Category Archives: Regenerative Medicine

When it comes to the different ways of exercising the human body, walking is about as accessible as they come, and new research suggests it could be a powerful way to tackle osteoarthritis in the knees. A study examined the benefits of a regular saunter in people aged over 50, and found not only can it reduce pain, but it may also slow the damage that takes place in the joint.

The research was carried out by scientists at the Baylor College of Medicine and draws on a multi-year observational study of more than 1,200 subjects aged 50 or older with knee osteoarthritis, the most common form of arthritis. The subjects self-reported their walking habits, including the time spent walking and frequency, enabling the researchers to classify 73 percent of the cohort as "walkers" and the remainder as "non-walkers."

Those who walked for exercise had a 40 percent decrease in the likelihood of experiencing frequent knee pain. X-ray images were also used to assess the progression and severity of osteoarthritis in the knees, with the walkers less likely to experience medial joint space narrowing, a measure of arthritis. This suggests that a regular stroll could not just limit the pain associated with the condition, but apply the brakes to osteoarthritis in the knees.

"These findings are particularly useful for people who have radiographic evidence of osteoarthritis but dont have pain every day in their knees, said first author of the paper Dr. Grace Hsiao-Wei Lo.This study supports the possibility that walking for exercise can help to prevent the onset of daily knee pain. It might also slow down the worsening of damage inside the joint from osteoarthritis.

There is no cure or way of reversing osteoarthritis, and although treatments are available these predominantly focus on relieving pain and improving mobility, such as through medication and physical therapy. Exciting advances are being made in regenerative medicine that could one day see grafts help reinforce troubled joints and reduce inflammation, but studies such as this one demonstrate there are already useful tools at our disposal.

People diagnosed with knee osteoarthritis should walk for exercise, particularly if they do not have daily knee pain, advised Lo. "If you already have daily knee pain, there still might be a benefit, especially if you have the kind of arthritis where your knees are bow-legged.

The research was published in the journal Arthritis & Rheumatology.

Source: Baylor College of Medicine

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Walking found to reduce pain and slow damage in arthritic knees - New Atlas

BOSTON & ZUG, Switzerland & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announce presentation of new data on exa-cel, formerly known as CTX001, from CLIMB-111, CLIMB-121 and CLIMB-131 highlighting the potentially transformative profile of this investigational therapy for people with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD) and provided additional program updates.

New Data for exa-cel from CLIMB Clinical StudiesThe data presented at the European Hematology Association (EHA) Congress are from 75 patients (44 with TDT and 31 with SCD) with follow-up ranging from 1.2 to 37.2 months after exa-cel dosing.

Of the 44 patients with TDT, 26 had beta-zero/beta-zero or other beta-zero-like severe genotypes. Forty-two of 44 patients with TDT were transfusion-free with follow-up ranging from 1.2 to 37.2 months after exa-cel infusion. Two patients who were not yet transfusion-free had 75% and 89% reductions in transfusion volume. TDT patients had substantial mean increases in fetal hemoglobin (HbF) and corresponding increases in mean total hemoglobin (Hb) with mean total Hb levels increasing to >11 g/dL by Month 3 and maintained thereafter.

All 31 patients with severe SCD characterized by recurrent vaso-occlusive crises (VOCs) (mean of 3.9 VOCs per year over the prior two years) were free of VOCs after exa-cel infusion through duration of follow-up, with follow-up ranging from 2.0 to 32.3 months. SCD patients had mean HbF (as a proportion of total Hb) of approximately 40% by Month 4 and maintained thereafter.

The safety was generally consistent with myeloablative conditioning with busulfan and autologous stem cell transplant. All patients engrafted neutrophils and platelets after exa-cel infusion. Among the 44 patients with TDT, two patients had serious adverse events (SAEs) considered related to exa-cel. As previously reported, one patient had three SAEs considered related to exa-cel, hemophagocytic lymphohistiocytosis (HLH), acute respiratory distress syndrome and headache, and one SAE of idiopathic pneumonia syndrome that was considered related to both exa-cel and busulfan. All four SAEs occurred in the context of HLH and have resolved. One patient had SAEs of delayed neutrophil engraftment and thrombocytopenia, both of which were considered related to exa-cel and busulfan, and both SAEs have resolved. Among the 31 patients with SCD, there were no SAEs considered related to exa-cel.

Additional details were presented during the EHA media briefing and can be found in the published abstract and presentation.

Late-breaking abstract #LB2367 entitled Efficacy and Safety of a Single Dose of CTX001 For Transfusion-Dependent eta-Thalassemia and Severe Sickle Cell Disease, will be an oral presentation on Sunday, June 12 at 09:45-11:15 CEST.

These robust data from 75 patients, of which 33 have one year or more of follow-up after exa-cel infusion, further demonstrate the potential of this investigational therapy as a one-time functional cure for patients with transfusion-dependent beta thalassemia or severe sickle cell disease, said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

By reactivating a naturally occurring developmental process, exa-cel restores fetal hemoglobin production and thereby can ameliorate the course of these diseases, said Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares The Childrens Hospital at TriStar Centennial Medical Center. The remarkable results based on this approach give me great optimism and confidence in the potential of this treatment for patients.

I have seen first-hand the impact that this investigational therapy has had on patients in these clinical trials and continue to be impressed by the totality of the data, said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Sapienza University of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Ges Childrens Hospital. Given the urgency for highly effective and curative therapies for patients with hemoglobinopathies, I am excited to be part of the team working towards the goal of addressing this unmet need.

Exa-cel Study UpdatesFollowing ongoing discussions with regulators, the clinical trial protocols for CLIMB-111 and CLIMB-121 were amended to incorporate feedback on the primary endpoints for regulatory submission. Specifically, the primary endpoint in CLIMB-111 for TDT has been amended from proportion of subjects achieving transfusion reduction after exa-cel infusion to proportion of subjects maintaining weighted average Hb 9 g/dL without red blood cell (RBC) transfusions for at least 12 consecutive months after exa-cel infusion.

