Category Archives: Gene Medicine

Nov. 2, 2019 13:30 UTC

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Veracyte, Inc. (Nasdaq: VCYT) today announced new data that advance understanding of the frequency, positive predictive value and co-occurrence of genomic alterations that are targeted by newly available and investigational precision medicine therapies for thyroid cancer. The findings were enabled by Afirma Xpression Atlas analyses, which uses RNA sequencing, of Veracytes extensive biorepository of thyroid nodule fine needle aspiration (FNA) samples from patients undergoing evaluation for thyroid cancer. The data were presented this week during the 89th Annual Meeting of the American Thyroid Association (ATA).

In one study, researchers assessed the frequency of ALK, BRAF, NTRK and RET fusions in nearly 48,000 consecutive patients whose thyroid nodule FNA samples were deemed indeterminate, suspicious for malignancy or malignant (Bethesda III/IV, V and VI categories, respectively) by cytopathology. The researchers found that 425 (0.89 percent) of the FNA samples harbored one of the alterations, with NTRK fusions the most common at 0.38 percent, followed by RET (0.32 percent), BRAF (0.13 percent) and ALK (0.06 percent). Additionally, RNA whole transcriptome sequencing demonstrated differences in the prevalence of these four fusions across Bethesda categories, with Bethesda V being the highest.

NTRK fusion inhibitors have received pan-cancer FDA approval and clinical trials have included selective inhibitors of ALK, BRAF, NTRK and RET, which makes their detection in patients with thyroid cancer of interest to physicians, said Mimi I. Hu, M.D., professor at The University of Texas MD Anderson Cancer Center, who presented the findings in a poster. As our understanding of the role of genomics in thyroid cancer advances, this information offers the potential to optimize initial treatment, predict response to treatment and prioritize selective targeted therapy should systemic treatment be needed.

In another study, researchers evaluated the positive predictive value of the NTRK, RET, BRAF and ALK fusions in 58 patients with indeterminate thyroid nodules (Bethesda III/IV categories) from Veracytes biorepository for whom surgical pathology diagnoses were available. They found that NTRK and RET fusions were associated with malignancy in 28 of 30 nodules, while risk of malignancy was lower among nodules with ALK (67 percent) or BRAF (75 percent). In a third study, researchers found that when using RNA sequencing data on a large sample of nearly 48,000 thyroid nodule FNA samples (Bethesda categories III-VI), they identified 263 co-occurrences of gene fusions and variants that were previously considered mutually exclusive.

The findings from these three studies underscore the power of our extensive biorepository of thyroid nodule FNA samples and our optimized RNA sequencing platform to advance understanding of the genomic underpinnings of thyroid cancer and to better capture the biology of thyroid lesions, said Richard T. Kloos, M.D., senior medical director, endocrinology, at Veracyte. As precision medicine therapies that target specific gene alterations emerge, understanding individual patients genomic profiles becomes increasingly important to physicians. Our Afirma Xpression Atlas provides this information at the same time as initial diagnosis with the Afirma Genomic Sequencing Classifier, or GSC, to help inform treatment decisions.

Also during the ATA meeting, Veracyte unveiled its new Afirma patient report, which in addition to identifying patients with benign or suspicious-for-cancer nodules among those deemed indeterminate by cytopathology, based on Afirma GSC results, now provides individualized and actionable variant and fusion information on each patient. This information includes: risk of malignancy, associated neoplasm type, relative risk of lymph node metastasis and extrathyroidal extension; availability of FDA-approved therapy; and genetic counseling and germline testing considerations. This information is also provided for patients with cytopathology results that are suspicious for malignancy or malignant (Bethesda V and VI).

