StemCell Therapy

Puma Biotechnology, Inc. (NASDAQ:PBYI) went up by 8.00% from its latest closing price compared to the recent 1-year high of $15.00. The companys stock price has collected 6.54% of gains in the last five trading sessions. Press Release reported on 09/08/20 that Puma Biotechnology to Present at the H.C. Wainwright 22(nd) Annual Global Investment Conference

Plus, the 36-month beta value for PBYI is at 1.17. Opinions of the stock are interesting as 1 analysts out of 11 who provided ratings for Puma Biotechnology, Inc. declared the stock was a buy, while 0 rated the stock as overweight, 6 rated it as hold, and 3 as sell.

The average price from analysts is $11.67, which is $1.41 above the current price. PBYI currently public float of 34.31M and currently shorts hold a 21.70% ratio of that float. Today, the average trading volume of PBYI was 894.18K shares.

PBYI stocks went up by 6.54% for the week, with a monthly jump of 3.53% and a quarterly performance of 7.43%, while its annual performance rate touched -9.20%. The volatility ratio for the week stands at 5.27% while the volatility levels for the past 30 days are set at 5.67% for Puma Biotechnology, Inc.. The simple moving average for the period of the last 20 days is 1.00% for PBYI stocks with a simple moving average of 3.19% for the last 200 days.

Many brokerage firms have already submitted their reports for PBYI stocks, with BofA/Merrill repeating the rating for PBYI by listing it as a Underperform. The predicted price for PBYI in the upcoming period, according to BofA/Merrill is $9 based on the research report published on June 25th of the current year 2020.

Goldman, on the other hand, stated in their research note that they expect to see PBYI reach a price target of $8, previously predicting the value of $24. The rating they have provided for PBYI stocks is Sell according to the report published on October 8th, 2019.

Citigroup gave a rating of Neutral to PBYI, setting the target price at $24 in the report published on May 10th of the previous year.

After a stumble in the market that brought PBYI to its low price for the period of the last 52 weeks, the company was unable to rebound, for now settling with -31.60% of loss for the given period.

Volatility was left at 5.67%, however, over the last 30 days, the volatility rate increased by 5.27%, as shares surge +4.37% for the moving average over the last 20 days. Over the last 50 days, in opposition, the stock is trading -2.56% lower at present.

During the last 5 trading sessions, PBYI rose by +6.54%, which changed the moving average for the period of 200-days by +9.73% in comparison to the 20-day moving average, which settled at $10.18. In addition, Puma Biotechnology, Inc. saw 17.26% in overturn over a single year, with a tendency to cut further gains.

Reports are indicating that there were more than several insider trading activities at PBYI starting from BRYCE RICHARD PAUL, who sold 90 shares at the price of $9.77 back on Sep 02. After this action, BRYCE RICHARD PAUL now owns 76,907 shares of Puma Biotechnology, Inc., valued at $879 using the latest closing price.

AUERBACH ALAN H, the PRESIDENT AND CEO of Puma Biotechnology, Inc., sold 347 shares at $9.78 during a trade that took place back on Sep 02, which means that AUERBACH ALAN H is holding 4,303,569 shares at $3,393 based on the most recent closing price.

Current profitability levels for the company are sitting at:

The net margin for Puma Biotechnology, Inc. stands at -27.77. The total capital return value is set at -24.14, while invested capital returns managed to touch -47.06. Equity return is now at value -246.90, with -17.40 for asset returns.

Based on Puma Biotechnology, Inc. (PBYI), the companys capital structure generated 688.48 points at debt to equity in total, while total debt to capital is 87.32. Total debt to assets is 51.18, with long-term debt to equity ratio resting at -2.60. Finally, the long-term debt to capital ratio is 673.45.

When we switch over and look at the enterprise to sales, we see a ratio of 1.24, with the companys debt to enterprise value settled at 0.35. The receivables turnover for the company is 10.71 and the total asset turnover is 1.10. The liquidity ratio also appears to be rather interesting for investors as it stands at 1.83.

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Is a Correction Looming Ahead for Puma Biotechnology, Inc. (PBYI) - The News Heater

The presented market report on the global Opthalmic Surgical Systems market published by Fact.MR is a comprehensive analysis of the leading parameters that are likely to determine the growth of the Opthalmic Surgical Systems market in the forthcoming decade. Further, the study dives in deep to investigate the micro and macro-economic factors that are projected to influence the global scenario of the Opthalmic Surgical Systems market during the forecast period (2019-2029).

The market study reveals that the Opthalmic Surgical Systems market is expected to grow at a CAGR of ~XX% and reach a value of ~USXX by the end of 2029. The report examines the current trends, growth opportunities, restraints, and market drivers that are projected to influence the overall dynamics of the Opthalmic Surgical Systems market in the assessment period. The market study predicts the course of the global Opthalmic Surgical Systems market post the COVID-19 pandemic and offers resourceful insights to market players pertaining to their business continuity strategies and more.

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Opthalmic Surgical Systems Market Reporting and Evaluation of Recent Industry Developments 2018 to 2028 - Scientect

The new report on the global Opthalmic Drugs growth gives estimations of the size of the international growth and the overall size and share of key regional segments during the historical assessment period of 2014 2018. The research report gives projections of different shares and opportunities, both in terms of projected value (US$Mn/Bn) as well as volume (n units), of different fragments in the Opthalmic Drugs Growth during the estimated timeframe of 2019 2029. The business knowledge study offers users with a granular analysis of key development elements, promising business avenues, and the overall dynamics of the vendor landscape of the global Opthalmic Drugs growth.

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The recent report on the global Opthalmic Drugs growth published by the Fact.MR includes the impact of COVID-19 on the Opthalmic Drugs growth. Severe economic crisis is being faced by each and every country in the world. This has affected each and every growth in the world and it will take a good amount of time to recover. The Opthalmic Drugs growth study includes the current growth scenario on the global platform and also forecasts growth development during the forecast period. We leverage space-age industrial and digitalization tools to provide avant-garde actionable insights to our clients regarding the Opthalmic Drugs growth. For enhancing readers experience, the report starts with a basic overview of the Opthalmic Drugs and its classification.

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Opthalmic Drugs Growth to Witness Positive Growth owing to Outbreak of COVID-19, Projects Fact.MR - The Cloud Tribune

Written by Johnson T A | Bengaluru | Updated: September 14, 2020 8:12:29 amInvestigations have revealed that Masood, then 27, who left behind his parents, wife and two young children, was closely associated with the IS in Syria and Iraq and was a key contact in Syria for Bengaluru youths trying to join the terror outfit. (Representational)

A business management graduate belonging to a wealthy Bengaluru family who has been missing for around seven years and was suspected to have joined the Islamic State is now known to have been killed in Syria.

Sources said the death of Faiz Masood had been confirmed by a doctor arrested in Bengaluru recently by the NIA in connection with an Islamic State Khorasan Province case. Abdur Rahman, an opthalmologist, was among the Bengaluru youths who travelled to Syria in 2013-14 to join the IS.

Investigations have revealed that Masood, then 27, who left behind his parents, wife and two young children, was closely associated with the IS in Syria and Iraq and was a key contact in Syria for Bengaluru youths trying to join the terror outfit.

