Monthly Archives: March 2022

NEW YORK

U.S. regulators on Monday cleared the way for the sale of beef from gene-edited cattle in coming years after the Food and Drug Administration concluded the animals do not raise any safety concerns.

The cattle by Recombinetics are the third genetically altered animals given the green light for human consumption in the U.S., after salmon and pigs. Many other foods already are made with genetically modified ingredients from crops like soybeans and corn.

The cattle reviewed by the FDA had genes altered with a technology called CRISPR to have short, slick coats that let them more easily withstand hot weather. Cattle that arent stressed by heat might pack on weight more easily, making for more efficient meat production.

The company did not say when home cooks or restaurants might be able to buy the beef, but the FDA said it could reach the market in as early as two years.

Unlike the salmon and pigs, the cattle did not have to go through a years-long approval process. The FDA said the cattle were exempt from that because their genetic makeup is similar to that of other existing cattle and the trait can be found naturally in some breeds.

Dr. Steven Solomon, director of the FDAs Center for Veterinary Medicine, said the agencys review of Recombinetics cattle took several months. He said theres no reason that meat from the animals or their offspring would need to be labeled differently.

Solomon said a genetically altered animal marketed as having a special advantage such a higher than normal ability to withstand heat might need to go through the full approval process.

This opens up a completely different pathway, he said, noting the decision could be encouraging for other biotech companies, many of which are small startups.

The gene-edited trait in the Recombinetics cattle can be passed down so semen and embryos from them could be used to produce offspring with the same shorter coats.

The trait will make beef production more sustainable and could improve animal welfare in warmer climates, Recombinetics said in a statement without providing further details.

Greg Jaffe, who specializes in biotechnology at the Center for Science in the Public Interest, said the FDAs announcement made clear it wasnt exempting all gene-edited animals from the longer approval process.

They reinforce the idea that this is a case-by-case review, Jaffe said.

He said the agency should be more transparent about the review process so people know whats in the works. That could lead to better public acceptance and minimize any potential economic disruptions from global trade, since other countries might consider the animals genetically modified foods that need to be labeled, he said.

Jaydee Hanson, of the Center for Food Safety, said the agency should keep track of the animals for several generations to ensure there arent any unintended issues.

The genetically modified pig is intended mainly for medical purposes, not meat, according to the company that developed it. The firm recently provided a pig heart that was transplanted into a dying man in an experimental surgery.

The company behind the modified salmon said the fish are being sold to distributors in the Midwest and Northeast.

Alison Van Eenennaam, an animal geneticist at UC Davis who has worked with Recombinetics, said requiring all companies to go through the lengthy approval process could end the possibility of commercializing gene-edited animals in the U.S.

For the gene-edited cattle cleared by the FDA, she said it could take about two years for beef from the offspring to reach the market.

Once the semen is used to create embryos, she said gestation would take about nine months and the resulting calves might be slaughtered after about 10 months. She noted the market isnt limited to the U.S., given the way cattle are bred.

Lets face it, bull semen goes all over the world, she said.

Continued here:
Gene-edited beef cattle receive regulatory clearance in U.S. - Los Angeles Times

Image of the female mouse that gave birth via asexual reproduction.PNAS

Chinese scientists have managed to get a female mouse to produce live viable offspring from an unfertilized egg thanks to gene editing. This is in theory the first case of a mammal that has achieved asexual reproduction, technically known as parthenogenesis or virgin creation, which is common in many insects and reptiles, and even in some bird species. But until very recently, it was believed that mammals could not reproduce without male genetic material.

In 2004, the world learned about Kaguya, a mouse born without the need for sperm or sexual reproduction. Mammals have two copies of each gene in their genome one from their mother and one from their father. When a sperm cell penetrates an egg, something called genomic imprinting happens, meaning that some genes behave differently depending on whether they were inherited from the father or the mother.

Many of the genes involved in the process have to do with growth, which may mean, for evolutionary reasons, that the fathers imprint favors growth in order to have larger offspring, while the mothers may do the opposite to ensure that the pregnancy can be brought to term. Some of these genes are also associated with a greater risk of suffering cancer, among other diseases. The Japanese scientists who created Kaguya used two eggs: one worked as the real egg while the other one imitated the sperms contribution. The result was a baby that was born to two females.

The new study, led by Yanchang Wei, a reproductive medicine researcher at Ren Ji Hospital in Shanghai, took the concept a step further, creating a pregnancy from a single, unfertilized egg. Chinese scientists used a gene-editing technique very similar to CRISPR, which is used in medicine and biotechnology. The authors of the study used the tool to imitate the genomic imprint in seven different points of the genome of an egg that already had two copies of each gene. This triggered a biochemical process that was the equivalent of fertilization: the egg grew from one single cell to a 140-cell blastocyst.

Theyve managed something fascinating but terribly inefficient

The scientists implanted 192 embryos made with this technique in as many female mice, but only one of them gave birth to a healthy pup that survived, although it weighed less than average. Two others died shortly after birth. The survivor reached adulthood and was able to reproduce normally.

In their study, published in the scientific journal PNAS, researchers noted that parthenogenesis can be achieved by targeted epigenetic rewriting of multiple critical imprinting control regions. The team believes that the technique could be perfected to achieve a higher live birth success rate. The success of parthenogenesis in mammals opens many opportunities in agriculture, research, and medicine, says the study.

It is a groundbreaking study, agrees Lluis Montoliu, a researcher at Spains National Research Council (CSIC), who underscored that the eggs had a copy of their genes and another copy taken from another egg that came from a different species of mouse, allowing scientists to check the effects of the editing. In theory at least, further perfecting this technique might allow a woman to have children on her own, admits Montoliu. But the researcher adds that its use on humans is a very long way off. The main thing now is to follow the lives of these mice born through these techniques, and see whether they have problems with their lives and reproduction. There are still a lot of unanswered questions.

Theyve managed something fascinating but terribly inefficient, adds David Haig, a researcher at Harvard University who developed a theory about genomic imprinting several years ago.

