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End of Palestinian Authority coordination with Israel in response to annexation threat: decision already impacting medical referrals – occupied…

On 19 May 2020, Palestinian President Mahmoud Abbas announced that the Palestinian Authority (PA) considered itself absolved of all agreements and understandings it has signed with Israel and the United States. This decision was in response to Israels announcement of its intention to annex parts of the West Bank as early as 1 July, subject to the full agreement of the current U.S. administration. The threat of annexation has been widely condemned by the international community and the UN Secretary-General has stated that annexation would constitute a most serious violation of international law, grievously harm the prospect of a two-State solution and undercut the possibilities of a renewal of negotiations.

The PA has subsequently halted almost all bilateral contacts with Israel, including security coordination.[1] The PA has also stopped accepting the clearance revenues that Israel collects on its behalf which, adding to the economic uncertainty caused by the COVID-19 pandemic, has resulted in a loss of about 80 per cent of its monthly revenues, and is limiting its capacity to pay employees salaries.

The suspension of coordination has already affected humanitarian operations across the occupied Palestinian territory (oPt), including preparedness and response to the COVID-19 pandemic. Since the beginning of June, the import of essential supplies by humanitarian agencies has been disrupted, affecting some of the major operational organizations, including the World Health Organization (WHO), the UN Childrens Fund (UNICEF), and the UN Development Programme (UNDP), in addition to some NGOs.

This Humanitarian Bulletin article, the first in a series addressing the implications of the halt in coordination between the PA and Israel, focuses on the access of Palestinian patients, in particular from the Gaza Strip, to health care in East Jerusalem and in Israel; two Gaza infants have died thus far, before their transfers to hospitals in Israel and in East Jerusalem, respectively, could be arranged.

Access to essential health care critically affected

Information and cases included in this section were provided by the World Health Organization (WHO)

Many Palestinian patients throughout the oPt, and their companions, require Israeli-issued permits to access essential health services unavailable in their locality. For residents of the Gaza Strip, permits are required to access hospitals in the West Bank, including East Jerusalem, as well as Israel and Jordan. In the West Bank, patients and their companions also require permits to enter East Jerusalem, Israel or Jordan.[2] In 2019 each month, there were more than 7,000 patient permit applications from the West Bank (outside East Jerusalem), and over 2,000 from Gaza. Almost a third of applications are for persons with cancer. Approximately half of all referrals by the Palestinian Ministry of Health (MoH) were to hospitals in East Jerusalem.

Despite the cessation of permits coordination, patients and their families from the West Bank have been able to go in person to the Israeli District Coordination Office (DCO) to apply directly for permits. Additionally, the Israeli authorities are promoting a new mobile phone application, which Palestinians can download to apply for permits remotely, although concerns remain regarding the privacy and security implications of this system. Since the halt of coordination, there is no oversight of the application process for patients and companions, with limited means to track or record the number of those who apply; how many permits are denied or delayed; or to facilitate appeals on behalf of applicants.

In Gaza, although financial approvals for patient referrals are still issued, the PA has ended its coordination of permits requests for residents, including for patients and their companions. The issuance of referrals had already declined since the outbreak of COVID-19 in the oPt in March, following the Palestinian MoHs decision to reduce health services delivery in order to decrease transmission risk. This included efforts to localize some essential services, such as the short-term provision of limited chemotherapy through private providers in Gaza. In addition to the limitations imposed by the Palestinian authorities, the Israeli authorities further restricted eligibility criteria for exit permits, limiting those only to cancer patients and emergency cases.[3] Consequently, in March, patient exits declined to 1,279, from a monthly average of 1,777 in January and February. April recorded only 159 applications, of which 113 were approved, and May, 160 applications, of which 105 were approved.

While restrictions eased after an initial decline in COVID-19 cases, the recent increase of cases in the West Bank and Israel may cause authorities to extend the limitations on exit from Gaza to prevent the occurrence of a community outbreak there.

The exit of a limited number of emergency cases had been facilitated through the intercession of the Palestinian Centre for Human Rights (PCHR), a Gaza-based NGO, among other organizations. Selma, a 64-year-old with Hodgkins Lymphoma, was referred to a West Bank hospital for self-stem cell transplant, a procedure unavailable in Gaza. With the help of PCHR, she obtained a permit to travel to the West Bank on 7 June, the day of her scheduled transplant operation. However, PCHR ended the facilitation of coordination of permits on 9 June, following an Israeli media report portraying the organization role as replacing the PAs role.[4] Currently, a number of civil society organizations and hospitals are coordinating for patients referred for urgent treatment.

Osama, a one-year-old boy from Gaza City, was diagnosed with leukaemia on 8 June and referred urgently to Augusta Victoria Hospital in East Jerusalem for immediate treatment. Through the considerable efforts of hospital staff, he received a permit on 11 June, and he was able to travel to the hospital the same day.