Column| UN report flagging IS threat underlines why India cannot lower its guard

Questioning by the NIA, other central and state agencies of Rahman and an alleged associate a fellow doctor, also from Bengaluru has reportedly revealed that the two of them had met Masood at the Syrian border town of Atme, when they crossed over from a Turkey refugee camp in end 2013.

Rahman and his associate, both 22 and medical students at the time, said Masood died in an attack on a camp that left one of them with minor injuries. This prompted them to give up plans of joining the IS and return within days of making it to Syria, seeking financial help of their parents, officials said.

Investigators claimed Rahman and his associate, who went to become doctors after returning to India, admitted that Masoods death, the injury to one of them, as well as the serious infighting they saw among IS cadre had disillusioned them.

An Expert Explains| Why it is necessary to watch the emergence of ISIS in the region

Another alleged associate of Rahman, an aeronautical engineer, has reportedly also told investigators that Masood had facilitated his travel to Syria to join the IS. He left before the two medical students and stayed longer before suffering an injury to his arm and returning.

Masood had left for Qatar in September 2013 and disappeared soon after. His disappearance was not reported to the police by his family. Security agencies stumbled upon his name in 2014-15, as being one of those from India possibly killed in fighting in Syria, when they began looking closely at identities of IS recruits from India.

Investigations being carried out by multiple agencies have revealed that Masood was part of a group of wealthy Muslim youths from east Bengaluru who met often in 2012-13 and discussed religion. Several from the group later left to join the IS when it was established around mid-2013.

An online blog Masood maintained between 2010 and 2011 shows he was deeply affected by the plight of poor Muslims in Bengaluru. In blog posts in 2010 he sought funds for an orphanage, extolled Muslims to donate blood and narrated stories of a child and a young man in dire need of funds for medical care to save their lives.

In March 2010, he wrote, The Muslim community might be a minority in this country but when it comes to the population of the slums we are the majority. Many efforts are being made to improve the condition of the people. But unless the realization and awareness of their condition doesnt spread among the family (Muslims) things will not change.

Investigations have revealed that the Syria trip in 2013-14 of Rahman and others was also facilitated by a dentist and a computer applications graduate who earlier lived in Bengaluru and are currently working in Saudi Arabia.

Rahman was arrested on August 17 by the NIA on charges of conspiring with Jahanazaib Wani and his wife, held from New Delhi in March, to carry out activities of the Islamic State of Khorasan Province unit in India. In its statement, the NIA said Rahman was in the process of developing a medical application for helping injured ISIS cadres in conflict-zones and a weaponry-related application for the benefit of ISIS fighters.

In the first chargesheet filed in the case last week, the NIA alleged that Wani, who belongs to Kashmir, and others were also provoking some gullible youth to participate in anti-CAA protests actively. In case these protests failed to provoke the Muslims, they were planning for arsoning of Government buildings & public property so that riots could happen and they could exploit the sentiments of Muslims, the NIA said.

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Missing Bengaluru youth helped many join IS, killed in Syria, says probe - The Indian Express

DUBLIN--(BUSINESS WIRE)--The "Global Regenerative Medicine Market Analysis & Forecast to 2025; Stem Cells, Tissue Engineering, BioBanking & CAR-T Industries" report has been added to ResearchAndMarkets.com's offering.

This report provides a comprehensive overview of the size of the regenerative medicine market, segmentation of the market (stem cells, tissue engineering and CAR-T therapy), key players and the vast potential of therapies that are in clinical trials. The analysis indicates that the global regenerative medicine market was worth $35 billion in 2019 and will grow to over $124 billion by 2025, with a CAGR of 23.3% between this time frame. This report describes the evolution of such a huge market in 15 chapters supported by over 350 tables and figures in 700 pages.

Key Questions Answered

Key Topics Covered:

1.0 Report Synopsis

2.0 Introduction

3.0 Stem Cells and Clinical Trials

4.0 Stem Cells, Disruptive Technology, Drug Discovery & Toxicity Testing

5.0 Stem Cell Biomarkers

6.0 Manufacturing Stem Cell Products

7.0 Investment & Funding

8.0 Regenerative Medicine Market Analysis & Forecast to 2025

9.0 Stem Cell Market Analysis & Forecast to 2025

10.0 Tissue Engineering Tissue Engineering Market Analysis and Forecast to 2025

11.0 Biobanking Market Analysis

12.0 Global Access & Challenges of the Regenerative Medicine Market

13.0 Cell and CAR T Therapy

14.0 Company Profiles

15.0 SWOT Industry Analysis

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/dfpyeg

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Global Regenerative Medicine Market Analysis & Forecast to 2025 - ResearchAndMarkets.com - Business Wire

VALENCIA, Calif. & MELBOURNE, Australia--(BUSINESS WIRE)--AVITA Therapeutics, Inc. (NASDAQ: RCEL, ASX:AVH), a regenerative medicine company that is developing and commercializing a technology platform that enables point-of-care autologous skin restoration for multiple unmet needs, announced today the initiation of the pivotal study assessing the use of the RECELL System to treat stable vitiligo with the enrollment of the first patient at Miami Dermatology and Laser Institute in Miami, FL. The study will evaluate the safety and effectiveness of AVITA Therapeutics RECELL System to repigment skin in patients who have vitiligo that has been stable for at least one year.

The initiation of the vitiligo clinical study is a milestone in advancing AVITA Therapeutics pipeline to leverage the utility and full potential of our innovative RECELL technology platform to address unmet medical needs in dermatological applications, said Dr. Mike Perry, AVITA Therapeutic Chief Executive Officer. Globally, there have been several published case series and pilot randomized clinical trials reporting positive results with the use of RECELL for treating patients with stable vitiligo and repigmenting depigmented skin lesions. We are pleased to initiate this pivotal study as a next step toward offering a treatment option for the millions of Americans who live with vitiligo.

Vitiligo is an autoimmune disease that attacks the epidermis layer of skin resulting in loss of color or pigmentation. This serious skin condition affects up to 2% of the population worldwide, including an estimated 6.5 million Americans.i Vitiligo has a comparable market size & psychosocial impact to other major dermatology diseases including psoriasis (thick, scaly skin) and atopic dermatitis (red, cracked skin). IV-VI Like these diseases, patients with vitiligo may suffer from poor body image along with low self-esteem, leading to an impaired quality of life.ii There is currently no cure for vitiligo, nor a universally accepted method for limiting the spread of the disease. Although many treatments are being used for the management of vitiligo, they are often temporary with a high rate of recurrence.iii

While often considered a cosmetic issue, vitiligo can greatly impact the quality of life of those living with the disease, and treatment options are limited, said Jill Waibel, MD, owner and Medical Director of Miami Dermatology and Laser Institute. We look forward to assessing the safety and efficacy of the RECELL System in restoring skin color in stable vitiligo lesions and potentially offering those who live with vitiligo hope with a new, easy in-office treatment.

The multi-center pivotal study will assess the safety and effectiveness of the RECELL System in treatment of depigmented vitiligo lesions at 24 weeks in patients whose vitiligo is stable, meaning they have not had new vitiligo lesions or lesions that have expanded for at least one year. Clinicians will obtain a small amount of the study participants own healthy skin at the point-of-care to prepare a suspension of Spray-On Skin Cells using the RECELL System that will then be applied to the vitiligo lesion. Additional long-term safety and effectiveness data, including sustained repigmentation of the vitiligo lesion, will be collected over the course of the study.