English version by Susana Urra.

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Female mouse gives birth without sex or sperm - EL PAS in English

The first person to have his failing heart replaced with that of a genetically altered pig in a groundbreaking operation died Tuesday afternoon at the University of Maryland Medical Center, two months after the transplant surgery.

David Bennett Sr., who lived in Maryland, was 57. He had severe heart disease, and had agreed to receive the experimental pigs heart after he was rejected from several waiting lists to receive a human heart.

It was unclear whether his body had rejected the foreign organ. There was no obvious cause identified at the time of his death, a hospital spokeswoman said.

Hospital officials said they could not comment further on the cause of death, because his physicians had yet to conduct a thorough examination. They plan to publish the results in a peer-reviewed medical journal.

Dr. Bartley Griffith, the surgeon who performed the transplant, said the hospitals staff was devastated by the loss of Mr. Bennett.

He proved to be a brave and noble patient who fought all the way to the end, Dr. Griffith said. Mr. Bennett became known by millions of people around the world for his courage and steadfast will to live.

The heart transplant was one of a number of pioneering procedures in recent months in which organs from genetically altered pigs were used to replace organs in humans. The process, called xenotransplantation, offers new hope for tens of thousands of patients with ailing kidneys, hearts and other organs, as there is an acute shortage of donated organs.

Mr. Bennetts transplant was initially deemed successful. It is still considered a significant step forward, because the pigs heart was not immediately rejected and continued to function for well over a month, passing a critical milestone for transplant patients.

Some 41,354 Americans received a transplanted organ last year, more than half of them kidneys, according to the United Network for Organ Sharing, a nonprofit that coordinates the nations organ procurement efforts.

But there is a dire shortage of organs, and a dozen or more people on waiting lists die each day. About 3,800 Americans received human donor hearts last year as replacements, more than ever before, but demand remains high.

Scientists have been trying to produce pigs whose organs would not be rejected by the human body, a research effort that has picked up steam over the past decade because of new gene editing and cloning technologies.

New York surgeons announced in October that they had successfully attached a kidney grown in a genetically altered pig to a brain-dead human patient, finding that the organ worked normally and produced urine for 54 hours.

In January, surgeons at the University of Alabama at Birmingham reported that they had for the first time successfully transplanted kidneys from a genetically modified pig into the abdomen of a 57-year-old brain-dead man. The kidneys functioned and produced urine for three days.

U.A.B. surgeons said they hoped to launch a small clinical trial with live human patients by the end of the year.

Shortly after Mr. Bennetts heart surgery in January, The Washington Post reported that he had a criminal record stemming from an assault 34 years ago, in which he repeatedly stabbed a young man in a fit of jealousy, leaving him paralyzed.

The victim, Edward Shumaker, spent two decades in a wheelchair, paralyzed from the waist down, and suffered numerous medical complications including a stroke that left him cognitively impaired before he died in 2007 at age 40, according to his sister, Leslie Shumaker Downey, of Frederick, Md.

Mr. Bennetts son, David Bennett Jr., who was a child at the time of the stabbing, has said that he does not want to discuss his fathers past, and emphasized that his father was contributing to medical science by undergoing the experimental transplant and hoped to potentially save patient lives in the future.

The heart given to Mr. Bennett came from a genetically altered pig provided by Revivicor, a regenerative medicine company based in Blacksburg, Va.

The pig carried 10 genetic modifications. Four genes were knocked out, or inactivated, including one that encodes a molecule that causes an aggressive human rejection response.

Another gene was also inactivated to prevent the pigs heart from continuing to grow after it was implanted. In addition, six human genes were inserted into the genome of the donor pig modifications designed to make the pigs organs more tolerable to the human immune system.

On New Years Eve, the Food and Drug Administration granted an emergency authorization for the experimental surgery, which was done a week later.

The transplanted heart performed well initially, and there were no signs of rejection for several weeks. Mr. Bennett spent time with his family, did physical therapy and watched the Super Bowl, hospital officials said.

But he was not discharged, and several days ago his condition started to deteriorate, hospital officials said.

His son issued a statement thanking the hospital and staff for their exhaustive efforts on behalf of his father.

We hope this story can be the beginning of hope and not the end, Mr. Bennett said. We also hope that what was learned from his surgery will benefit future patients and hopefully one day, end the organ shortage that costs so many lives each year.

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Patient Who Recieved Groundbreaking Pig Heart Transplant Dies - The New York Times

LONDON--(BUSINESS WIRE)--Ixaka Ltd, an integrated cell and gene therapy company, announces that Advanced Therapy Medicinal Product (ATMP) classification has been granted by the European Medicines Agency (EMA) for its Chemically Encapsulated Lentiviral vector for Targeted In Vivo CAR T-cell therapy (CELTIC-19) targeted nanoparticle (TNP) product.

ATMP status was granted to CELTIC-19 due to its potential as a gene therapy medicinal product that targets specific cells and expression of the gene of interest directly within the patients body. Such an approach makes it possible to eliminate all the ex vivo stages of genetic modification, that are required for the production of currently marketed cell therapy products.

CELTIC-19 is Ixakas lead TNP program for CD19 haematological malignancies. It consists of a polymer nanoparticle encapsulating a bald lentiviral vector encoding for a T-cell specific promoter and the chimeric antigen receptor (CAR). The nanoparticle is coated with a CD3 binding molecule allowing in vivo targeting and transduction of the T-cells. The construct can then be infused systemically into the bloodstream to target and genetically modify T-cells within the body. This approach allows the generation of CAR T-cells which are potentially more efficacious, safer, and considerably less expensive to produce than established CAR T-cell therapies, which have been shown to be effective and have been approved for use in CD19-malignancies.

CELTIC-19s numerous advantages over established ex vivo CAR T-cells therapies, include but are not limited to its high specificity and transduction efficiency, retreatment possibility, no cytokine requirement and persistent expression.

