StemCell Therapy

Critical considerations in ensuring patient access to cell and gene therapies, and continued industry growth, include proper understanding of how to manage source material quality and supply chain continuity. The following areas will be covered:

The importance of defining source material requirements for translation of allogeneic advanced therapies The value of a reliable and recallable donor network for consistent source material access The critical requirements for obtaining GMP-compliant source materials for commercial manufacture Best practice in handling, monitoring and delivering temperature-sensitive source materials The importance of early planning, coordination and communication with all stakeholders

The webinar will take place on 12 March 2020 at 9:00 PDT / 12:00 EDT / 16:00 GMT / 17:00 CET.

Biocairs industry-leading service offering for cell and gene therapy logistics includes:

Dedicated project management teams including customer support specialists A global network of liquid nitrogen service stations 24/7 collection and delivery across the world A level of flexibility in service provision unrivalled in the industry

To register for the webinar visit http://www.biocair.com/events or contact marketing@biocair.com for more information.

Image: The webinar will be hosted by BIOINSIGHTS

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Biocair participates in cell and gene webinar alongside HemaCare and Beam Therapeutics - Cambridge Network

NEW YORK, Feb. 6, 2020 /PRNewswire/ --

Gene Therapy Market by Vector Type (Viral Vector and Non-viral Vector), Gene Type (Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others), and Application (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Disease, and Other Diseases): Global Opportunity Analysis and Industry Forecast, 20192026

Read the full report: https://www.reportlinker.com/p05844072/?utm_source=PRN

The global gene therapy market was valued at $393.35 million in 2018, and is estimated to reach $6,205.85 million by 2026, registering a CAGR of 34.8% from 2019 to 2026.Gene therapy is a technique that involves the delivery of nucleic acid polymers into a patient's cells as a drug to treat diseases. It fixes a genetic problem at its source. The process involves modifying the protein either to change the genetic expression or to correct a mutation. The emergence of this technology meets the rise in needs for better diagnostics and targeted therapy tools. For instance, genetic engineering can be used to modify physical appearance, metabolism, physical capabilities, and mental abilities such as memory and intelligence. In addition, it is also used for infertility treatment. Gene therapy offers a ray of hope for patients, who either have no treatment options or show no benefits with drugs currently available. The ongoing success has strongly supported upcoming researches and has carved ways for enhancement of gene therapy.Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is on an increase, despite the limited number of products that have successfully reached the market. In addition, benefits of gene therapy over conventional cancer therapies and increase in government support fuel the growth of the gene therapy market.The gene therapy market is a widely expanding field in the pharmaceutical industry with new opportunities. This has piqued the interests of venture capitalists to explore this market and its commercial potential. Major factors that drive the growth of this market include high demands for DNA vaccines to treat genetic diseases, targeted drug delivery, and high incidence of genetic disorders. However, the stringent regulatory approval process for gene therapy and the high costs of gene therapy drugs are expected to hinder the growth of the market. On the contrary, increase in the pipeline developments for gene therapy market are expected to provide lucrative opportunity during the forecast period.The global gene therapy market is segmented based on vector type, gene type, application, and geography. Based on vector type, it is categorized into viral vector and non-viral vector. Viral vector is further segmented into retroviruses, lentiviruses, adenoviruses, adeno associated virus, herpes simplex virus, poxvirus, vaccinia virus, and others. Non-viral vector is further categorized into naked/plasmid vectors, gene gun, electroporation, lipofection, and others. Based on gene type, the market is classified into antigen, cytokine, tumor suppressor, suicide, deficiency, growth factors, receptors, and others. Based on application, the market is divided into oncological disorders, rare diseases, cardiovascular diseases, neurological disorders, infectious disease, and other diseases. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

KEY MARKET BENEFITS FOR STAKEHOLDERS This report offers a detailed quantitative analysis of the current market trends from 2018 to 2026 to identify the prevailing opportunities. The market estimations provided in this report are based on comprehensive analysis of the key developments in the industry. In-depth analysis based on geography facilitates in analyzing the regional market to assist in strategic business planning. The development strategies adopted by key manufacturers are enlisted in the report to understand the competitive scenario of the market.

KEY MARKET SEGMENTS

By Vector Type Viral vectoro Retroviruseso Lentiviruseso Adenoviruseso Adeno Associated Viruso Herpes Simplex Viruso Poxviruso Vaccinia Viruso Others Non-viral vectoro Naked/Plasmid Vectorso Gene Guno Electroporationo Lipofectiono Others

By Gene Type Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Others

By Application Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

By Region North Americao U.S.o Canadao Mexico Europeo Germanyo UKo Franceo Spaino Italyo Rest of Europe Asia-Pacifico Japano Chinao Australiao Indiao South Koreao Rest of Asia-Pacific LAMEAo Brazilo South Africao Saudi Arabiao Rest of LAMEA

KEY PLAYERS PROFILED Adaptimmune Therapeutics Plc. Anchiano Therapeutics Ltd. Achieve Life Sciences, Inc. Adverum Biotechnologies, Inc. Abeona Therapeutics Inc. Applied Genetic Technologies Corporation Arbutus Biopharma Corporation, Audentes Therapeutics, Inc. AveXis, Inc. Bluebird Bio, Inc. Celgene Corporation CRISPR Therapeutics AG Editas Medicine, Inc. Editas Medicine, Inc. GlaxoSmithKline Plc. Intellia Therapeutics, Inc. Merck & Co., Inc. Novartis AG REGENXBIO Inc. Spark Therapeutics, Inc. Sangamo Therapeutics, Inc. Uniqure N. V.Voyager Therapeutics, Inc

The other players of the gene therapy market include (companies not profiled in the report): Amgen Epeius Biotechnologies Sanofi Juno Therapeutics Advantagene

Read the full report: https://www.reportlinker.com/p05844072/?utm_source=PRN

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

__________________________Contact Clare: clare@reportlinker.comUS: (339)-368-6001Intl: +1 339-368-6001

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The global gene therapy market was valued at $393.35 million in 2018, and is estimated to reach $6205.85 million by 2026, registering a CAGR of 34.8%...

The cell and gene therapy sector is booming. At the end of 2019, there were 1,069 registered clinical trials in the field. By 2024, the cell and gene therapy market is estimated to reach revenues of $6.6B (5.9B). But there is a problem. As a result of the rapid growth of gene and cell therapies, the demand for plasmids is skyrocketing. And the industry is struggling to meet it.

