Archive for the ‘Gene Therapy Research’ Category

Abstract / Synopsis:

AAV8-RPGR gene therapy for X-linked retinitis pigmentosa showed early responses to treatment at one month with increased retinal sensitivity with retinal toxicity.

This article was reviewed by Paulo E. Stanga, MD

The results of treatment of X-linked retinitis pigmentosa (XLRP) with AAV8-RPGR gene therapy proved to be early and effective with durable improvements in vision occurring as early as one month following treatment.

XLRP, a rare disease that comprises 10% to 20% of RP worldwide, affects mostly males, and 70% of the cases are caused by RPGR gene mutations and 60% of those are caused by gene mutations in RPGR-ORF15. In this form of RP, the median age of blindness is 45 years, which is younger than the other forms.

Disease progression occurs in stages, with nyctalopia manifesting in the early stage, peripheral visual field constriction in the middle stage, and central visual deterioration and visual loss in the end stage, according to Paulo E. Stanga, MD, professor of Ophthalmology and Retinal Regeneration, Manchester Royal Eye Hospital and University of Manchester, London, UK.

Related: Gene therapy offering hope for retinal, corneal patients

The RPGR mutations cause abnormal transport across the cilium, where RPGR is located, and this abnormal transport results in photoreceptor death.

Obviously, this leads to loss of retinal sensitivity across the visual field and loss of visual acuity, he said.

The treatment that Dr. Stanga and colleagues devised has the goal of correcting the full length of the RPGR-ORF15 mRNA.

We aim for yields of high expression levels that are four times higher than the expression levels of the wild-type RPGR, he explained.

Related: Research targets precision data for gene, cell therapy

Six-month Phase I resultsDr. Stanga and colleagues are currently conducting a two-year dose-escalation clinical trial. The study included men who were 18 years and older with genetically confirmed XLRP. All patients had active disease that was visible bilaterally in the maculas. The study included six cohorts, with the following levels of affect vision: 1, better than light perception; 2 and 3, 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters; and 4, 5, and 6, greater than 34 ETDRS letters.

The primary endpoint was the incidence of dose-limiting toxicities and treatment-emergent adverse events. The secondary endpoints were the changes in microperimetry, visual stability, and changes in the ellipsoidal zone on spectral-domain optical coherence tomography, Dr. Stanga recounted.

The patients underwent a surgical procedure that included creation of a bleb followed by injection of the virus vector within the bleb.

Related: New vitreoretinal tools advancing surgical outcomes

The investigators evaluated the early effects of changes in the retinal sensitivity in the central retina using microperimetry (Maia, Centervue). The central 16 retinal loci represent 8 degrees of vision; an improvement of five of the central 16 loci equals a 30% improvement in the central visual field. An improvement of 7 dB represents five times greater light sensitivity, he explained.

One month after treatment, Dr. Stanga reported that there was a significant improvement in microperimetry in six of the 12 treated eyes in cohorts 3 to 6 that occurred at one month after vector injection; these cohorts received therapeutic doses. Cohorts 1 and 2, which received subtherapeutic doses, showed no changes.

Cohort 3 showed a mean improvement in the mean retinal sensitivity of 5 to 6 dB in the central 16 retinal loci between the treated and untreated eyes. The improvement became apparent at one month and remained relatively stable at three and six months, Dr. Stanga reported.

According to Dr. Stanga, these changes in retinal sensitivity differed from those observed in untreated eyes in the central 16 retinal loci. The untreated eyes showed decreases in retinal sensitivity over time.

Related: Surgeons provides pearls for handling retinal tears

The microperimetry heat maps also reflected the changes in the treated eyes with enlargement of the sensitive areas.

The investigators also reported that they also determined that the gene therapy with AAV8-RPGR gene therapy for XLRP was generally well tolerated.

No patients left the study and no dose-limiting toxicities were readily apparent.

Transient inflammation developed in the higher cohorts that responded to systemic steroid therapy. Two ocular adverse effects were related to the procedure or drug.

ConclusionsWe demonstrated proof of concept with durable dose-related improvements that appeared as early as one month after treatment across multiple microperimetry analyses, Dr. Stanga concluded. The preliminary efficacy signals were exhibited in cohorts 3 to 6, which responded to the highest doses.

Read more by Lynda Charters

Paulo E. Stanga, MDE: [emailprotected]This article is based on Dr. Stanga's presentation at the American Academy of Ophthalmology 2019 annual meeting. Dr. Stanga has no financial interest in this subject matter.

Excerpt from:
Gene therapy offers treatment for X-linked retinitis pigmentosa - Modern Retina

AUSTIN, Texas & CAMBRIDGE, Mass. (Feb. 11, 2020) Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and other serious diseases, today announced that it signed an exclusive license agreement with the University of Pittsburgh for a diabetes gene therapy that may have the potential to cure Type 1 and Type 2 diabetes, which together currently affect approximately 30.3 million people in the U.S, or 9 percent of the U.S. population.