In parallel with the clinical study, AVITA Therapeutics is partnering with the University of Massachusetts Medical School on a complementary and more scientifically-oriented vitiligo feasibility study.

Of note: Use of the RECELL System in patients undergoing reconstruction of skin defects not associated with a burn injury is limited by the Federal law to investigational use.

Authorized for release by the Chief Executive Officer of AVITA Therapeutics, Inc.

ABOUT AVITA THERAPEUTICS, INC.

AVITA Therapeutics is a regenerative medicine company with a technology platform positioned to address unmet medical needs in burns, chronic wounds, and aesthetics indications. AVITA Therapeutics patented and proprietary collection and application technology provides innovative treatment solutions derived from the regenerative properties of a patients own skin. The medical devices work by preparing a RES REGENERATIVE EPIDERMAL SUSPENSION, an autologous suspension comprised of the patients skin cells necessary to regenerate natural healthy epidermis. This autologous suspension is then sprayed onto the areas of the patient requiring treatment.

AVITA Therapeutics first U.S. product, the RECELL System, was approved by the U.S. Food and Drug Administration (FDA) in September 2018. The RECELL System is indicated for use in the treatment of acute thermal burns in patients 18 years and older. The RECELL System is used to prepare Spray-On Skin Cells using a small amount of a patients own skin, providing a new way to treat severe burns, while significantly reducing the amount of donor skin required. The RECELL System is designed to be used at the point of care alone or in combination with autografts depending on the depth of the burn injury. Compelling data from randomized, controlled clinical trials conducted at major U.S. burn centers and real-world use in more than 8,000 patients globally, reinforce that the RECELL System is a significant advancement over the current standard of care for burn patients and offers benefits in clinical outcomes and cost savings. Healthcare professionals should read the INSTRUCTIONS FOR USE - RECELL Autologous Cell Harvesting Device (https://recellsystem.com/) for a full description of indications for use and important safety information including contraindications, warnings and precautions.

In international markets, our products are marketed under the RECELL System brand to promote skin healing in a wide range of applications including burns, chronic wounds and aesthetics. The RECELL System is TGA-registered in Australia and received CE-mark approval in Europe.

To learn more, visit http://www.avitamedical.com.

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

This letter includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as anticipate, expect, intend, could, may, will, believe, estimate, look forward, forecast, goal, target, project, continue, outlook, guidance, future, other words of similar meaning and the use of future dates. Forward-looking statements in this letter include, but are not limited to, statements concerning, among other things, our ongoing clinical trials and product development activities, regulatory approval of our products, the potential for future growth in our business, and our ability to achieve our key strategic, operational and financial goal. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Each forward- looking statement contained in this letter is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others, the timing of regulatory approvals of our products; physician acceptance, endorsement, and use of our products; failure to achieve the anticipated benefits from approval of our products; the effect of regulatory actions; product liability claims; risks associated with international operations and expansion; and other business effects, including the effects of industry, economic or political conditions outside of the companys control. Investors should not place considerable reliance on the forward-looking statements contained in this letter. Investors are encouraged to read our publicly available filings for a discussion of these and other risks and uncertainties. The forward-looking statements in this letter speak only as of the date of this release, and we undertake no obligation to update or revise any of these statements.

PR20200914

i Advances in Vitiligo: An Update on Medical and Surgical Treatments. A. Dillon, et al. J Clin Aesth Derm. 2017ii Willingness-to-pay and quality of life in patients with vitiligo. Radtke, et al. BJD. 2009iii Vitiligo Research Foundation Treatment Guidelines. https://vrfoundation.org/treatment_guidelines Accessed 4/18/20 V The burden of vitiligo: Patient characteristics associated with quality of life. Homan, et al. JAAD. 2009VI Comparison of the Psychological Impacts of Asymptomatic and Symptomatic Cutaneous Diseases: Vitiligo and Atopic Dermtitis. Noh, et al. Annals of Derm. 2013

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First Patient Enrolled in AVITA Therapeutic's Pivotal Study Evaluating the RECELL System for Repigmentation of Stable Vitiligo - Business Wire

ARLINGTON, Va. & REYKJAVIK, Iceland--(BUSINESS WIRE)--Kerecis, the company pioneering the use of fish skin and fatty acids for tissue regeneration and protection, is donating its Kerecis Omega3 Burn product for burn victims of the fires in California, Oregon and Washington. Qualified medical personnel wanting to take advantage of this offer should contact wildfires@kerecis.com.

These horrific fires are having a devastating effect on human lives and habitat, said G. Fertram Sigurjonsson, founder and CEO of Kerecis. We want to help those who have been burned to heal as quickly and easily as possible. We encourage medical professionals to contact us to get a supply of Kerecis Omega3 Burn for their patients.

About Kerecis Omega3 Burn

Kerecis Omega3 Burn is intact fish skin that, when grafted onto damaged human tissue, recruits the bodys own cells and ultimately is converted into living tissue. Because no disease-transfer risk exists between cold-water fish and humans, the Kerecis fish skin is only gently processed and retains its similarity to human skin, making it an ideal skin substitute. The fish skin contains Omega3 fatty acids and multiple proteins that help the product to become incorporated into the body quickly, while providing a much-needed bacterial barrier to protect the wound bed. Clinical studies have found that the Kerecis products heal wounds faster than competitive products, so patients can be ready for additional tissue building and/or move directly to split-thickness skin grafting in record time. Kerecis Omega3 Burn is available for the treatment of humans as well as for animals.

About Kerecis

Kerecis is pioneering the use of fish skin and fatty acids in the globally expanding cellular- therapy and regenerative-medicine market. The Kerecis fatty-acid-rich intact fish skin protects the bodys tissues and enables the body to regenerate tissues. The Kerecis sprayable fatty-acid topical and oral formulations protect the body from bacterial and viral infections.

The Kerecis products, which are based on fish skin and fatty acids, are currently being used to regenerate tissue in diabetic and trauma wounds (including burns), and for infection control. Kerecis is also developing products for areas such as oral surgery, plastic surgery and neurological applications.

The companys mission is to extend human life by supporting the bodys own ability to regenerate, and its vision is to become the world leader in tissue regeneration by sustainably harnessing natures own remedies. For more information, visit http://www.kerecis.com

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Kerecis to Donate its FDA-Approved Fish Skin Treatment for Burn Victims of West Coast Fires - Business Wire

TOKYO--(BUSINESS WIRE)--SanBio Co., Ltd. (headquarters: Chuo-ku, Tokyo, Representative Director and President: Keita Mori, hereafter SanBio) hereby announces that it has obtained new analytical results from the Phase 2b clinical trial (the trial) of SB623 for the treatment of chronic motor deficit resulting from ischemic stroke the SanBio Group (SanBio Co., Ltd. and its subsidiary SanBio, Inc.) conducted in the US. It also announces that based on the newly obtained results, it has updated its development plans, including in regard to late-stage clinical trials for the ischemic stroke and hemorrhagic stroke programs of SB623 in Japan.