Gilbert Wagener, Senior Vice President, Chief Medical Officer at Ixaka commented:

TNP-based in vivo CAR T-cell therapies such as Ixakas represent a significant advance over recently approved ex vivo CAR T-cell therapies, and hold the promise of delivering more effective, universal, and safer treatment option for patients. It is great to see this potential recognized by the Europeans Medicine Agency.

Joe Dupere, CEO at Ixaka commented:

Our nanoparticle-based in vivo gene delivery technology is ideally positioned to deliver on the promise of in vivo CAR-T therapies to transform cancer treatment without the need for costly dedicated manufacturing sites for T-cell modification. The designation of CELTIC-19 as an Advanced Therapy Medicinal Product further signifies its potential as a ground-breaking new treatment option and is an important step on our continued journey.

About Ixaka

Ixaka is a cell and gene therapy company focused on using the natural powers of the body to cure disease.

Ixakas proprietary technologies enhance the naturally therapeutic power of cells by increasing the presence of curative cells at the site of disease, or by directly modifying cells within the body to improve disease targeting and boost their restorative effect.

Ixakas technologies concentrated multi-cell therapies and nanoparticle therapeutics demonstrate potential for the treatment of a broad range of serious diseases across oncology, cardiovascular, neurological and ocular diseases, and genetic disorders.

Ixaka has offices in London, UK with R&D and manufacturing operations in Seville, Spain and Paris, France and additional manufacturing capability in Frankfurt, Germany.

For more information, please visit http://www.ixaka.com

Connect with us: Twitter: https://twitter.com/ixaka_Ltd; LinkedIn: https://www.linkedin.com/company/ixaka-limited/

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CELTIC-19 Granted Advanced Therapy Medicinal Product Classification by European Medicines Agency - Business Wire

In the United States, 1 in 10 people has type 2 diabetes. Someone dies every 36 seconds from cardiovascular disease (CVD) in the U.S., and CVD causes one-third of all deaths worldwide.

Both conditions may develop slowly, and the symptoms are not always evident. Doctors assess a persons risk of developing them by taking a patient history and observing current lifestyle factors.

Now, a study led by Prof. Dr. Chris Lauber, of Twincore in Germany, has shown that lipids in blood plasma can predict future risk of type 2 diabetes and CVD.

The method known as lipidomic profiling can provide a quantitative measurement of risk long before symptoms develop.

The authors of this study have added to the growing evidence that lipidomics profiling could very well usher in the next generation of detection of cardiovascular diseases as well as diabetes.

Dr. Suneet Singh, Medical Director of CareHive.

The longitudinal study included just over 4,000 healthy, middle-aged Swedish people from the Malm Diet and Cancer-Cardiovascular Cohort.

Participants ranged from 4668 years at the start of the study. Around 10% had a body mass index (BMI) of more than 30.

The researchers took one baseline blood sample from each participant. They then centrifuged these blood samples to separate out the plasma for analysis.

Researchers measured concentrations of 184 different lipids in the blood plasma using mass spectrometry. They used these lipidomic profiles to calculate risk scores and assign participants into one of six risk groups.

Dr. Singh told Medical News Today the study could add to previous methods of assessing risk:

This new approach allows for a novel framework of risk stratification which also allows for enhancement when used in conjunction with the measurement of standard clinical variables.

He did, however, note that there was little diversity in the study population: It is advisable to continue to expand upon this research by increasing the patient population to include several more variables. This includes different patient ages, racial groups, ethnicities, and physical activity levels.

The researchers followed up participants for more than 20 years from baseline assessments made between 1991 and 1994. In that time, 13.8% of the cohort developed type 2 diabetes, and 22% developed CVD.

For those in the highest risk groups, these values rise to 37% for developing type 2 diabetes and 40.5% for developing CVD as compared to the group averages above.

People in the lowest risk groups had a decreased risk of developing both conditions.

The researchers also found that people with high lipid risk scores had a much greater likelihood of developing obesity during the follow-up period. Obesity is a risk factor for both type 2 diabetes and CVD.

The authors suggest that this risk-score-based approach could indicate the risk of both type 2 diabetes and CVD many years before disease onset.

[T]his earlier disease detection occurs at a time where the disease has not become clinically evident. As a result, researchers and clinicians can now take on diseases from a more proactive preventive lens and not simply one that starts at the treatment stage, Dr. Singh commented.

Using mass spectrometry to measure blood plasma lipids is quick, relatively inexpensive, and gives quantitative results.

The authors of this study suggest that it could identify lipids that contribute most to the risk of developing subsequent health conditions and, potentially, lead to new drug treatments.

Dr. Singh told MNT: Research [] demonstrated in this paper helps to advance the field of clinical mass spectrometry as a whole by continuing to demonstrate the potential for earlier disease detection in advance of conventional means.

The authors stress that further research is needed to verify their hypothesis and demonstrate how informative the plasma lipidome is for health and disease.

The lipidome may provide insights much beyond diabetes and cardiovascular disease risk.

Prof. Chris Lauber, lead author of the study.

So, this simple test could tell people their risk of developing both type 2 diabetes and CVD. By making diet and lifestyle changes, they may then be able to decrease that risk.

More here:
Blood test may predict long-term risk of diabetes and heart disease - Medical News Today

IRVINE, Calif. & BASEL, Switzerland--(BUSINESS WIRE)--Urovant Sciences, a wholly-owned subsidiary of Sumitovant Biopharma Ltd., today announced positive topline results from its Phase 2a, double-blind, placebo-controlled exploratory study of URO-902, an investigational, novel, locally injected gene therapy product (plasmid human cDNA encoding maxi-K channel), in patients with overactive bladder (OAB), who were not well managed by oral therapies.

URO-902 showed a clinically meaningful and statistically significant effect on a number of relevant outcome measures in OAB including number of micturitions, urgency episodes, and quality of life indicators compared to placebo, 12 weeks post-administration, said Cornelia Haag-Molkenteller, MD, PhD, executive vice president and chief medical officer of Urovant Sciences. URO-902 was well tolerated, compared to placebo. The most common adverse event was urinary tract infection, in both treatment groups. We are encouraged by these positive results and pending the completion of the study in Fall 2022 and we look forward to discussing next steps for the URO-902 clinical development plan.