About Afirma

The Afirma Genomic Sequencing Classifier (GSC) and Xpression Atlas provide physicians with a comprehensive solution for a complex landscape in thyroid nodule diagnosis. The Afirma GSC was developed with RNA whole-transcriptome sequencing and machine learning and helps identify patients with benign thyroid nodules among those with indeterminate cytopathology results in order to help patients avoid unnecessary diagnostic thyroid surgery. The Afirma Xpression Atlas provides physicians with genomic alteration content from the same fine needle aspiration samples that are used in Afirma GSC testing and may help physicians decide with greater confidence on the surgical or therapeutic pathway for their patients. The Afirma Xpression Atlas includes 761 DNA variants and 130 RNA fusion partners in over 500 genes that are associated with thyroid cancer.

About Veracyte

Veracyte (Nasdaq: VCYT) is a leading genomic diagnostics company that improves patient care by providing answers to clinical questions that inform diagnosis and treatment decisions without the need for costly, risky surgeries that are often unnecessary. The company's products uniquely combine RNA whole-transcriptome sequencing and machine learning to deliver results that give patients and physicians a clear path forward. Since its founding in 2008, Veracyte has commercialized seven genomic tests and is transforming the diagnosis of thyroid cancer, lung cancer and idiopathic pulmonary fibrosis. Veracyte is based in South San Francisco, California. For more information, please visit http://www.veracyte.com and follow the company on Twitter (@veracyte).

Cautionary Note Regarding Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "expect," "believe," "should," "may," "will" and similar references to future periods. Examples of forward-looking statements include, among others, the ability of Veracytes Afirma Xpression Atlas to analyze FNA samples to help diagnose thyroid cancer, the expected impacts of Veracytes collaboration with Johnson & Johnson in developing interventions for lung cancer, on Veracytes financial and operating results, on the timing of the commercialization of the Percepta classifier, and on the size of Veracytes addressable market. Forward-looking statements are neither historical facts nor assurances of future performance, but are based only on our current beliefs, expectations and assumptions. These statements involve risks and uncertainties, which could cause actual results to differ materially from our predictions, and include, but are not limited to: our ability to achieve milestones under the collaboration agreement with Johnson & Johnson; our ability to achieve and maintain Medicare coverage for our tests; the benefits of our tests and the applicability of clinical results to actual outcomes; the laws and regulations applicable to our business, including potential regulation by the Food and Drug Administration or other regulatory bodies; our ability to successfully achieve and maintain adoption of and reimbursement for our products; the amount by which use of our products are able to reduce invasive procedures and misdiagnosis, and reduce healthcare costs; the occurrence and outcomes of clinical studies; and other risks set forth in our filings with the Securities and Exchange Commission, including the risks set forth in our quarterly report on Form 10-Q for the quarter ended September 30, 2019. These forward-looking statements speak only as of the date hereof and Veracyte specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

Veracyte, Afirma, Percepta, Envisia and the Veracyte logo are trademarks of Veracyte, Inc.

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Veracyte Announces New Data That Advance Understanding of Genomic Alterations Targeted by Precision Medicine Therapies for Thyroid Cancer - BioSpace

Type 2 diabetes affects almost 400 million people across the world. Diabetes is caused by a combination of lifestyle as well as genetic factors which together result in high blood sugar levelsOne such genetic factor that can influence blood sugar levels is a variation in a gene called SLC30A8, which encodes a protein which carries zinc.

This protein is important, because zinc is essential for ensuring that insulin, (the only hormone that can reduce blood sugar levels) has the right shape in the beta-cells of the pancreas.

Researchers have known for almost ten years that changes in this gene can reduce the risk of getting type 2 diabetes, but not how this happened. They now recruited new members from families with a rare mutation in the SLC30A8 gene to study how they responded to sugar in a meal.

Departmental chief doctor Tiinamaija Tuomi from the Helsinki University Hospital, who co-led the study, said: A definite strength of our study is we could study families. We could compare people with the mutation with their relatives who do not have the mutation, but who have similar genetic background and life-style.

This way, we could make sure that the effects we were seeing were definitely because of this gene, and not because of another genetic or life-style factor.