Plasmids are the key building blocks needed to manufacture viral vectors, which are the most common strategy to develop gene therapies, explains Stefano Baila, Director of Operations and Business Development at Anemocyte. Plasmids are not only crucial for the development of gene therapies, but also for therapies that involve the genetic modification of cells, such as CAR-T. This has greatly increased the demand for plasmids and I would say that the industry was probably not ready to address this demand.

While the industry is struggling with the lack of plasmids on the market, another challenge is quality. Those providers that can meet the high-quality requirements for plasmids have long waiting lists of up to 12 months, says Baila. For companies moving into phase 3 or commercial production, it becomes crucial that the good manufacturing practice (GMP) requirements are met.

But another result of the rapidly evolving gene and cell therapy space is the fact that the regulatory requirements are not quite up to scratch. The regulatory framework around plasmid production is very confusing for the industry at the moment, says Baila. The main guidelines refer to the quality of the product, but the level of quality remains open to interpretation. More clarity would definitely help once and for all to define the exact quality levels required at different stages of drug development.

Consequently, plasmid providers have to be able to address all quality levels required at each stage of drug development. As the first biotech manufacturing organization (BMO) worldwide, Italian company Anemocyte has met this challenge by focusing greatly on the industrys needs. Their keyword is flexibility.

Before starting their work on plasmid manufacturing, the team spent several months interviewing companies about their difficulties and needs regarding the bottleneck in plasmid production and regulatory issues.

For us, it was key to understand the needs of the industry and find a possible solution, Baila explains. Our research resulted in a brand new facility, which is designed with adaptable manufacturing spaces that enable flexible time management. This ensures that the manufacturing process continues to roll without creating a bottleneck.

As a next step, the Anemocyte team had to decide whether to use a classified cleanroom or just a regular lab for the manufacturing process. We decided to keep the bar pretty high, so we are working with a cleanroom facility where we apply the GMP standard, says Baila. We maintain a high quality and also address the time issues that all companies seem to share as their main challenge in cell and gene therapy development.

The flexibility of its manufacturing facility allows the Anemocyte team to easily adapt to its customers needs. Our customers have control over what we do, explains Marco Ferrari, CEO of Anemocyte. They have the opportunity to be involved in the process, and decisions and actions are discussed and shared with them to ensure their product is produced at the high standard they expect.

Moreover, Anemocyte pays attention to new technologies and innovations. The fast evolution of the cell and gene therapy industry greatly increases the demand for new solutions, Ferrari explains. Staying on top of innovation is therefore mandatory today. Our approach is to stay ahead of the trends and be capable of deploying useful solutions for our customers.

Anemocytes manufacturing facility is built in such a modular way that it can be replicated and adapted to meet the rising demand for plasmids. This, as Ferrari puts it, ensures that the Anemocyte team will not be caught off guard when more companies come knocking at our door. This is an advantage for long-term customers because the manufacturing facility can be duplicated and built according to the customers needs.

As the worlds first BMO, Anemocyte pays specific attention to innovation and the ongoing trends in the industry. One of the emerging trends is the use of nonviral vectors for the development of gene therapies.

Even in the nonviral approach, plasmids play a key role, explains Baila. A part of the nonviral strategy is the transfer of plasmids into cells via mechanical or chemical methods. So, in one way or another, plasmids will always be needed.

Our investment in the nonviral gene modification space is an example of how we are tackling potential future trends that are still under the radar or explored at an academic level, adds Ferrari.

Dont sit on a waiting list to get your plasmids produced! Get in touch with the team at Anemocyte or learn more about the company and the development of plasmids for cell and gene therapies here!

Images via Shutterstock.com

Author: Larissa Warneck, Science Journalist at Labiotech.eu

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How Can We Tackle the Bottleneck in Plasmid Production for Gene... - Labiotech.eu

The collaboration with Childrens Medical Research Institute will boost the efficiency of AAV purification, leading to increased access to the viral vectors needed to manufacture gene therapies

GE Healthcare Life SciencesandChildrens Medical Research Institutewill jointly drive the development of new affinity ligands for the purification of adeno-associated viral (AAV) vectors used in gene therapies. The focus of the collaboration is to bring to market-specific ligands for multiple AAV types, enhancing the chromatographic separation of AAV-based vectors. This will improve the manufacturing efficiency and scalability of gene therapies, enabling the availability of viral vectors on a global scale.

With more than 800 gene therapies currently in clinical trials, there is an increasing demand for the raw materials needed in the manufacturing process of viral vectors. AAVs are viral vectors used in more than 70% of the in vivo gene therapy clinical trials. According to GlobalData, the 2025 gene therapy in vivo therapeutic market is expected to reach USD 32 billion with an estimated CAGR of 105% between 2019-2025.

The collaboration combines the expertise from the latest available research on AAVs with application testing, advancing a comprehensive understanding of the clinical functionality and the commercial opportunities of AAV-based gene therapies. Childrens Medical Research Institute will share with GE Healthcare Life Sciences AAV capsid variants targeting different tissues. GE Healthcare Life Sciences will then design and test ligand prototypes, which Childrens Medical Research Institute will assess. Based on the performance results, GE Healthcare Life Sciences will manufacture and commercialize novel improved AAV affinity ligands.

Dr Leszek Lisowski, the lead gene therapy scientist at Childrens Medical Research Institute, says: Bringing the fruits of our work to the patients requires a joint effort between academia and the industry. The collaboration with GE Healthcare Life Sciences will allow us to expedite the development of novel clinical options at a lower cost.

Olivier Loeillot, General Manager, Bioprocess at GE Healthcare Life Sciences, says: The industry needs better and more personalized technologies to speed biopharmaceuticals through clinical trials and bring them to market. Our long biomanufacturing expertise combined with Childrens Medical Research Institutes pioneering research will lead to purification technologies that will streamline the production of gene therapies.

Catarina Flyborg, General Manager, Cell and Gene Therapy at GE Healthcare Life Sciences, says: Collaborations with organizations such as Childrens Medical Research Institute are critical to developing the technologies needed to move the industry forward. By working directly with world-class researchers, GE Healthcare Life Sciences can develop the purification technologies that will contribute to increasing the availability of viral vectors globally.

Childrens Medical Research Institute in Australia is globally recognized for its work on microsurgery, cancer research, neurobiology, embryology and gene therapy. The AAV affinity ligands resulting from this collaboration will be compatible with GE Healthcare Life Sciences resin-based chromatography portfolio used in the purification of most FDA-approved biopharmaceuticals.