The Phase 2a study included 80 female patients and was designed to evaluate the efficacy, safety, and tolerability of a single, physician administered dose of URO-902 of 24 milligrams (mg) and 48 mg, compared with placebo with a primary timepoint at week 12 post-administration. Patients were followed for up to 48 weeks post-administration. URO-902 has the potential to be the first gene therapy for patients with OAB.

These promising results suggest that URO-902 could potentially offer a new treatment option for patients with overactive bladder who have been inadequately managed by oral pharmacologic therapy, said Kenneth Peters, MD, principal investigator, and chief of the department of urology at Beaumont Hospital, Royal Oak; Medical director of the Beaumont Womens Urology and Pelvic Health Center and professor and chair of urology of the Oakland University William Beaumont School of Medicine in Rochester, Michigan.

The company plans to present the topline results of the study at the American Urological Association annual meeting being held May 13-16, 2022 in New Orleans, LA.

About the Phase 2a Study

The study was a randomized, double blind, placebo-controlled trial to evaluate the efficacy, safety, and tolerability of a single physician administered dose of URO-902, a novel gene therapy being developed for patients with OAB who have not been adequately managed with oral or transdermal pharmacologic therapy for OAB. URO-902 is administered via direct intradetrusor injections into the bladder wall under local anesthesia in patients who are experiencing OAB symptoms and urge urinary incontinence (UUI).

The Phase 2a trial enrolled 80 female patients in two cohorts: the first cohort received either a single administration of 24 mg of URO-902 or matching placebo, and the second cohort received 48 mg of URO-902 or matching placebo into the bladder wall. Multiple outcome measures were explored, including the effect on the number of micturitions, urgency episodes, and quality of life indicators compared to placebo, 12 weeks post-administration, as well as an assessment of the safety and tolerability of this potential new therapy. Patients were followed for up to 48 weeks after initial administration.

About URO-902

URO-902 has the potential to be the first gene therapy for patients with OAB. If approved, this innovative treatment may address an unmet need for patients who have not been adequately managed by oral or transdermal pharmacologic OAB therapies and are concerned with potential urinary retention with other minimally invasive therapies or surgical interventions related to existing third-line OAB treatments.

About Urovant Sciences

Urovant Sciences is a biopharmaceutical company focused on developing and commercializing innovative therapies for areas of unmet need, with a dedicated focus in Urology. The Companys lead product, GEMTSA(vibegron), is an oral, once-daily (75 mg) small molecule beta-3 agonist for the treatment of adult patients with overactive bladder (OAB) with symptoms of urge urinary incontinence, urgency, and urinary frequency. GEMTESA was approved by the U.S. FDA in December 2020 and launched in the U.S. in April 2021. GEMTESA is also being evaluated for the treatment of OAB in men with benign prostatic hyperplasia. The Companys second product candidate, URO-902, is a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. Urovant Sciences, a wholly owned subsidiary of Sumitovant Biopharma Ltd., intends to bring innovation to patients in need in urology and other areas of unmet need. Learn more about us at http://www.urovant.com or follow us on Twitter or LinkedIn.

About Sumitovant Biopharma Ltd.

Sumitovant is a global biopharmaceutical company leveraging data-driven insights to rapidly accelerate development of new potential therapies for unmet patient conditions. Through our unique portfolio of wholly-owned Vant subsidiariesUrovant, Enzyvant, Spirovant, Altavantand use of embedded computational technology platforms to generate business and scientific insights, Sumitovant has supported the development of FDA-approved products and advanced a promising pipeline of early-through late-stage investigational assets for other serious conditions. Sumitovant, a wholly-owned subsidiary of Sumitomo Dainippon Pharma, is also the majority-shareholder of Myovant (NYSE: MYOV). For more information, please visit our website at http://www.sumitovant.com or follow us on Twitter and LinkedIn.

About Sumitomo Dainippon Pharma Co., Ltd.

Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China, and other Asian countries. Sumitomo Dainippon Pharma is based on the 2005 merger between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 7,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com.

About GEMTESA

GEMTESA is a prescription medicine for adults used to treat the following symptoms due to a condition called overactive bladder:

It is not known if GEMTESA is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Do not take GEMTESA if you are allergic to vibegron or any of the ingredients in GEMTESA.

Before you take GEMTESA, tell your doctor about all your medical conditions, including if you have liver problems; have kidney problems; have trouble emptying your bladder or you have a weak urine stream; take medicines that contain digoxin; are pregnant or plan to become pregnant (it is not known if GEMTESA will harm your unborn baby; talk to your doctor if you are pregnant or plan to become pregnant); are breastfeeding or plan to breastfeed (it is not known if GEMTESA passes into your breast milk; talk to your doctor about the best way to feed your baby if you take GEMTESA).

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

What are the possible side effects of GEMTESA?

GEMTESA may cause serious side effects including the inability to empty your bladder (urinary retention). GEMTESA may increase your chances of not being able to empty your bladder, especially if you have bladder outlet obstruction or take other medicines for treatment of overactive bladder. Tell your doctor right away if you are unable to empty your bladder.

The most common side effects of GEMTESA include headache, urinary tract infection, nasal congestion, sore throat or runny nose, diarrhea, nausea and upper respiratory tract infection. These are not all the possible side effects of GEMTESA. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please click here for full Product Information for GEMTESA.

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Urovant Sciences Announces Positive Topline Results of Phase 2a Trial of its Potential Novel Gene Therapy, URO-902 - Business Wire

JAVEN, France, March 10, 2022 /PRNewswire/ --HTL Biotechnology, a pioneer and world leader in the development and production of innovative pharmaceutical grade biopolymers, today announced its incubator's first equity investment in GelMEDIX Inc., an early-stage biotechnology company committed to developing the next generation of ocular and regenerative therapies.