The results showed that people with the mutation have higher insulin and lower blood sugar levels, reducing their risk for diabetes.

An international collaboration of 50 researchers also studied pancreatic cells with and without the mutation in the lab and carried out experiments in mice and human cellular material to understand exactly what was happening when the function of the SLC30A8 gene changed.

Professor Anna Gloyn, who co-led the study, from the Wellcome Centre for Human Genetics, University of Oxford, said: The work is a collaborative effort bringing pharma and academia together and researchers from multiple European Countries. It is a tour de force, since we were able to measure the impact of the mutation in many different systems, including human beta-cells.

Dr Benoit Hastoy, co-first author from the Oxford Centre for Diabetes, Endocrinology & Metabolism, University of Oxford said: We found that this mutation had collateral consequences on key functions of pancreatic beta cells and during their development. Importantly, this study exposes the extraordinary molecular complexity behind a specific gene variation conferring risk or protection from type 2 diabetes.

Taken together, the human and model system data show enhanced glucose-stimulated insulin secretion combined with enhanced conversion of the prehormone proinsulin to insulin as the most likely explanation for protection against type 2 diabetes, said Om Prakash Dwidedi, the co- first author of the study from the Institute for Molecular Medicine Finland (FIMM), University of Helsinki.

Better understanding of the genetic and pathological mechanism behind diabetes can open up new ways of preventing or treating type 2 diabetes.

Professor Leif Groop from the University of Helsinki and the Lund University who directed the study, added: Our results position this zinc transporter as an appealing and safe target for antidiabetic therapies. If a drug can be developed that mimics the protective effect of this mutation, beta-cell function could be preserved and the insulin secretion capacity in diabetic patients maintained.

Read more:
New study decodes gene function that protects against type 2 diabetes - Health Europa

Following the successful fundraising for Zente and Levente, two Hungarian toddlers suffering from SMA (spinal muscular atrophy) for their 700 million HUF treatment, themost expensive medicine in the world, Hungary has come together again for a third boy suffering from SMA Noel, who lives in rkrtvlyes in Bihar county, Romania.

Noels mother reported on a Facebook page created for the baby, who is only a few months old, is suffering from the same illness as the two other boys who recently received the support of a whole country, which made it possible for them to receive treatment. The family has created a foundation, but people can donate to multiple bank accounts as well.

Fundraiser Set up for Another Toddler to Receive Worlds Most Expensive Medicine

The mother emphasized that she would like to point out that I not only ask Hungarian people and those living in Hungary to help. We created the site in Hungarian because it is our mother tongue and because there are many helpful Hungarian people living here in Romania as well. Of course, translations are being made.

They have already taken the necessary steps and are waiting for an approval for Noel to receive another medicine, Spinraza, which will help him to develop, and for his condition to not further deteriorate. The Spinraza injection is financed completely by the National Health Insurance Fund (NEAK). The vaccine was patented just last year. This treatment is not cheap either, as an injection costs 23 million HUF, but the cost is entirely borne by NEAK. However, this medicine is needed by patients for the rest of their lives.

Fundraising for Toddlers Expensive Treatment Moves Hungary

This is why they have also set up fundraising to receive a medicine which is only needed once to improve the babys condition. However, this expensive medicine, called Zolgensma, is currently the most expensive medicine in the world and it has only been on the market since May.

SMA-1 is an extremely rare genetic disorder which affects only one in eight to ten thousand people. Because of a defective gene, their body does not produce the protein that protects muscle cells, so their muscles slowly deteriorate. Symptoms of SMA-1 usually occur during the first months of the patients life. In most cases, due to respiratory paralysis, children do not reach age two. There are approximately 120 SMA patients officially registered in Hungary (this applies to all types of SMA, not just SMA-1) but due to the outdated registration system, professionals say that the actual number is around 300.