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GE Healthcare joins CMRI to optimize gene therapy manufacturing - BSA bureau

With more than 900 investigational new drug (IND) applications for ongoing clinical studies related to gene therapies, and with the number of advanced therapy medicinal products at clinical stage worldwide exceeding 1,000, the US Food and Drug Administration (FDA) this week released a number of policies.

The policies, addressed to developers and manufacturers, include six final guidance documents on gene therapy manufacturing and clinical development of products, following up to respective draft guidance documents released in 2018, and a draft guidance related to orphan drug designations for therapeutic candidates.

Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review, commented Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, adding The framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field.

Regarding the draft guidanceInterpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations, the agency explained that it focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease.

The final guidance titled Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) aims to inform sponsors on how to provide sufficient CMC information, in order to assure product safety, identity, quality, purity, and strength (including potency) of the investigational product and to be able to claim market authorization from the regulatory body.

Addressed to developers and manufacturers of retroviral vector-based human gene therapy products, the second document titled Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up determines testing for RCR during manufacture, as well as the regulations for follow-up monitoring of patients who have received such treatments.

Titled Long-Term Follow-Up After Administration of Human Gene Therapy Products, the third document includes recommendations by the FDA regarding the design of long-term follow-up studies for the collection of data on delayed adverse events.

Specifically, the FDA suggests that, as a result of long-term exposure to an investigational gene therapy, patients may be at increased risk of undesirable and unpredictable outcomes, and therefore they may be monitored for an extended period of time past the active follow-up period. The document outlines several factors based on which a risk assessment should be performed to determine the necessity of long-term monitoring for each product.

Another guidance of the FDA is focused on Human Gene Therapy for Hemophilia, and it provides recommendations regarding the clinical trial design for such therapies, as well as addressing discrepancies between Hemophilia A and B coagulation factors activity assays.

Focusing on Human Gene Therapy for Retinal Disorders, the fourth FDA guidance includes recommendations related to product development, preclinical testing, and clinical trial design for such gene therapy products.

Finally, the guidance on Human Gene Therapy for Rare Diseases, with suggestions on the clinical design for such products, is needed, according to the FDA, due to the limited study population size and potential feasibility and safety issues. Moreover, the FDA cites issues related to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the product.

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FDA guidance on gene therapies development and manufacturing - BioPharma-Reporter.com

Nationwide Childrens Hospital is asking for tax abatements on a new for-profit gene therapy center. If granted, it would exempt the company from paying money to Columbus City Schools for 15 years.

Andelyn Biosciences has been proposed as a for-profit company that would manufacture gene therapy products.

Those products would be developed and distributed to companies that sell them for medical treatment, explains Libbey Hoang, vice president of planning and business development with Nationwide Childrens.

"Our focus will be to bring rare genetic disorder treatment for children to the market," Hoang says. "We believe that theres a great opportunity for Nationwide Childrens to be the leader in that because of our transformational science that has occurred here."

The center would be part of Ohio State Universitys West Innovation Campus, at the corner of Lane and Carmac. Hoang says it will be specifically designed for biologics manufacturing.

That combined with having to hire an expected 150 employees before they can turn a profit is why theyre asking for a 15-year, 100% tax abatement.

"The facility cost us about $64 million in improvements to construct," Hoang says. "Because the company will take approximately four years to actually then produce treatment, we will have nearly $30 million in taxable payroll with very limited income, so thats the major reason were seeking the tax abatement."

However, Nationwide Children's ask is drawing criticism.

"Our stance has always been that we oppose tax abatements for corporations that dont need them," says John Coniglio, president of the Columbus Education Association.

The teachers union has long opposed the city forfeiting tax dollars to spur development. He points to Nationwide Childrens hospitals profit margin an average of about $327 million per year.

"The first thing that comes to my mind is: Does Childrens Hospital really need this tax abatement?" he says.

The hospital runs several programs in partnership with Columbus City Schools, including STEM programs and primary care services. Nationwide Childrens is also offering an estimated $53 million in health care services in the schools during the 15-year abatement period.

"Childrens Hospital does do good things in Columbus City Schools," Coniglio says. "But my question would be: Are you doing this just because you dont want to take the risk that regular individuals have to take every time they want to open a business or do something new?"

Michael Stevens, interim director of development for the City of Columbus, says the land proposed for Andelyn Biosciences is owned by the state, so its not currently producing any tax revenue.

"Without this incentive there would not be the project," Stevens says. "And at this point this parcel does not generate any revenue for the schools or the city, and as a result of incentivizing and making this investment, then were going to see revenue coming into the income tax for the city that then we share with the schools over the 15-year period."

Stevens says the city and the schools will split land and income taxes from Andelyn Biosciences projected to be about $2.5 million each. That price is still significantly less than would have been collected if the project went through without the abatement.

To receive the tax abatement, Nationwide Childrens needs approval from both the Columbus Board of Education and Columbus City Council.

The proposal hasnt been presented to City Council, yet so member Elizabeth Brown declined to make a judgement. But Brown says its important to consider the motivation behind Andelyn Biosciences.

"The genetic disorders are so narrowly presented in the average population that pharmaceutical companies cant make money on developing those things," she says. "Which is why Childrens Hospital is going after it, to my understanding."

The school board plans to take up the abatements on Tuesday. In a statement, board president Jennifer Adair says Nationwide Children's has a strong partnership with the district, and they are considering the abatement. Council will have the final say.

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Nationwide Children's Seeks City Tax Breaks On For-Profit Gene Therapy Facility - WOSU

Thought leaders in cancer cell and gene therapy research will share the latest advances, address the greatest challenges and showcase the most innovative programs in progress today.

Alliance for Cancer Gene Therapy (ACGT) will host its inaugural ACGT 2020 Cancer Summit on April 16, 2020 at the Alexandria Center for Life Science in New York City. The ACGT 2020 Cancer Summit which launches ACGTs 20th anniversary will bring together researchers, companies, investors and advocates in cancer cell and gene therapy to discuss the latest advances, with a focus on combating solid tumors.

A partial list of ACGT 2020 Cancer Summit speakers includes:

Presenting sponsors include Alexandria Real Estate Equities, Inc./Alexandria Venture Investment. Additionally, STAT will be a media partner for the ACGT 2020 Cancer Summit. For more sponsorship information and early bird registration, please visit https://summit2020.acgtfoundation.org

Media Registration

Media registration is free of charge for all valid press card holders or via provision of formal journalist credentials. Register early by contacting ACGT.pr@HDMZ.com to begin receiving advance meeting materials, media alerts, and access to meeting presenters.