Although Osamas mother was still breastfeeding him, he was accompanied by his grandmother on the grounds that older people find it easier to pass the security conditions required for permits. The medical team at Augusta Victoria Hospital reported his severe distress for days after his separation from his mother. Osamas father commented, We were told he is crying most of the time. Someone from the hospital has contacted me to try to coordinate a permit for Osamas mother to travel and be close to him, but we are still waiting.

The first fatality in Gaza following the halt in coordination was reported on 18 June, when eight-month-old Omar Yaghi, died of cardiac complications, before he was able to receive a permit to travel for an operation to Sheba Medical Center in Israel. On 22 June, a nine-day-old infant, Anwar Harb, who also suffered from heart problems, died before he could receive treatment in East Jerusalem. The Israeli authorities had agreed to his transfer to Al-Makassed Hospital that day, but the hospital was unable to coordinate the ambulance transfer in time.[5]

Following the death of Omar Yaghi, a coalition of Israeli-based organizations sent an urgent letter to the Israeli Defence Minister, Attorney General, and the head of COGAT, demanding that Israel, as the occupying power, immediately ensure Palestinian travel through the Erez Crossing. The organizations specifically requested that Israel remove all bureaucratic restrictions on the submission of permit applications by patients and other humanitarian cases and allow Gaza residents to enter Israel and the West Bank immediately; immediately clarify how travel permit applications may be filed by Gaza residents; and publish clear, detailed, and accessible information about the alternative application procedure.

Roles and responsibilities of the parties

There have been suggestions that international organizations, particularly the UN, may be requested to perform the coordination activities, which the PA has been previous responsible for. However, as stated by UN Special Coordinator, Nikolay Mladenov, in his most recent briefing to the UN Security Council; while the UN is prepared to provide support on an emergency basis, the UN cannot replace the Palestinian Authority. It is critical that humanitarian and other assistance not be delayed or stopped.

The emergency support to be provided by the UN does not absolve Israel or the PA of their primary obligations towards the Palestinian population in the oPt. Under international law, Israel, as occupying power, bears the primary responsibility for the protection and welfare of this population, including access to essential health services. Even prior to the suspension of coordination, Palestinian patients, and their companions, faced major barriers to timely access to essential healthcare, due to delays and denials of the Israeli-issued permits needed for access to major specialist centres in East Jerusalem and in Israel.

The PA, having ratified human rights treaties, also has certain duties towards this population, within the limits of its control, as does the de facto authority in the Gaza Strip. These include ensuring respect and protection for the right to the highest attainable standard of health, and ensuring that policies introduced, or the withdrawal from previous responsibilities, do not cause harm.

Based on these considerations, the UN has agreed with both the Palestinian and Israeli authorities the establishment of a temporary mechanism, as part of which the UN will facilitate the transfer of the required documentation between the sides, to ensure access for Gaza patients to essential health services in the West Bank, including East Jerusalem, and Israel. This mechanism is expected to start operating during the second half of July.

[1] The only exception is coordination relating to the Kerem Shalom crossing for goods in and out of Gaza, which continues to operate as before.

[2] The majority of men over 55 years of age, and women over 50 from the remainder of the West Bank, as well as children 13 years and under, and accompanied by an adult, do not require permits to enter Israel or East Jerusalem.

[3] https://www.ochaopt.org/content/additional-treatment-option-available-ca...

[4] See https://www.pchrgaza.org/en/?m=20200609

[5] http://www.alhaq.org/cached_uploads/download/2020/06/27/200626-joint-urg...

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End of Palestinian Authority coordination with Israel in response to annexation threat: decision already impacting medical referrals - occupied...

Impact of COVID-19 on Autologous Stem Cell and Non-Stem Cell Based Therapies Market Rapid Growth And Forecast to 2027 | Fibrocell Science, Inc.,…

A new business intelligence report released by CMI with title Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Research Report 2019-2027 is designed covering micro level of analysis by manufacturers and key business segments. The Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market survey analysis offers energetic visions to conclude and study market size, market hopes, and competitive surroundings. The research is derived through primary and secondary statistics sources and it comprises both qualitative and quantitative detailing. Some of the key players profiled in the study are Caladrius Biosciences, Vericel Corporation, Fibrocell Science, Inc., Genzyme Corporation, BrainStorm Cell Therapeutics, Regeneus Ltd., and Dendreon Corporation.

Whats keeping Caladrius Biosciences, Vericel Corporation, Fibrocell Science, Inc., Genzyme Corporation, BrainStorm Cell Therapeutics, Regeneus Ltd., and Dendreon Corporation. Ahead in the Market? Benchmark yourself with the strategic moves and findings recently released by CMI

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Market Overview of Global Autologous Stem Cell and Non-Stem Cell Based Therapies

If you are involved in the Global Autologous Stem Cell and Non-Stem Cell Based Therapies industry or aim to be, then this study will provide you inclusive point of view. Its vital you keep your market knowledge up to date segmented by Applications and major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports we can provide customization according to your requirement.