The essence of the treatment is that the patients are given a virus by gene therapy that infects and replaces the gene pool of defective or missing motor neurons, thus preventing muscular atrophy.In Hungary, another treatment may have given hope to SMA patients as of last year, but for the time being, it is only funded on a case-by-case basis, exclusively for children.

featured photo: Noel and his family. (photo:Kicsi Noel SMA 1 Facebook)

Continued here:
Fundraising Started for Another Baby with SMA to Receive World's Most Expensive Medicine - Hungary Today

Alignment With FDA on GMP Commercial Manufacturing Process for KB103 (Bercolagene telserpavec, B-VEC)KB105 Granted Fast Track Designation by the FDAPlatform Patent for Delivering any Effector to the Skin Granted by USPTO

PITTSBURGH, Nov. 04, 2019 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (Krystal) (KRYS), a gene therapy company developing medicines to treat dermatological diseases, announced financial results for third quarter 2019, recent corporate highlights and upcoming milestones.

"Following our CMC meeting, we believe we have a scalable GMP commercial process in place to fulfill future patient demand and anticipate a modest impact to our previously disclosed B-VEC timeline, said Krish S. Krishnan, chairman and chief executive officer of Krystal Biotech. We plan on announcing our agreement with the FDA on trial design and endpoints prior to initiating the B-VEC pivotal trial.

Corporate Highlights

CMC alignment and End of Phase 2 meeting

Positive results from Phase 1/2 trial of B-VEC

KB105

Patents

Pipeline

Financial results for the quarter endedSeptember 30, 2019

For additional information on the Companys financial results for the quarter ended September 30, 2018, refer to form 10-Q filed as with the SEC.

About KB103KB103 is Krystals lead product candidate, currently in clinical development, seeks to use gene therapy to treat dystrophic epidermolysis bullosa, or DEB, an incurable skin blistering condition caused by a lack of collagen in the skin.KB103 is a replication-defective, non-integrating viral vector that has been engineered using the HSV-1 virus employing Krystals STAR-D platform to deliver functional human COL7A1 genes directly to the patients dividing and non-dividing skin cells.Krystals vector can penetrate skin cells more efficiently than other viral vectors.Its high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.

About Dystrophic Epidermolysis Bullosa, or DEBDystrophic epidermolysis bullosa, or DEB, is an incurable, often fatal skin blistering condition caused by a lack of collagen protein in the skin. It is caused by mutations in the gene coding for type VII collagen, or COL7, a major component of the anchoring fibrils, which anchor the epidermis to the underlying dermis, and provide structural adhesion in a normal individual. The lack of COL7 in DEB patients causes blisters to occur in the dermis as a result of separation from the epidermis.This makes the skin incredibly fragile, leading to blistering or skin loss at the slightest friction or knock. It is progressive and incredibly painful.

The most severe form of DEB is recessive DEB, or RDEB, which is caused by null mutations in the COL7A1 gene.DEB also occurs in the form of dominant DEB, or DDEB, which is considered to be a milder form of DEB.There are no known treatments which affect the outcome of either form of the disease, and the current standard of care for DEB patients is limited to palliative treatments.Krystalis developing KB-103 for the treatment of the broad DEB population, including both recessive and dominant forms of the disease.

About KB105KB105 is Krystals second product candidate, currently in clinical development, seeks to use gene therapy to treat patients with TGM-1 deficient ARCI. KB105 is a replication-defective, non-integrating viral vector that has been engineered employing Krystals STAR-D platform to deliver functional human TGM-1 gene directly to the patients dividing and non-dividing skin cells.