About Alliance for Cancer Gene Therapy

For nearly 20 years, Alliance for Cancer Gene Therapy (ACGT) has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Founded by Barbara and Edward Netter after their daughter-in-laws death from breast cancer, ACGT funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. One hundred percent of all public funds raised by ACGT directly support program and research, thanks to separate funding to support administrative expenses.

For more information, visit acgtfoundation.org, call 203-358-5055, or join the ACGT community on Facebook, Twitter, Instagram and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200203005084/en/

Contacts

Media Inquiries: Emily Maxwell 312-506-5220emily.maxwell@hdmz.com

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Save the Date: Alliance for Cancer Gene Therapy 2020 Cancer Summit to be Held in New York City - Yahoo Finance

The two big pharma companies joined, among others, the investing group for Vineti, the developer of a digital platform for personalized therapeutics enabling the distribution of regenerative medicine, both at clinical and commercial scale.

More specifically, the cloud-based digital platform connects healthcare providers, suppliers, and biopharmaceutical developers by providing them with real-time access to data.

Focusing on personalized medicine, the platform is designed to provide simplicity to end users. Vinetis CEO, Amy DuRoss explained to us why simplicity is important for such treatments:Cell and gene therapies have many, many different types of people involved in the manufacturing and delivery process, including healthcare providers who arent typically part of manufacturing supply chains.

These unfamiliar, highly regulated processes need to be as simple for healthcare providers and other stakeholders as possible, so that these therapies can be produced for patients quickly and without error, she added.

According to the company, Vineti is the only platform of record currently enabling both clinical and commercial personalized therapeutics, such as chimeric antigen receptor (CAR) T-cell therapies and allogeneic cell therapies, into the supply chain.

Both Gilead and Novartis hold assets at commercial stage, the marketing of which can benefit from the use of the platform. Such assets are Novartis gene therapy Zolgensma (onasemnogene abeparvovec-xioi) and Kymriah (tisagenlecleucel), as well as Gileads Yescarta (axicabtagene ciloleucel).

Vinetis Series C financing round closed at $35m (31.8m), with the software developer announcing that it will use the funding for the expansion of its business, including new software product development and commercial expansion in Europe and Asia-Pacific.

The financing was led by Cardinal Health, with participation from Novartis and Gilead, through its subsidiary, Kite, as well as existing investors. Following the completion of the funding, representatives of Novartis and Kite will join Vinetis leadership as board observers.

Kites global head of technical operations, Charles Calderaro, stated that Kite played a foundational role as Vineti's first biopharma partner, when the company prepared for the launch of Yescarta.

Asked about how the platform will be expanded after the funding, DuRoss explained that it will offer improved configurability, enabling users to add new or updated features and adjust them at will, ultimately saving them cost and time.

In biopharma, its all about speed to market, and our configuration approach supports that, DuRoss told us.

Moreover, the platform will position the company towards global expansion, by including more languages as well as support for more regional regulations and standards.

According to DuRoss, the companys partnersincreasingly operate worldwide, in international medical centers and manufacturing sites, and many patient-based drug products cross borders on their journey to becoming a therapy.

Additionally, the company plans to add to the platform more of the standards and integrations required to industrialize cell and gene therapies. Vinetis CEO told us that It may sound contradictory to standardize such deeply personalized drug products, but for scale, standardization is absolutely essential.

DuRoss claimed that even though its still early days in cell and gene therapy, some standard approaches are emerging, as are pre-built integrations that connect critical technology stacks.

We want to help our partners focus on the uniqueness of their science, and not have to reinvent the wheel on supply chain management with every new drug product, she added.

Finally, the company plans to expand the range of therapies that the platform supports to include other high-value therapeutics. Well keep our focus on cell therapy, gene therapy, and personalized cancer vaccines, but expand into other therapeutic areas requiring precision coordination, said DuRoss.

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Novartis and Gilead provide funding to Vineti - BioPharma-Reporter.com

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Aperio Clinical Outcomes, a leading clinical research organization (CRO), announced today that Jonathan Yusi, an expert in the coordination and management of cell and gene therapy clinical trials, has joined the company as Senior Clinical Trials Manager to support their biotechnology clients in the immuno-oncology space.

Yusi has been managing immune-based therapy trials for over seven years. Prior to joining Aperio, he was a program manager for CAR-T studies and oversaw the first CAR-T program at a large CRO. He has provided independent trial management consulting for CAR-T trials, and his expertise has resulted in lasting KOL relationships within the immuno-oncology space. In addition to his adoptive cell therapy knowledge, Yusi brings over 20 years of clinical research experience to Aperio, with a focus on trial logistics, management, and monitoring of targeted and immune therapies in oncology trials.

Says Suzanne Kincaid, Aperios COO and an oncology industry veteran herself, FDA expects to see over 200 INDs for cell and gene therapies in 2020, so it is imperative that our biotech clients have expertise like Jonathans to manage their trials. He has a strong understanding of the complexities of cell and gene therapy studies and can break down the components for ideal study set-up. We are so excited to have Jonathan help our immuno-oncology clients as they explore these groundbreaking treatments.

Cell and gene therapy trials are a logistical maze, and one missed endpoint can be catastrophic to the study, says Yusi. These programs allow me to utilize everything Ive learned about clinical research and oncology, and my medical and scientific background brings an understanding to the science behind the treatments. The bulk of my career has involved oncology trials, so as the treatments have evolved and become more personalized, my experience has evolved as well. These are life-saving breakthroughs, and Im happy to bring this experience to Aperio and our immune-based therapy clients.

About Aperio Clinical Outcomes

In a data driven industry, Aperio remains dedicated to transparency with clients and focused on the most important part of the process: people. Aperio provides full, customizable clinical research services across multiple therapeutic areas, as well as consulting services in Quality Assurance, Strategic Resourcing and Clinical Trial Technology. For more information: http://www.aperioclinical.com.

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Aperio Hires CAR-T Trials Expert Jonathan Yusi to Support Cell and Gene Therapy Studies - Business Wire

GenScript Biotech Global Forum Highlights Advances in Cell and Gene Therapy and Opportunities in China

GenScript Biotech Corp., a leading global biotechnology group and a pioneer in the field of gene synthesis, held its inaugural "Global Forum on Cell & Gene Therapy and the Booming China Market," during the JP Morgan Healthcare Conference week, attracting hundreds of industry leaders, investors and others to address the challenges and opportunities in this innovative field.

