Category Archives: Genetic Therapy

SOUTH SAN FRANCISCO, Calif., Feb. 11, 2020 /PRNewswire/ --Encoded Therapeutics, Inc.(Encoded), a precision gene therapy company,today announced the appointment of Salvador Rico, M.D., Ph.D., as chief medical officer and the promotion of Martin Moorhead, Ph.D., to chief technology officer. Dr. Rico joins Encoded from Audentes Therapeutics, where he led clinical development of the company's pipeline of gene therapies for neuromuscular disorders. In his three years at Encoded, Dr. Moorhead has guided the development of the company's technology platform for creating innovative AAV-based gene therapies. He previously led the development of clonoSEQ, the FDA-approved next-generation sequencing assay for detecting minimal residual disease in lymphoid malignancies, at Adaptive Biotechnologies.

"Sal is an accomplished physician-scientist with deep experience advancing novel therapeutics through clinical development, and Martin is a strong leader who brings a genomics mindset to all aspects of gene therapy development," said Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D."With these appointments, we now have some of the most qualified gene therapy experts in the industry with a proven track record of delivering for patients in need. Their collective experience includes bringing multiple AAV-based gene therapies through clinical development, FDA filings, and approval. I am more confident than ever that our novel gene therapies can make a major impact on patients suffering from debilitating diseases, starting with Dravet Syndrome."

At Encoded, Dr. Rico will lead medical strategy and clinical development of ETX101, which is being developed for patients with SCN1A+ Dravet Syndrome. Dr. Moorhead will lead the technical team that enables Encoded's innovative research platform.

"I am delighted to join an organization that is so committed to transforming patients' lives with the development of next-generation gene therapies," said Dr. Rico. "I look forward to working closely with both the team at Encoded, and with the Dravet Syndrome community, to advance ETX101 through clinical development and ultimately, deliver it to patients in need."

"In building a technology platform that combines the power of genomics and computation with AAV-based gene therapy, Encoded is forging the path for the next generation of precision genetic medicines," said Dr. Moorhead. "I am very proud of what we have accomplished to date and am thrilled at the opportunity to help advance multiple programs for diseases where no treatment options currently exist."

New Leadership Team Appointments

About Encoded

Encoded Therapeutics, Inc., is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. We are advancing our lead asset, ETX101, for the treatment of SCN1A-positiveDravet Syndrome. For more information, please visitwww.Encoded.com.

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SOURCE Encoded Therapeutics, Inc.

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Encoded Therapeutics Expands Gene Therapy Leadership with Key Appointment and Promotion - PRNewswire

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

Ari Berkowitz, University of Oklahoma

(The Conversation is an independent and nonprofit source of news, analysis and commentary from academic experts.)

Ari Berkowitz, University of Oklahoma

(THE CONVERSATION) An emergency room physician, initially unable to diagnose a disoriented patient, finds on the patient a wallet-sized card providing access to his genome, or all his DNA. The physician quickly searches the genome, diagnoses the problem and sends the patient off for a gene-therapy cure. Thats what a Pulitzer prize-winning journalist imagined 2020 would look like when she reported on the Human Genome Project back in 1996.

A new era in medicine?

The Human Genome Project was an international scientific collaboration that successfully mapped, sequenced and made publicly available the genetic content of human chromosomes or all human DNA. Taking place between 1990 and 2003, the project caused many to speculate about the future of medicine. In 1996, Walter Gilbert, a Nobel laureate, said, The results of the Human Genome Project will produce a tremendous shift in the way we can do medicine and attack problems of human disease. In 2000, Francis Collins, then head of the HGP at the National Institutes of Health, predicted, Perhaps in another 15 or 20 years, you will see a complete transformation in therapeutic medicine. The same year, President Bill Clinton stated the Human Genome Project would revolutionize the diagnosis, prevention and treatment of most, if not all, human diseases.

It is now 2020 and no one carries a genome card. Physicians typically do not examine your DNA to diagnose or treat you. Why not? As I explain in a recent article in the Journal of Neurogenetics, the causes of common debilitating diseases are complex, so they typically are not amenable to simple genetic treatments, despite the hope and hype to the contrary.

Causation is complex

The idea that a single gene can cause common diseases has been around for several decades. In the late 1980s and early 1990s, high-profile scientific journals, including Nature and JAMA, announced single-gene causation of bipolar disorder, schizophrenia and alcoholism, among other conditions and behaviors. These articles drew massive attention in the popular media, but were soonretractedorfailedattemptsatreplication. These reevaluations completely undermined the initial conclusions, which often had relied on misguided statistical tests. Biologists were generally aware of these developments, though the follow-up studies received little attention in popular media.