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Furthermore, the years considered for the study are as follows:

Historical year 2014 2018

Base year 2019

Forecast period** 2019 to 2027 [** unless otherwise stated]

**Moreover, it will also include the opportunities available in micro markets for stakeholders to invest, detailed analysis of competitive landscape and product services of key players.

Detailed Segmentation:

On the basis of application, the global autologous stem cell and non-stem cell based therapies market is classified into:Neurodegenerative diseasesCardiovascular diseasesCancer and autoimmune diseasesSkin transplantationInfectious disease

Top Players in the Market are: Caladrius Biosciences, Vericel Corporation, Fibrocell Science, Inc., Genzyme Corporation, BrainStorm Cell Therapeutics, Regeneus Ltd., and Dendreon Corporation.

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o North America (United States, Canada, and Mexico)

o Europe (Germany, France, UK, Russia, and Italy)

o Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

o South America (Brazil, Argentina, Colombia)

o Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

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Important Features that are under offering & key highlights of the report:

Detailed overview of Autologous Stem Cell and Non-Stem Cell Based Therapies market

Changing market dynamics of the industry

In-depth market segmentation by Type, Application etc

Historical, current and projected market size in terms of volume and value

Recent industry trends and developments

Competitive landscape of Autologous Stem Cell and Non-Stem Cell Based Therapies market

Strategies of key players and product offerings

Potential and niche segments/regions exhibiting promising growth

A neutral perspective towards Autologous Stem Cell and Non-Stem Cell Based Therapies market performance

Market players information to sustain and enhance their footprint

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Major Highlights of TOC:

Chapter One: Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Industry Overview

1.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Industry

1.1.1 Overview

1.1.2 Products of Major Companies

1.2 Autologous Stem Cell and Non-Stem Cell Based Therapies Market Segment

1.2.1 Industry Chain

1.2.2 Consumer Distribution

1.3 Price & Cost Overview

Chapter Two: Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Demand

2.1 Segment Overview

2.1.1 APPLICATION 1

2.1.2 APPLICATION 2

2.1.3 Other

2.2 Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Size by Demand

2.3 Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Forecast by Demand

Chapter Three: Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market by Type

3.1 By Type

3.1.1 TYPE 1

3.1.2 TYPE 2

3.2 Autologous Stem Cell and Non-Stem Cell Based Therapies Market Size by Type

3.3 Autologous Stem Cell and Non-Stem Cell Based Therapies Market Forecast by Type

Chapter Four: Major Region of Autologous Stem Cell and Non-Stem Cell Based Therapies Market

4.1 Global Autologous Stem Cell and Non-Stem Cell Based Therapies Sales

4.2 Global Autologous Stem Cell and Non-Stem Cell Based Therapies Revenue & market share

Chapter Five: Major Companies List

Chapter Six: Conclusion

Key questions answered

o Who are the Leading key players and what are their Key Business plans in the Global Autologous Stem Cell and Non-Stem Cell Based Therapies market?

o What are the key concerns of the five forces analysis of the Global Autologous Stem Cell and Non-Stem Cell Based Therapies market?

o What are different prospects and threats faced by the dealers in the Global Autologous Stem Cell and Non-Stem Cell Based Therapies market?

o What are the strengths and weaknesses of the key vendors?

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Impact of COVID-19 on Autologous Stem Cell and Non-Stem Cell Based Therapies Market Rapid Growth And Forecast to 2027 | Fibrocell Science, Inc.,...

Stem Cell Reconstructive Market Capacity, Production, Revenue, Price and Gross Margin, Industry Analysis & Forecast by 2025 – Jewish Life News

Stem Cell Reconstructive Market report 2018, discusses various factors driving or restraining the market, which will help the future market to grow with promising CAGR. The Stem Cell Reconstructive Market research Reports offers an extensive collection of reports on different markets covering crucial details. The report studies the competitive environment of the Stem Cell Reconstructive Market is based on company profiles and their efforts on increasing product value and production.

This Report covers the manufacturers data, including: shipment, price, revenue, gross profit, interview record, business distribution etc., these data help the consumer know about the competitors better. This report also covers all the regions and countries of the world, which shows a regional development status, including market size, volume and value, as well as price data.

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The study objectives of this report are: To analyze global Stem Cell Reconstructive status, future forecast, growth opportunity, key market and key players. To present the Stem Cell Reconstructive development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America. To strategically profile the key players and comprehensively analyze their development plan and strategies. To define, describe and forecast the market by type, market and key regions.