"As an industry, we are on the brink of achieving some extraordinary breakthroughs in cell and gene therapy for cancer and other diseases," said GenScript Biotech CEOFrank Zhang, PhD. "Four gene and cell therapies have recently been approved by the FDA, bringing new hope to patients, and this is only the beginning. Our vision is to make cancer a chronic or curable disease rather than a deadly one, and to transform the treatment of cancer, autoimmune and other diseases by leveraging the advantages of cell and gene therapy."

While significant advances are being made, the Forum also tackled some of the more pressing challenges, such as mitigating treatment side effects, improving treatment efficacy in solid tumors and scaling up manufacturing. Panelists from Kite Pharma, GE Healthcare Life Sciences, Ziopharm Oncology, Oxford Biomedica, Genethon, CARsgen Therapeutics, J&J Innovation Asia Pacific, the American Society of Gene & Cell Therapy, Loncar Investment, Lilly Asia Ventures, and many others participated in the event.

In the U.S. alone, the U.S. Food and Drug Administration is expected to approve 40-60 cell and gene therapies by 2030. During a panel discussion focused on regulatory issues, experts considered what regulators will need to do to keep up with the rapid pace of innovation, the new hospital-based regulatory pathway inChina, how to ensure quality through the manufacturing process, and the challenges and opportunities that come with regulatory harmonization among different countries.

Chinacontinues to attract significant attention from industry and investors and is poised to grow even more. During his welcoming remarks, Zhang notedChina'semergence as a global economic leader, with a projected$1.1 trillionspend on healthcare this year, as well as the growing disease burden inChina. By 2030, an estimated 4.3 million Chinese will be diagnosed with one of the 14 major cancers, according to research from IMS Health. Panelists addressed issues such as the amount of capital required to achieve scale inChina, and advantages of the market inChina.

"The drug development business is changing rapidly andChinais at the fore in a number of ways," Zhang said. "Biotech and pharma companies do not need or desire to have the infrastructure to scale their drugs through commercialization. With lower costs,Chinais a natural place for companies to contract out costly development and manufacturing to organizations that have the expertise and experience to collaborate with them through the entire discovery to development lifecycle."

For its part, GenScript has put significant resources into its Contract Development and Manufacturing Organization (CDMO) business to meet the increasing demand. In 2018, the company officially launched its biologics CDMO segment, and last year opened a new GMP compliant biologics research center. GenScript is also leading the way in cell therapy through its antibody discovery service and plasmid and virus production capabilities.

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Advances in Cell and Gene Therapy and Opportunities in China - BSA bureau

The UK accounts for over 12% of global cell and gene therapy clinical trials, according to new data published by a government-backed agency.

The Cell and Gene Therapy Catapult (CGT Catapult) today released its 2019 UK Advanced Therapy Medicinal Products (ATMPs) clinical trials database showing that the 127 ongoing trials represent a 45% increase compared with 2018.

According to the CGT Catapult this means the NHS and UK life sciences R&D centres are working well to allow innovative therapies to progress through to the clinic.

Publication of the figures comes the day after the government announced a deal with Novartis to bring cholesterol drug inclisiran to patients in England, and the announcement of an updated life sciences industrial strategy aimed at stimulating private investment in the sector.

Cell and gene therapies require specialist systems and infrastructure and according to the CGT Catapult the UK is being recognised internationally with the majority of commercially sponsored trials being backed by international organisations.

The database shows that 77% of UK cell and gene therapy trials are now sponsored by commercial organisations compared to only 25% in 2013.

The main therapy area for cell and gene therapies clinical trials remains oncology (39%) followed by ophthalmology (13%) and haematology (12%).

While the majority of trials are in the recruitment phase, the number of trials recruiting is considerably larger than previous years.

In 2019, there were 90 cell therapy trials recruiting patients, compared with around 55 in 2018, and the data suggest that these trials are quickly moving through planning and regulatory approvals to recruitment stage.

Around 65% of trials involve autologous cells, sourced from individual patients, with the remainder being off the shelf allogeneic products.

Established as an independent centre of excellence the CGT Catapult is funded by the government through Innovate UK.

It aims to encourage inward investment from big pharma and other international companies by bridging the gap between scientific research and full-scale marketing.

Keith Thompson, CEO of the Cell and CGT said: The total number of cell and gene therapy clinical trials in the UK has been increasing consistently by an average of 25% year on year since 2013. This has been enabled by the development of the UKs fantastic ecosystem to support the development and clinical adoption of cell and gene therapies.

The full report is available here.

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UK accounts for 12% of cell and gene therapy trials - report - - pharmaphorum

Gene therapies are in the in-demand medicine, due to their potential to treat life-threatening diseases, including illnesses classified as rare genetic diseases. Examples include treating sickle cell anemia and anti-tumor immunotherapy. As well as being demand, the market is also lucrative, expected to grow to $11.96 billion by 2025. The growth with gene therapies is represented by the range of gene therapy products in the clinical pipeline, where Frost and Sullivan estimate some 400 cell and gene therapy products in development. The last year alone has seen a 27 percent increase in the number of clinical trials involving gene therapy products. This is also reflected with the number of emergent start-up companies entering the gene therapy space. Most start-up companies elect to outsource the downstream manufacturing stages, due to the relatively low costs involved, as off-set against the costs involved with constructing specialist facilities. Big Pharma is also investing in the field. For example, Pfizer has injected $500 million into one of its plants in North Carolina and Fujifilm Diosynth has made a similarly large investment to boost production capability. READ MORE: Revolutionizing the CRISPR methodThe growth has been fuelled by support from governments and regulatory agency. Here the U.S. 21st Century Cures Act as been a key driver, supported by the U.S. Food and Drug Administration (FDA). There are, however, some limitations to be overcome. One limitation with progressing gene therapy products is with costs and pricing structures, which is resulting in many therapies being unstainable. This is also limiting the transition to scale, given the complexities tied to growing, sourcing and transporting cells. Costs are high partly as a result of the development and partly because many gene therapies offer curative solutions, making them an in-demand and expensive medicine. A solution to the costs factors is, according to Vered Caplan (Orgensis Inc) the development of point-of-care automation, including the application of closed systems for processing cell therapies. These systems require the use of fewer personnel and permit real-time quality control testing to take place. A second limitation is with the availability of rare materials of sufficient quality, such as plasmid DNA and tranfection agents), which need to be manufactured under the same standards applied to mainstream pharmaceuticals (that is, Good Manufacturing Practices. With new technology emerging to improve product yield and to drive down costs, coupled with increased investments, the expansion of gene therapy products looks set to be one of the big drivers within healthcare over the next five years.