The primary endpoint in CLIMB-121 for SCD has been updated from proportion of subjects with HbF 20% after exa-cel infusion, to proportion of subjects who have not experienced any severe VOCs for at least 12 consecutive months after exa-cel infusion.

Both clinical trials are now in Phase 3 and are fully enrolled. All patients will have the opportunity to join CLIMB-131, a long-term follow-up study, after completing participation in the initial studies.

Additional Pediatric StudiesIn line with the companys strategy of developing therapies for patients of all ages, two additional Phase 3 studies of exa-cel have begun. Earlier this year, the Independent Data Monitoring Committee (DMC) met to review the data in adults and adolescents and endorsed expanding into younger pediatric patients. CLIMB-141 and CLIMB-151 are Phase 3 open-label trials designed to assess the safety and efficacy of a single dose of exa-cel in patients ages 2 to 11 years with TDT or SCD, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years and will plan to extend to patients 2 to less than 5 years of age at a later date. Each trial will enroll approximately 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a long-term follow-up trial.

Vertex also presented three additional abstracts on the burden of disease in sickle cell disease and beta thalassemia at the EHA Congress.

About exagamglogene autotemcel (exa-cel)Exacel, formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 geneedited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent VOCs, in which a patients own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygencarrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exacel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, exacel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among geneediting approaches being evaluated for TDT and SCD, exacel is the furthest advanced in clinical development.

About CLIMB111 and CLIMB121The ongoing Phase 1/2/3 openlabel trials, CLIMB111 and CLIMB121, are designed to assess the safety and efficacy of a single dose of exacel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exacel infusion. Each patient will be asked to participate in CLIMB131, a longterm followup trial.

About CLIMB-131This is a longterm, openlabel trial to evaluate the safety and efficacy of exacel in patients who received exacel in CLIMB111, CLIMB121, CLIMB141 or CLIMB151. The trial is designed to follow participants for up to 15 years after exacel infusion.

About CLIMB141 and CLIMB151The ongoing Phase 3 open-label trials, CLIMB141 and CLIMB151, are designed to assess the safety and efficacy of a single dose of exacel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to patients 2 to less than 5 years of age at a later date. Each trial will enroll approximately 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a longterm followup trial.

About the GeneEditing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, exacel, will then be infused back into the patient as part of an autologous hematopoietic stem cell transplant (HSCT), a process which involves a patient being treated with myeloablative busulfan conditioning. Patients undergoing HSCT may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of exacel. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of exacel on multiple measures of disease and for safety.

About the VertexCRISPR CollaborationVertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exacel represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialization of exacel and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1mediated kidney disease, pain, type 1 diabetes, alpha1 antitrypsin deficiency and Duchenne muscular dystrophy.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

(VRTX-GEN)

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, (i) statements by Dr. Carmen Bozic, Dr. Haydar Frangoul, and Dr. Franco Locatelli in this press release, (ii) our plans and expectations to present clinical data from the ongoing exa-cel clinical trials during the EHA Congress, (iii) the progress of the ongoing exa-cel clinical trials, including expectations regarding the abstracts that will be made available on the virtual platform including anticipated projections and estimates related to the various economic impacts of SCD and TDT, (iv) the potential benefits, efficacy, and safety of exa-cel, including the potentially transformative nature of the therapy and the potential of the treatment for patients, (v) our plans and expectations for our clinical trials and pipeline products, and (vi) the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites, patient enrollment, and expectations regarding clinical trial follow-up. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety and/or efficacy, or other reasons, that internal or external factors could delay, divert, or change our plans and objectives with respect to our research and development programs, that future competitive or other market factors may adversely affect the commercial potential for exa-cel, and other risks listed under the heading Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at http://www.vrtx.com and on the SECs website at http://www.sec.gov. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(CRSP-GEN)

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR THERAPEUTICS word mark and design logo and CTX001 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements made by Dr. Carmen Bozic, Dr. Haydar Frangoul, and Dr. Franco Locatelli in this press release, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: i) the safety, efficacy and clinical progress of the ongoing exa-cel clinical trials, including expectations regarding the abstracts that will be made available on the virtual platform and our plans and expectations to present and the clinical data that are being presented during the EHA Congress, as well as the potentially transformative nature of exa-cel and the potential of the treatment for patients; and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain, are neither promises nor guarantees and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with exa-cel at this time) not to be indicative of final or future trial results; the potential that the exa-cel clinical trial results may not be favorable or may not support registration or further development; that future competitive or other market factors may adversely affect the commercial potential for exa-cel; CRISPR Therapeutics may not realize the potential benefits of its collaboration with Vertex; potential impacts due to the coronavirus pandemic, such as to the timing and progress of clinical trials; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With exagamglogene autotemcel (exa-cel) at the 2022...

The new report by Expert Market Research titled, Global Live Cell Imaging Market Report and Forecast 2022-2027, gives an in-depth analysis of the global live cell imaging market, assessing the market based on its segments like product type, application, technology, and major regions. The report tracks the latest trends in the industry and studies their impact on the overall market. It also assesses the market dynamics, covering the key demand and price indicators, along with analysing the market based on the SWOT and Porters Five Forces models.

Request a free sample copy in PDF or view the report [emailprotected] https://bit.ly/3mtMGEU

The key highlights of the report include:

Market Overview (2017-2027)

As the number of stem cell research projects grows, so does the use of live cell imaging tools to analyse the location, purity, and amount of cells and their components, boosting market growth. The use of live cell imaging tools to precisely detect protein levels for optimal medication therapy is rising, as it is critical to determine the interaction between stem cells and tissues during stem cell research. The introduction of numerous government initiatives to support research and development (R&D) activities is fueling the live cell imaging industrys expansion. For example, in March 2020, the Canadian government announced a $6.9 million investment to promote stem cell research efforts in the country through the Stem Cell Networks research financing programme.