In this study, the years considered to estimate the market size of Stem Cell Reconstructive are as follows: History Year: 2015-2019 Base Year: 2019 Estimated Year: 2020 Forecast Year 2020 to 2026 For the data information by region, company, type and application, 2019 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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Stem Cell Reconstructive Market Capacity, Production, Revenue, Price and Gross Margin, Industry Analysis & Forecast by 2025 - Jewish Life News

GEMoaB and Intellia Therapeutics Enter into Research Collaboration and Licensing Agreement on Next-Generation Cellular Immunotherapy Based on…

GEMoaB and Intellia to Combine Proprietary Technologies and Research and Development Expertise to Create Transformative Allogeneic Cellular Immunotherapies against Promising Targets in Oncology and Inflammatory Diseases

GEMoaB to Receive Upfront Payments, Regulatory and Commercial Milestone Payments Plus Royalties Based on Net Sales

DRESDEN, Germany, July 20, 2020 /PRNewswire/ -- GEMoaB, a biopharmaceutical company focused on the development of next-generation immunotherapies for hard-to-treat cancers, announced today that it has entered into a research collaboration and license agreement with Intellia (Nasdaq: NTLA), a global leader in the field of genome editing treatments.

The companies will conduct joint research to combine GEMoaB's proprietary RevCAR technology platform with Intellia's proprietary genome editing technologies to discover and develop next-generation allogeneic cellular immunotherapies for hard-to-treat cancers and inflammatory diseases. The companies will focus on immunotherapies directed against a selected number of targets.

Under the terms of the agreement, GEMoaB will receive payments for each product based on target reservation and selection, achievement of regulatory, clinical and commercial milestones as well as tiered royalties based on net sales. Intellia will lead the research collaboration, and be responsible for clinical development and commercialization.

"We are very pleased to enter into the agreement with Intellia, which is, similarly to GEMoaB, using its cutting-edge technology to develop breakthrough products for patients with a high unmet medical need," said Armin Ehninger, Ph.D., Chief Scientific Officer at GEMoaB. "Not only does the collaboration reflect the unique optionality of GEMoaB's technological platform, but it will also provide rapid clinical proof-of-concept for RevCAR, our second cellular immunotherapy platform."

Andrew Schiermeier, Ph.D., Intellia's Chief Operating Officer, added, "We are very pleased to work with the talented GEMoaB team, and believe that their RevCAR platform represents a unique and powerful advance in how we think about engineering cell therapies. We are confident that the combined power of GEMoaB's cellular immunotherapy platform and Intellia's numerous and proprietary CRISPR-based approaches to T-cell engineering will accelerate the development of unique and highly-differentiated, genome-edited products for patients with a high unmet medical need."

About GEMoaB

GEMoaB is a privately-owned, clinical-stage biopharmaceutical company that isaiming to become a globally leading, fully integrated biopharmaceutical company. By advancing its proprietary UniCAR, RevCAR and ATAC platforms, the company will discover, develop, manufacture and commercialize next-generation immunotherapies for the treatment of cancer patients with a high unmet medical need.

GEMoaB has a broad pipeline of product candidates in pre-clinical and clinical development for the treatment of hematological malignancies as well as solid tumors. Its clinical stage assets GEM333, an Affinity-Tailored Adaptor for T-Cells (ATAC) with binding specificity to CD33 in relapsed/refractory AML, and GEM3PSCA, an ATAC with binding specificity to PSCA for the treatment of castrate-resistant metastatic prostate cancer and other PSCA expressing late stage solid tumors, are currently investigated in Phase I studies and globally partnered with Bristol-Myers Squibb. A Phase IA dose-finding study of the first UniCAR asset, UniCAR-T-CD123 for treatment of relapsed/refractory AML and ALL is ongoing, UniCAR-T-PSMA against CRPC and other PSMA-expressing late-stage solid tumors, is planned to be tested in a Phase IA study initiated by H2 2020.

Manufacturing expertise, capability and capacity are key for developing cellular immunotherapies for cancer patients. GEMoaB has established a preferred partnership with its sister company Cellex, a world leader in manufacturing hematopoietic blood stem cell products and a leading European CMO for CAR-T cells, co-operating in that area with several large biotech companies.

About UniCAR and RevCAR

GEMoaB is developing rapidly switchable universal CAR-T platforms, UniCAR and RevCAR, to improve the therapeutic window and increase efficacy and safety of CAR-T cell therapies in more challenging cancers, including solid tumors as well as hard-to-treat inflammatory diseases. Standard CAR-T cells depend on the presence and direct binding of cancer antigens for activation and proliferation. An inherent key feature of the UniCAR and RevCAR platforms is a rapidly switchable on/off mechanism enabled by the short pharmacokinetic half-life and fast internalization of soluble adaptors termed targeting modules (TMs). These TMs provide the antigen-specificity to activate UniCAR/RevCAR gene-modified T-cells (UniCAR-T, RevCAR-T) and consist of a highly flexible antigen-binding moiety, linked to a peptide motif recognized by UniCAR-T/RevCAR-T. Different to UniCAR, where the CAR consists of a scFv, RevCAR uses an inert peptide attached to the CAR. RevCAR TMs consist of a highly flexible antigen-binding moiety, linked to a scFv domain binding to the inert peptide attached to the CAR of the RevCAR effector T-cell.