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Why gene therapy is set to transform medicine - Digital Journal

Precision BioSciences, a Durham-based genome editing company, has reached two regulatory milestones for its potential therapy against multiple myeloma, a chronic cancer of white blood cells.

The U.S Food and Drug Administration (FDA) has accepted the companys Investigational New Drug (IND) application and also granted the therapy Orphan Drug Designation, a status that gives companies tax reductions and other incentives to develop treatments for rare diseases.

The therapy, designated as PBCAR269A, is Precisions third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate.

FDA acceptance of the IND for PBCAR269A further underscores the ongoing progress in our allogeneic CAR T pipeline, said Matt Kane, co-founder and chief executive officer of Precision BioSciences. We have now moved three CAR T programs from preclinical to clinical stage development since April 2019, and we look forward to continuing to advance our allogeneic CAR T portfolio to bring these novel therapeutic candidates to patients.

Matthew Kane

The company plans to begin a Phase 1 clinical trial of the therapy this spring at multiple sites using material produced at its own manufacturing facility in Durham. About 48 patients are expected to be enrolled.

For more information about the trial, visitwww.clinicaltrials.gov, and enter study identifier number NCT04171843.

In preclinical disease models, PBCAR269A has demonstrated no evidence of graft-versus-host disease at doses that resulted in potent anti-tumor activity, said Chris Heery, M.D., chief medical officer of Precision BioSciences. There remains significant unmet need in the treatment of relapsed/refractory multiple myeloma, and we are excited to begin clinical trials with an off-the-shelf CAR T therapy candidate in this setting.

The IND for PBCAR269A builds on the initial clinical data Precision presented in late 2019 for its lead program, a CAR T therapy for treating non Hodgkins lymphoma and B-cellacute lymphoblastic leukemia, and the FDAs acceptance of an IND for another CAR T therapy for treating non-Hodgkins lymphoma, chronic lymphocytic leukemia and small lymphocytic lymphoma.

Precision BioSciences harnesses T cells, a type of white blood cell that is vital to the adaptive immune systems ability to identify specific antigens and destroy pathogens. Through the companys CAR T technology, the T cells can be directed to kill cancer cells.

Precision produces the CAR T therapies by selecting T cells derived from healthy donors as starting material. Then, using its proprietary ARCUS genome-editing technology, the company modifies the donor T cells.

Scientists insert the CAR gene at the T cell receptor locus, enabling the T cell to target a specific marker on a cancer cell, while knocking out the T cell receptor to prevent the patients immune system from recognizing and attacking the T cells.

The company optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of certain types of CAR T cells throughout the manufacturing process and in the final product.

The process creates a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease, normally a major challenge when inserting foreign or altered cells or tissues into the body.

The company has posted a four-minutevideoon its website to explain CAR T therapy, using Samurai warriors as an analogy.

Last July Precision opened its Manufacturing Center for Advanced Therapeutics (MCAT), the first in-house current Good Manufacturing Process (cGMP)-compliant manufacturing facility in the United States dedicated to genome-edited, off-the-shelf chimeric antigen receptor CAR T cell therapy products.

Precision Bio facility

Given the potential output of our platform, weve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients, Kane said when the facility opened. In addition to our clinical work, it also has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR T cell therapies and 4,000 doses of gene therapies per year.

The facility can produce three different drug substances: allogeneic CAR T cells, messenger RNA and adeno-associated viral vectors. It was designed to meet regulatory requirements in the United States, Europe and Japan.

In addition to health care, Precisions ARCUS genome-editing platform has applications in food and agriculture.

In 2018 the company created a new name and brand identity, Elo Life Systems, for its food and agriculture business, previously known as Precision PlantSciences, based in Research Triangle Park.

Elo is using the ARCUS platform and other new technologies for applications in crop improvement, animal genetics, industrial biotechnology and sustainable agriculture.

Since it was spun out of Duke University in 2006, Precision raised about $300 million in venture capital, government grants and collaboration agreements. The company went public in March 2019, grossing $145.4 million in an initial public offering of stock.

The companys shares are listed on the Nasdaq Global Select Market under the ticker symbol DTIL, shorthand for the companys marketing tagline, Dedicated to improving life.

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Precision BioSciences hits two key FDA milestones in advancing gene therapy for cancer - WRAL Tech Wire

ASC Therapeutics ("ASC"), a privately-held gene therapy company focused on developing transformative gene-based medicines for serious diseases, announced today that it has entered into a long-term strategic manufacturing partnership with Vigene Biosciences ("Vigene"), a Maryland-based Contract Develop and Manufacturing Organization (CDMO). Vigene will provide ASC with access to GMP manufacturing including viral vectors and plasmid DNA for its hemophilia A gene therapy clinical program, as well as a manufacturing platform for future gene therapy programs.

"The genetic platform technology developed by ASC is going to change the way serious diseases are treated in the future. We have seen remarkable potency data in our Hemophilia A IND-enabling gene therapy studies," said Dr. Ruhong Jiang, ASC Founder & CEO.

Dr. Jiang continued, "We are proud to welcome Vigene, a global leader in gene therapy process development and GMP manufacturing with a proven track record and expertise in the field of viral vector manufacturing, to become an integral part of ASC long-term plan. Our partnership provides ASC access to Vigenes world-class team with expertise for both plasmid DNA and viral vector manufacturing as well as high-caliber QC and QA teams."

"Vigene is excited to become a long-term CMC partner for ASC with multiple newly built, state-of-the-art GMP suites and high-titer virus producer cell lines, we are well positioned to support ASC for both early-stage and commercial virus production. This partnership will ensure that all ASC clinical deliverables are achieved in a timely fashion," said Dr. Zairen Sun, Vigenes President and CEO. "We have a world-class manufacturing team, and this agreement is a testimony for our recognition by top-tier biopharmaceutical companies."

About ASC Therapeutics

ASC Therapeutics is a biopharmaceutical company dedicated to advancing an integrated and sustainable pipeline focused on gene therapies that correct the root cause of complex and intractable diseases. We are leveraging our AAV-based gene therapy, CRISPR-Cas9 and proprietary gene editing platforms consolidated over 10 years to develop transformative gene-based medicines. ASC is accelerating through clinical trials several lead gene therapies for inherited blood disorders. Our team of industry veterans in discovery, pre-clinical, clinical and CMC gene therapy development and world-class academic and biopharmaceutical partnerships are potentiating our gene therapy capabilities. To learn more about ASC Therapeutics, please visit http://www.asctherapeutics.com.

