BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND, enabling the company to proceed with initiating a clinical trial for VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell therapy to treat T1D. Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2021 in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.

As we celebrate the 100th anniversary of the discovery of insulin this year, we are excited to bring a first-in-class cell therapy to the clinic with the potential to meaningfully impact people living with T1D, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We look forward to getting our clinical program underway and testing our unique approach of replacing pancreatic islet cells, which are destroyed in people with type 1 diabetes, with our stem cell-derived fully differentiated insulin-producing pancreatic islet cells.

About VX-880VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy that is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the bodys ability to regulate glucose levels by restoring pancreatic islet cell function, including insulin production.

The VX-880 clinical trial will involve an infusion of fully differentiated, functional islet cells, as well as the chronic administration of concomitant immunosuppressive therapy, to protect the islet cells from immune rejection.

About the Phase 1/2 Clinical TrialThe clinical trial is a Phase 1/2, single-arm, open-label study in subjects who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This will be a sequential, multi-part clinical trial to evaluate the safety and efficacy of different doses of VX-880. Approximately 17 patients will be enrolled in the clinical trial.

About Type 1 DiabetesT1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death. Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in patients with T1D. Hypoglycemia remains a critical limiting factor in glycemic management, and severe hypoglycemia can cause loss of consciousness, coma, seizures, injury, and can be fatal.

There are currently limited treatment options beyond insulin for the management of T1D.

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 medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. 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.

Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Bastiano Sanna, Ph.D., in this press release, statements regarding the development, plans and expectations for our T1D pipeline program, including our plans to initiate a Phase 1/2 clinical trial in people with T1D and expected timeline of our clinical trials, statements regarding patient enrollment and dosing, statements regarding potential clinical trial results and anticipated benefits of VX-880, and our plans to provide further updates on our T1D pipeline program. 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 the FDA may not approve our IND, 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 may not support registration or further development due to safety, efficacy or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trials, 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 at http://www.sec.gov and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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Vertex Announces FDA Clearance of Investigational New Drug (IND) Application for VX-880, a Novel Cell Therapy for the Treatment of Type 1 Diabetes...

Since the approval of the first five gene or gene-modified cell therapies in the US Luxturna, Yescarta, Tecartus, Kymriah and Zolgensma this new technology has been evolving rapidly and more recent approval applications have faced challenges.

The FDAs requirement on Biomarin to provide an additional two years of clinical data of Valrox before approval signals an increase in regulatory scrutiny for the regenerative medicine industry.

Other safety and manufacturing events such as three patient deaths in the high-dose arm of the Audentes ASPIRO trial and an FDA refusal to review the biologics licence application for Bristol Myers Squibb and bluebird bios idecabtagene vicleucel CAR-T therapy further emphasise this point.

A crucial challenge faced by cell and gene therapy developers is the necessity to balance clinical considerations with logistical ones such as manufacturing.

Naturally, clinical considerations will always surpass logistical ones, but it is not so simple. Thats why manufacturers must guarantee the development of drug manufacturing processes that are vigorously in compliance with all relevant FDA guidance.

An example issue that the industry is facing is that drug developers are adopting different approaches to produce recombinant AAVs (rAAVs). The majority of AAV gene therapies in clinical trials are produced using transient transfection.

However, there is a challenge in scaling up such procedures, as it is not easy to maintain transient transfection consistency from batch to batch, especially at scales over 500 litres.

Thats why developers have started exploring other techniques, for example using a baculovirus with an insect producer cell, as done by BioMarin to produce its gene therapy for haemophilia.

While this provided greater scalability, using different platforms to produce AAVs could have clinical implications.When comparing rAAVs produced using transiently transfected human cells with baculovirus infected insect cells, alterations in several functionally relevant AAV characteristics are exposed, including post-translational modifications, such as glycosylation, acetylation, phosphorylation and methylation.

It is vital that any baculoviruses used to produce the AAVs are entirely removed from the final product, as any such contamination would have very adverse effects in patients. Therefore, while one may achieve higher production, the incorporation of other manufacturing steps can add complication, and therapeutic dose may also need to increase.

At the same time, any adventitious viruses that could infect the producer cells must also be removed from the final product. Although no patient has contracted a viral infection from a monoclonal antibody or recombinant protein drug to date, this is because of the vigorous procedures in place to remove the unwelcome viruses.

At the same time, cell and gene therapies that utilise AAVs and lentiviruses could face difficulty employing these processes, as it would destroy the viral vector that is either the final drug product or crucial to producing it.

Drug developers are therefore turning to other methods of adventitious virus control such as pass all incoming process liquids through virus filters. These kinds of processes will only become more important in a climate of increasing regulatory examination.