About Autosomal Recessive Congenital IchthyosisTransglutaminase 1 (TGM-1) is an essential epidermal enzyme that facilitates the formation of the epidermal barrier, which prevents dehydration, and protects the skin from unwanted toxins and surface microorganisms. The loss of TGM-1-activity results in the severe genetic skin disease autosomal recessive congenital ichthyosis (ARCI). Most patients with a TGM-1-deficiency exhibit life-long pronounced scaling with increased trans epidermal water loss (TEWL). The scales are plate-like, often of a dark color, and cover the whole-body surface area. Erythroderma is either absent or minimal. Such patients usually have ectropion and, at times, eclabium, hypoplasia of joint and nasal cartilage, scarring alopecia, especially at the edge of the scalp, and palmoplantar keratoderma. Additional complications include episodes of sepsis, fluid and electrolyte imbalances due to impaired skin barrier function, and failure to thrive, especially during neonatal period and infancy. Severe heat intolerance, and nail dystrophy are also frequently observed. TGM-1-deficient ARCI is associated with increased mortality in the neonatal period and has a dramatic impact on quality of life. No efficient treatment is available; current therapy only relieves some symptoms. There are approximately 23,000 cases of TGM1 deficient ARCI worldwide and about 400 new cases per year globally.

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About the STAR-D Gene Therapy PlatformKrystalhas developed a proprietary gene therapy platform, the Skin TARgeted Delivery platform, or STAR-D platform, that consists of an engineered HSV-1 viral vector and skin-optimized gene transfer technology, to develop off-the-shelf treatments for dermatological diseases. We believe that the STAR-D platform provides an optimal approach for treating dermatological conditions due to the nature of the vector. Certain inherent features of the HSV-1 virus, combined with the ability to strategically modify the virus in the form employed as a gene delivery backbone, provide the STAR-D platform with several advantages over other viral vector platforms for use in dermatological applications.

AboutKrystal BiotechKrystal Biotech, Inc.(KRYS) is a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases. For more information, please visithttp://www.krystalbio.com.

Forward-Looking StatementsThis press release includes certain disclosures that contain forward-looking statements, including, without limitation, statements regarding development timelines for KB103, the ability of KB103 to be a transformative treatment option for DEB patients and the ability of our Ancoris manufacturing facility to supply KB103 for the forthcoming clinical trial . You can identify forward-looking statements because they contain words such as anticipate, believes and expects. Forward-looking statements are based on Krystals current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees nor assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Krystals filings with theSecurities and Exchange Commission, including its registration statement on Form S-3, and in its Forms 10-K and 10-Q, as modified or supplemented from time to time, under the caption Risk Factors.

CONTACTS:

Investors:Ashley R. RobinsonLifeSci Advisorsarr@lifesciadvisors.com

Media:Darren Opland, PhD LifeSci Public Relationsdarren@lifescipublicrelations.com

Source: Krystal Biotech, Inc.

Link:
Krystal Biotech Reports Third Quarter 2019 Financial and Operating Results - Yahoo Finance

Ger Brophy, PhDExecutive Vice PresidentBiopharma ProductionAvantor

Genuine progress is being made in the longstanding battle to effectively treat and control cancer. The National Cancer Institute projects that nearly five million more U.S. citizens are expected to survive cancer in 2029 than in 2019.1 Therapeutic tools such as next-generation sequencing and advances in immunotherapy are just two ways that fundamental scientific breakthroughs and innovative thinking are realizing the potential for new cancer treatments.

One of the most revolutionary breakthroughs in this new era is cell and gene therapy. At its most basic definition, gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patients cells as a drug to treat disease. According to a paper published in the Journal of Gene Medicine, somewhere around 2600 gene therapy clinical trials had been undertaken in 38 countries around the world as of November 2017.2

These clinical trials demonstrate that the recent attention being paid to gene and cell therapy is not just hype. Some have noted that a select number of approved cell and gene therapies are for relatively small patient groups. However, its exciting to see the number of trials grow, especially when one considers this technologys ability to impact patients lives.

Its true that the number of patients receiving treatment is relatively small compared to other therapeutic regimens, but thats to be expected. Many of the biopharmaceutical researchers and manufacturers started with smaller, defined patient populations and, in particular, those with pediatric relapse refractory acute lymphoblastic leukemia. In part, these early efforts were directed at this type of cancer because the researchers wanted to deal with small populations that they understood well and, in many cases, had few or no other options for treatments.

