Category Archives: Genetic Therapy

Millions of people are affected by blood disorders, and the prevalence is expected to grow as our population ages.

It is not surprising that, according to the American Society of Hematology, the FDA approved several new therapies or new indications for previously approved therapies in 2019 for people living with non-malignant blood disorders. Those included two disease-modifying treatments for sickle cell disease and the first anticoagulant for venous thromboembolism management in children.

In the wings and poised to reshape the treatment landscape for hematologic disorders are two potential blockbuster drugs - Valrox (valoctocogene roxaparvovec) for hemophilia A and vadadustat for anemia related to chronic kidney disease - that are among the 11 included in the Cortellis Drugs to Watch analysis predicted to achieve annual sales of $1 billion by 2024.

Hemophilia A

Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII, a clotting protein. People with that disorder are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. According to the U.S. CDC, the condition occurs in approximately one in 5,000 live births, and about 20,000 people are living with hemophilia in the U.S. Accurate data on the worldwide incidence of hemophilia is estimated at more than 400,000.

People suffering from the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints and that group makes up approximately 43% of the hemophilia A population. The standard of care (SOC) for such individuals is a prophylactic regimen of replacement factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite those treatments, many people continue to experience bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

One and done treatment

San Rafael, Calif.-based Biomarin Pharmaceutical Inc. has submitted a BLA to the FDA for its investigational AAV gene therapy, Valrox, for adults with hemophilia A. The therapy is designed to deliver functional copies of the FVIII gene into patients cells, enabling them to make the previously missing or defective FVIII protein. If approved, Valrox would be the first potentially curative (one and done) approach to hemophilia A, eliminating the need for blood transfusions and FVIII replacement therapy after a single infusion. Subject to completion of the agency's filing review, the company anticipates the BLA review to start in February.

The FDA has granted breakthrough therapy and orphan drug designations; and the EMA has validated the company's marketing authorization application with the review under accelerated assessment.

According to the Cortellis Drugs to Watch analysis, the filings were based on interim data from the phase III GENEr8-1 study as well as three-year phase I/II trial data. In GENEr8-1, Valrox met the prespecified criteria for U.S. and EU regulatory review, with eight patients in a 20-patient cohort achieving FVIII levels of at least 40 IU/dl at 23 to 26 weeks; the ongoing trial has the goal of evaluating superiority of Valrox to the current standard of care, prophylactic therapy. The high-dose cohort in the phase I/II study showed 100% resolution of target joints, a 96% reduction in mean annualized FVIII usage, and all patients remained off FVIII prophylaxis. No thrombotic events or development of FVIII inhibitors have been reported to date.

Transformative for patients

"People with severe hemophilia A continue to experience clinically relevant breakthrough bleeds despite the current standard of care and can be limited in their physical activities," noted John Pasi, chief investigator for the phase I/II study and a principal investigator for the phase III study. "Valoctocogene roxaparvovec represents a potentially transformative investigative therapy that could improve patients' quality of life, including consequences of bleeding, physical functioning, role functioning, emotional impact, treatment concern and worry."

In January, the company published three years of follow-up data in the phase I/II study showing the median use of exogenous factor VIII at the 6e13-vg/kg dose was reduced from 138.5 infusions per year to zero infusions per year in year three. In the year before study entry, the mean annualized number of factor VIII infusions per participant was 136.7+22.4; at the end of year three, the mean annualized use of exogenous factor VIII decreased by 96% to a mean of 5.5+9.4 infusions.

"As a treating physician, I am excited about the potential of the field of gene therapy to make a meaningful difference in the lives of people with hemophilia A," Pasi noted on the results.

First-mover advantage

In a presentation at the annual J.P. Morgan Healthcare Conference in January 2020, Biomarin Chairman and CEO Jean-Jacques Bienaime was excited to reveal that the company is ramping up productivity at its Novato, Calif., plant, more than doubling its capacity to 10,000 doses annually. He noted that the capacity upgrade is important as the firm wants to be able to supply the gene therapy market as quickly as possible "because first-mover advantage in gene therapy is fundamental in a sense that every time you treat a patient that patient is off the market."

Gene therapy certainly has the potential to revolutionize treatment for patients with hemophilia A. However, according to the Canadian Hemophilia Society, Never have so many coagulation therapies been in development. In addition to the large number of therapies recently introduced, we have identified another 18 new therapies in development or soon to be marketed, including six clotting factor concentrates, four bypassing therapies to treat patients with inhibitors, two non-factor coagulation products and five gene therapy products.

If approved, Valrox will certainly face competition from well-established FVIII replacement therapies, in addition to those that are nearing the market, the Cortellis analysis suggests.

Recently approved therapies such as Roche Holding AG's Hemlibra (emicizumab) and Bayer AG's Jivi (antihemophilic factor [recombinant] pegylated) are improving the options available to those patients. The hemophilia market is conservative in adopting new therapies, the analysis notes, and patients may be reluctant to switch to new products if their current replacement therapy works well. However, an unmet need remains, as approximately 75% of hemophilia A patients do not respond adequately to their treatment or even do not receive treatment at all. Despite a relatively low worldwide prevalence of patients, with orphan pricing, hemophilia A represents a large market.