About ATAC

GEMoaB's platform of Affinity-Tailored Adaptors for T-Cells (ATAC) is characterized by high binding affinity to tumor antigens and lower affinity to the CD3 antigen on effector T-cells, preventing T-cell auto-activation in pre-clinical models. Safety and tolerability of the treatment are also increased by the relatively short serum half-life (60 min). The use of fully humanized antibodies reduces the risk of immunogenicity even in case of chronic dosing. Half-life extended ATACs are in pre-clinical development.

More information can be found at http://www.gemoab.com.

For further information please contact

Jana Fiebiger j.fiebiger@gemoab.com Tel.: +49 351 4466-45012

Investor Contact

Michael Pehl m.pehl@gemoab.com Tel.: +49 351 4466-45030

Forward-looking Statements

This announcement includes forward-looking statements that involve risks, uncertainties and other factors, many of which are outside of our control, that could cause actual results to differ materially from the results and matters discussed in the forward looking statements. Forward looking statements include statements concerning our plans, goals, future events and or other information that is not historical information.

The Company does not assume any liability whatsoever for forward-looking statements. The Company assumes that potential partners will perform and rely on their own independent analyses as the case may be. The Company will be under no obligation to update the Information.

Logo - https://mma.prnewswire.com/media/947610/GEMoaB_Logo.jpg

http://www.gemoab.com

SOURCE GEMoaB

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GEMoaB and Intellia Therapeutics Enter into Research Collaboration and Licensing Agreement on Next-Generation Cellular Immunotherapy Based on...

Courageous Oscar Saxelby-Lee from Worcester celebrates ringing end of treatment bell with huge party – Birmingham Live

A brave Worcestershire six-year-old has rung the bell to signal the end of his cancer treatment after a long battle.

Oscar Saxelby-Lee who travelled more than 6,500 miles to get treatment in Singapore this year and flew back last month, held a party with family, friends and neighbours on Sunday (July 19) to mark the occasion.

In a poignant moment his headteacher, Kate Wilcock from Pitmaston Primary School read The End of Treatment Bell poem followed by three cheers hip hip hooray; from those gathered. Mrs Wilcock said his classmates were so excited to have Oscar back at school when the new year starts in September.

In a post on the Hand in Hand for Oscar Facebook page the moment the familys joy and thanks were expressed.

His mum Olivia said: Go Ozzy Bear!!! So so so sooooo proud of you!

We tried to keep it sweet and to a minimum but it kind of got a little mad out on our street today!!!

Thank you to all of our close family and friends (neighbours too) for popping down to cheer our super star on!

Thank you to the special people who helped make this the BEST possible bell ringing day for our boy! Especially his headteacher Mrs Wilcock, reception teachers Mrs Keating, Miss Senter and Mrs Bladen who is an absolute angel!

Also to Something Party for helping us with the balloon set up, Neil for our banners, Ruth for our cakes, Yours Truly Creations and Gifts for Oscars perfect T-shirt and Ellys Bakes for even more goodies!

Oscar was diagnosed with an aggressive bloody cancer - T-cell acute lymphoblastic leukaemia - in December 2018 after his parents noticed unusual bruising on his legs.

The youngster from St Johns in Worcester was given just months to find a stem cell match to save his life which sparked a fundraising mission by his parents Olivia Saxelby, 25, and Jamie Lee, 28.

The nationwide campaign raised more than 600,000 to pay for pioneering treatment CAR-T (chimeric antigen receptor T-cell) therapy not available in the UK, with Oscar the second child in the world to have it. He then had a second bone marrow transplant using stem cells from dad Jamie.

And just three months after the operation Oscar flew back home on Thursday, June 25. The youngster was then driven to see his classmates who cheered him as he went past the school gates.

The familys home had been decked in balloons, including a green dinosaur and welcome home signs with young Oscar beaming.

That day his mum posted: Cant quite believe it.... crying bucket loads!

"The day weve dreamt of for so very long has now become a reality! Were actually bringing him home!!!

You are our HERO Ozzy Bear, you truly truly are the most amazing little miracle. Enjoy every last minute of this journey darling because YOU have owned it!

Singapore, Oh, Singapore.... You hold a very special place in our hearts. To each and everyone of you who have helped us and supported us, THANK YOU!

Throughout his treatment, Oscar has kept in touch with his classmates and teachers from his hospital bed via an innovative robot - nicknamed "Ozzybot".

His headteacher Kate Wilcock, who organised a huge donor registration event, which saw 4,855 volunteers queue in the freezing rain at his Worcestershire school, and Ozzybot lessons, said: Theres been plenty of ups and downs to get here but this is the absolute icing on the cake that hes come home, cancer free and its worked.

"Our future plans now will be to support Oscar and his family and even start some learning at home.

"Then we will be working really closely with Olivia, Jamie and Oscar for some transition into school.

"That will be the best day when he walks back through the door at Pitmaston Primary."Oscar is due to return to Singapore in six-months-time to check his progress.