The success of these initial efforts has led to broader programs targeting larger populationsstarting with leukemia and lymphoma. Ultimately, the most challenging opportunityand the one with the greatest potential for beneficial outcomesis multiple myeloma. If these patients begin to see benefits from cell and gene therapies, it will justify the incremental approach the industry has been taking.

The genuine, almost unprecedented potential for cell and gene therapy cannot be understated. For the first time, people are talking about curing these ruthless, relentless diseases. In a way never before possible, were taking control of and harnessing the patients own immune system to fight these cancers. Among the first patients treated for acute lymphoblastic leukemia, several are alive and thrivingfour, five, and six years later.

The game changer here is that cell and gene therapy uses the bodys own systems, either the cellular immune system or the ability to repair and replace defective or missing genes. CAR T-cell therapy is arguably among the most personalized medicines one can consider. The patients own T cells are extracted, modified, activated, expanded, purified, and returned to the patient.

Significant growth is underway in the size and sophistication of companies and organizations entering the cell and gene therapy markets. Many of the early movers in cell and gene therapy were small biotech startups. In some cases, their treatments were supported by major hospital centers.

Increasingly, weve all seen a greater interest from the major biopharma companies. Novartis was probably the biggest; it started earliest and was successful in getting approval for Kymriah. Since last year, weve seen several important acquisitions by Gilead and Bristol-Myers Squibb, and major biopharma companies are participating in large strategic partnerships in China. As companies of this size get involved, the hope is that they will leverage their increased breadth and depth to develop novel products, instigate new trials, and find ways to manufacture therapies at scale.

If the cell and gene therapy industry is to succeed, it must overcome challenges of two kinds: scalability and manufacturability. These challenges may be summarized in a set of questions: Can we manufacture cell and gene therapies at scale? If we can manufacture these treatments at scale, then can we do so safely? Can we do so at a reasonable cost so the populations that are affected by these diseases can access treatments?

With cell therapy, the single biggest point of variability is the patients own cells. By their very nature, these cells are individual to the patient, and their health implications for the patient should be considered as an integral part of cell processingat least as far back as the time of leukapheresis.

Variables and failure modes must be taken out of cell processing systems. We can standardize and miniaturize these systems, and we can enclose them so that theyre not exposed to failure modes. Also, we can improve technologies, like sterile fluid transfer, if we use excipient technology to further stabilize production. Finally, we can use analytical technology to understand the factors that contribute to a therapys success or failure.

Cell therapy producers and the companies that support and supply them need to become more innovative. In areas such as cell culture components, production chemicals, single-use technologies, sterile fluid transfer, and excipientsand the technology surrounding those process componentsthere is value to improving collaboration and trying new solutions to address the issues of manufacturability and scale.

We need to better analyze and understand the variability that comes from the research data, even at the early stages of these trials, and use it to correlate with clinical and process outcomes. Taking out manual steps as early as possible is important, as well as creating closed systems using sterile fluid transfer technologies.

One of the most significant challenges is finding solutions around side effects. As we understand how to provide a more efficacious dose, perhaps using fewer cells, some of the side effects of these drug therapies may improve. Furthermore, we must find scalable ways to reduce costs.

Ultimately, these drugs must be developed in a more cost-effective manner. Thats an area where technology providers and suppliers can play a significant role, by closing and automating systems and by understanding the contribution of labor and overhead and possible economies of scale from reducing processes.

There have been encouraging improvements in the way various regulatory groups have supported gene and cell therapy. To a certain degree, groups representing different regionsNorth America, Europe, and Asiahad been perceived to be setting precedents independently. More recently, it appears that regulatory bodies have been very open and collaborative in acknowledging that cell and gene therapies differ from more mature treatments such as biopharma drugs for cancer. The regulatory bodies have shown that they are willing to put the appropriate regulatory system into place to streamline the approval process and institute the ongoing monitoring of cell and gene therapies.