In addition, Valrox is not without its own challenges, the Cortellis analysis notes. The drug fell short in the initial cohort of the phase III GENEr8-1 trial: of 17 evaluable patients, three failed to achieve FVIII levels above 5 IU/dl. The mean and median FVIII levels across the cohort were also lower than seen in phase I/II, at 33 and 36 IU/dl, respectively. Failure to improve the response once full data are available may impact the potential commercial uptake

The market

The global market for hemophilia A last year was estimated to be close to $10 billion, and Bienaime in his presentation said that an estimated 121,000 patients are located in the territories covered by the company. Although no pricing has yet been set for the gene therapy, the company has done a lot of payer research. He indicated that payers attribute a high value to the physiological correction of hemophilia A, and it appears the U.S. payer community would be comfortable with a price between $2 million and $3 million.

Given the fact that wholesale acquisition pricing for Hemlibra in non-inhibitor adult patients is between $600,000 to $800,000 per year, Biomarin has looked at pricing of between $1 million and $5 million. Since by launch date it will have four years of data on Valrox, the price could be set at four times the average price of Hemlibra ($700,000), establishing a price of $2.8 million. That would make it the most expensive one-time therapy, topping spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi) from Novartis AG at $2.1 million. The company says that this first gene therapy for any form of inherited hemophilia could save health care systems more than $20 million over a typical patients lifetime.

For that reason, even if the penetration is modest, Valrox could post significant revenue. The Cortellis analysis predicts sales of $17.45 million forecast for this year, rising to $1.297 billion in 2024. However, while Biomarin is in the pole position with its gene therapy, potential competition in the pipeline from FVIII gene therapies in development may temper sales forecasts in the mid- to long term. Spark Therapeutics Inc.'s SPK-8011 entered phase III development in February 2019, having shown a 94% reduction in bleeds and a 95% reduction in FVIII infusions in a previous phase I/II study. Other direct competitors in phase I/II development include Ultragenyx Pharmaceutical Inc.s DTX-201, Sparks SPK-8016, Shire plcs SHP-654 and University College London (UCL)/St. Jude Children's Research Hospitals AAV2/8-HLP-FVIII-V3.

Anemia in chronic kidney disease

Anemia is one of the many complications of chronic kidney disease (CKD), which worsens as kidney disease progresses; most patients whose CKD has progressed to kidney failure have significant anemia. It is estimated that CKD affects 200 million people worldwide. The anemia in CKD is currently treated with injectable recombinant erythropoiesis-stimulating agents (ESAs), which are often associated with inconsistent hemoglobin responses and safety risks.

The need for more effective and safer therapies has led to the discovery of hypoxia-inducible factor prolyl hydroxylase (HIF-PH) enzyme inhibitors, a new class of agents for the treatment of anemia in CKD (see sidebar story). Those agents work by stabilizing the HIF complex and stimulating endogenous erythropoietin production even in patients with end-stage kidney disease.

Several HIF-PH enzyme inhibitors are currently in development targeting the estimated $3.5 billion renal anemia market, including Cambridge, Mass.-based Akebia Therapeutics Inc.s vadadustat, which has advanced to late-stage clinical trials. In July, its strategic partner, Osaka-based Mitsubishi Tanabe Pharma Corp., filed an NDA with the Japanese Ministry of Health, Labour and Welfare, seeking approval for the product as a treatment for anemia due to CKD.

The filing is based on data from four studies in Japanese patients: an active-controlled study in non-dialysis-dependent CKD anemia (J01), another active-controlled study but in dialysis-dependent patients with CKD anemia (J03), and two single-arm studies in patients with peritoneal and hemodialysis-dependent CKD anemia (J02 and J04). Vadadustat demonstrated non-inferiority with respect to hemoglobin level versus the active comparator (darbepoetin alfa) in both dialysis- and non-dialysis-dependent patients (J01 and J03 studies; 11.66 vs 11.93 g/dL, and 10.61 vs 10.65 g/dL, respectively) and showed therapeutic effect in the single-arm studies.

If approved, the company expects commercial launch during mid-2020, and filings in the U.S. and the EU are planned, those territories covered by Akebias alliance with Otsuka Pharmaceutical Co., Ltd.

Competitors

Within the HIF-PH inhibitor class itself, and particularly from Fibrogen Inc.s Evrenzo (roxadustat), the first-in class HIF-PH inhibitor, vadadustat will face direct competition. Fibrogen has partnered with Astellas Pharma Inc. and Astrazeneca plc for the development and marketing of Evrenzo, which has been approved in Japan (in dialysis patients) and China (in dialysis- and nondialysis-dependent patients) in the third quarter of 2019. An additional filing in Japan in non-dialysis patients is expected in the short term upon completion of a second pivotal study in that setting. The drug has also been filed in the U.S (for both patient populations) and filings in the EU are expected by March, according to Cortellis, following positive top-line data from the ALPS (nondialysis-dependent) and HIMALAYAS (dialysis-dependent) studies.