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Courageous Oscar Saxelby-Lee from Worcester celebrates ringing end of treatment bell with huge party - Birmingham Live

Notch Therapeutics Appoints David Main as President and Chief Executive Officer to Advance the Company’s Novel Gene-Edited, iPSC-Derived Immune Cell…

TORONTO, July 20, 2020 /PRNewswire/ --Notch Therapeutics Inc., a biotechnology company creating universally compatible, off-the-shelf T cell therapies for cancer and immune disorders from renewable stem cell sources, is pleased to announce the appointment of David Main as President and Chief Executive Officer.

Notch is applying its scalable Engineered Thymic Niche (ETN) technology platform to develop homogeneous and universally compatible, stem cell-derived T cell therapies. To date, Notch has assembled a world-class scientific team and built a fully integrated, tightly controlled platform for generating and editing immune cells from clonal stem cells to enable development of a broad range of T cell therapeutics. Notch has also entered into a partnership with Allogene Therapeutics (NASDAQ: ALLO) to apply Notch's proprietary ETN platform to develop CAR-targeted, induced pluripotent stem cell (iPSC)-derived, off-the-shelf T cell or natural killer (NK) cell therapies for hematologic cancer indications.

"We have a clear goal at Notch: To create universally compatible, safe, and effective immunotherapies with the capability to treat thousands of patients from a single manufacturing run," said David Main. "The company has an internationally recognized team, a groundbreaking technology positioned to redefine and expand the clinical and commercial potential of cell therapy, and has already attracted a leading corporate partner. This is an exciting time to join and lead the company, which is now strongly positioned to advance our own pipeline of products as we also pursue additional partnering opportunities."

"We have spent the past year building a leading company developing next-generation, off-the-shelf immunotherapies driven by outstanding science and focused execution," said Ulrik Nielsen, Ph.D., Chairman of Notch. "David provides Notch with a proven industry leader and strategic thinker who has extensive experience driving and financing biotech innovation from early-stage research through commercial readiness. We are excited to bring in such outstanding leadership that is ideally suited to lead the company as it continues to advance its new class of off-the-shelf T cell therapy products."

Mr. Main is a highly experienced biopharmaceutical executive who brings to Notch more than 30 years of industry leadership experience with a strong track record of value creation and company growth. Most recently, as co-founder, Chairman, and CEO of Aquinox Pharmaceuticals, Mr. Main oversaw the advancement of the company's lead product from target validation through Phase 3 clinical trials. He also led the transition of Aquinox from a private to a NASDAQ-listed public company with approximately $300 million raised in equity capital and then completed the successful merger of Aquinox with Neoleukin Therapeutics. Prior to his leadership of Aquinox, Mr. Main served as President and CEO of INEX Pharmaceuticals and as a Vice President of QLT.

About Notch Therapeutics (www.notchtx.com) Notch is an immune cell therapy company creating universally compatible, allogeneic (off-the-shelf) T cell therapies for the treatment of cancer and immune disorders. Notch's technology platform uses genetically tailored stem cells as a renewable source for creating allogeneic T cell therapies that expand treatment options and has the potential to deliver safer, consistently manufactured and more cost-effective cell immunotherapies to patients. At the core of Notch's technology is the synthetic Engineered Thymic Niche (ETN) platform, which precisely controls the expansion and differentiation of stem cells in a process suitable for large-scale manufacturing, delivering fully defined, consistent, feeder-free and serum-free T cells that can be genetically tailored for any T cell-based immunotherapeutic application. This technology was invented in the laboratories of Juan-Carlos Ziga-Pflcker, Ph.D. at Sunnybrook Research Institute and Peter Zandstra, Ph.D., FRSC at the University of Toronto. Notch was founded by these two institutions, in conjunction with MaRS Innovation (now Toronto Innovation Acceleration Partners) and the Centre for Commercialization of Regenerative Medicine (CCRM) in Toronto.

Contact:Mary Moynihan M2Friend Biocommunications 802-951-9600 [emailprotected]

SOURCE Notch Therapeutics

Notch Therapeutics

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Notch Therapeutics Appoints David Main as President and Chief Executive Officer to Advance the Company's Novel Gene-Edited, iPSC-Derived Immune Cell...

embryonic stem cell NIH Director’s Blog

Posted on September 20th, 2016 by Dr. Francis Collins

Many people probably think of mice as unwanted household pests. But over more than a century, mice have proven to be incredibly valuable in medical research. One of many examples is how studies in mice are now helping researchers understand how mammalian genomes work, including the human genome. Scientists have spent decades inactivating, or knocking out, individual genes in laboratory mice to learn which tissues or organs are affected when a specific gene is out of order, providing valuable clues about its function.