The trial evaluated efficacy and safety of SB623 in 163 patients suffering from chronic motor dysfunction from ischemic stroke. On January 29, 2019, SanBio announced that the trial did not meet its primary endpoint, as it failed to demonstrate statistical significance in the difference in the proportion of patients whose Fugl-Meyer Motor Scale (FMMS) score improved by 10 or more points from the baseline (primary endpoint) between the treatment group that received SB623 and the control group. Since then, the SanBio Group had continued to work on additional analysis of the trial data, and results of the additional analysis are as follows.

In conducting the additional analysis, from the perspective of minimal clinically important difference (MCID, or the minimal change in scores or other metrics that could be interpreted to mean the change in a patient is clinically meaningful) and based on the results of the Phase 2 clinical trial of SB623 for the treatment of chronic motor deficit from traumatic brain injury (TBI; STEMTRA trial), the company reevaluated trial data using composite FMMS. Of the total 163 patients enrolled in the trial, the company specifically looked at 77 patients who had infarct areas smaller than a certain size (47% of all patients enrolled in this trial). The SanBio Group evaluated the proportion of patients that met one or more of the following FMMS score improvement criteria 24 weeks after treatment: 6-point improvement on FMMS score for upper extremity, 4-point improvement on FMMS score for lower extremity, and 9-point improvement on FMMS total score (all from the baseline). Of the 51 patients in the treatment group that received SB623, improvement was seen in 49%, versus in 19% of 26 patients in the control group that received sham surgery, the difference between the two groups being statistically significant (p-value of 0.02). SanBio Group thinks that even compared to the primary endpointthe proportion of patients whose FMMS score improved by 10 or more points over the baseline six months after treatmentthe endpoint using composite FMMS can adequately explain clinical significance of the treatment efficacy. Details of the additional analysis results will be announced at the financial results briefing for institutional investors and the media held on September 15, 2020. The briefing video will be made available to the public on our website on the 16th of September or thereafter.

Based on the above results, the SanBio Group has begun preparations for the next late-stage clinical trials in the ischemic stroke and hemorrhagic stroke programs of SB623. 2021. Specific designs of the clinical trials and the contents of development for those two programs will be announced promptly upon being finalized. To maximize the value of SB623 at an early stage by selecting areas to focus the Groups management resources on, the SanBio Group plans to prioritize the development of the ischemic stroke and hemorrhagic stroke programs in Japan at the same time as it prepares to file for approval of SB623 for the treatment of chronic motor deficit resulting from TBI in Japan by the end of the current fiscal year (ending January 2021). The Group, however, postponed the global Phase 3 clinical trial for the TBI program of SB623 it had planned to commence this fiscal year to the next or subsequent fiscal years.

Many patients suffering from the chronic effects of ischemic stroke are said to be regularly taking drugs to prevent recurrence. However, because there is no drug that can fundamentally cure motor dysfunction, there is high unmet need for therapeutic drugs to restore motor functions for patients in the chronic phase of stroke. The SanBio Group aims to contribute to improving the lives of these patients, as well as of their family members, suffering from motor impairment and difficulties it causes in carrying out their daily lives through SB623.

About SB623

SB623 is an allogeneic mesenchymal stem cell produced by modifying and culturing bone marrow derived from healthy donors. Implantation of SB623 cells into nerve tissues is expected to promote regeneration of damaged nerve cells. Because SB623 is made from allogeneic cells, large-scale production is possible and there is no need for complex cell processing required for treatments using autologous cells, e.g., cell preparation for each patient at medical institutions. Hence, pharmaceutical products made from allogeneic cells, such as SB623, can be provided to many patients in uniform quality.

About SanBio Co., Ltd. and SanBio, Inc.

SanBio Group is engaged in the regenerative cell medicine business, spanning research, development, manufacture, and sales of regenerative cell medicines. The Companys propriety regenerative cell medicine product, SB623, is currently being investigated for the treatment of several conditions including chronic neurological motor deficit resulting from traumatic brain injury and ischemic stroke. The Company is headquartered in Tokyo, Japan and Mountain View, California, and additional information about SanBio Group is available at https://sanbio.com.

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Additional Analytical Results of the US-Based Phase 2b Clinical Trial of Regenerative Cell Medicine SB623 for the Treatment of Chronic Motor Deficit...

"On behalf of our entire board of directors, we wholeheartedly welcome Terry to his new role as President and CEO of Vizgen," said Dr. David R. Walt, Cofounder of Vizgen; Hansjrg Wyss Professor of Biologically Inspired Engineering, Harvard Medical School; Professor of Pathology, Brigham and Women's Hospital; Core Faculty, Wyss Institute for Bioinspired Engineering, Harvard University; HHMI Professor. "Terry's technical savvy combined with his demonstrated business acumen in building out product and service offerings on a global scale will benefit Vizgen as the Company enters the next phase of commercial development."

Prior to joiningVizgen, Terry was President of Akoya Biosciences, a post he assumed after an acquisition from PerkinElmer where he served as General Manager, Quantitative Pathology Solutions. Previously, he held several executive positions at global biopharma and diagnostic companies including Roche, Hologic, and Bristol-Myers Squibb, and has extensive experience in launching innovative technologies in new markets. He holds an MBA from the University of Chicago Booth School of Business, an MS in Microbiology from Virginia Tech, and dual BS degrees in Molecular Genetics and Psychology from The Ohio State University.

"I'm excited to take the helm at Vizgen to work together with an incredibly talented team of scientists and innovators to bring an unsurpassed spatial profiling technology to market," said Mr. Lo. "Gene expression with spatial context has now become the new research frontier in unlocking core biological questions, and Vizgen's technology is regarded as a premier solution to gain insight into the molecular underpinnings of health, the progression to disease, and the development of new therapies and vaccines."

Vizgen's MERFISHtechnology was developed in the laboratory of Dr. Xiaowei Zhuang, a Howard Hughes Medical Institute Investigator and David B. Arnold, Jr. Professor of Science at Harvard University. Dr. Zhuang and Dr. Jeffrey Moffitt, a former postdoctoral fellow in Dr. Zhuang's lab andnow an Assistant Professor at the Program in Cellular and Molecular Medicine at Boston Children's Hospital and the Department of Microbiology at Harvard Medical School, are also cofounders of Vizgen. MERFISH enables spatially resolved, single-cell genomic profiling at extremely high levels of throughput and accuracy. The novel technology is used as a tool for several Human Cell Atlasprojects and was named a "Technology to Watch" by Nature for mapping the transcriptome.

Vizgen launched in January 2020 with a $14M Series A Financing led by ARCH Venture Partners and Northpond Ventures. Last month the Company announced an early release program for its spatial genomics platformto provide scientific investigators an opportunity to gain access to the proprietary technology to accelerate their research. Vizgen's technology is already being employed by world-leading academic research institutions including the Broad Institute of MIT and Harvard and The Rockefeller University.

For more information email: [emailprotected]

About Vizgen Vizgen is developing the next generation of spatially resolved genomic profiling tools that enable researchers to gain new insight into the biological systems that underlie human health and disease. The company's patented MERFISH technology enables massively multiplexed, genome-scale nucleic acid imaging with high accuracy and unrivaled detection efficiency at subcellular resolution. MERFISH provides transformative insight into a wide range of tissue-scale basic research and translational medicine in oncology, immunology, neuroscience, infectious disease, developmental biology, and regenerative medicine. For more information, go towww.vizgen.com, connect on social media

@Twitter,@LinkedInandFacebook, and join the MERFISH Group at: https://bit.ly/merfishgroup.