Furthermore, the increasing use of live cell imaging in the discovery of new medications is propelling the market forward. The development of new technologies that allow for the precise analysis of RNA, nucleic acid, proteins, and DNA, among other things, is driving demand for many diagnostic methods, moving the market forward. Furthermore, the rise in the prevalence of chronic diseases like cancer is driving up demand for live cell imaging in both diagnosis and treatment. The expanding research and development (R&D) activities to detect cancer cells in bone marrow while also allowing for the identification of specific cancer cells are likely to boost market growth.

Industry Definition and Major Segments

The study of living cells using microscope technology to obtain images of live cells and tissues is known as live cell imaging. It is essential in a variety of laboratory operations in biological and biomedical research because it gives real-time and reliable information on cells and tissues, making it suitable for stem cell research and regenerative medicine development.

Explore the full report with the table of [emailprotected] https://bit.ly/3tpEoSd

By technology, the market can be divided into:

The market can be categorised based on its applications into:

The major product types of live cell imaging are:

The regional markets include:

Market Trends

Artificial intelligence (AI), deep learning, and 3D printing are progressively being integrated into live cell imaging techniques, as technology improvements are a key antecedent of scientific research and development efforts. The expanding use of artificial intelligence (AI) allows for more precise, simpler, and time-efficient cell imaging. Furthermore, AI-based microscopy can recognise and analyse minor cell components like nuclei, allowing researchers to analyse data more quickly and effectively. AI-based microscopes also automate and optimise many functions for quantifying live cells, resulting in increased cell viability and faster image capture. This is fueling the expansion of the live cell imaging sector by increasing demand for such microscopes in research centres.

Furthermore, the increasing use of 3D printing in a variety of medical and biological applications is fueling market expansion. Because air bubbles are a common problem in perfusion chambers used in live cell imaging, the demand for fluidic devices made with 3D printing technology is increasing dramatically. Furthermore, the cost-effectiveness of 3D printing is increasing the affordability of live cell imaging research operations, which is propelling the market forward. In the forecast future, the development of portable and low-profile devices that can be directly installed on optical microscopes to improve cell imaging precision is expected to drive market expansion for live cell imaging.

Latest News on Global Live Cell Imaging [emailprotected] https://bit.ly/3HaaQ0z

Key Market Players

The major players in the market are Carl Zeiss AG, Leica Microsystems GmbH, Nikon Instruments Inc., Becton, Dickinson and Company, GE Healthcare and Others.

About Us:

Expert Market Research is a leading business intelligence firm, providing custom and syndicated market reports along with consultancy services for our clients. We serve a wide client base ranging from Fortune 1000 companies to small and medium enterprises. Our reports cover over 100 industries across established and emerging markets researched by our skilled analysts who track the latest economic, demographic, trade and market data globally.

At Expert Market Research, we tailor our approach according to our clients needs and preferences, providing them with valuable, actionable and up-to-date insights into the market, thus, helping them realize their optimum growth potential. We offer market intelligence across a range of industry verticals which include Pharmaceuticals, Food and Beverage, Technology, Retail, Chemical and Materials, Energy and Mining, Packaging and Agriculture.

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Global Live Cell Imaging Market to be Driven by Growing Stem Cell Research Market in the Forecast Period of 2022-2027 mbu timeline - mbu timeline

By Tom Piombino

For the cell and gene therapy (CGT) sector, recent years have been nothing less than phenomenal. For example, according to the Alliance for Regenerative Medicine, the sector has witnessed skyrocketing investment. The $7.5 billion raised in 2017 was more than tripled by the $22.7 billion raised in 2021.

Besides creating opportunities for CGT companies, such funding is creating a crunch for both real estate and talent. For example, in San Francisco, more than half a billion square feet of manufacturing space has been acquired by CGT companies in the past year. In Philadelphia, where academia is generating intellectual property in the CGT space at a torrid pace, more than one million square feet of new laboratory space is in development.

In North Carolinas Research Triangle regionwhere vacancy rates have kept per-square-foot development in the $30s range (as opposed to downtown Bostons $100/square foot)the competition for resources has placed a premium on selection and planning, as CGT programs shift from R&D, which occurs in science clusters, to manufacturing, which occurs in attractive suburban markets.

This shift is a huge challenge for CGT companies. The transition from research and preclinical development to process development and clinical manufacturing is rough. Some stakeholders underestimate the difficulties of taking their businesses to the manufacturing phase, and they end up treading water in a sea of limited options. While the pandemic significantly impacted the office market, many of the available and proposed building conversions cannot provide the rigor or meet the cost expectations of a manufacturing facility.

Site selection in todays market goes beyond engineering and real estate. It requires bringing knowledge and talent to appreciate the perspectives of more diverse workforces and company cultures while sustaining market growth, promotion, and compliance. From an organizations project inception, unity of the resources early in a planning process offers owners the perspectives needed to make the right decisions for their businesses while focusing on the big picture of bringing groundbreaking therapies to market. All too often, these decisions are predicated on immediate needs (2 years out) or currently available resources, instead of a vision of technological progress (10 years out).

Site selection can be daunting for the most seasoned real estate professionals, and even more so for people whose expertise is outside of the field. Selecting an attractive siteone with the proper structural capacity, heights, utilities, workforce availability, access to transportation, access to parking, and amenitiescan quickly become an all-consuming task for an operating company. Additionally, predicting the needs and wants of a workforce in a transitionary state, post-pandemic, adds another dimension of complexity for peoples whose day job is attending to developing science.

Much like an engineering design process, the building out of early-stage GCT facilities requires a deliberate approach with input from experts whose knowledge in various fields benefits the project as a whole. It must start at the business case inception, utilizing a well-rounded team that can streamline the process and thoroughly explore solutions while addressing needs and challenges across the organization. While speed is incredibly important at this stage, attempts at seeing this inceptionary process as a transaction often result in a misrepresentation of the required outcome.