This investment supports continued development of the GelMEDIX platform, which enables the delivery of therapeutics from small molecules to cell and gene therapies. Initial research focuses on ophthalmology with lead programs in vision restoring cornea and retina cell therapies and sustained release small molecule therapies.

GelMEDIX's most advanced program is focused on developing a sustained release corticosteroid subconjunctival implant which aims to improve patient care in ocular surface inflammation (postoperative pain and inflammation, dry eye disease, allergic conjunctivitis). One drug-loaded implant replaces 70 patient administered eye drops over the course of one month.

"Instead of using drops, one easy treatment will be administered in the operating theater or in the clinic without any loss of efficacy. In addition to anti-inflammatories, the platform technology can be used for delivery of pro-regenerative therapies that restore ocular health" said Reza Dana, M.D., M.P.H., M.Sc., Scientific Co-founder of GelMEDIX. "As such, this product represents one of the most promising innovations deriving from our proprietary hydrogel platform."

This initial implant constitutes only one of the several research opportunities deriving from GelMEDIX's proprietary photocrosslinkable hydrogel platform, which uniquely enables tunable bioadhesion, tissue regeneration, and biodegradation. HTL's partnership with GelMEDIX also facilitates the development of new applications in regenerative medicine both in ophthalmology and other therapeutic areas.

HTL's incubator will support GelMEDIX through a direct investment and industrial and scientific support throughout its development thanks to its expertise in biopolymers and ophthalmology sectors. HTL will also produce methacrylate hyaluronic acid, a key component for tailoring application specific parameters across the GelMEDIX pipeline including viscosity, bioadhesion, and therapeutic release profiles. Additionally, HTL will help GelMEDIX in the industrialization of its hydrogel production.

"It is an honor to have the support of such a renowned company as HTL. Beyond the financial aspect, its keen understanding of ophthalmology issues, its industrial know-how and the high quality of its products are all crucial assets to accelerate GelMEDIX's development," said Arthur Driscoll, President and Chief Development Officer of GelMEDIX.

HTL's participation will be joined by another equity investment from the venture fund Safar Partners. "The pioneering advancements GelMEDIX is making in the development of ocular and regenerative therapies will lead to dramatic improvements in how these treatments are administered to patients," said Nader Motamedy, a Safar Managing Partner. "The GelMEDIX hydrogel platform is the kind of transformative healthcare technology that Safar Partners highly values as both a long-term position for our portfolio as well as a development that will improve global health."

HTL's incubator is a financial vehicle allowing HTL to take minority investments in innovative biotechs in the biopolymer sector, either as seed funds or as series A investments. The incubator also aims to support these biotechs thanks to HTL's unique knowledge and expertise in the production of biopolymers.

"Innovation is the core of HTL's DNA, which is why we aim at supporting tomorrow's medicine by investing in biotechs that are pushing away the limits of biopolymer use in the medical sector," said Charles Ruban, Deputy CEO of HTL. "We are really excited about this partnership with GelMEDIX, which is a perfect example of the type of biotechs to which we wish to provide strategic and financial support".

This incubator represents one of the strategic axes of HTL's ambitious R&D strategy which positions the French company as the global driver of innovation in the biopolymer sector, developing new markets and applications for biopolymers to address unmet medical needs. The company also relies on its state-of-the-art research facility and numerous partnerships with entities at the forefront of world research to nurture its biopolymer platform for the healthcare industry.

About biopolymers and hyaluronic acidBiopolymers include several types of substances which are naturally produced by the cells of living organisms. Among them, glycosaminoglycans (GAGs) are known for their lubricating and shock-absorbing characteristics, as well as their natural biodegradability within the human body. This is the case, for example, of hyaluronic acid (hyaluronan or HA), a natural substance present in the human body with many biological functions such as skin hydration or lubrication of joints and eye tissue.

HTL produces GAGs by biofermentation, an alternative to animal extraction that maintains the quality required for pharmaceutical grade, allowing the biopolymers to be injectable into patients and used as ingredients for the development of medical treatments. The chemical properties of biopolymers can also be customized by HTL's R&D teams in order to precisely meet the needs of customers and their patients.

Today, the biopolymers which are developed and produced by HTL are used to produce treatments that improve the lives of millions of patients in many fields, such as ophthalmology (cataract surgery, treatment of glaucoma, treatment of dry eye ...), rheumatology (treatment of osteoarthritis), urology (treatment of vesico-ureteral reflux, a rare pediatric congenital disease), or in aesthetic medicine (dermal fillers). Biopolymers are also at the heart of several research programs focused on disruptive innovations in medicine such as bioprinting and regenerative medicine, tissue engineering as well as drug and stem cell delivery.

About GelMEDIXGelMEDIX Inc. is an early-stage biotechnology company committed to innovating the next generation of ocular and regenerative therapies through its proprietary hydrogel platform. GelMEDIX's programs are based upon its photo crosslinked hydrogels, originally developed by Prof. Nasim Annabi (UCLA) and Prof. Reza Dana (Mass Eye and Ear, Harvard Medical School). These hydrogels uniquely enable bioadhesion, tissue regeneration, tunable mechanics, and therapeutic loading across modalities from small molecules to cell and gene therapies.GelMEDIX is developing drug products for indications across the eye focused on cell-based therapies for vision restoration, intraocular sustained release of small molecules and peptides, and in situ forming bioadhesives.GelMEDIX is backed by Safar Partners and HTL Biotechnology along with leading angel investors and is currently raising a Series-A financing. GelMEDIX is based in Cambridge, MA., USA For additional information please inquire with info@gelmedix.com or visit https://gelmedix.com

About HTLHTL is a leading biotech and industrial player in the development and production of innovative, pharmaceutical-grade biopolymers that are used by leading pharmaceutical and medical device companies to transform the lives of millions of patients in multiple therapeutic areas such as ophthalmology, dermatology, medical aesthetics, rheumatology, and urology.

HTL is at the forefront of innovation in the biopolymer industry to meet tomorrow's medical needs by creating new types of biopolymers and chemical modifications, while exploring the untapped potential of biopolymers in innovative applications such as bioprinting or drug delivery.

