, DNA-based approach to skincare. Recent studies suggest our genetics account for 50 to 60 per cent of ageing, says Dr Elisabet Hagert, co-founder of Alll. This means how we age really is dependent on our genetic predispositions. Based on an in-depth DNA analysis, a bespoke skincare line is created, targeting individual concerns at a granular level regardless of gender.

As we progress into the new decade, were being spoiled for choice when it comes to skincare and grooming solutions with an emphasis on skin type and areas of concern, rather than gender. The only question that remains is this: with us men being fiercely brand loyal, will we be willing to leave our Sharknado-scented deodorants behind?

Is customisable skincare the future of the beauty industry?

A man explains why more men need to pay attention to skincare

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Skin deep: Is the future of skincare gender neutral? - VOGUE India

The Medical Research Council (MRC) and independent medical research charity LifeArc are streaming 16 million into establishing a network of gene therapy innovation hubs.

The centres will offer clinical grade viral vectors as well as translational and regulatory guidance to support academic-led patient trials of new gene therapies.

Operating as 'centrally coordinated facilities', the hubs aim to address challenges faced by academics as they seek to advance novel gene therapy research into early stage clinical trials, such as a shortage of viral vector production capacity and a complex and evolving translational pathway for gene therapies.

LifeArc and the MRC said they will create the network by providing UK-based research organisations with grants for up to five years, to support the costs associated with expanding or repurposing existing viral vector production centres.

The selected centres, or hubs, will also have access to LifeArcs translation advice and support.

We hope that through this unique collaboration with the MRC, LifeArc can offer its funding and expertise in technology transfer and translational science to support the progression of promising gene therapies, said Dr Melanie Lee, the charity's chief executive. Translation of advanced therapies will be a core focus of LifeArcs future strategy for delivering significant new patient benefits.

MRC executive chair Professor Fiona Watt added: Through this partnership, we aim to support clinical development of the most exciting gene therapy projects from the UKs world-leading academic researchers. This investment will streamline and accelerate progress towards a new generation of genetic medicines for patients.

Read more:
LifeArc, MRC create 16m fund to set up gene therapy hubs - PharmaTimes

SAN RAFAEL, Calif., May 3, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the company has entered into a preclinical collaboration and license agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR will receive an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales. The company did not disclose financial terms. BioMarin management reiterated its 2020 GAAP net income guidance of $20 to $80 million, inclusive of this collaboration.

The license initially covers DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM). Additionally, the companies will collaborate on several of DiNAQOR's other pipeline programs, and BioMarin has the option to extend the license to include these additional programs on similar terms. Reflecting the long-term commitment to the collaboration, BioMarin is simultaneously investing in DiNAQOR.

"With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer at BioMarin. "This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies."

"We are thrilled to collaborate with the researchers at DiNAQOR to conduct this pioneering work on the development of gene therapies for inherited cardiomyophathies," said Lon Cardon, Chief Scientific Strategy Officer and Senior Vice President at BioMarin. "We believe there is tremendous potential in combining our experience in gene therapy research and development with DiNAQOR's in-depth knowledge of genetic heart diseases."

DiNAQOR was founded and is led by several leading pharmaceutical and biotechnology executives and academics with deep cardiology and gene therapy expertise. The company's holistic approach to gene therapy is focused on gene therapies for the heart that deliver a medical solution that can safely deliver gene therapies to the heart muscle, ensure transduction of the cardiac cells, and limit the exposure of the therapy to other organs.

"BioMarin is a global leader in rare disease research, development and commercialization, and their commitment to DiNA-001 is a powerful validation of DiNAQOR's gene therapy platform," said Dr. Johannes Holzmeister, Co-Founder, Chairman and CEO at DiNAQOR. "We believe our platform has many potential applications and this milestone agreement will enable us to invest in expanding our genetic medicine pipeline."