More than a decade ago, NIH initiated a project called KOMPthe Knockout Mouse Project [1]. The goal was to use homologous recombination (exchange of similar or identical DNA) in embryonic stem cells from a standard mouse strain to knock out all of the mouse protein-coding genes. That work has led to wide availability of such cell lines to investigators with interest in specific genes, saving time and money. But its one thing to have a cell line with the gene knocked out, its even more interesting (and challenging) to determine the phenotype, or observable characteristics, of each knockout. To speed up that process in a scientifically rigorous and systematic manner, NIH and other research funding agencies teamed to launch an international research consortium to turn those embryonic stem cells into mice, and ultimately to catalogue the functions of the roughly 20,000 genes that mice and humans share. The consortium has just released an analysis of the phenotypes of the first 1,751 new lines of unique knockout mice with much more to come in the months ahead. This initial work confirms that about a third of all protein-coding genes are essential for live birth, helping to fill in a major gap in our understanding of the genome.

Posted In: Health, Science

Tags: conserved genes, embryonic development, embryonic stem cell, essential genes, genes, genetic conditions, genetics, genomics, homologous recombination, humans, International Mouse Phenotyping Consortium, knockout mice, Knockout Mouse Project, KOMP, miscarriages, mouse, phenotype, stem cells, stillbirths

Posted on July 19th, 2016 by Dr. Francis Collins

Caption: From stem cells to bone. Human bone cell progenitors, derived from stem cells, were injected under the skin of mice and formed mineralized structures containing cartilage (1-2) and bone (3). Credit: Loh KM and Chen A et al., 2016

To help people suffering from a wide array of injuries and degenerative diseases, scientists and bioengineers have long dreamed of creating new joints and organs using human stem cells. A major hurdle on the path to achieving this dream has been finding ways to steer stem cells into differentiating into all of the various types of cells needed to build these replacement parts in a fast, efficient manner.

Now, an NIH-funded team of researchers has reported important progress on this front. The researchers have identified for the first time the precise biochemical signals needed to spur human embryonic stem cells to produce 12 key types of cells, and to do so rapidly. With these biochemical recipes in hand, researchers say they should be able to generate pure populations of replacement cells in a matter of days, rather than the weeks or even months it currently takes. In fact, they have already demonstrated that their high-efficiency approach can be used to produce potentially therapeutic amounts of human bone, cartilage, and heart tissue within a very short time frame.

Posted In: Health, Science

Tags: bioengineering, Bone, cartilage, development, embryonic stem cell, heart cells, human embryonic stem cell, mesoderm, muscle cells, regenerative medicine, replacement tissue, RNA sequencing, scoliosis, stem cell differentiation, stem cells, tissue engineering

Posted on June 2nd, 2015 by Dr. Francis Collins

If youre curious what innovations are coming out of the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative, take a look at this video shot via a microscope. What at first glance looks like water dripping through pipes is actually a cool new technology for swiftly and efficiently analyzing the gene activity of thousands of individual cells. You might have to watch this video several times and use the pause button to catch all of the steps, but it provides a quick overview of how the Drop-seq microfluidic device works.

First, a nanoliter-sized droplet of lysis buffer containing a bead with a barcoded sequencing primer on its surface slides downward through the straight channel at the top of the screen. At the same time, fluid containing individual cells flows through the curved channels on either side of the bead-bearing channelyou can catch a fleeting glimpse of a tiny cell in the left-hand channel about 5 seconds into the video. The two streams (barcoded-bead primers and cells) feed into a single channel where they mix, pass through an oil flow, and get pinched off into oily drops. Most are empty, but some contain a bead or a celland a few contain both. At the point where the channel takes a hard left, these drops travel over a series of bumps that cause the cell to rupture and release its messenger RNAan indicator of what genes are active in the cell. The mRNAs are captured by the primer on the bead from which, after the drops are broken, they can be transcribed, amplified, and sequenced to produce STAMPS (single-cell transcriptomes attached to microparticles). Because each bead contains its own unique barcode that enables swift identification of the transcriptomes of individual cells, this process can be done simultaneously on thousands of cells in a single reaction.

Posted In: Health, Science, Video

Tags: BRAIN Initiative, Drop-seq, embryonic stem cell, genomics, inDrop, neurology, retina, single cell analysis, technology, transciptome

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embryonic stem cell NIH Director's Blog

Clinton Vows to Fund Embryonic Stem Cell Research as …

By Peter J. Smith

WASHINGTON, D.C., October 9, 2007 (LifeSiteNews.com) - Democratic presidential candidate Hillary Rodham Clinton has promised to sign an executive order overturning President Bushs restrictions on federal funding for embryonic stem cell research once she is elected President.

The former First Lady and current junior senator from New York told her audience at the Carnegie Institution for Science that President Bush was waging a "war on science" that hindered the United States from becoming the "innovation nation."

"I will lift the current ban on ethical stem cell research," Clinton said. "The presidents ban on stem cell funding amounts to a ban on hope."

However the US has no actual ban on embryonic stem cell research. Regulations established by the Bush administration in August 2001 prohibit researchers from using federal funds to create new lines of embryonic stem cells, but it does not hinder private companies from funding their work.