HTL has a long history in France and in Javen (Ille-et-Vilaine, Brittany) where its production and R&D activities are located. Nearly 180 employees work at this site.

To learn more about HTL: https://htlbiotech.com/

About Safar PartnersSafar Partners is a seed- to growth-stage venture fund investing primarily in technology companies out of MIT, Harvard, and the University of Rochester. Safar takes advantage of the principles of private equity to create value as our companies scale beyond initial prototypes. We accelerate the scaling of our portfolio companies through the formation of spinouts or joint ventures to address additional markets, industries, or geographies. For more about Safar Partners, visit https://www.safar.partners

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Precision medicine involves more than molecularly sequencing a tumor and matching it with a targeted therapy, according to Kelvin P. Lee, MD, who argued that bulk sequencing may become an antiquated approach when single-cell RNA sequencing technology becomes available because it can provide a clearer picture of the complexity and heterogeneity of tumors.

Once we move away from bulk sequencing, which essentially says, This whole tumor is all the same and get down to a much finer specificity that [enables us to identify] tumor cells that have this mutation and look like this vs tumor cells that have that mutation and look like that, we will get more and more sophisticated in how we treat patients. The more heterogeneous the tumorand they become more heterogeneous as they are later in treatmentthe harder they are to treat, and the less helpful our current precision genomics technology is in that setting, Lee said in an interview with OncLive following an Institutional Perspectives in Cancer webinar on Precision Medicine.

In the interview, Lee, director of the Indiana University (IU) Simon Comprehensive Cancer Center, the H.H. Gregg Professor of Oncology, professor, Department of Medicine,

Division of Hematology/Oncology, associate dean for cancer research, IU School of Medicine, discussed the nuances of an effective molecular tumor board, explained the practical application of precision oncology genomics, and highlighted the pathways that have the potential to change the treatment landscape for precision oncology.

Lee: The take-home message is that every tumor should be sequenced. If you dont do it now, you may not be able to do it later. The information can be useful in designing a treatment plan, but it also helps us understand that cancer. The more information we have on lung cancers from patients, the better we can design new therapies and move that field forward. If a patient has a tumor specimen that you can send to precision genomics, send it because maybe we dont have an answer today, but maybe in 2 weeks we will have an answer, and that becomes important for that patient.

At IU there is a precision medicine team, and our patients are referred to them. That precision medicine team sets up the testing and the [molecular] sequencing and then does tumor boards, where they go through the results, they talk about what abnormalities and what mutations were found and then they go through what [mutations] are potentially actionable and how we want to utilize [treatment for] those [alterations]. The nice thing about tumor boards is that all the experts are in the room, and now that we have extended [ours] to be virtual, not only can we do precision medicine downtown at University Hospital, but now we have clinics that are in our suburban and metropolitan regions. Were moving those facilities out farther, so we can do precision medicine, hopefully, out in the community. Hopefully, with the expansion, primary care physicians and community oncologists can also utilize this [opportunity to] get the referrals and then sit in virtual tumor boards and get answers for their patients.

The key piece is not only doing the [molecular] sequencing and understanding what the mutations are, but also what youre doing with that information. For many of the mutations, the therapeutic that might be applicable to that patient or that mutation are in clinical trials. An effective precision medicine program has to not only have the expertise as to what these mutations are, what these established targets or established therapeutics are that we might be able to use, but also has to have an ongoing real-time knowledge of all the clinical trials that are available and in the literature.

Not only are there new compounds that a patient might be eligible for in a clinical trial, but there are also drugs that have been used for other things that are being repurposed for targeting mutations that we had not previously expected those drugs to be able to do. The precision medicine team must be aware of the literature, not only published literature but abstracts and journal or meeting work that says, Maybe you can use this malaria drug to target this mutation, which may be something that had not been previously understood. That is, overall, a key piece of what makes precision medicine so effective and so important, but so difficult to do. Its not just sequencing stuff, and then saying, There it is and then figuring out how to manage that [patient].

Next-generation sequencing is now allowing us to get at whole genomes, instead of just testing for BRCAmutations or [other single-gene mutations]. We are identifying other mutations, and now instead of having to re-sequence somebodys tumor, we have all that data thats there. If something pops up that later becomes this polymorphism or this mutation that is important in this cancer, we have that data; we can go back and look at those aspects. What it allows us to do is really what I would consider the next generation of precision medicine: to understand how mutations work together.

For immunotherapy, [tumor] mutational burden has been a key driver of whether checkpoint inhibitors are important. Now as we understand more of what a persons cancer has, in terms of mutations, our informatics, our artificial intelligence, and our machine learning technology is poised to take that data and say, if you have these 2 mutations: Are they compensating for each other, and do you have to target both? Now, if a patient has 1 mutation, they get this 1 drug, but biology is much more complicated. There are mutations that may work in concert with each other that may develop other additional vulnerabilities that we didnt anticipate because one mutation is causing the cell to do something and another mutation is stressing the cells, so maybe there is a target thats not either one of those 2 mutations but that is in the pathways that those mutations are driving that can be gone after. The exciting thing for me is understanding that. Im an immunologist, so understanding the complexity of cells, because immune systems see lots of things simultaneously, is really what we are looking forward to.

Its a very active process; patients are identified at the beginning of the week, and then the team asks: What are the mutations? What are the [alterations] that are actionable? Then they begin to sort through what treatment options are available and what the adverse effects [AEs] are. PharmDs have to ask: What are the AEs, particularly for experimental agents? What are the interactions with other drugs? How do we get these things and what literature supports the use of this agent? Then, are there odd things that we have to understand? Are there subsets of patients who have particularly bad responses that had been reported in the literature? All that gets pulled together.