"Momentum for gene therapies continues to build, and BioMarin has demonstrated tremendous scientific, clinical, and manufacturing leadership and expertise in the space," said Thomas Voit, M.D., Ph.D., Co-Founder and Chief Scientific Officer at DiNAQOR and Director of the Biomedical Research Centre at the Great Ormond Street Hospital and the UCL Institute of Child Health, University College London. "We are looking forward to combining our strengths to expand the promise of gene therapy treatments by targeting the heart muscle to treat rare genetic cardiomyopathies."

About HCM and MYPBC3

Hypertrophic cardiomyopathy (HCM) is one of the most common genetic heart diseases, with about 500,000 patients diagnosed with HCM worldwide. Up to 60% of HCM cases have a genetic origin, and it is estimated that 40% of those have mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C (MyBP-C).

HCM affects the heart muscle, causing the muscle to enlarge. HCM patients have an increased risk of developing heart failure and life-threatening arrhythmias. There are no approved pharmacological treatment options available that address the underlying disease biology of HCM and invasive surgery or heart transplantation may be the only options available for patients with advanced disease.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit http://www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

About DiNAQOR

Founded in 2019, DiNAQOR AG is a global gene therapy platform company focused on advancing novel solutions for patients suffering from heart disease. The company's lead preclinical program, DiNA-001 is focused on the treatment of MYBPC3-linked cardiomyopathy. DiNAQOR is headquartered in Pfffikon, Switzerland, with additional presence in London, England and Boston, Massachusetts (US). For more information visit http://www.dinaqor.com.

Forward Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: BioMarin's expectations regarding the announced collaboration, the prospects for the lead and follow on pipeline products and it's 2020 GAAP profitability. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning the programs; the ability to manufacture the product candidates, BioMarin's revenue for 2020, especially with the possible impact of COVID-19, and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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Company Codes: NASDAQ-NMS:BMRN

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BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic...

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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BioMarin broadens its gene therapy horizons with a new R&D alliance in rare cardio cases - Endpoints News

When the axons that extend from neurons break during a spinal cord injury, the result is often a lifelong loss of motor functioning, because vital connections from the brain to other body parts cannot be restored. Now, researchers from Temple Universitys Lewis Katz School of Medicine say they may have found a way to recover some functions lost to axon breaks.

The researchers discovered that boosting levels of a protein called Lin28 in injured spinal cords of mice prompts the regrowth of axons and repairs communication between the brain and body. Lin28 also helped repair injured optic nerves in the animals, they reported in the journal Molecular Therapy.

The Temple team zeroed in on Lin28 because its a known regulator of stem cells, meaning it controls their ability to differentiate into various cells in the body. The researchers examined the effects of Lin28 on spinal cord and optic nerve injuries using two mouse models: one that was engineered to express extra Lin28 and another that was normal and was given the protein after injury via a viral vector.

Maintaining Momentum: Applying Recent Regulatory Guidance in the Midst of the Coronavirus

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All of the mice experienced axon regeneration, the researchers reported. But they found that the best results occurred in the normal mice that received Lin28 injections post-injury. In fact, in animals with optic nerve injuries, the axons regrew to the point where they filled the entire tract of the nerve.

Lin28 treatment after injury improved coordination and sensation in the mice, the researchers reported.

"We observed a lot of axon regrowth, which could be very significant clinically, since there currently are no regenerative treatments for spinal cord injury or optic nerve injury," said senior author Shuxin Li, M.D., Ph.D., professor of anatomy and cell biology at the Lewis Katz School of Medicine, in a statement.

RELATED: Gene therapy with 'off switch' restores hand movement in rats with spinal cord injury

Lin28 is already a target of interest, though it has garnered the most attention so far in cancer research. Startup Twentyeight-Seven Therapeutics is developing a small molecule that inhibits the protein in the hopes that doing so will boost Let-7, a cancer-suppressing microRNA. The company raised more than $82 million in a series A financing last year.

Several new approaches for repairing spinal cord injuries are under investigation, most notably gene therapy. King's College researchers are working on a gene therapy that repairs axons by prompting the production of the enzyme chondroitinase. A UT Southwestern team is targeting the gene LZK to increase levels of supportive nervous system cells called astrocytes in response to spinal injuries.