SOURCE Vizgen

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Vizgen Taps Akoya Biosciences Executive Terry Lo As New President and CEO - PRNewswire

VANCOUVER, B.C., Sept. 14, 2020 /PRNewswire/ -- The Global Femtech Marketis expected to reach USD 60.01 Billion by 2027, according to a new report by Emergen Research. Demand for the femtech industry is motivated mainly by the growing burden of both chronic and infectious diseases among the world's female population. An increase in the number of health problems relating to women would stimulate competition for technologically innovative healthcare solutions. Growing women's emphasis on reproductive health and sexual empowerment in developing economies would further encourage development in the industry.

Increasing awareness among women of the detection and management of early illness as part of the patient care program would improve the market outlook. Various efforts by government and other agencies in developing countries to raise awareness of women's health would accelerate the development of the industry. Furthermore, an increasing tendency towards daily preventive care check-ups, as well as the advancement of user-friendly technology to track individual health problems, may prove beneficial to the developments in the women's health industry.

While more and more people today choose to be more transparent about their health concerns and treatment, in some of the lesser developed regions, women's health issues remain stigmatized. For these places, Femtech applications are likely to be favored because the scanning is less invasive and more secure. Increasing population growth is related to being one of the main factors behind the case.

Request free sample of this research report at: https://www.emergenresearch.com/request-sample/37

Key Highlights From The Report

Read more at: https://www.emergenresearch.com/industry-report/femtech-market

For the purpose of this report, Emergen Research has segmented into the Global Femtech Market on the basis of type, end-use, application, and region:

Type Outlook (Revenue: USD Billion; 2017-2027)

End Use Outlook (Revenue: USD Billion; 2017-2027)

Application Outlook (Revenue: USD Billion; 2017-2027)

Regional Outlook (Revenue, USD Billion; 2017-2027)

Find more research reports on healthcare and pharmaceuticals industry, by Emergen Research:

Regenerative Medicine MarketRegenerative Medicine Market By Product (Tools, Therapeutics), By Therapeutic Category (Musculoskeletal, Dermatology, Immunology & Inflammation, Cardiovascular), and By Applications (Wound Care, Musculoskeletal Disorders, Ocular Disorders), Forecasts to 2027

Next-Generation Sequencing MarketNext-Generation Sequencing Market by Technology (Whole Exome, Whole Genome, Others), By Workflow (Sequencing, Pre-Sequencing, Others), By Application (Consumer Genomics, HLA Typing, Others) and By End-Use (Academic, Clinical, Others), Forecasts to 2027

RFID in Healthcare MarketBy Product (Tags, Systems & Software) and By Application (Asset Tracking, Patient Tracking, Pharmaceutical Tracking, Blood Tracking, Others), Forecasts to 2027

Non-Invasive Prenatal Testing MarketBy Method, By End-Use, By Application, By Region, Forecasts to 2017-2027

Viral Vector and Plasmid Manufacturing MarketBy Vector Type (Retrovirus, Adenovirus, Others), By Workflow (Upstream, Downstream), By Disease (Cancer, Genetic Disorders, Others), By Application (Gene Therapy, Retailers) and By End-User, Forecasts to 2027

Interoperability Solutions in Healthcare MarketBy Level (Foundational, Structural, Semantic), By Product Type (Services, Solutions), and By Application (Diagnostics, Treatments, Others), Forecasts to 2027

About Emergen Research

At Emergen Research, we believe in advancing with technology. We are a growing market research and strategy consulting company with an exhaustive knowledge base of cutting-edge and potentially market-disrupting technologies that are predicted to become more prevalent in the coming decade.

With market-leading insights and an in-depth understanding of leading and niche technologies, our solutions address the most pertinent questions for your business needs. A major technological shift has been witnessed towards creating a 'Circular Economy,' fuelled by factors, such as the increased adoption of bio-based materials, along with other methods for achieving carbon neutrality. We are conversant in technologies, viz., Artificial Intelligence (AI), Augmented Reality (AR), Virtual Reality (VR), Robotic Process Automation (RPA), Smart Manufacturing, Internet of Things (IoT), Big Data Analytics, Machine learning, Nanotechnology, Edge Computing, Blockchain Technology, Cloud Computing, Vehicle Electrification, Advanced Maintenance Analytics, and Predictive Maintenance, among other prevalent and emergent technologies.

Contact Us:Eric LeeCorporate Sales SpecialistEmergen Research | Web: https://www.emergenresearch.comE-mail: [emailprotected]

Read full Press Release at :https://www.emergenresearch.com/press-release/global-femtech-market

SOURCE Emergen Research

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Femtech Market to Reach USD 60.01 Billion By 2027 | CAGR of 15.6%: Emergen Research - PRNewswire

Silence Therapeutics Appoints Mark Rothera as President and Chief Executive Officer

Experienced biotech executive to lead the next phase of growth

14 September 2020

LONDON, Silence Therapeutics plc, AIM:SLN and Nasdaq: SLN (Silence or the Company), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, today announces the appointment of Mark Rothera as President and Chief Executive Officer (CEO) and Board member, effective immediately. Iain Ross, who has been Executive Chairman since December 2019, has today assumed his previous position of Non-Executive Chairman.

Mr. Rothera brings more than 30 years of experience in the biopharmaceutical industry, with a strong record of commercial and operational leadership, including driving the successful build of multiple biotech companies, predominantly in the field of rare or specialty diseases. Prior to joining Silence, Mr. Rothera served as CEO of Orchard Therapeutics (Orchard), where he oversaw its transformation from a small U.K.-based, privately held company with two clinical-stage programmes into a leading gene therapy company with seven clinical-stage programmes and fully integrated capabilities. Under his leadership, Orchard completed an initial public offering of American Depositary Shares on the Nasdaq Global Market and during his tenure that company secured more than $600 million in financing and grew from a market capitalization of $250 million to more than $1.7 billion at its peak.

Prior to Orchard, Mr. Rothera served as Chief Commercial Officer of PTC Therapeutics (PTC), where he helped transition that company from a privately held R&D biotechnology company to a publicly traded, commercial-stage company with a global footprint, including the successful launch of two rare disease therapies. He also previously served as Global President of Aegerion Pharmaceuticals Inc. and Vice President and General Manager of commercial operations at Shire Human Genetic Therapies for Europe, Middle East and Africa. Mr. Rothera received an M.A. in Natural Sciences from Cambridge University and an M.B.A. from the European Institute for Business Administration (INSEAD).

Based out of Silences New York City office, Mr. Rothera will lead the continued global expansion of the Company. His appointment follows the completion of Silences Nasdaq listing on 8 September 2020 and aligns with the strategy of increasing the Companys presence in the United States.

Iain Ross, Chairman of Silence Therapeutics plc, said: "On behalf of the Silence Board and the entire Silence team, I welcome Mark to the Company. Following a thorough search, Marks appointment reflects his proven leadership skills and strong track record in growing successful biotechnology companies and building shareholder value. I believe he will now provide the leadership necessary to grow Silence into a leading international biotechnology company built upon our innovative siRNA technology platform, proprietary product pipeline and validating industry partnerships.