These virus filters use a membrane barrier to remove virus particles. Its a size-based removal technique using a specially designed polymeric membrane to keep virus particles on the surface and within the pores of the membrane. The addition of viral filters to incoming media streams could help deliver a virus-control strategy that is suitable for these kinds of therapeutics.

The first cell and gene therapies were developed to treat rare and fatal diseases, which is why patients were possibly more likely to accept higher-risk treatment options.

But todays cell and gene therapies are being produced for more common diseases, to act as therapies that go beyond the traditional approach to disease treatment.For that reason, we must create manufacturing processes for cell and gene therapies that arent just rapidly scalable but also guarantee maximum patient safety.

Pall is a global supplier of filtration, separations and purification products for drug developers.

Clive Glover is Director, Strategy at Pall Corporation

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Keeping up with the developing regulatory requirements for gene and cell therapies - PMLiVE

A Cambridge UK-Korea joint venture promising great things in nextgen cell and gene therapy technology has been rewarded with major cash backing in a Series A round.

Avactas JV with Daewoong Pharmaceutical AffyXell Therapeutics has secured $7.3 million to further develop its pipeline of next generation cell and gene therapies.

AffyXell was established in January 2020 to develop novel mesenchymal stem cell therapies. The business is combining Avactas Affimer platform with Daewoongs MSC platform such that the stem cells are genetically modified to produce and secrete therapeutic Affimer proteins in situ in the patient.

The Affimer proteins are designed to enhance the therapeutic effects of the MSC creating a novel, next generation cell therapy platform.

The Series A funding has been raised from a group of venture funds including Samsung Venture Investment Corporation, Shinhan Venture Investment, Smilegate Investment, Shinhan Investment Corporation, Kolon Investment, Stonebridge Ventures and Gyeongnam Venture Investment.

The proceeds will be used by AffyXell to further the development of MSCs engineered to produce Affimer molecules generated by Avacta that suppress immune response and restore immune balance.

While initially focusing on inflammatory and autoimmune diseases and prevention of organ transplant rejection, longer term goals could also include applications in regenerative medicine, infectious diseases and oncology.

Avacta's R & D costs associated with the generation of the Affimer proteins are funded by AffyXell whilst Avacta retains the rights to commercialise the Affimer proteins outside the field of cell therapies.

Avacta CEO Dr Alastair Smith said: The potential for AffyXells new class of MSC therapies to deliver improved treatments for a wide range of inflammatory and autoimmune diseases is significant, in a market estimated to be worth $16 billionn by 2025.

We expect these novel engineered MSCs to show a more powerful therapeutic effect than existing antibodies and stem cells and they therefore have the potential to lead the rapidly growing field of cell and gene therapy.

AffyXell is uniquely positioned to develop novel and powerful cell therapies through the combination of two world-class technologies: Avactas Affimer platform and Daewoongs proprietary technology for generating off-the-shelf allogeneic MSC therapies.

Completion of the Series A funding is a strong validation of this concept and moves us closer to providing these new therapies to the patients who need them.

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Avacta JV raises $7.3m for cell and gene therapy push | Business Weekly - Business Weekly

Quote from Andrew Winner, Product Manager, Lonza Bioscience:"The short viability window of fresh Leukopaks means researchers are at the mercy of donor and shipping schedules and any logistical delays can wreak havoc on project costs and the quality of research results. By offering cryopreserved Leukopaks, we are expanding our ability to deliver high-viability cell products internationally, and the rapid availability of stored cryopreserved Leukopaks means our customers are better able to adhere to uncompromising research timelines."

Basel, Switzerland, 28 January 2021 Lonza has expanded its renowned human primary cell offerings with the launch of fully customizable, high-quality cryopreserved Leukopaks. The frozen Leukopaks will enable long-distance shipping of leukapheresis products without the concern of reduced cell viability encountered with fresh Leukopaks. Being suitable for long-term storage in research labs, the cryopreserved Leukopaks will also allow immediate access to viable cells for greater convenience and workflow flexibility. The cryopreserved Leukopaks come in a range of sizes, and multiple donor characteristics and testing options are available through a unique costing structure that allows customers to only pay for the customization that they need.

A Leukopak is an enriched leukapheresis-derived product containing high concentrations of peripheral blood mononuclear cells like T cells, B cells and monocytes. Such cells are a critical raw material in immunotherapy research and for optimizing cell therapy process development before progressing to full clinical manufacture. However, fresh Leukopaks can be hard to access and must be used rapidly to avoid cell degradation. International transportation options are thus severely limited, and logistical delays or donor cancellations can have catastrophic impacts on research costs and quality. Cryopreserved Leukopaks allow reliable global shipping while maintaining cell viability and functionality, and the ability to thaw cryopreserved Leukopaks when needed means researchers are better able to plan ahead for more cost-efficient therapy development.