Using applied experience and several iterations, an experienced project team with an understanding of the business case can recognize trends, similarities, and the opportunities to quickly adapt the solutions that can be acted upon in weeks rather than months. If this approach is taken from the inception of a project, owners can realize considerable benefits and avoid distractions from their day jobs.

First, it brings into focus a clear multiyear plan addressing the operational needs of the organization: Who will need to be hired for research, quality control, manufacturing, maintenance, packaging, and shipping, among other positions? How can an organization build its culture during the growth period? What kinds of efficiencies are needed in the facility? What impact will the operational costs have on funding resources? How does residual value of the asset impact the investor appetite in a facility?

Second, it brings more value to the effort by helping owners explore costs and schedules from the outset. If owners begin months later, during conceptual design, they may belatedly discover that they could have been better positioned to seize growth opportunities.

Planning for companies in a fast-growth period can be daunting, especially in a real estate environment as competitive as the one that currently exists. Company leaders versed in other aspects of the business such as R&D may not have a sense of how much space will be needed in the long term, or of how much effort will be needed to ensure a space meets requirements such as staying up to code.

Onboarding the project team early on can be key to making a project successful because it streamlines the iterative process. By working closely with owners at the inception of the project, team members learn what works for organizations and what does not, building upon the plan as it progresses.

It also develops trust and confidencetwo elements that are especially valuable when the team needs to be flexible in the exploratory phase of project planning. Not only do these elements empower team members to present multiple options, including unconventional options, it gives them the space to learn from feedback and converge on the right solution.

When evaluating options, organizations have to focus on important factors for each element of the plan:

Site: This is about more than the location of the facility. The site plays a key role in meeting the organizations current goals and serves its future growth. With those near-term and long-term goals prioritized, the team can address details such as the need to include multi-floor manufacturing facilities or loading docks capable of accommodating particular types of vehicles.

Also, not to be overlooked in site selection is customer access. Ensuring that an organizations key audience can easily access these facilitieswhether they are in urban areas, which may present traffic and parking difficulties, or in smaller markets, which may not be as easily accessible by commercial transportationwill be a factor in the decision-making process.

Space: The rapid growth in the CGT market puts space at the forefront of planning for small companies. How big does a new facility need to be? What features will need to be included? Can the current building stock in a market serve an organizations needs, or will a greenfield development be required? Much of the real estate market is developer-driven rather than company-driven, which has both benefits and challenges.

In a developer-driven project, a facility may be fitted out to serve as a manufacturing nexus for cell therapies, gene therapies, and biologics. Alternatively, a facility may meet a particular manufacturing need, once the developer determines the appropriate scale reviews a few solutions. While there are few standard solutions that fit all CGT companies, by taking standard ideas and measuring the options to the needs, organizations can streamline the process by tailoring elements to their requirements.

Brand: More than being about aesthetics, a facilitys appearance has the ability to tell an organizations story, from its culture to its future plans. While a warehouse can serve a company like Amazon quite effectively, for growing CGT companies wanting to attract top talent and top funders, the impression that a facility presents impacts the mission of the company as well as how an organization can differentiate itself from other companies in a competitive marketplace.

Expectations: There are a lot of companies that can get derailed early on in the process, as their expectations can deviate from reality.

For owners coming out of institutions that are not well placed, moving to a central location might put them in greater proximity to talent. It also could come with significant extra costs in rent, construction, and operations. Rents within regions, not justbetween regions, can swing significantly.

However, organizations also must be honest when considering the costs. For example, is $22/square foot for rent in the suburbs more cost effective compared to $55/square foot near a city core if it means sacrificing the ability to recruit talented people who refuse to work in the suburbs?

In addition, with space at a premium, can an organization live with a real estate strategy that might require a stepped approach to growth? In the rapidly expanding CGT markets, many companies moving from research to trials to manufacturing have compromised on facilities that are too small or in the wrong location, putting growth strategies at risk.

The demands on CGT companies are different from those on other companies. Even as biopharma players have moved into the market, the competition has only increased the difficulty of coping with the markets growth.

The move from research to preclinical trials to clinical manufacturing is occurring at such a pace that it is leaving some companies waiting at a crossroads without a complete strategy on moving forward. By engaging technical and subject matter experts as part of a project team early in the process, organizational leaders can make decisions that will position their organizations for years of growth.

Tom Piombino (tpiombino@ipsdb.com) is managing director of the Americas for IPSIntegrated Project Services. For more information on the Inceptioneering process, please visit http://www.ipsdb.com/expertise/services/inceptioneering.

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Overcoming the Challenges of Cell and Gene Therapy Facilities - Genetic Engineering & Biotechnology News

A ghost heart grown with scaffolding from a pigs organ and human cells may soon be used in human transplants, experts say.

A molecular biologist said that after seeing heart cells beat in unison in a petri dish, she can truly imagine building a personalized human heart.

Doris Taylor, director of regenerative medicine research at Texas Heart Institute, said that by using a patients own tissues, they were able to create a viable option with pig cells that the body will not reject.

It actually changed my life, Taylor toldCNN. I said to myself, Oh my gosh, thats life. I wanted to figure out the how and why, and re-create that to save lives.

Taylor said the innovative replacement could potentially become a planned procedure rather than a last-minute surgery.

That reduces your risk by eliminating the need for [antirejection] drugs, by using your own cells to build that heart it reduces the cost .. and you arent in the hospital as often so it improves your quality of life.

Taylor also presented a robot that was taught to administer the human stem cells into the ghost heart in a sterile environment.

The biologist showed a video of the translucent manufactured heart turning pink after it was injected.

Its the first shot at truly curing the number one killer of men, women, and children worldwide heart disease. And then I want to make it available to everyone, the scientist excitedly shared.