On a personal note, and on behalf of the Board, I would like to thank the management team and staff at Silence for their support, hard work and tremendous resilience during the current COVID-19 pandemic and over the past nine months whilst I have been Executive Chairman. The Company has made great strides during this period, and is now in a strong position, both operationally and financially, and ready for Mark to take the helm.

Mark Rothera, President and CEO of Silence Therapeutics plc, added: It is an honour to take the role of leading Silence at this time in the Companys history. I believe the Company is poised to capitalise on its important siRNA technology platform, pipeline and research capabilities built over 18 years, and position itself as a leader in the RNAi field. The Company has made great strides under Iains leadership and I look forward to working with the Board, the management team and Silence employees to build upon this momentum.

Director disclosures

The following information is being disclosed pursuantto Rule 17 and paragraph (g) of Schedule 2 of the AIM Rules for Companies.

Mark Rothera

Full name and age: Mark Andrew Rothera (aged 58)

Current Directorships or Partnerships:Genpharm

Previous Directorships or Partnerships in the last 5 years:Orchard Therapeutics plcPTC Therapeutics International LimitedAlliance for Regenerative Medicine

No further information in connection with his appointment is required to be disclosed under Schedule Two, paragraph (g) of the AIM Rules for Companies.

Enquiries:

About Silence TherapeuticsSilence Therapeutics is developing a new generation of medicines by harnessing the bodys natural mechanism of RNA interference, or RNAi, to inhibit the expression of specific target genes thought to play a role in the pathology of diseases with significant unmet medical need. Silences proprietary technology can be used to engineer short interfering ribonucleic acids (siRNAs) that bind specifically to and silence, through the RNAi pathway, almost any gene in the human genome to which siRNA can be delivered. Silences wholly owned product candidates include SLN360 designed to address the high and prevalent unmet medical need in reducing cardiovascular risk in people born with high levels of Lipoprotein(a) and SLN124 to address beta-thalassemia and myelodysplastic syndrome. Silence is also developing SLN500 in partnership with Mallinckrodt Pharmaceuticals to reduce the expression of the C3 protein for the treatment of complement pathway-mediated diseases. Silence maintains ongoing research and collaborations with AstraZeneca, Mallinckrodt Pharmaceuticals and Takeda. For more information, please visit: https://www.silence-therapeutics.com/

The person who arranged for the release of this announcement on behalf of the Company was Rob Quinn, Chief Financial Officer.

Forward-Looking StatementsCertain statements made in this announcement are forward-looking statements, including with respect to the Companys clinical and commercial prospects. These forward-looking statements are not historical facts but rather are based on the Company's current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as 'anticipates,' 'expects,' 'intends,' 'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company's control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.

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Silence Therapeutics Appoints Mark Rothera as President and Chief Executive Officer - GlobeNewswire

Daniel Dennett has inspired many people, but perhaps none more than his former student Jeff Stibel, A95, an entrepreneur and brain scientist. Now Stibel is ensuring that the influential philosophers legacy will continue at Tufts, with a generous gift to create a consortium at the university focused on cognitive and brain science.

Stibels gift will launch the Stibel Dennett Consortium for Brain and Cognitive Science, which will bring together important research and teaching in the field. The consortium will cross university departments and schools, including psychology, biology, philosophy, education, engineering, and medicine, and will serve students, faculty, alumni, and the wider community.

I was inspired to create a new consortium at Tufts that will serve as a center of gravity, to explore important and groundbreaking cognitive and brain science issues through teaching and research, said Stibel, who is the author of two books and a USA Today column on the workings of the mind.

With his fellow partners Stibel has also given to the university BrainGate, Inc., a company that holds intellectual property enabling technologies to read and translate brain signals through a computer interface. The brain's motor cortex sends out electrical pulses that can be recorded by this technology and decoded into motor commands. In the future, the companys technology could help people with spinal injuries or locked-in syndrome control devices, such as a robotic arm or an exoskeleton that would allow a paralyzed person to walk.

The donation of BrainGate, Inc., combined with Stibels support for faculty, promises to spur new research at the university. Stibels gift includes funds to endow two professorships in the School of Arts and Sciences. Gina Kuperberg has been appointed the inaugural Dennett Stibel Professor of Cognitive Science, and Stephanie Badde has been recruited to the faculty as the Stibel Family Assistant Professor of Brain and Cognitive Science.

Kuperberg and Badde plan to build upon the BrainGate technology, exploring a deeper understanding of how the brain processes language as well as how the brain gathers information from our senses and tells our body how to move. Other faculty at Tufts also plan to explore research opportunities related to the BrainGate intellectual property.

What Jeff Stibel has given us is priceless, said James Glaser, dean of the School of Arts and Sciences. The two professorships have enabled us to recognize the excellence of Gina Kuperberg, an important cognitive science faculty member, and to recruit a talented new colleague to the program. And the gift of BrainGate, Inc., will help solidify Tufts international reputation as a locus of excellence in cognitive science.

In just one of its potential applications, the Stibel gift may lead to a deeper understanding of the very nature of human memory, said Michael Levin, A92, the Vannevar Bush Professor of Biology and director of the Allen Discovery Center at Tufts . Levin studies regenerative biologythe process of replacing or "regenerating" human or animal cells, tissues, or organs to restore or establish normal function.

Advances in regenerative medicine depend on understanding electrical anatomical memory, which is like memory in the brain. BrainGates technology could offer key insights into how cells communicate to signal growth, adaptation to trauma, or even the storage of memories.

Using the technology to interpret the communication between cells, we could find out how memories are stored and encoded in tissue, and learn to decode them, Levin said. We also could learn how memories can survive remodeling of the tissue, how memories can be moved or copied, and how memories can belong to a unified self. Levin is currently collaborating with Dennett on research and a publication related to cellular memory and cognition

Tufts will launch the Stibel Dennett Consortium in the fall, through an online event for the Tufts community with other programming to follow.

Angela Nelson can be reached at angela.nelson@tufts.edu.

Stephanie Badde, Stibel Family Assistant Professor of Brain and Cognitive Science: We think our senses give us a good impression of the physical reality around us, but that's just not true. The information were getting is actually very spottyit's as if were wearing blurry glasses with a small hole in the middle. The brain performs a lot of work to take in this input and give us the impression we have. My research asks, How does the brain do this miracle?

With the BrainGate patents, we hope to find out more about how movements and sensory information are connected in the brain, and how this might be leveraged so that people can regain the sense of touch and sense of body posture where theyve lost them.

Gina Kuperberg, Dennett Stibel Professor of Cognitive Science: We take it for granted that as we talk to one another, we are literally transferring thoughts from one mind to another. Language is what enables humans to communicate, and ultimately, language is a code. My research program is aimed at understanding this code. We're trying to figure out not only where, when, and how the brain uses language to communicate, but also the nature of the neural code itself.

The whole idea of decoding brain activity is so important, not only medically for people in the future, but for understanding the nature of the human brain and thought. And were really on the cutting edge of being able to do that, particularly now with the BrainGate intellectual property.