Lonzas cryopreserved Leukopaks are available in a range of sizes, including packs of 2.5, 5 and 9.5 billion cells, which can be subdivided into separate smaller bags for greater convenience. Specific donor characteristics like age, gender and Human Leukocyte Antigen (HLA) type are also available, with a wide range of recallable donors and several product testing options. Customization follows a unique, tailored pricing structure, where customers only pay for the customization they require. Customers will also have access to Lonzas globally renowned technical support services to facilitate optimized product usage and greater research success.

To find out more about Lonzas cryopreserved Leukopak offerings, please clickhere.

About LonzaLonza is the preferred global partner to the pharmaceutical, biotech and nutrition markets. We work to prevent illness and enable a healthier world by supporting our customers to deliver new and innovative medicines that help treat a wide range of diseases. We achieve this by combining technological insight with world-class manufacturing, scientific expertise and process excellence. These enable our customers to commercialize their discoveries and innovations in the healthcare sector.

Founded in 1897 in the Swiss Alps, today Lonza operates across three continents. With approximately 14,000 full-time employees, we are built from high-performing teams and of individual talent who make a meaningful difference to our own business, as well as to the communities in which we operate. The company generated sales of CHF 4.5 billion in 2020 with a CORE EBITDA of CHF 1.4 billion. Find out more atwww.lonza.com

Follow @Lonza onLinkedInFollow @LonzaGroup onTwitter

Lonza Contact Details

Dr. Sanna FowlerHead of External CommunicationsLonza Group LtdTel +41 61 316 8929sanna.fowler@lonza.com

Dirk OehlersInvestor RelationsLonza Group LtdTel +41 79 421 1609dirk.oehlers@lonza.com

Additional Information and DisclaimerLonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited ("SGX-ST"). Lonza Group Ltd is not subject to the SGX-STs continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

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Lonza Announces High-Quality Cryopreserved Leukopaks for More Flexibility in Immunology and Cell Therapy Research - BioSpace

The Stem Cell Therapy for Osteoarthritis Market grew in 2019, as compared to 2018, according to our report, Stem Cell Therapy for Osteoarthritis Market is likely to have subdued growth in 2020 due to weak demand on account of reduced industry spending post Covid-19 outbreak. Further, Stem Cell Therapy for Osteoarthritis Market will begin picking up momentum gradually from 2021 onwards and grow at a healthy CAGR between 2021-2025.

Deep analysis about Stem Cell Therapy for Osteoarthritis Market status (2016-2019), competition pattern, advantages and disadvantages of products, industry development trends (2019-2025), regional industrial layout characteristics and macroeconomic policies, industrial policy has also been included. From raw materials to downstream buyers of this industry have been analysed scientifically. This report will help you to establish comprehensive overview of the Stem Cell Therapy for Osteoarthritis Market

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Key product type:MonotherapyCombination Therapy

Key applications:Osteoarthritis (unspecified)Knee OsteoarthritisShoulder OsteoarthritisHip Osteoarthritis

Key players or companies covered are:MesoblastRegeneusU.S. Stem CellAnterogenAsterias Biotherapeutics

The report provides analysis & data at a regional level (North America, Europe, Asia Pacific, Middle East & Africa , Rest of the world) & Country level (13 key countries The U.S, Canada, Germany, France, UK, Italy, China, Japan, India, Middle East, Africa, South America)

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Key questions answered in the report:1. What is the current size of the Stem Cell Therapy for Osteoarthritis Market, at a global, regional & country level?2. How is the market segmented, who are the key end user segments?3. What are the key drivers, challenges & trends that is likely to impact businesses in the Stem Cell Therapy for Osteoarthritis Market?4. What is the likely market forecast & how will be Stem Cell Therapy for Osteoarthritis Market impacted?5. What is the competitive landscape, who are the key players?6. What are some of the recent M&A, PE / VC deals that have happened in the Stem Cell Therapy for Osteoarthritis Market?

The report also analysis the impact of COVID 19 based on a scenario-based modelling. This provides a clear view of how has COVID impacted the growth cycle & when is the likely recovery of the industry is expected to pre-covid levels.

Contact us:i2iResearch info to intelligenceLocational Office: *India, *United States, *GermanyEmail: [emailprotected]Toll-free: +1-800-419-8865 | Phone: +91 98801 53667

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Stem Cell Therapy for Osteoarthritis Market Research Report 2021: Market Competition Trend and Price by Manufacturers till 2026 with key players...