The U.S. FDAs support on CAR T-cell technologies is a good example. Regulators are allowing flexibility in the normal hierarchy of how clinical trials are performed, particularly in Phase II and III trials, but the companies must still address the FDAs postmarketing comments and safety issues.

Some have suggested that, ultimately, almost all cancer treatments will be based on gene and cell therapy approaches since they represent the most personalized form of treatment, which is, theoretically, the one with the highest potential for success.

Thats probably overly ambitious. Both large molecules and small molecules will continue to provide trusted, effective solutions with each type of drug product finding its niche. For example, large molecules are being developed for areas like neurodegeneration and are still offering great potential.

Its worth remembering that monoclonal-based therapies and biopharmaceuticals have really only started to make a significant impact in the last 15 to 20 years. Cell and gene therapies are just starting and have yet to make a significant market impact. But considerable effort is going into developing, understanding, and characterizing drug targets, as well as the development of technology to make targeted drugs in production-level volumes.

All these developments are exciting and offer a great deal of hope. It is clear that gene and cell therapies work and save lives. The challenge now is to scale their production. It is also clear that cell and gene therapies can emulate other therapeutic approaches that have transitioned from theoretical possibility to practical reality. As a similar transition occurs for cell and gene therapies, the production issues that need to be addressed will be seen more clearly, prompting action that will bring us to the next stage of development.

References1. Bluethmann SM, Mariotto AB, Rowland, JH. Anticipating the Silver Tsunami: Prevalence Trajectories and Comorbidity Burden among Older Cancer Survivors in the United States. Cancer Epidemiol. Biomarkers Prev. 2016; 25: 10291036.2. Ginn SL, Amaya AK, Alexander IE, et al. Gene therapy clinical trials worldwide to 2017: An update. J. Gene Med. 2018; 20(5): e3015.

Ger Brophy, PhD, is Executive Vice President, Biopharma Production at Avantor

Here is the original post:
Opportunities and Challenges in Cell and Gene Therapy Development - Genetic Engineering & Biotechnology News

Healthcare is an industry that is currently being transformed using the latest technology, so it can meet the challenges it is facing in the 21st century. Technology can help healthcare organizations meet growing demand and efficiently operate to deliver better patient care. Here are 9 technology trends that will transform medicine and healthcare in 2020.

The 9 Biggest Technology Trends That Will Transform Medicine And Healthcare In 2020

AI and Machine Learning

As the world population continues to grow, and age, artificial intelligence, and machine learning offer new and better ways to identify disease, diagnose conditions, crowdsource and develop treatment plans, monitor health epidemics, create efficiencies in medical research and clinical trials, and make operations more efficient to handle the increased demands on the healthcare system. By 2020, medical data will double every 73 days. McKinsey estimates that there could be $100 billion in annual savings for medicine and pharma by leaning on big data as well as the artificial intelligence and machine learning tools to process it. Artificial intelligence algorithms powered by recent advances in computational power learn from the data and can predict the probability of a condition to help doctors provide a diagnosis and treatment plans. Ultimately, AI and machine learning can assist with many clinical problems as long as governing and regulatory bodies can determine how to regulate the use of algorithms in healthcare.

Robotics

When it comes to life or death, would you trust a robot with yours? Currently, collaborative robotssuch as the da Vinci surgical robot are already assisting humans with tasks in the operating room. However, the potential for robots in healthcare expands beyond surgical uses. With tremendous growth expected in the industrythe global medical robotics market is expected to reach $20 billion by 2023theres no doubt that robots used in healthcare will continue to conduct more varied tasks. These already include helping doctors examine and treat patients in rural areas via telepresence," transporting medical supplies, disinfecting hospital rooms, helping patients with rehabilitation or with prosthetics, and automating labs and packaging medical devices. Other medical robots that are promising include a micro-bot that can target therapy to a specific part of the body, such as radiation to a tumor or clear bacterial infections.