Our tumor board is later in the week where all that information is presented: the patient case is presented, the genomic description or the description of the genetic changes are reviewed, and then the treatment options are also reviewed if theyre available, and the strength of each [drug] in terms of the data that says that this drug would be particularly good in this patient, or this drug would not be something that wed be looking for in this patient. A lot of what makes tumor boards effective is that research. Its not just, I have a piece of paper says I can give this drug and then you are done. It really is a lot of thoughtful research that goes into understanding the options that a patient might have.

The case study showed what the right process is to analyze the data you get to reach a meaningful action plan. As the technology goes forward, and as our ability to detect things gets more sophisticated, as we start to move toward single-cell sequencing, for example, RNA sequencing, when we start to look at the epigenome, we will have substantially more data than we have now. Then well start looking at what the patients immune system looks like when we start [molecularly] sequencing that. The amount of data that will be collected and the kinds of data that will be collected will grow exponentially.

The key piece of molecular tumor boards and the key piece of precision medicine is: How do you analyze that data? The analytical pipeline is going to be the same, the structure is going to be the same. How do you act? How do you take that data in? How do you analyze it, and then how do you use that analysis to come to specific treatment recommendations for that patient? That framework, that pipeline is going to be the same regardless of what the data coming in is. The key thing that was important in that whole process of going through these case studies is to recognize what the steps were that were taken from the very beginning, from the actual case itself where the patient comes in with their history. How were those data put together and analyzed? How was that used to make decisions for the treatment plan for that patient? Its the structure of the analytical process that was the most important aspect of going through those case studies.

The data suggests that with early genomic testing, when you apply it to precision medicine to identify therapeutics, the anti-cancer effect, or the ability to impact a persons cancer is greater on early diagnosis than late diagnosis. There probably are a variety of biologies that are implicated by that. When a tumor has been exposed to lots of things, it probably not only has its initial mutations, but probably has a lot of adaptations that have happened because of chemotherapy that has been given that we dont necessarily pick up; it may not be a genomic abnormality, but it may be overexpression of a particular gene that confers resistance. As those tumors become more resistant to therapy, the initial driver mutations may become less important as things go forward.

It speaks to biology also, because in the beginning, probably, in tumors that have not been treated or not been heavily pretreated at the time of diagnosis, theyre probably less genetically complicated, so maybe they have just one mutation. Maybe all of them have that one mutation, you treat them with a drug, and they all die. As tumors go along, they become much more heterogeneous, so instead of one population of cancer cells, now you have 75 different populations or tribes, for example, that are living, and some of them are sensitive, some of them are different. Some of them have different mutations, and precision genomics is moving towards being able to understand complex tumors, such that some of the cells have one mutation and some of them have a completely different set of mutations. We dont pick that up right now, simply because we dont have the single-cell RNA sequencing technology yet, although thats coming.

We are beginning to see precision medicine in the context of immunotherapy. In that sense, the change in framework is its not the cancer alone thats important because not only do you have to sequence the cancer, and understand its genetic makeup, you also have to sequence the immune systemthe normal part of the patient that is essentially the effector part. Instead of giving chemotherapy, where youre saying, I have a drug, I know everything about that drug, and all I need to do is figure out whats going on in the cancer, if I can find that this drug will hit this piece of the cancer, then lets put those two together.

For immunotherapy, you have the cancer, which is doing stuff and its dynamic, and its activating the immune system and suppressing the immune system. We have to understand that, and some of the suppression that it does is not because it is suppressing the immune system, its making the normal tissue around it suppress the immune system.

People say that cancer is a non-healing wound, so essentially, for wound healing, you dont want your immune system to fire up and start destroying all the tissue around a healing wound. Otherwise, youll never heal. The normal body has perfectly good mechanisms to shut off your immune response and cancers take advantage of that, but that phenomenon is not in the cancer. Its in the surrounding tissue. You have to look in the surrounding tissue to see whats going on there.

Then, you have to look at the immune system because the immune system is the thing thats going to kill the cancer cell, and people have different immune systems. Its very clear that there are lots of genetic variabilities in that. Maybe that genotype within somebodys immune system is not good at getting activated by this immunotherapy. Maybe we should try something different. Its another level of complexity that precision medicine has a tremendous role in, but it becomes that much more complicated because now youre not just looking at the cancer, were now looking at the cancer, the cancers effect on its surrounding microenvironment, and the immune systems ability to target that cancer and perhaps live in that environment that surrounds the tumor. Its an additional level of analysis and complexity. Thats coming though. With the expansion of immunotherapy that will be a much bigger piece of what we do in terms of therapy. Those kinds of analyses and guidance by precision medicine will be a key component of how we deploy immunotherapy in patients with cancer.

More here:
Precision Genomics Moves Toward Increased Granularity in Molecular Sequencing - OncLive

ReportLinker

Abstract: What`s New for 2022? -Global competitiveness and key competitor percentage market shares. -Market presence across multiple geographies - Strong/Active/Niche/Trivial.

New York, March 11, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Gene Therapy Industry" - https://www.reportlinker.com/p05817594/?utm_source=GNW -Online interactive peer-to-peer collaborative bespoke updates -Access to our digital archives and MarketGlass Research Platform -Complimentary updates for one year

Global Gene Therapy Market to Reach US$3.4 Billion by the Year 2027

Amid the COVID-19 crisis, the global market for Gene Therapy estimated at US$970.5 Million in the year 2020, is projected to reach a revised size of US$3.4 Billion by 2027, growing at a CAGR of 19.5% over the period 2020-2027.Viral, one of the segments analyzed in the report, is projected to grow at a 19.7% CAGR to reach US$3 Billion by the end of the analysis period. After an early analysis of the business implications of the pandemic and its induced economic crisis, growth in the Non-Viral segment is readjusted to a revised 17.6% CAGR for the next 7-year period. This segment currently accounts for a 11.1% share of the global Gene Therapy market.