WATERTOWN, Mass.--(BUSINESS WIRE)--SQZ Biotechnologies (NYSE: SQZ), a cell therapy company developing novel treatments for multiple therapeutic areas, today announced that the companys Investigational New Drug (IND) application for SQZTM Activating Antigen Carriers (SQZ AACs) in HPV+ tumors was cleared by the U.S. Food and Drug Administration (FDA). The clinical trial will investigate SQZ-AAC-HPV, a cell therapy candidate generated from red blood cells (RBCs) engineered with tumor-specific antigen to treat HPV+ tumors. This trial, SQZ-AAC-HPV-101, marks the first clinical program from the companys wholly-owned SQZ AAC platform.

SQZ AACs are a novel cellular immunotherapy candidate designed to transport tumor-specific antigen and TLR agonists to the patients endogenous, professional, antigen presenting cells in vivo. These antigen presenting cells are capable of potent T cell activation that could potentially drive an anti-tumor effect. In preclinical studies, SQZ AACs in mouse models have demonstrated robust immune responses, CD8 T cell infiltration, and correlated tumor reduction.

Advancing our SQZ AAC program into the clinic is a significant milestone for our team, said Armon Sharei, PhD, founder and chief executive officer of SQZ Biotechnologies. This program further illustrates the broad cell engineering capabilities of our core technology and the diversity of our pipeline. SQZ AACs potentially open another dimension of biology where SQZ cell therapy candidates could drive patient impact.

The Phase 1 multi-center trial will enroll multiple cohorts to assess SQZ-AAC-HPV as both monotherapy and in combination with other immunoncology therapies. HLA-A*02+ patients with recurrent, locally advanced or metastatic HPV16+ head & neck, cervical, anal, penile, vulval and vaginal cancers are all eligible for the study.

We are dedicated to leveraging our unique capabilities and novel cell therapy candidates to try to improve patients lives by offering them potential outcomes that they need and deserve. SQZ AACs are our next approach for a potential differentiated cell therapy targeting solid tumors that could represent an exciting evolution in the cancer patient experience, said Oliver Rosen, MD, chief medical officer.

About SQZ-AAC-HPV

SQZ AACs are generated by squeezing red blood cells (RBCs) with an antigen and activating adjuvant. The process is tuned to make the engineered RBCs appear aged. Once administered to patients, SQZ AACs aim to be rapidly taken up by professional antigen presenting cells through a natural process to destroy aged RBCs in the body known as eryptosis. To take advantage of this process, SQZ AACs are designed to act as a Trojan horse to deliver significant quantities of antigen and activation factors to the professional, endogenous antigen presenting cells in the lymphoid organs and drive subsequent activation of T cells specific to HPV+-tumors. SQZ-AAC-HPV is the first product candidate from the SQZ AAC platform.

About SQZ Biotechnologies

SQZ Biotechnologies is a clinical-stage biotechnology company developing transformative cell therapies for patients with cancer, infectious diseases, and other serious conditions. Using its proprietary technology, SQZ Biotechnologies offers the unique ability to deliver multiple materials into many patient cell types to engineer what we believe can be an unprecedented range of potential therapeutics for a variety of diseases. SQZ Biotechnologies has the potential to create well-tolerated cell therapies that can provide therapeutic benefit for patients and to improve the patient experience over existing cell therapy approaches. With accelerated production timelines under 24 hours and the opportunity to eliminate preconditioning and lengthy hospital stays, our goal is to use the SQZ approach to establish a new paradigm for cell therapies. Our first therapeutic applications aim to leverage the potential to generate target-specific immune responses, both in activation for the treatment of solid tumors and immune tolerance for the treatment of unwanted immune reactions and autoimmune diseases. For more information please visit http://www.sqzbiotech.com.

Forward Looking Statement

This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained that do not relate to matters of historical fact should be considered forward-looking statements, including statements relating to upcoming events and presentations, our product candidates, preclinical and clinical activities, regulatory requirements, clinical efficacy and therapeutic impact. These forward-looking statements are based on management's current expectations. The words may, will, should, expect, plan, anticipate, could, intend, target, project, estimate, believe, predict, potential or continue or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

These and other important factors discussed under the caption "Risk Factors" in our Form 10Q filed with the U.S. Securities and Exchange Commission (SEC) on December 10, 2020 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements. Any forward-looking statements represent management's estimates as of this date. New risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. While we may elect to update forward-looking statements in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct.

Certain information contained in this press release relates to or is based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and research. While we believe these third-party sources to be reliable as of the date of this press release, we have not independently verified, and we make no representation as to the adequacy, fairness, accuracy or completeness of any information obtained from third-party sources.

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SQZ Biotechnologies Announces FDA Clearance of IND Application to Allow for Clinical Trial with SQZ Activating Antigen Carriers (SQZ AACs) in Patients...