Computer and Machine Vision

Training computers to "see" the world and understand visual input is no small feat. Since there has been significant progress in machine vision, there are more ways computers and machine vision are being used in medicine for diagnostics, viewing scans and medical images, surgery, and more. Machine vision is helping doctors definitively know how much blood a woman loses in childbirth so that appropriate care can be given to reduce the mortality of mothers from post-partum hemorrhaging. Computers provide accurate intel, while previously this was a guessing game. The applications where computers are being used to view CT scans to detect neurological and cardiovascular illnesses and spot tumors in X-ray images are growing rapidly.

Wearable Tech

Wearable fitness technology can do much more than tell you how many steps you walk each day. With more than 80% of people willing to wear wearable tech, there are tremendous opportunities to use these devices for healthcare. Today's smartwatches can not only track your steps but can monitor your heart rhythms. Other forms of wearable devices are ECG monitors that can detect atrial fibrillation and send reports to your doctor, blood pressure monitors, self-adhesive biosensor patches that track your temperature, heart rate, and more. Wearable tech will help consumers proactively get health support if there are anomalies in their trackers.

Genomics

Artificial intelligence and machine learning help advance genomic medicinewhen a person's genomic info is used to determine personalized treatment plans and clinical care. In pharmacology, oncology, infectious diseases, and more, genomic medicine is making an impact. Computers make the analysis of genes and gene mutations that cause medical conditions much quicker. This helps the medical community better understand how diseases occur, but also how to treat the condition or even eradicate it. There are many research projects in place covering such medical conditions as organ transplant rejection, cystic fibrosis, and cancers to determine how best to treat these conditions through personalized medicine.

3D Printing

Just as it's done for other industries, 3D printing enabled prototyping, customization, research, and manufacturing for healthcare. Surgeons can replicate patient-specific organs with 3D printing to help prepare for procedures, and many medical devices and surgical tools can be 3D printed. 3D printing makes it easier to cost-effectively develop comfortable prosthetic limbs for patients and print tissues and organs for transplant. Also, 3D printing is used in dentistry and orthodontics.

Extended Reality (Virtual, Augmented and Mixed Reality)

Extended reality is not just for entertainment; its being used for important purposes in healthcare. The VR/AR healthcare market should reach $5.1 billion by 2025. Not only is this technology extremely beneficial for training and surgery simulation, but it's also playing an important part in patient care and treatment. Virtual reality has helped patients with visual impairment, depression, cancer, and autism. Augmented reality helps provide another layer of support for healthcare practitioners and aided physicians during brain surgery and reconnecting blood vessels. In mixed reality, the virtual and real worlds are intertwined, so it provides important education capabilities for medical professionals as well as to help patients understand their conditions or treatment plans.

Digital Twins

A digital twin is a near real-time replica of something in the physical worldin healthcare, that replica is the life-long data record of an individual. Digital twins can assist a doctor in determining the possibilities for a successful outcome of a procedure, help make therapy decisions, and manage chronic diseases. Ultimately, digital twins can help improve patient experience through effective, patient-centric care. The use of digital twins in healthcare is still in its early stages, but its potential is extraordinary.

5G

As the capabilities for healthcare centers to provide care in remote or under-served areas through telemedicine increase, the quality and speed of the network are imperative for positive outcomes. 5G can better support healthcare organizations by enabling the transmission of large imaging files so specialists can review and advise on care; allow for the use of AI and Internet of Things technology; enhance a doctor's ability to deliver treatments through AR, VR and mixed reality; and allow for remote and reliable monitoring of patients.

These technologies offer incredible opportunities to provide better healthcare to billions of people and make help our healthcare systems cope with the ever-increasing demands.

More:
The 9 Biggest Technology Trends That Will Transform Medicine And Healthcare In 2020 - Forbes