The Temple team has a two-pronged approach to further developing their Lin28-directed treatment. They hope to develop a vector that can be safely delivered by injection and that would deliver the therapy directly to damaged neurons. They also plan to study other molecules in the Lin28 signaling pathway.

"Lin28 associates closely with other growth signaling molecules, and we suspect it uses multiple pathways to regulate cell growth," Li said, potentially revealing other therapeutic molecules that could further boost neuron repair.

Originally posted here:
Repairing spinal cord injuries with a protein that regulates axon regeneration - FierceBiotech

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Summary

Its an understatement to say that Facebook has changed the world its created an ability for people to be transparent about their experiences, lives, and opinions, for better or worse. In the case of Homology, its for the latter. Homology, a gene therapy company whose technology has already been scrutinized by scientists as untrue, has just one product in clinical trials, HMI-102, for the rare disease phenylketonuria ("PKU"). As Med Genie reported back in January, FIXXs trial update showed interim phenylalanine (Phe) results that suggested HMI-102 was not efficacious for low and mid-dose patients. Our note today highlights a data point the company would very much like you not to know the only patient in FIXXs high dose cohort posted her results on Facebook, and they show that HMI-102 is unlikely to reach trial endpoints even at a high dose. We believe this patient was forced to take her posts down as a result, and management selectively disclosed the issue to the sell side, providing comments about the drugs safety, and conveniently ignoring the implications to efficacy and the business. Maybe they were hoping to raise capital before officially announcing trial results. Who knows? We believe that the HMI-102 program is dead in the water, and since its progress is the major driver of FIXXs value, we believe that the stock should trade to cash value, or $5.80, down 56% from the April 27th close.

Background

The best shorts are often one-trick ponies, but rarely do we find one where a single data point completely upends the long investment case. Homology Medicines, FIXX, is one of those rare finds.

Homology is a gene therapy company which has their first and lead product candidate, HMI-102, in a dose-escalation Phase 1/2 trial (pheNIX) for phenylketonuria. FIXX also has 2 IND-enabling programs and some discovery stage programs, but the HMI-102 trial is the companys main shot at viability.

PKU is a relatively rare disease, with a U.S. incidence of approximately 350 cases per year and a prevalence of just 16,500, per FIXXs 10-k. PKU is tied to mutations in the gene that control PAH, an enzyme that metabolizes phenylalanine, or Phe. The condition results in a deficiency in the enzymatic activity of PAH, causing an excess in Phe in the bloodstream that can result in intellectual disability. PKU patients are identified soon after birth, and are primarily treated with a Phe-restricted diet. FIXXs HMI-102 seeks to modify the underlying genetic cause of PKU, effectively curing the disease and allowing patients to eat normally and not experience the cognitive and metabolic issues from higher than normal Phe.

FIXXs technology, which does not use the CRISPR approach to gene therapy, has already been subject to scrutiny, with David Russell, a researcher at the University of Washington, saying, Whats surprising is this company raised so much money on something thought to be untrue in the scientific community, in a piece in MIT Technology Review.

We believe that recent revelations about the efficacy of HMI-102 support this skepticism and show that the drug is not efficacious, kicking out the one leg holding up FIXXs business, and that FIXX should trade to cash value, or $5.80 per share, down 56% from the April 27th closing price.

The pheNIX trial

The pheNIX trial for HMI-102 launched in June 2019, and its primary efficacy endpoint is two plasma Phe measurements below 360 umol/L (or 6 mg/dL) between 16 and 24 weeks after dosing. Following evaluation of the first two patients in a cohort, a decision can be made to either escalate to the next dose level, add a third patient or expand the cohort at the selected dose level.

Now before we review whats new, its helpful to note that a mouse study presented at the 21st Annual Meeting of the American Society of Gene & Cell Therapy titled Sustained Correction of Phenylketonuria by a Single Dose of AAVHSC Packaging a Human Phenylalanine Hydroxylase Transgene showed that HMI-102 showed an effect to mouse Phe levels just one week after dosing in fact, mouse Phe levels remained relatively flat after that first drop.