About Vigene Biosciences

Vigene Biosciences is an award-winning and private equity backed leader in gene delivery development and manufacturing. Vigene has 16 years of cGMP viral vector production experience. Vigenes mission is to make gene therapy affordable. Vigene offers IND-enabling and IND-supporting materials as well as FDA- and EMA-compliant commercial products for plasmid, AAV, lentivirus, and adenovirus with proven technologies and track records. The GMP facility features 10 GMP suites including 5 brand-new cGMP suites with 200L-500L single-use stir tank bioreactors for suspension cells as well as iCellis 500 for adherent cells. In 2018 and 2019 Vigene was recognized by INC500. In 2018, Vigene was chosen as the ACG Emerging Company of the Year Award. To learn more about Vigene Biosciences, please visit http://www.vigenebio.com

View source version on businesswire.com: https://www.businesswire.com/news/home/20200114005788/en/

Contacts

ASC Contact Information: Oscar Segurado, MD, PhDChief Medical OfficerASC TherapeuticsMilpitas, CA650.490.5199oscar.segurado@asctherapeutics.com

Vigene Contact Information: Jeffrey Hung, Ph.D.Chief Commercial OfficerVigene BiosciencesRockville, MD301.251.6638jhung@vigenebio.com

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ASC Therapeutics and Vigene Announce Long-Term Strategic Partnership for Gene Therapy Development and Manufacturing - Yahoo Finance

Two Parkinsonspatients treated with AXO-Lenti-PD, an investigative gene therapy, in an ongoing clinical trial continue to show improvement 12 months later, Axovant, the therapys developer, said in a release.

These findings at one year after treatment are important because this timepoint allows for a better assessment of therapy durability, and a more assured differentiation between placebo effects and therapeutic response, the company added.

AXO-Lenti-PD has shown encouraging results in these two people given a first low dose in the SUNRISE-PD (NCT03720418) Phase 1/2 clinical trial, which is now enrolling up to 30 patients at sites in France and England.

The treatment works by delivering three genes involved in dopamine production directly to the brain via a surgical procedure.

Dopamine is a neurotransmitter a molecule involved in transmitting information between neurons that is critical to coordinating movement. Dopamine-producing (dopaminergic) neurons are lost in Parkinsons, and the resulting drop in dopamine levels is the cause of many disease symptoms.

By infecting brain cells with the genetic instructions to increase dopamine production, AXO-Lenti-PD aims to turn other cells into dopaminergic neurons.

Current dopamine replacement therapies require continual oral doses of dopamine, whose effectiveness fades over time. The period between when one doses effectiveness wanes and the taking of a next dose can result in off periods, wherein patients report a return of symptoms such as poor motor control, stiffness, fatigue and mood changes.

Helping the brain to again produce adequate levels of dopamine would, in theory, eliminate the need for periodic oral doses, which could significantly limit off periods.

Previous studies in primate models of Parkinsons found AXO-Lenti-PD to be safe and effective, and SUNRISE-PD results at three months post-treatment found that a one-time delivery of the therapy significantly improved patient scores on theUnified Parkinsons Disease Rating Scale (UPDRS), a standard assessment of motor and non-motor symptoms associated with Parkinsons.

The trial consists of two parts. Part A is an open-label, dose-escalation phase in which patients receive one of potentially three escalating doses of the gene therapy. In part B, a new group of patients will be randomized to either the ideal part A dose or to a sham procedure as an untreated control group. SUNRISE-PDs goal is to test the safety, tolerability, and effectiveness of the potentialtreatment.

Both patients here, the first two enrolled, received the lowest dose (4.2106transducing units) of AXO-Lenti-PD.

One-year results showpositive changes of 24 points and 20 points (respectively for the two patients) on the UPDRS Part III Off score, representing a 37% improvement in off-period motor symptoms, Axovant reported. Improvement at six months was 29%, as measured on the same scale.

These patients also showed an average 13-point positive change from baseline (study start) representing a 44% improvement on the UPDRS Part II Off score, which assesses daily life activities. On the PDQ-39 score index, another quality-of-life measure in Parkinsons disease, these two showed an average 15-point positive change, or a 30% improvement from baseline to 12 months.

Both patients tolerated AXO-Lenti-PD well, and neither reported any serious side effects. One maintained a diary of on/off periods, which is useful in evaluating changes that might be due to therapy across time.

People being enrolled in SUNRISE-PD have had Parkinsons for at least five years, have motor fluctuations and dyskinesia (jerky, involuntary movements), and are between the ages of 48 and 70. More information can be found here.

The company expects to soon release six-month results on the first two patients given a second and higher dose of AXO-Lenti-PD. This dose is three times higher than that given the first cohort.

If dose-escalation results allow, Axovant expects to begin the randomized and placebo-controlled part B of the SUNRISE-PD as a Phase 2 study by the close of 2020.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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One-year Results in 2 Given Gene Therapy at Low Dose Showing... - Parkinson's News Today

Dublin, Jan. 13, 2020 (GLOBE NEWSWIRE) -- The "Gene Therapy Market (3rd Edition), 2019-2030" report has been added to ResearchAndMarkets.com's offering.

The Gene Therapy Market, 2019-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, and genome editing therapies. The study also features an elaborate discussion on the future potential of this evolving market. Amongst other elements, the report features:

Key Topics Covered:

Some of the Companies Mentioned include:

For more information about this report visit https://www.researchandmarkets.com/r/nuaak0

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

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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Global Gene Therapy Market 2019-2030: Promising Therapeutics Areas - Yahoo Finance

Published: February, 2020

Q. My family tends to be long-lived. I hear longevity is due to our DNA, and I also hear it's due to lifestyle. Which is it, and how do they make us live longer?

A. Both DNA and lifestyle can affect longevity, and they both do so in the same way: by altering our body chemistry. DNA controls the production of each of the natural chemicals in our body. It controls both the shape (and, hence, the effectiveness) of each chemical, and also controls how much of that chemical is made. So, it's not surprising that DNA could affect longevity. In the past 20 years, astonishing progress has been made in understanding the body chemistry that controls the aging process. And that knowledge has allowed scientists to extend the life of various animals through simple genetic manipulations.

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What has the most impact on longevity? - Harvard Health

The team at Frazier Healthcare did a number of deals that exemplified its wide-ranging strategy in 2019: Tachi Yamada worked with gene therapy pioneer Jim Wilson to launch Passage Bio; Mike Gallatin sold Mavupharma and its STING-targeted small molecule to AbbVie; and Bhaskar Chaudhuri flipped Arcutis to an IPO just months after introducing it to the world via a crossover round close to $100 million. Theyre now kicking off 2020 with a new, bigger fund that will give them firepower to do more.