CEO of Advanced Solution Michael Golway applauded Taylors work and tenacity in the years-long project.

At any point, Dr. Taylor could have easily said Im done, this just isnt going to work,' Golway told CNN.

But she persisted for years, fighting setbacks to find the right type of cells in the right quantities and right conditions to enable those cells to be happy and grow.

Taylor first came interested in growing hearts when she worked on a team in 1998 at Duke University.

The team injected cells into arabbits failed heartand created new heart muscle.

However, whenever she attempted to translate the success onto human biology it was hit or miss.

We were putting cells into damaged or scarred regions of the heart and hoping that would overcome the existing damage, Taylor said.

I started thinking: What if we could get rid of that bad environment and rebuild the house?

In 2008, Taylor found real success when she and the team at the University of Minnesota rid a rats heart of cells and started working with the translucent skeleton left behind.

After this breakthrough, she moved into pigs hearts because of their anatomical similarity to humans.

Im just humbled and privileged to do this work, and proud of where we are, Taylor said.

The technology is ready. I hope everyone is going to be along with us for the ride because this is game-changing.

This story originally appeared on The Sun and was reproduced here with permission.

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Ghost heart grown with scaffolding from a pig's organ and patients cells - New York Post

--100% ORR at both dose levels; deep and durable responses observed in patients with poor prognostic factors --

-- 22 of 31 (71%) evaluable patients reached CR/sCR --

-- 13 of 16 patients (81%) dosed more than 12 months ago reached CR/sCR; 8 (50%) with EMD; 9 (56%) remain in ongoing response with a median follow up of 17.7 months --

-- No cases of Grade 3 CRS and no delayed neurotoxicity or parkinsonian-like events observed at RP2D (n=25) --

-- Phase 2 pivotal study on track to initiate by YE 2022 --

-- Management to host live webcast event on Sunday, June 5, 2022, at 7:00 p.m. CDT to discuss new positive CART-ddBCMA data with a panel of clinician experts --

FOSTER CITY, Calif., June 3, 2022 /PRNewswire/ -- Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, today announced new positive clinical data from the ongoing Phase 1 expansion study of its novel, autologous, CART-ddBCMA therapy for the treatment of patients with relapsed or refractory multiple myeloma. The clinical results are being presented during an oral presentation at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting.

Evaluable for efficacy and safety analysis were 31 patients, based on follow-up of at least one month, following treatment. These evaluable patients comprised the dose escalation cohorts for the first dose level (100 million CAR+ T cells, n=6), the second dose level (300 million CAR+T cells, n=6), and a dose expansion cohort at the recommended Phase 2 dose (RP2D) of 100 million CAR+T cells (n=19). All patients enrolled in the study have poor prognostic factors with 21 of 31 (68%) patients penta-refractory, 12 of 31 (39%) extramedullary disease (EMD), and all 31 patients having had at least three prior treatments.

The interim CART-ddBCMA clinical results (May 3, 2022 cutoff date) demonstrate deep and durable responses in patients with poor prognostic factors.

Matthew J. Frigault, M.D., CART-ddBCMA study investigator and Assistant Director of the Cellular Therapy Service at Mass General Cancer Center and Instructor at Harvard Medical School said, "The demand for clinically meaningful and safe CAR-T therapies outweighs what's currently available to multiple myeloma patients. It is encouraging to see these data continue to demonstrate deep responses and provide a benefit to patients. I look forward to enrolling patients in the Phase 2 pivotal study."

"We're excited by these long-term results, particularly given the challenging patient demographics, and believe these promising results reflect the potential for our lead program, CART-ddBCMA, to be a best-in-class treatment for patients with multiple myeloma," said Rami Elghandour, Arcellx's Chairman and Chief Executive Officer. "We believe there's a significant unmet need for cell therapies and we're committed to providing physicians with a safe and effective treatment option for multiple myeloma patients. We're honored to have our data presented at ASCO by Dr. Frigault and look forward to beginning enrollment in our Phase 2 pivotal study by the end of this year as the next step in the path towards regulatory approval."

The presentation can be accessed on the company's corporate website here.

Oral Presentation Details:

Title:Phase 1 Study of CART-ddBCMA in Relapsed or Refractory Multiple MyelomaSpeaker:Matthew J. Frigault, M.D., Assistant Director of the Cellular Therapy Service at Mass General Cancer Center, and Instructor at Harvard Medical SchoolSession Type/Title:Oral Abstract Session/Hematologic MalignanciesPlasma Cell DyscrasiaSession Date:Sunday, June 5, 2022Session Time: 8:00 a.m. 11:00 a.m. CDTLocation:McCormick Place Convention Center, Chicago, IllinoisAbstract Number:8003

Webcast Event:

Arcellx will host a live webcast event with an expert panel of clinicians to discuss the clinical results on Sunday, June 5, 2022, at 7:00 p.m. CDT. The event will be accessible from Arcellx's website atwww.arcellx.comin the Investors section. A replay of the webcast will be archived and available for 30 days following the event.

About Multiple Myeloma

Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient's immune function. Multiple myeloma is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About CART-ddBCMA

CART-ddBCMA is Arcellx's BCMA-specific CAR-modified T-cell therapy utilizing the company's novel BCMA-targeting binding domain for the treatment of patients with relapsed or refractory multiple myeloma. CART-ddBCMA is currently in a Phase 1 study. Arcellx's proprietary binding domains are novel synthetic proteins designed to bind specific therapeutic targets. CART-ddBCMA has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.

About Arcellx, Inc.