The U.S. Accounts for Over 53.7% of Global Market Size in 2020, While China is Forecast to Grow at a 25.1% CAGR for the Period of 2020-2027

The Gene Therapy market in the U.S. is estimated at US$521.3 Million in the year 2020. The country currently accounts for a 53.71% share in the global market. China, the world second largest economy, is forecast to reach an estimated market size of US$107.9 Million in the year 2027 trailing a CAGR of 25.1% through 2027. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 17.3% and 18.9% respectively over the 2020-2027 period. Within Europe, Germany is forecast to grow at approximately 18.6% CAGR while Rest of European market (as defined in the study) will reach US$107.9 Million by the year 2027.Select Competitors (Total 154 Featured) -

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Abeona Therapeutics Inc.

Adaptimmune Therapeutics Plc

Advantagene, Inc.

Adverum Biotechnologies, Inc

Akcea Therapeutics

Alnylam Pharmaceuticals, Inc.

Amgen Inc

Anchiano Therapeutics, Inc.

AnGes, Inc.

Applied Genetic Technologies Corporation

Audentes Therapeutics, Inc.

Biogen

bluebird bio, Inc.

Chiesi Farmaceutici S.p.A

CRISPR Therapeutics AG

Editas Medicine, Inc.

Gilead Sciences, Inc.

Intellia Therapeutics, Inc.

Jazz Pharmaceuticals, plc.

Juno Therapeutics, Inc

Merck KGaA

MolMed S.p.A.

Novartis Gene Therapies

Orchard Therapeutics plc

REGENXBIO Inc.

Sangamo Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Sibiono GeneTech Co. Ltd.

Spark Therapeutics, Inc.

uniQure N.V.

Voyager Therapeutics

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=GNW

I. METHODOLOGY

II. EXECUTIVE SUMMARY

1. MARKET OVERVIEW Influencer Market Insights World Market Trajectories Impact of Covid-19 and a Looming Global Recession A Prelude to Gene Therapy Classification of Gene Therapies COVID-19 Causes Gene Therapy Market to Buckle & Collapse COVID-19 Impact on Different Aspects of Gene Therapy Manufacturing & Delivery Research & Clinical Development Commercial Operations & Access Managing Derailed Operations Focus on Clinical Development Programs Targeting Manufacturing & Delivery Strategies Securing Supplies Remote Working Gene Therapy Set to Witness Rapid Growth Post COVID-19 Gene Therapy - Global Key Competitors Percentage Market Share in 2022 (E) Competitive Market Presence - Strong/Active/Niche/Trivial for Players Worldwide in 2022 (E) By Vector Type VIRAL VECTORS ACCOUNT FOR A MAJOR SHARE OF THE MARKET Adeno-Associated Virus Vectors Lentivirus NON-VIRAL VECTORS TO WITNESS FASTER GROWTH US and Europe Dominate the Gene Therapy Market Oncology Represents the Largest Indication for Gene Therapy Market Outlook WORLD BRANDS

2. FOCUS ON SELECT PLAYERS Recent Market Activity Select Innovations

3. MARKET TRENDS & DRIVERS Availability of Novel Therapies Drive Market Growth Select Approved Gene Therapy Products Adeno-associated Virus Vectors - A Leading Platform for Gene Therapy Lentiviral Vectors Witness Increasing Interest Rising Cancer Incidence Worldwide Spurs Demand for Gene Therapy Global Cancer Incidence: Number of New Cancer Cases in Million for the Years 2018, 2020, 2025, 2030, 2035 and 2040 Global Number of New Cancer Cases and Cancer-related Deaths by Cancer Site for 2018 Number of New Cancer Cases and Deaths (in Million) by Region for 2018 Compelling Level of Technology & Innovation to Ignite Gene Therapy Promising Gene Therapy Innovations for Treatment of Inherited Retinal Diseases Gene Therapy Pivots M&A Activity in Dynamic Domain of Genomic Medicine M&As Rampant in Gene Therapy Space Gene Therapy Deals: 2018 and 2019 Emphasis on Formulating Robust Regulatory Framework Strong Gene Therapy Pipeline Gene Therapy: Phase III Clinical Trials OHSU Implements First-Ever LCA10 Gene Therapy Clinical Trial with CRISPR Growing Funding for Gene Therapy Research Market Issues & Challenges

4. GLOBAL MARKET PERSPECTIVE Table 1: World Recent Past, Current & Future Analysis for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 2: World Historic Review for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 3: World 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2017, 2021 & 2027

Table 4: World Recent Past, Current & Future Analysis for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 5: World Historic Review for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 6: World 10-Year Perspective for Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 7: World Recent Past, Current & Future Analysis for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 8: World Historic Review for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 9: World 10-Year Perspective for Non-Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 10: World Recent Past, Current & Future Analysis for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 11: World Historic Review for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 12: World 10-Year Perspective for Oncological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 13: World Recent Past, Current & Future Analysis for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 14: World Historic Review for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 15: World 10-Year Perspective for Rare Diseases by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 16: World Recent Past, Current & Future Analysis for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 17: World Historic Review for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 18: World 10-Year Perspective for Neurological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 19: World Recent Past, Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 20: World Historic Review for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 21: World 10-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

III. MARKET ANALYSIS

UNITED STATES Gene Therapy Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United States for 2022 (E) Table 22: USA Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 23: USA Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 24: USA 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 25: USA Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 26: USA Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 27: USA 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

CANADA Table 28: Canada Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 29: Canada Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 30: Canada 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 31: Canada Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 32: Canada Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 33: Canada 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

JAPAN Gene Therapy Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Japan for 2022 (E) Table 34: Japan Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 35: Japan Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 36: Japan 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 37: Japan Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 38: Japan Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 39: Japan 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

CHINA Gene Therapy Market Presence - Strong/Active/Niche/Trivial - Key Competitors in China for 2022 (E) Table 40: China Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 41: China Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 42: China 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 43: China Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 44: China Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 45: China 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

EUROPE Gene Therapy Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Europe for 2022 (E) Table 46: Europe Recent Past, Current & Future Analysis for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 47: Europe Historic Review for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 48: Europe 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2017, 2021 & 2027

Table 49: Europe Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 50: Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 51: Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 52: Europe Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 53: Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 54: Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

See the article here:
Global Gene Therapy Market to Reach US$3.4 Billion by the Year 2027 - Yahoo Finance