At $617 million, Fund X is fairly dramatically bigger than their last fund, said managing partner Patrick Heron.

Were increasingly going after new therapeutic modalities like gene therapy, cell therapy, neoantigens and because those need significant manufacturing and CMC investment or investing more dollars per company, he told Endpoints News.

He sees Frazier pouring around $40 million into each company supporting them through every stage, whether its helped with the launch or joined through a later syndicate though that could vary if, say, they sell a company right after Series A. By that estimate, the new fund could touch anywhere from 15 to 25 biotechs.

About a third of the portfolio is reserved for homegrown startups, another 15% to 25% for public securities, and the rest is in-between.

Heron is one of three leading the fund; hes joined by managing partner James Topper and Dan Estes, whos just been promoted to general partner. Also involved will be Jamie Brush, newly made partner after spearheading investments in public securities for the past three years.

Frazier is happy to both create and syndicate with its venture brethren, Heron added, citing OrbiMed as a friend.

The congregation has grown exponentially since Frazier first put its feet down three decades ago.

When I started at Frazier, there were probably 10 to 15 life sciences focused VC funds, said Heron, whos just celebrated his 20th year at the firm, and now theres probably 100.

It gives Frazier a lengthy track record to boast which can be particularly helpful when they pitch bigger players on biotech spinoffs such as Phathom Pharmaceuticals, now developing one of Takedas GI drugs.

Pharma companies have become more receptive to that when they see substantial value accrual to them, Heron said. And its public now: Takeda owns probably about $200 million worth of stock in Phathom, and so they are basically deriving a lot of economic value from the partnership, and what theyre also focused on is the quality of teams we can put around their asset such that the program will reach the clinic and benefit patients.

Two former Celgene execs from the global inflammation and immunology franchise have been recruited to the C-suite at Phathom, including CEO Terrie Curran and CCO Martin Gilligan.

Despite the lack of big check M&A at the beginning of the year and an election looming in November, Heron remains optimistic as their deal flow has been in line with the expectation of 2 to 3 sales per year. And the same goes for IPO.

I think you will see a lot of companies sort of backing up the truck and loading up with as much capital as they can, with probably less robust activity in the second half of the year, he said.

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Frazier closes 12th fund in 30 years, with $617M to bet on cell/gene therapy, Big Pharma spinoffs and more - Endpoints News

LONGMONT, Colo. A few months ago, 2-year-old Maisie Forest was finally able to sit up on her own for the first time. Her development has been delayed by a rare genetic disorder called Spinal Muscular Atrophy, but last August, she received a groundbreaking treatment for the condition.

"It's a miracle drug," said Maisie's mother, Ciji Green. "It's not the cure, but we're talking about a disease that had no treatments four years ago," she added.

The "miracle drug" Green is referring to Zolgensma, a gene therapy for Spinal Muscular Atrophy made by Novartis-owned AveXis. On Tuesday, AveXis cut the ribbon on a new facility in Longmont where it will soon produce Zolgensma.

"Zolgensma is this first product weve had approved by the FDA for the treatment of kids with Spinal Muscular Atrophy," said AveXis President David Lennon.

The FDA approval came last May, just in time for Maisie to receive the treatment. But her mother still had to fight for the insurance company to pay for it. At $2.1 million per dose, Zolgensma is the most expensive drug or treatment ever made. Lennon said Novartis has invested half a billion dollars in the production of Zolgensma.

For Green, the cost is well worth the changes she's already seen in her daughter. Speaking to employees at the AveXis ribbon cutting, she called them heroes.

"To all of you it may just be a treatment, but to my family and so many others, its so much more," said Green.

AveXis says the same platform they used to produce Zolgensma might be applied to other therapies for other diseases in the future. The company is looking at developing treatments for Rett Syndrome, Friedreichs Ataxia, and an inherited form of Amyotrophic Lateral Sclerosis, or ALS.

"There are actually thousands of these kinds of diseases. Usually they impact a few hundred kids or adults every year, but altogether there are potentially millions of patients who have genetic diseases around the world," said Lennon.

Lennon said AveXis chose Longmont for its production facility in part because of the infrastructure already in place. The building at 4000 Nelson Rd. was previously occupied by pharmaceutical companies AstraZeneca and Amgen. He said the available talent was also a factor.

AveXis retained most of the employees from the previous tenants. With new hires, the Longmont facility currently has a staff of around 300 employees and expects to grow to 400 by the end of 2020.

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Gene therapy company begins operations in Longmont - The Denver Channel

Ultragenyx Pharmaceutical saw its shares jump around 27% in trading Friday after announcing positive top-line data out of its gene therapy trial.

Its a small number, just three patients that form part of a third cohort for the phase 1/2 study, as well as another small test but a longer-term look from the second cohort.

In cohort three testing the biotechs drug DTX301, an adeno-associated virus gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency, there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status.

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Meanwhile, in cohort two, one female patient saw a new response after a year. The biotech added that the two previously disclosed responders in cohort one and two also remain clinically and metabolically stable at 104 and 78 weeks, respectively. Across all nine patients dosed in the study, up to six patients have demonstrated a response, it said in a statement.

RELATED: BIO: In conversation with Emil Kakkis, Ultragenyx CEO

OTC deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme OTC. Excess ammonia, which is a neurotoxin, travels to the central nervous system through the blood,

According to the National Organization for Rare Disorders, the severity and age of onset of OTC deficiency vary from person to person, even within the same family. A severe form of the disorder affects some infants, typically males, shortly after birth (neonatal period). A milder form of the disorder affects some children later in infancy. Both males and females may develop symptoms of OTC deficiency during childhood. Most carrier females are healthy, but may be prone to severe headaches following protein intake.

Analysts at Jefferies said the data looked better than expected and could be a positive spark to help turn the stock heading into 2020 events. It certainly did in the immediate term, with the biotechs shares up by 27% in mid-morning trading Friday.

We are encouraged to see a more uniform response at the higher doses including three female responders. To date, three patients in the study have discontinued alternate pathway medication and liberalized their diets while remaining clinically and metabolically stable, said Eric Crombez, M.D., chief medical officer of the Ultragenyx Gene Therapy development unit.

We are moving to prophylactic steroid use in the next cohort as we believe this could further enhance the level and consistency of expression that we have demonstrated so far.

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Ultragenyx shares jump on 'better than expected' gene therapy data - FierceBiotech