Arcellx, Inc. is a clinical-stage biotechnology company reimagining cell therapy by engineering innovative immunotherapies for patients with cancer and other incurable diseases. Arcellx believes that cell therapies are one of the forward pillars of medicine and Arcellx's mission is to advance humanity by developing cell therapies that are safer, more effective, and more broadly accessible. Arcellx's lead product candidate, CART-ddBCMA, is being developed for the treatment of relapsed or refractory multiple myeloma (r/r MM) in an ongoing Phase 1 study. CART-ddBCMA has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations by the U.S. Food and Drug Administration.

Arcellx is also advancing its dosable and controllable CAR-T therapy, ARC-SparX, through two programs: a Phase 1 study of ACLX-001 for r/r MM, initiated in the second quarter of 2022; and ACLX-002 in relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndrome, expected to enter the clinic in the second half of 2022.

Visitwww.arcellx.comfor more information.

Forward-looking StatementsThis press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements in this press release that are not purely historical are forward-looking statements. The forward-looking statements contained herein are based upon Arcellx's current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including those set forth in Part I, Item 1A (Risk Factors) of Arcellx's Annual Report on Form 10-K and in other reports, such as Quarterly Reports on Form 10-Q and Current Reports on Form 8-K, that Arcellx may file from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Arcellx assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Investor Contact:Myesha LacyArcellx, Inc.[emailprotected]510-418-2412

Media ContactLaura Morgan Sam Brown Inc.[emailprotected] 951-333-9110

SOURCE Arcellx, Inc

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Arcellx Presents Continued Robust Long-Term Responses from its CART-ddBCMA Phase 1 Expansion Trial in Patients with Relapsed or Refractory Multiple...

A revolutionary theoretical approach of aging that suggests that individuals might gradually turn feeble, following nearing their 70s; has opened the promise of novel medicines for age-related drop and illnesses.

Cambridge scientists concluded a mechanism that causes a fatal shift in hormone levels in old maturity level, raising the likelihood of bleeding malignancies and leukemia as well as reducing the ability of white blood cells to develop resistance, reported The Guardian.

Significant improvements, the experts claim, transpire in body parts across the system, from the epidermis to the nervous system, and might explain why individuals frequently mature decently for years prior to actually undergoing a relatively steep decrease in their 70s and 80s.

While according to Dr. Peter Campbell, corresponding author on the paper and director of the malignancy, ageing, and cancer stem cell project at Cambridge's Sanger Center, what was really intriguing with this research is that there could be a shared key mechanism at action. And the overall purpose is to halt or intervene in the aging phenomenon, although scientists foresee a potential to utilize this to quantify life expectancy.

Furthermore, aging is a complicated phenomenon, numerous researchers assume that the steady accumulation of alterations in tissues diminishes the body's natural capacity to operate effectively. The new study reveals such assumption is incorrect, or at finest partial, and mainly blames selfish genes that become dominant in old life.

Specialists analyzed leukocytes from infants through persons in their 70s and 80s in collaboration with academics from the Wellcome-MRC Cambridge Stem Cell Laboratory.

Researchers discovered that persons around the age of 65 had a diversified populace of 20,000 to 200,000 distinct varieties of progenitor cells in their brain tissue that generated a broad spectrum of red and white blood cells.

The scenario was drastically distinct among the over-65s. Nearly half of their lymphocytes were derived from a mere 10 or 20 unique regenerative medicine, significantly lowering the richness of the individual's blood cells, with negative effects for their wellness.

The authors discussed in Naturethat while stem cells associated with blood production accumulate alterations through history, the majority of these alterations are innocuous.

As per the website Time To Time, as an individual is in their 30s and 40s, the proliferation benefit of the mutant progenitor cells is negligible, however when they attain retirement point of 70, these fast-growing lymphocytes begin to predominate erythrocytes creation.

Also read: Activists Rescue Scared Dog From Meat Shop in China Ahead of the Yulin Festival

The huge increase answers why there is a rapid shift in morbidity beyond the maturity level of 70, why withering strikes at that period. Faster-growing rbcs have been related to blood malignancies and leukaemia, although they potentially render patients more susceptible to illness and experimental interventions like radiotherapy.

"Researchers recognize that all of these facts extend to other body systems," Campbell remarked. The group plans to hunt for the similar system in epidermis to comprehend why creases and delayed injury repair occur with maturity.

According to Dr. Elisa Laurenti, an associate lecturer at the Wellcome-MRC Cambridge Stem Cell Institute and a co-senior investigator on the investigation, systemic inflammatory, tobacco, disease, and medication all could create cancer-causing growth factors.

Researchers anticipate that these variables will similarly hasten the reduction in blood stem cell variety connected with aging.

It's probable that there are other things that could delay this machine down as well. Researchers also have the interesting challenge of determining how these recently found variations influence blood function in the seniors so that we may discover how to reduce illness injury and ensure overall health, as per Flipboard.

Related article: Hepatitis Outbreak in the US Linked to Contaminated Strawberries

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Aging to Your 70s and Above Makes a Catastrophic Change in Your Body, Says Research - Nature World News

BOSTON & ZUG, Switzerland & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) today announced new late-breaking clinical data accepted for oral presentation at the 2022 European Hematology Association (EHA) Congress. Vertex also announced three abstracts accepted for poster presentation at EHA.

Late-breaking abstract #LB2367 entitled Efficacy and Safety of A Single Dose of CTX001 For Transfusion-Dependent eta-Thalassemia and Severe Sickle Cell Disease, will be an oral presentation on Sunday, June 12 at 09:4511:15 CEST. The abstract from Vertex and CRISPR Therapeutics includes data on patients treated in CLIMB111 and CLIMB121 and followed in CLIMB131 with CTX001, now known as exagamglogene autotemcel (exa-cel). This abstract has been selected for the media briefing program and is therefore embargoed until Saturday, June 11 at 09:00 am CEST.

In addition, three real-world evidence and health economics abstracts from Vertex have been accepted for poster presentation.

The accepted abstracts are now available online on the EHA website.

Exacel is being investigated in multiple ongoing clinical trials as a potential one-time therapy for patients with either TDT or SCD.