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Getting Smarter About the Mind | Tufts Now - Tufts Now

One of the major challenges facing gene therapy a way to treat disease by replacing a patients defective genes with healthy ones is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia, and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the currently available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

[Read: These tech trends defined 2020 so far, according to 5 founders]

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses from which dangerous disease-causing genes have been removed as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene-editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts as a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up our genome. This means that researchers like me can leverage CRISPR technology to revolutionize gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for the immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

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CRISPR can precisely remove even single units of DNA. KEITH CHAMBERS/SCIENCE PHOTO LIBRARY/Getty Images

A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the number of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibodies against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually, the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology, provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

This article is republished from The Conversation by Samira Kiani, Associate Professor of Pathology, University of Pittsburghunder a Creative Commons license. Read the original article.

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How CRISPR is tackling the troubling immune response thats plagued gene therapy until now - TNW

BOSTON, Sept. 15, 2020 /PRNewswire/ --GreenLight Bioscienceshas received a $3.3 million grant from the Bill & Melinda Gates Foundation to develop new mRNA-based gene therapies for Sickle Cell Disease and other global health challenges.

The funding will support GreenLight's research and testing of affordable therapies using the company's novel messenger RNA (mRNA) approach to gene editing. mRNA technology is already being used to develop vaccine candidates for infectious diseases, including the COVID-19 pandemic.

While initial research will focus on a cure for Sickle Cell Disease, GreenLight plans to develop a versatile gene editing platform to address a variety of diseases affecting underserved patient populations, such as treating HIV in developing countries.

Sickle Cell Disease is a group of inherited blood disorders in which red blood cells develop abnormally, causing pain and anemia. More than 4 million people currently suffer from the disease, with another 40+ million having the sickle cell trait, which can be passed on to future generations. The disease primarily targets people of African, Hispanic, or Middle Eastern descent. Current treatment regimens including blood transfusions and bone marrow transplants are costly, invasive, and impractical for treating large segments of affected patient populations.

"Funders are recognizing the potential of our innovative approach to gene editing that, in combination with our proprietary RNA manufacturing capability, has the potential to deliver accessible gene therapies and improve human health globally," said Marta Ortega-Valle, senior vice president of Human Health and Corporate Development at GreenLight Biosciences. "Finding a safe and effective therapy is critical, but equally important is the ability to produce it affordably for broader access. We are grateful for the Gates Foundation's support to advance novel gene editing approaches for populations in which those therapies are currently out of reach."

Gene editing therapies hold significant promise in the treatment of Sickle Cell Disease since it is a disorder caused by gene mutation. Using RNA as its core, GreenLight Biosciences is working to develop an in vivo gene therapy that could ultimately offer a cure to the disease.

Once the therapy candidate is validated and moves into clinical use, GreenLight Biosciences' biomanufacturing platform will accelerate production of affordable treatments at scale. "Manufacturing sufficient quantities of high-quality RNA at an accessible cost is critical for achieving the full potential of new therapies that aim to reach a global patient population. That capability does not yet exist in the market, but GreenLight's end-to-end, self-contained manufacturing platform aims to make that possible for all mRNA-based therapies and vaccines," Ortega-Valle added.

About GreenLight Biosciences, Inc.GreenLight is a bio-performance company with a unique, cell-free production platform that delivers high-performing RNA solutions to human, plant and animal challenges. GreenLight develops RNA products for plant and life science applications, and collaborates with industry leaders to advance vaccine development, pandemic preparation, crop management, and plant protection. The cutting-edge, natural platform delivers higher-quality RNA at a lower cost and higher speed than was ever before possible. The GreenLight team values diversity, inclusion, and equality and promises to use collaboration to remain scientifically imaginative and passionately focused on making a difference in the world. For more information, visithttps://www.greenlightbiosciences.com/.

SOURCE GreenLight Biosciences

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GreenLight Biosciences Receives $3.3 Million Grant to Develop Sickle Cell Disease Cure Using mRNA Gene Therapy - PRNewswire

JOHNSON CITY, Tenn., Sept. 10, 2020 /PRNewswire/ --LabConnect, the preeminent provider of clinical trial central lab services, today announced the expansion of its Johnson City facility to support its significant growth in cell and gene therapy and immuno-oncology studies. The company,which recentlyrelocated its headquarters to Tennessee, is doubling the capacity of its biorepository for sample storage and its clinical trial kit building capacity. LabConnect welcomed Tennessee Governor Bill Lee, Congressman Phil Roe, Economic Development Commissioner Bob Rolfe, elected officials, and other dignitaries to celebrate this milestone in the company's growth.

"While we have all had to adjust and adapt during these unprecedented circumstances, Tennessee's business climate has remained strong and welcoming to companies around the globe," said Gov. Lee. "I applaud LabConnect for continuing to invest and create jobs in our state and for choosing to bring its headquarters to Tennessee. I look forward to the many great things that will come from this facility in Johnson City."

"We are pleased that another company has chosen to expand its presence in Tennessee," Commissioner Rolfe said. "LabConnect is committed to leading the way in central laboratory services, which will have a global impact from its Tennessee=based facility. We appreciate LabConnect for its continued innovation and for creating high quality jobs."

"We are excited that the incentives with the State, Tennessee Valley Authority, and Northeast Tennessee Regional Economic Partnership have enabled LabConnect to expand its operations," said Tom Sellig, LabConnect CEO. "Our location offers several advantages which has allowed us to provide unique services to pharmaceutical and biotech clients. We are currently serving 200 leading biopharmaceutical clients and proud of the more than 20 products we have supported that are now FDA approved and used to treat patients around the world. The expanded capacity will allow us to scale our organization to meet LabConnect's growing demand for our clients' cell & gene therapy, rare and orphan diseases, and immuno-oncology projects."

For more information, visit http://www.labconnect.com.

About LabConnectConnect with LabConnectthe preeminent provider of central laboratory support services for analytically and logistically complex studies such as immuno-oncology, cell and gene therapies, and rare & orphan diseases. The company offers unique and innovative services that have been specifically designed to meet the exacting demands of today's clinical trials. The worldwide scope of services includes routine and specialized testing, real-time sample tracking, data integration, biorepository, sample processing and specialized functional outsourcing. Leading the evolution in central laboratory services since 2002, LabConnect's services are customized to fit the unique needs of biopharmaceutical clients. Get connected by requesting a proposal at http://www.labconnect.com or via email at [emailprotected].

SOURCE LabConnect

http://www.labconnect.com

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LabConnect Announces Expansion to Support Cell & Gene Therapy Growth - PRNewswire

New Jersey contract manufacturer Catalent has been right in the mix in the COVID-19 response effort, signing pacts to help produce frontrunners in the vaccine hunt. Now, the company is fleshing out a Maryland facility to aid in that effortand position Catalent's cell and gene therapy offerings well into the future.

Catalent will infuse $130 million into its cell and gene therapy manufacturing facility in Harmans, Maryland, to broaden the CDMO's late-stage production capacity, the company said Wednesday.

The newest investment will add five late-stage clinical and commercial manufacturing suites to the Harmans site, expected to go online in the first half of 2022, Catalent said.