"In her rush to attack the president, Hillary Clinton has conveniently forgotten that George W. Bush is the only president who has ever made federal money available for stem cell research," said Republican National Committee spokesman Danny Diaz according to Reuters.

Clintons speech also gave the impression that "ethical" stem cell research was synonymous with embryonic stem cell research, although this premise is hotly contested within the scientific community. A number of stem cell researchers reject on a practical basis any need to drive into ethically dubious territory, since stem cell therapies are continuing to be produced from non-controversial sources (e.g. adult stem cells, umbilical cord blood). On the other hand, the promise of cures from experimentation with embryonic stem cells is filled with more hot air than hope, since the cells derived from the destruction of a human embryo are naturally designed to work in the fast-developing embryonic environment and have been shownto be incompatible and tumor-causing in adult tests.

See related coverage by LifeSiteNews.com

Adult Stem Cell Research: True Potential Sacrificed for Other Possibilities Says Biotech Writer http://www.lifesitenews.com/ldn/2006/jun/06061311.html

UK Researcher: Cord Blood Real Potential for Cures, Not Embryonic Stem Cells - Part 1 http://www.lifesitenews.com/ldn/2006/aug/06081804.html

UK Researcher: Embryonic Stem Cells Have Never Been Used to Treat Anyone and no Plans Exist to do so http://www.lifesitenews.com/ldn/2006/aug/06082401.html

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Clinton Vows to Fund Embryonic Stem Cell Research as ...

Orchard Therapeutics Announces Orphan Drug and Rare Pediatric Disease Designations for OTL-203 for the Treatment of MPS-I – GlobeNewswire

July 20, 2020 07:00 ET | Source: Orchard Therapeutics (Europe) Limited

BOSTON and LONDON, July 20, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company has received both orphan drug designation and rare pediatric disease designation from the U.S Food and Drug Administration (FDA) for OTL-203, anex vivoautologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I).

We are pleased by the FDAs acknowledgement of the critical and urgent need to develop additional treatments for MPS-I given the severe, life-limiting nature of the disease, said Bobby Gaspar, M.D., PhD., chief executive officer of Orchard. The underlying causes of lysosomal storage disorders such as MPS-I have been notably difficult to address, and we are encouraged by the early evidence of our hematopoietic stem cell gene therapys approach to potentially treating this condition. The orphan drug and rare pediatric disease designations provide important momentum for the OTL-203 clinical program, which we remain committed to advancing as quickly as possible for patients in need.

The FDA grants orphan designation, also referred to as orphan status, to drugs intended for the treatment of rare diseases that affect fewer than 200,000 people in the US.1 This designation affords Orchard certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.2 Separately, rare pediatric disease designations are granted for rare diseases that primarily affect children under 18 years old with recipients of this designation being awarded a priority review voucher, upon approval.3 The priority review voucher may be redeemed, transferred, or sold.4

Orchard recently announced new interim data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203. The first primary outcome measure was met with all eight patients achieving hematologic engraftment. Additionally, improved motor skills compared to baseline, stable cognitive scores, and normal growth was seen in the first two patients with at least one year of follow-up. Orchard expects to release full proof-of-concept results and initiate the registrational study for OTL-203 in 2021.

About OTL-203 and MPS-I Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births. There are three subtypes of MPS-I; approximately 60 percent of children born with MPS-I have the most severe subtype, called Hurler syndrome, and rarely live past the age of 10 when untreated.

Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

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

Availability of Other Information About Orchard Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, and Orchards expectations regarding the timing of clinical trials and announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development and commercial programs; the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended March 31, 2020, as filed with the U.S. Securities and Exchange Commission (SEC) on May 7, 2020, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

___________________________________ 1 316 Orphan Drug Act & 316.20-21: Verification of orphan-drug status (https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=316.20)

2FDA Industry Guidance: Designating an Orphan Product: Drugs and Biological Products (https://www.fda.gov/industry/developing-products-rare-diseases-conditions/designating-orphan-product-drugs-and-biological-products)

3FDA Rare Pediatric Disease Designation Voucher Programs (https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs)

4360ff Title 21 Food and Drugs (https://www.govinfo.gov/content/pkg/USCODE-2012-title21/pdf/USCODE-2012-title21-chap9-subchapV-partB-sec360ff.pdf)

Contacts

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

Media Molly Cameron Manager, Corporate Communications +1 978-339-3378 media@orchard-tx.com

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Orchard Therapeutics Announces Orphan Drug and Rare Pediatric Disease Designations for OTL-203 for the Treatment of MPS-I - GlobeNewswire

Growing Infrastructure Development Projects in Asia-Pacific to Fuel Growth of the Animal Stem Cell Therapy Market 2017 2025 – Bulletin Line

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Growing Infrastructure Development Projects in Asia-Pacific to Fuel Growth of the Animal Stem Cell Therapy Market 2017 2025 - Bulletin Line