About exagamglogene autotemcel (exa-cel)

Exacel, formerly known as CTX001, is an investigational, autologous, ex vivo CRISPR/Cas9 geneedited therapy that is being evaluated for patients with TDT or SCD characterized by recurrent VOCs, in which a patients own hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygencarrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. The elevation of HbF by exacel has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, exacel has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. Exa-cel has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among geneediting approaches being evaluated for TDT and SCD, exacel is the furthest advanced in clinical development.

About CLIMB111 and CLIMB121

The ongoing Phase 1/2/3 openlabel trials, CLIMB111 and CLIMB121, are designed to assess the safety and efficacy of a single dose of exacel in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after exacel infusion. Each patient will be asked to participate in CLIMB131, a longterm followup trial.

About CLIMB-131

This is a longterm, openlabel trial to evaluate the safety and efficacy of exacel in patients who received exacel in CLIMB111, CLIMB121, CLIMB141 or CLIMB151. The trial is designed to follow participants for up to 15 years after exacel infusion.

About CLIMB141 and CLIMB151

The ongoing Phase 3 open-label trials, CLIMB141 and CLIMB151, are designed to assess the safety and efficacy of a single dose of exacel in patients ages 2 to 11 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now open for enrollment and currently enrolling patients ages 5 to 11 years of age and will plan to extend to ages 2 to less than 5 years of age at a later date. Each trial will enroll up to 12 patients. Patients will be followed for approximately two years after infusion. Each patient will be asked to participate in CLIMB-131, a longterm followup trial.

About the GeneEditing Process in These Trials

Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, exacel, will then be infused back into the patient as part of an autologous hematopoietic stem cell transplant (HSCT), a process which involves a patient being treated with myeloablative busulfan conditioning. Patients undergoing HSCT may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of exacel. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of exacel on multiple measures of disease and for safety.

About the VertexCRISPR Collaboration

Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. Exacel represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing and commercialization of exacel and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1mediated kidney disease, pain, type 1 diabetes, alpha1 antitrypsin deficiency and Duchenne muscular dystrophy.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

(VRTX-GEN)

Vertex Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, our plans and expectations to present clinical data from the ongoing exa-cel clinical trials during the EHA Congress, expectations regarding the abstracts that will be made available on the virtual platform and the clinical data that will be presented during the EHA Congress, including anticipated projections and estimates related to the various economic impacts of SCD and TDT, the potential benefits, efficacy, and safety of exa-cel, including the potentially transformative nature of the therapy and the potential of the treatment for patients, our plans and expectations for our clinical trials and pipeline products, the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites, patient enrollment and expectations regarding clinical trial follow-up. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety and/or efficacy, or other reasons, that internal or external factors that could delay, divert, or change our plans and objectives with respect to our research and development programs, that future competitive or other market factors may adversely affect the commercial potential for exa-cel, and other risks listed under the heading Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at http://www.vrtx.com and on the SECs website at http://www.sec.gov. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(CRSP-GEN)

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement

This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: i) the safety, efficacy and clinical progress of the ongoing exa-cel clinical trials, including expectations regarding the abstract that will be made available on the virtual platform and our plans to present and the clinical data that are being presented during the EHA Congress; and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain, are neither promises nor guarantees and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with exa-cel at this time) not to be indicative of final or future trial results; the potential that the exa-cel clinical trial results may not be favorable or may not support registration or further development; that future competitive or other market factors may adversely affect the commercial potential for exa-cel; CRISPR Therapeutics may not realize the potential benefits of its collaboration with Vertex; potential impacts due to the coronavirus pandemic, such as to the timing and progress of clinical trials; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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Vertex and CRISPR Therapeutics Announce Acceptance of Late-Breaking Abstract for CTX001 at the 2022 Annual European Hematology Association (EHA)...

CAMBRIDGE, Mass., June 2, 2022 /PRNewswire/ -- Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"), a development-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programing platform, today announced that Mahesh Karande, President and CEO, will participate in fireside chats at the following upcoming investor conferences in June.

Jefferies Healthcare Conference Date: June 10, 2022Time: 9:30 a.m. ET

Goldman Sachs 43rd Annual Global Healthcare ConferenceDate: June 15, 2022Time: 5:40 p.m. ET / 2:40 p.m. PT

Live webcasts of the fireside chats will be available on the Investors & Media section of the Company's website at http://www.omegatherapeutics.com. An archived replay of the fireside chats will be available on the same website for approximately 90 days.

About Omega Therapeutics

Omega Therapeutics, founded by Flagship Pioneering, is a development-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as a new class of programmable epigenetic medicines. The company's OMEGA Epigenomic Programming platform harnesses the power of epigenetics, the mechanism that controls gene expression and every aspect of an organism's life from cell genesis, growth, and differentiation to cell death. Using a suite of technologies, paired with Omega's process of systematic, rational, and integrative drug design, the deterministic OMEGA platform enables control of fundamental epigenetic processes to correct the root cause of disease by returning aberrant gene expression to a normal range without altering native nucleic acid sequences. Omega's modular and programmable mRNA epigenetic medicines, Omega Epigenomic Controllers, target specific epigenomic loci within insulated genomic domains, EpiZips, from amongst thousands of unique, mapped, and validated genome-wide DNA-sequences, with high specificity to durably tune single or multiple genes to treat and cure diseases through Precision Genomic Control. Omega is currently advancing a broad pipeline of development candidates spanning a range of disease areas, including oncology, regenerative medicine, multigenic diseases including immunology, and select monogenic diseases.

For more information, visitomegatherapeutics.com, or follow us onTwitterandLinkedIn.

Contacts

Media contact: Jason BracoLifeSci Communications646.751.4361[emailprotected]

Investor contact: Kevin MurphyArgot Partners212.600.1902[emailprotected]

SOURCE Omega Therapeutics

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Omega Therapeutics to Participate in the Jefferies Healthcare Conference and Goldman Sachs 43rd Annual Global Healthcare Conference in June 2022 - PR...