This would imply that the therapy shows its effect soon after dosing and the longer timelines contemplated in the study endpoints are to indicate that the effect is long lasting. Sure enough, we see a similar dynamic in the pheNIX trial data released by FIXX in December 2019. There were 3 patients examined here from the 10-k: (n=2 patients in the low-dose Cohort 1 and n=1 patient in the mid-dose Cohort 2) as of the data cutoff of December 2, 2019. A fourth patient was dosed in Cohort 2 subsequent to the data cutoff date and was therefore not included in the analysis.

In this release, we see the two patients in the low-dose cohort experienced no improvement in fasting Phe after dosing or even 12 weeks later. The cohort 2 patient, getting a mid-dose, showed an improvement in Phe level immediately after dosing, but their Phe level did not fall below the 360 umol/L threshold defined as the primary endpoint (it stayed around 500) calling into question the efficacy of the treatment, which Med Genie mentions in their piece (from the 10-k):

Damning revelations

On March 5, 2020, a woman we will call Miss A started a Facebook group (since made private or taken down on April 15, 2020) to discuss her experience getting gene therapy for PKU:

On March 9, 2020, Miss A, who lives in Normal, IL tells us shes going to Chicago, which happens to be one of the pheNIX trial sites:

The next day, Miss A provides us with enough to data to know that she is Patient 5, part of the high dose cohort 3 in FIXXs HMI-102 trial (cohort 3, mentioned by Miss A, would be the next dose up from the cohort 2, the mid-dose cohort):

Dr. Burton appears to be Dr. Barbara K. Burton, a physician focused on PKU at the Ann & Robert H. Lurie Childrens Hospital of Chicago, a trial site mentioned in the pheNIX trial description on clinicaltrials.gov:

This, taken together with the fact that Biomarins own PKU trial was in too early a stage to enroll a Cohort 3 patient in March 2020, its probably safe to say that Miss A is taking part in FIXXs pheNIX trial.

On March 11, Miss A receives her infusion:

Miss A then shares a series of updates on how she is feeling and the progress of her weekly visits post-infusion. Six days post infusion, she says she hasnt gotten any test results back, but that it may take 2 or 3 weeks to get a Phe level:

27 days post-infusion, Miss A tells a FB commenter that she wont get Phe levels till six weeks post-infusion:

And then 35 days post-infusion, on April 15, 2020, a bombshell Miss A gets her Phe levels, and at 25 mg/dL or 1497uMol/L, they are well above the 360 uMol/L endpoint threshold after 5 weeks (and after the drug typically takes effect), suggesting that HMI-102 is not efficacious even for a high dose patient:

Just a few hours after her post, Miss As entire group is either taken down or made private, perhaps at the demands of FIXX itself.

Management's disclosure problem

We believe that management was aware of this post and tried to manage the perception of it by talking to the sell side and to select investors. In fact, Oppenheimers equity sales desk was sharing the below email conversation between their analyst Matt Biegler and FIXXs Theresa McNeely to explain the price action on April 15th:

Who was Theresa planning to speak to? We know that Bairds Madhu Kumar got a call:

But when we asked Theresa ourselves, she was much less forthcoming:

It appears to us that FIXX chose to inform the sell side and potential larger investors, who appear to be selling the stock (it has dramatically underperformed biotech broadly), but not the average investor. This is a significant red flag that investors should be aware of.

Why all this matters

Because the mouse study and the Cohort 2 data showed an effect to Phe levels one week after dosing rather than a gradual reduction, we can conclude that Miss As level, at 1497 uMol/L, is probably not going to get better, unfortunately. Further, her levels several weeks into the trial are still much higher than the threshold level specified in the primary endpoint of 360 uMol/L. This means that the therapy is showing zero efficacy even for a high dose patient. This data point is damning given the size of the trial and importance of the high dose patient in light of the lack of efficacy in the low and mid-dose cohorts.