The analysis indicates vadadustat would also face competition from other well-established therapeutic approaches in CKD such as intravenous iron replacement products and blood transfusions that offer rapid increases in Hb levels.

Future direct competition will also come from Glaxosmithkline plcs HIF-PH inhibitor daprodustat, which was submitted for approval in Japan in August last year.

The Cortellis analysis cites sales forecasts in 2024 of $1.188 billion for Evrenzo and $286 million for daprodustat. For vadadustat, sales of $2 million are forecast for this year, rising to $1.589 billion in 2024.

See a related article:

Out of basic science, a blockbuster: Vadadustat

To read more about the Cortellis Drugs to Watch potential blockbusters, visit BioWorlds collection of articles which are freely available.

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Therapies poised to reshape the treatment landscape for hematologic disorders - BioWorld Online

Drugmaker Novartis recently launched a lottery to help families access the novel gene therapy Zolgensma outside of the United Statesthe only country where it's currently on the market, but some have questioned whether a lottery is the most appropriate way to distribute the drug, Andrew Joseph and Ed Silverman report for STAT News.

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FDA last year approved Zolgensma to treat a spinal muscular atrophy (SMA), a genetic neuromuscular disease.

Patients with SMA produce an insufficient amount of SMN protein, which breaks down motor neurons that send messages to the muscles. In turn, those with the disease develop muscle weakness, which can lead to death. Without any treatment, children with Type 1 SMA, which is the most serious form and affects 1 in 15,000 babies, typically die by the age of two, mostly from respiratory problems, Joseph and Silverman report.

Zolgensma treats the condition by inserting a healthy copy of an SMN gene into the patient's motor neurons, which heals the neurons and restarts production of the SMN protein. The treatment is only needed once. In the United States, the drug is only approved for patients under age two.

However, access to the drug is currently limited.

The United States is the only country where regulators have approved the drug. According to Joseph and Silverman, European regulators are expected make an approval to decision this quarter, while officials in Canada and Australia may take until next year to make a decision.

In addition, the supply of Zolgensma is limited, according to Novartis, and the therapy is complicated to manufacture. On top of that, Novartis has just one facility making Zolgensma, Joseph and Silverman report. The company said it intends to open two more facilities to make this year.

The drug also has a $2.1 million price tag, which makes it the most expensive drug in the world, according to Joseph and Silverman. In countries where the drug is not approved, families have tried fundraising to buy the drug and get it injected by U.S. doctors, Joseph and Silverman report.

Novartis has said it will allow health insurers to pay for Zolgensma over the course of five years via annual installments of $425,000, but critics say the price is too high.

To help patients in countries where the drug is not yet approved, Novartis devised a lottery that will give some patients access to the drug for free through a compassionate use program, which enables patients to get unapproved medications. The company will give away 50 doses in the first six months of 2020 and plans to give away up to 100 doses total this year.

Some ethicists have said lotteries can be a fair way to dole out limited supplies of a resource, as they create an even playing field and keep those with more money or better connections from having an advantage over others. However, others questioned whether the lottery is truly fair, saying the company should favor the sickest children, as healthier children may be able to wait longer.

Holly Fernandez Lynch, a bioethicst at University of Pennsylvania's Perelman School of Medicine, said, "If it is really not possible to help all who are in need of help, then a lottery with priority to patients who are worst off is not a bad approachand definitely fairer than other things a company could do." She added, "The key is to first do everything possible to minimize the need for a lottery at alland it's not obvious to me that Novartis has done that here."

Genevieve Kanter, another bioethicist from the Perelman School, said she understands why some people feel uneasy about the lottery. "[I]t does become a zero-sum game, which is what bothers some people about the mechanism, even if at the end of the day, more kids get treated than in the alternate scenario where there's no lottery," Kanter said.

Dave Lennon, president of AveXis, the unit within Novartis that developed Zolgensma, said that Novartis thought about giving priority to the sickest patients, but after consulting with ethicists, the company decided they didn't want to influence who would receive the treatment in any way.

"It's the only fair way to allocate," Lennon said, acknowledging that it was "not an ideal situation." He added, "The alternative is not do anything, which we didn't feel like was a good option." According to Lennon, if supply allowed, Novartis wanted to expand the program (Joseph/Silverman, STAT News, 2/7).

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This life-saving infusion costs $2.1M. A new lottery will give out 100 doses for free. - The Daily Briefing

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 vector o Retroviruses o Lentiviruses o Adenoviruses o Adeno Associated Virus o Herpes Simplex Virus o Poxvirus o Vaccinia Virus o Others Non-viral vector o Naked/Plasmid Vectors o Gene Gun o Electroporation o Lipofection o 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 America o U.S. o Canada o Mexico Europe o Germany o UK o France o Spain o Italy o Rest of Europe Asia-Pacific o Japan o China o Australia o India o South Korea o Rest of Asia-Pacific LAMEA o Brazil o South Africa o Saudi Arabia o 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

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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%...

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Powerful Proof Anyone Can Invest for an Early Retirement - February 07, 2020 - Nasdaq

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

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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