Established through a generous $20-million gift from the Larry and Judy Tanenbaum Family Foundation, the Tanenbaum Institute for Science in Sport at the University of Toronto will be a global centre of excellence for high-performance sport science and sports medicine.

The Tanenbaum Institute will yield new knowledge at the intersection of research and practice, translating discoveries into innovations that dramatically impact health and performance across all athlete populations.

The Tanenbaum Institute will bring together the leading sport science research of U of Ts Faculty of Kinesiology & Physical Education, the sports medicine research expertise of the Temerty Faculty of Medicine, and the renowned clinical and research leadership of the Dovigi Orthopaedic Sports Medicine Clinic and the Lunenfeld-Tanenbaum Research Institute at Sinai Health.

Today marks a monumental step forward in support of Canadian high-performance athletics, one that will lead to improved athlete performance, safety and well-being, said U of T President Meric Gertler. Thanks to the extraordinary generosity of Larry and Judy Tanenbaum, the Tanenbaum Institute for Science in Sport will become one of the worlds leading centres in the field. And the Institute will be truly unique, combining the strengths of U of Ts top-ranked research programs and sports medicine departments with leading clinical care centres at Sinai Health, all in the heart of one of the worlds most celebrated sporting cities.

The Tanenbaum Institute for Science in Sport will help model and predict athlete performance and improve health outcomes based on a wealth of data from across the Greater Toronto Area. This new knowledge will support high-performance athletes across a spectrum that includes world-class professional, non-professional and para athletes, including from diverse and underrepresented communities, as well as athletes striving for high-performance optimization in recreational sports.

The institute will catalyze U of T and Sinai Healths sport science and sports medicine expertise, generating novel insights and innovative technologies and interventions that improve athlete performance, health, safety and well-being; reduce risk of injury; accelerate and optimize recovery and rehabilitation; and advance high-performance sport in a manner that is safe, welcoming, inclusive and accessible to all.

To this end, the Tanenbaum Institute will work in partnership with sports clinics, associations and organizations, including Maple Leaf Sports & Entertainment (MLSE) and its teams: the Toronto Maple Leafs, Toronto Raptors, Toronto FC and the Toronto Argonauts, as well as the Toronto Marlies, Raptors 905 and TFC 2.

I truly believe that sport unites us, inspires us, and offers all people a path toward becoming their best selves, said Larry Tanenbaum, chairman of Tanenbaum Family Foundation and MLSE. The Tanenbaum Institute will bring together sports medicine, sport science and data science to encourage athletic engagement, enhance performance and accelerate recovery and rehabilitation. Im proud to join with U of T and Sinai Health in transforming athlete health and well-being.

Larry and Judy Tanenbaums gift will be combined with more than $20 million in additional support from U of T and Sinai Health. This investment will establish a Directorship and Research Acceleration Fund to support bold, innovative research across the institute, the university, and Sinai Health; create a groundbreaking new Chair in Sport Science and Data Modelling, a Chair in Musculoskeletal Regenerative Medicine, and a Professorship in Orthopaedic Sports Medicine; and provide funding for a range of cutting-edge research, innovations and clinical programs.

The Tanenbaum Institute will enjoy a remarkable head start, thanks to the amazing research and clinical sports medicine leadership we have amassed here at Sinai Health through the Dovigi Orthopaedic Sports Medicine Clinic and across U of T, said Dr. Gary Newton, president and CEO of Sinai Health. Establishing this landmark Institute is only the beginning. We look forward to transforming high-performance sport together with our many industry, government, and community partners.

The Tanenbaum Institutes cutting-edge research will play a leading role in advancing high performance sport in a manner that is safe, welcoming, inclusive and accessible to all, said Gretchen Kerr, dean of the Faculty of Kinesiology & Physical Education at U of T. We are so excited to be joining in this important research enterprise by pooling together our academic research, large and diverse athlete base, and training facilities with the world-class clinicians of Sinai Health.

Were incredibly excited by the potential for the Tanenbaum Institute to transform sports medicine across Canada and to train future generations of sport science and sports medicine leaders, said Trevor Young, dean of the Temerty Faculty of Medicine at U of T. By bringing together so many disciplines, the Tanenbaum Institute will make breakthrough big data-driven findings that will lead to better athlete health, safety and performance.

The Institute combines a diverse array of sport science and sports medicine talent. The Institutes research and clinical foci will include mild traumatic brain injuries, orthopaedics, regenerative medicine, biomechanics, wearable physiological and training monitoring technologies, technologies in parasport, mathematical and statistical modelling applied to individual athlete and team analytics, nutrition, individual and team psychology and health, exercise physiology and more.

This latest gift from the Tanenbaum Family Foundation builds on an impressive philanthropic legacy at U of T, Sinai Health and beyond. Larry and Judy Tanenbaum and the Tanenbaum family have been long-time supporters of U of T. In 2014, they helped establish the Anne Tanenbaum Centre for Jewish Studies at the Faculty of Arts & Science, one of North Americas leading programs of its kind. They also have also established several scholarships in support of student athletes.

At Sinai Health, Larry and Judy Tanenbaum have made several transformative investments. In 2013, the Tanenbaums gave $35 million to rename the Lunenfeld-Tanenbaum Research Institute (LTRI), accelerating Sinai Healths biomedical research institute.

Larry and Judy Tanenbaum have also made major gifts in support of cutting-edge physical and mental health research across Canada. Their generosity led to the creation of the Tanenbaum Open Science Initiative at McGill University and the Tanenbaum Centre for Pharmacogenetics at the Centre for Addiction and Mental Health.

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U of T and Sinai Health announce new gift from Larry and Judy Tanenbaum to establish the Tanenbaum Institute for Science in Sport - University of...