Now the sell side may parrot management and say that there is no way to know whether Miss A is who she says she is, but the evidence is certainly strong supporting her case. They may say that there was no way for her to know her Phe level, but her posts show that she expected to receive them. They may also say that the drug is safe, and that liver enzyme elevation should be expected in a therapy like HMI-102, but thats all beside the point. The point is that the Phe level Miss A received 5 weeks after infusion show that HMI-102 is not efficacious.

These results support the skepticism around FIXXs use of the AAVHSC vector in liver directed gene therapy, which, based on the results thus far, and in particular Miss As results, appear to show zero efficacy and thus makes it inferior to Biomarins AAV5 vector.

Furthermore, if management thought this information was material enough to talk to the sell side analysts covering the stock, why not put it in an 8-k or even a press release for the benefit of their entire shareholder base? Given the materiality of the information, wouldnt all shareholders have benefited from the same level of disclosure rather than be kept in the dark? This is behavior consistent with that of MDXG and ALLK, both companies with executives formerly from reputed companies who have seen their stock prices demolished.

For FIXX, we believe that this is a huge problem the HMI-102 pheNIX trial is the ONLY program in their portfolio that is in the Phase 1/2 stage, and thus the primary path to viability for the company. With this piece of data showing that HMI-102 is not efficacious, we believe that the program is likely worthless and unlikely to proceed to commercialization. While FIXX may try to apply HMI-102 to other indications, we believe that doing so would essentially restart the trial and approval clock without making up for the lost time to market from a failed PKU trial.

Furthermore, social media matters. While FIXX management may be dismissive of people posting on Facebook, these posts have value. In the case of Allakos (ALLK), Seligman Research put together a barn burner of a report which included numerous Facebook posts questioning the efficacy, safety, and trial design of ALLKs drug candidate. Since that report, ALLK stock is down approximately 51%, and ALLK actually has several later stage trials.

In the case of FIXX, we believe that HMI-102 in PKU is the ONLY path to viability given the lack of efficacy of HMI-102 at high dose, we believe that the HMI-102 program is dead, and that the stock should trade to its cash value per share, or $5.80 per share, down 56% from the April 27th close price.

Disclosure: I am/we are short FIXX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: This piece is our opinion and not an offer to buy/sell any securities. We're biased, you're biased, so do your own work and make your own decisions.

Link:
Can't FIXX This - We Believe Homology's HMI-102 Is In Trouble - Seeking Alpha

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Global Hemophilia Gene Therapy Market 2020 Top Companies, Industry Demand, Business Review and Regional Analysis by 2025 Cole Reports - Cole of Duty

The Gene Therapy Market report market intelligence study intended to offer complete understanding of global market scenario with the Impact of COVID-19 (Corona Virus). It attempts to analyze the major components of the Market which have greater influence on it. This includes various elements of significant nature including market overview, segmentation, competition landscape, Market chain analysis, key players stratergyand more. Also, the report provides a 360-degree overview of global market on the basis of various analysis techniques including SWOT and Porters Five Forces. Approximations associated with the market values over the forecast period are based on empirical research and data collected through both primary and secondary sources. This might help readers to understand the strengths, opportunities, challenges and perceived threats of the market.

Based on Classification, each type is studied as Sales, Market Share (%), Revenue (Million USD), Price, Gross Margin and more similar information. The report can help to realize the market and strategize for business expansion accordingly. In the strategy analysis, it gives insights from marketing channel and market positioning to potential growth strategies, providing in-depth analysis for new entrants or exists competitors in the Gene Therapy industry.

The Gene Therapy Market report wraps:

There are 13 Chapters to thoroughly display the Gene Therapy market. This report included the analysis of market overview, market characteristics, industry chain, competition landscape, historical and future data by types, applications and regions.

In the end, The objective of the market research report is the current status of the market and in accordance classifies it into a few objects. The report takes into consideration the first market players in every area from over the globe.

Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

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Gene Therapy Market to witness impressive Growth in Production-Consumption Ratio through 2026 - Latest Herald