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That expansion will bring the total number of manufacturing suites to 15 at the planned 350,000-square-foot complexnear the Baltimore/Washington International airport. The Harmans facility recently received FDA approval for commercial production, and its initial 10 manufacturing suites are set to be fully operational by the first quarter of 2021.

The five new suites will be located in a second building at the site that will also house cold-storage warehousing and added office space, Catalent said. The Harmans complex is one of five Maryland sites for Catalent's cell and gene therapy manufacturing portfolio.

Adding capacity at its Harmans site is a future play for Catalent in the bustling cell and gene therapy space, but the facility could also benefit the CDMO's immediate COVID-19 response efforts.

RELATED:AstraZeneca ropes in Catalent gene therapy site to produce viral vectors for COVID-19 vaccine

Last month, British drugmaker AstraZeneca tappedCatalent to help produce bulk drug substance and viral vectors at the Harmans facility for the University of Oxford's adenovirus-based COVID-19 vaccine.

Catalent will start production there this quarter,buildingon its previous pact with AstraZeneca for fill-finish and packaging duties at its Anagni, Italy, site.

Catalent's work will include production of viral vectors for a genetically modified form of the adenovirus used in Oxford's shot, dubbed AZD1222. The modified virus, known as ChAdOx1 nCoV-19, aims to induce a lasting immune response to spike proteins added to the virus's surface.

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Catalent injects $130M into Maryland cell and gene therapy site drafted into COVID-19 vaccine hunt - FiercePharma

Sarepta has gone all-in on gene therapy over the last few years, racing with Pfizer and Solid Biosciences to be the first to develop a genetic fix for Duchenne muscular dystrophy, one of the most common rare diseases.

Sarepta has been comfortably in the lead, collecting the first positiveresults and snaring a $1.15 billion cash commercialization deal with Roche, but this week the company hit a snag. Late yesterday, Sarepta provided a program update for its gene therapy, revealing that in a scheduled meeting the FDA had raised concerns about the kinds of tests they would use to measure potency in the pivotal study and commercial supply for the gene therapy. The company has assays that might fit the criteria, they said, but needed additional dialogue with the agency to confirm.

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FDA knocks back Sarepta in Duchenne gene therapy race with Pfizer, but analysts urge caution - Endpoints News

CAMBRIDGE, Mass., Sept. 09, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has completed a Type C written response only meeting with the Office of Tissues and Advanced Therapies (OTAT), part of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA), to obtain OTATs concurrence on the commencement of its next clinical trial for SRP-9001 using commercial process material. SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) is Sareptas investigational gene transfer therapy for the treatment of Duchenne muscular dystrophy.

Among other items, OTAT has requested that Sarepta utilize an additional potency assay for release of SRP-9001 commercial process material prior to dosing in a clinical study. Sarepta has several existing assays and data that it believes could be employed in response to OTATs request. However, additional dialogue with the Agency is required to determine the acceptability of the potency assay approach.

We look forward to working with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study. We will provide further updates as we are able, said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. Every day, thousands of children degenerate from the irreversible damage caused by Duchenne muscular dystrophy. It is for that reason that we will work relentlessly with the Division to satisfy any requests of OTAT and continue the advancement of a potentially transformative therapy for these patients.

About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin)SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Sareptas belief that its existing assays and data could be employed in response to OTATs request; the acceptability of Sareptas potency assay approach by the FDA; our plan to work with OTAT to potentially satisfy their requests and to obtain clarity on the timing of the commencement of our commercial supply study; and the potential of SRP-9001 to be a transformative therapy for DMD patients.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: delays in the commencement of Sareptas next clinical study for SRP-9001 could delay, prevent or limit our ability to gain regulatory approval for SRP-9001; any inability to complete successfully clinical development could result in additional costs to Sarepta or impair Sareptas ability to generate revenues from product sales, regulatory and commercialization milestones and royalties; SRP-9001 may not result in a viable treatment suitable for commercialization due to a variety of reasons, including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

Original post:
Sarepta Therapeutics Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy -...

Collaboration partners,Neurocrine Biosciences (NASDAQ:NBIX) and Voyager Therapeutics (NASDAQ:VYGR) announced data from an early stage clinical trial with Parkinson's disease patients and NBIb-1817, an experimental gene therapy. Three years after a single administration, 14 out of 15 patients are still reporting motor function improvements.

At the moment, NBIb-1817 is in the middle of a phase 2 study that was put on hold in April due to the COVID-19 pandemic. If allowed to restart, the phase 2 RESTORE-1 trial will randomize patients to receive NBIb-1817 or a placebo, then measure for a change in "On" time without troublesome dyskinesia.

Image source: Getty Images.

This potential new treatment option uses a viral vector to deliver a gene encoding an enzyme that helps Parkinson's disease patients convert levodopa into the dopamine they need.

Administering NBIb-1817 requires magnetic resonance imaging (MRI) to guide a pair of infusions that deliver the therapy directly into the striatum, a structure deep in the center of the brain. Needles through the skull aren't anybody's idea of a good time, but the side effects that come with daily doses of levodopa aren't any fun either.

For patients with severe Parkinson's disease that has a diminishing response to levodopa, the temporary discomfort that comes with NBIb-1817 treatment seems like a trade-off most will be willing to accept. Three years after a single treatment with NBIb-1817, patients were able to reduce their daily levodopa doses from a baseline of 1500.9 milligrams per day to 1061.4 milligrams per day. Despite reducing their levadopa intake, patients receiving three different dosage strengths of NBIb-1817 improved average "On" time by up to 2.23 hours.

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Parkinson's Disease Patients Get Long-Term Benefits From Experimental Gene Therapy - The Motley Fool

CAMBRIDGE, Mass., Sept. 15, 2020 /PRNewswire/ --Obsidian Therapeutics, Inc., a biotechnology company pioneering controllable cell and gene therapies, today announced that Bristol Myers Squibb (NYSE:BMY) has exercised its option to an exclusive worldwide license to a cell therapy candidate based on Obsidian's cytoDRiVE technology for the controlled expression of the immunomodulatory factor CD40L. This announcement marks the first opt-in decision by Bristol Myers Squibbsince the companies announced their collaboration to develop novel cell therapies in January 2019. Under the terms of the agreement, Obsidian is eligible to receive potential future milestone and royalty payments.

"We are very interested in exploring innovative approaches to developing engineered cell therapies, including the cytoDRiVE platform," said Rupert Vessey, D. Phil., Executive Vice President, Research and Early Development, Bristol Myers Squibb. "By controllingthe expression of armed payloads like CD40L, Obsidian's cell therapy candidates may have the potential to overcome tumor microenvironment resistance and unlock the power of cell therapy in solid tumors and other malignancies."

"This announcement marks an important milestone validating Obsidian's cytoDRiVE platform, and we look forward to continuing to work with Bristol Myers Squibbto bring powerful new immunotherapies to patients," said Paul K. Wotton, Ph.D., Chief Executive Officer of Obsidian Therapeutics. "We are also pleased with the pace with which our own pipeline programs are progressing as we continue to advance our lead controllable tumor infiltrating lymphocyte (TIL) therapy to the clinic."

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

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Maggie BellerRusso Partners, LLC[emailprotected]646-942-5631

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Obsidian Therapeutics Announces Bristol Myers Squibb Opt-In of cytoDRiVE Cell Therapy Candidate - PRNewswire