There are indeed individual gene mutations that cause devastating disorders, such as Huntingtons disease. But most common debilitating diseases are not caused by a mutation of a single gene. This is because people who have a debilitating genetic disease, on average, do not survive long enough to have numerous healthy children. In other words, there is strong evolutionary pressure against such mutations. Huntingtons disease is an exception that endures because it typically does not produce symptoms until a patient is beyond their reproductive years. Although new mutations for many other disabling conditions occur by chance, they dont become frequent in the population.

Instead, most common debilitating diseases are caused by combinations of mutations in many genes, each having a very small effect. They interact with one another and with environmental factors, modifying the production of proteins from genes. The many kinds of microbes that live within the human body can play a role, too.

Since common serious diseases are rarely caused by single-gene mutations, they cannot be cured by replacing the mutated gene with a normal copy, the premise for gene therapy. Gene therapy has gradually progressed in research along a very bumpy path, which has included accidentally causing leukemia and at least one death, but doctors recently have been successful treating some rare diseases in which a single-gene mutation has had a large effect. Gene therapy for rare single-gene disorders is likely to succeed, but must be tailored to each individual condition. The enormous cost and the relatively small number of patients who can be helped by such a treatment may create insurmountable financial barriers in these cases. For many diseases, gene therapy may never be useful.

A new era for biologists

The Human Genome Project has had an enormous impact on almost every field of biological research, by spurring technical advances that facilitate fast, precise and relatively inexpensive sequencing and manipulation of DNA. But these advances in research methods have not led to dramatic improvements in treatment of common debilitating diseases.

Although you cannot bring your genome card to your next doctors appointment, perhaps you can bring a more nuanced understanding of the relationship between genes and disease. A more accurate understanding of disease causation may insulate patients against unrealistic stories and false promises.

[ Youre smart and curious about the world. So are The Conversations authors and editors. You can read us daily by subscribing to our newsletter. ]

This article is republished from The Conversation under a Creative Commons license. Read the original article here: https://theconversation.com/why-sequencing-the-human-genome-failed-to-produce-big-breakthroughs-in-disease-130568.

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Why sequencing the human genome failed to produce big breakthroughs in disease - Jacksonville Journal-Courier

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.

9 steps your organization can take on the path toward personalized medicine

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%...

Building sufficient financial resources to retire early may sound like a dream, but making that dream come true is not as hard as it may sound. The main thing is simply to save more money each month. No big deal, right? Well...

Typically, advisors peg 15% to 20% of total income saved each month as a goal - but if you want to retire earlier, you probably have to ratchet that number up to 40% or 50% of your income. Not a feat easily accomplished when you review your take into account that a good portion of your paycheck goes to essential, non-negotiable lifestyle items. However, if you are willing to make some serious lifestyle changes and sacrifices, it's possible.

A relatively new movement called Financial Independence, Retire Early (FIRE) has been developed around this "sacrifice and over-save now to retire early" concept. FIRE followers develop strict savings programs (up to 75% of income) and make associated sacrifices like living in small apartments, walking to work every day, restrictive diets, and so on. This path may be too restrictive for many, but the mindset offers some takeaways that might be worth considering.

The first point is to adhere to the key principles of long-term investing, including developing a diversified portfolio that includes stocks with various styles, sizes, sectors and regions.

To accelerate the retirement investment cycle, you can construct a portfolio designed with more risk - and the potential for higher returns - but it should still be appropriately diversified to protect against larger than average market drawdowns that can be difficult to recover from and ruin any chance to accomplish your early retirement goal. There are numerous ways to diversify a portfolio, and how you do so should depend on your age, your risk tolerance, your growth and income needs, and your long-term goals.

Once you've begun saving at a higher rate and you have an investment plan, put that money to work in your plan as quickly as you can. Don't worry about finding the "perfect time" to invest - simply put the money in and keep it in. Let compounding work to help you grow your retirement savings at an exponential rate.

Growth stocks with low beta, strong earnings estimates, positive sales growth, and expected future growth are an excellent way to determine investable growth stocks for your retirement.

The Zacks Rank routinely recognizes lower risk growth retirement portfolio picks, and here are a few that may be worth considering: Flushing Financial (FFIC), First Defiance Financial (FDEF) and Banco Santander-Brazil (BSBR). These growth stocks have strong Zacks Ranks and a beta of 1 or lower, with earnings and sales growth of at least 5% over the past 5 years.

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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