The diabetes gene therapy, which was developed by lead researcher and Harvard graduate, Dr. George Gittes, at the Rangos Research Center at UPMC Children's Hospital of Pittsburgh, works by reprogramming beta cells in the pancreas to restore their function, thereby replenishing levels of insulin. The novel infusion process uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. The proteins these genes express transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body's immune system.

The diabetes gene therapy has been tested in vivo in mice and nonhuman primates. In studies of diabetic mice, the gene therapy approach restored normal blood glucose levels for an extended period of time, typically around four months. According to Dr. Gittes, the duration of restored blood glucose levels in mice could translate to decades in humans. Following preclinical studies, Dr. Gittes and his team plan to begin a Phase I clinical trial in diabetic patients, which could be the first-ever gene therapy tested in humans for diabetes.

"One of the biggest advantages of this gene therapy is that it could eliminate the need for insulin replacement therapy for diabetic patients," said Dr. Gittes. "Lifting this huge burden for the millions of patients who must continuously monitor blood glucose levels and inject insulin daily would be a breakthrough in modern medicine. This therapy has the potential to truly disrupt the diabetes market."

Genprex will add this exciting technology to its research and development pipeline, diversifying its portfolio and expanding its clinical development programs. The company will continue its focus on developing its immunogene therapies for cancer, including Oncoprex immunogene therapy, its lead drug candidate for non-small cell lung cancer, in parallel with development of the new diabetes gene therapy.

"We are excited to announce the licensing agreement with The University of Pittsburgh, and we look forward to working with Dr. Gittes and his team to develop this groundbreaking treatment for diabetes," said Rodney Varner, Genprex's Chairman and Chief Executive Officer. "At Genprex, we have always put patient needs first, focusing on ways to bring new treatment options to patient populations who have large unmet medical needs. We believe this diabetes gene therapy may potentially become a new treatment option for the millions of diabetes patients who now must take insulin replacement therapy, and it may be effective for patients who do not benefit sufficiently from that therapy. Even more moving, the diabetes gene therapy could hold the potential to provide long term effectiveness, or even be a cure, for diabetes patients."

Genprex plans to pursue potential partnerships for the development of this therapy globally and in the U.S.

According to the American Diabetes Association, more than 30 million Americans have diabetes, and approximately 1.5 million Americans are diagnosed with diabetes every year. Diabetes patients have the continuous burden of checking and monitoring their blood glucose levels and injecting insulin on a daily basis. Without effective management of diabetes, patients are at risk of stroke, hyperglycemia, cardiovascular disease, diabetic ketoacidosis and extremity amputation. Diabetes is the seventh leading cause of death in the U.S.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and other serious diseases. Genprex's technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and other serious diseases who currently have limited treatment options. Genprex works with world-class institutions and collaborators to in-license and develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches for patients with cancer and other serious diseases. The company's lead product candidate, Oncoprex immunogene therapy for non-small cell lung cancer (NSCLC), uses the company's unique, proprietary platform which delivers cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. In January 2020, the FDA granted Fast Track Designation for Oncoprex in combination with AstraZeneca's Tagrisso for the treatment of NSCLC. For more information, please visit the company's website at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effects of the licensed gene therapy on diabetes and the effect of Genprex's other product candidates, alone and in combination with other therapies, on cancer, as well as Genprex's ongoing and planned preclinical and clinical studies and potential partnerships. Risks that contribute to the uncertain nature of the forward-looking statements include risks relating to the effects of the safety and effectiveness of the licensed gene therapy and of Genprex's other product candidates, alone and in combination with other therapies, as well as the success of Genprex's ongoing and planned preclinical and clinical studies and the success of Genprex's efforts in concluding potential partnering arrangements for product development and commercialization. Other risks and uncertainties associated with Genprex and its product candidates are described more fully under the caption "Risk Factors" and elsewhere in Genprex's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Genprex undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Read more here:
Genprex and University of Pittsburgh Sign License Agreement - Patch.com

Neurocognitive development of young MPS IIIA patients preserved up to two years post ABO-102 treatment

Dose-dependent and sustained reductions in disease-specific biomarkers denotes clear biologic effects of ABO-102 and ABO-101

First patient treated in cohort 3 of ABO-101 MPS IIIB trial; total enrollment eight patients

Favorable safety profile observed in both studies

NEW YORK and CLEVELAND, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that researchers from the Abigail Wexner Research Institute (AWRI) at Nationwide Childrens Hospital presented positive interim data from two ongoing Phase I/II clinical trials evaluating ABO-102 and ABO-101, the Companys investigational gene therapies for MPS IIIA and MPS IIIB, respectively, at WORLDSymposium. Results from the Transpher A study demonstrated that MPS IIIA patients younger than 30 months treated with ABO-102 in dose cohort 3 continue to show neurocognitive development 18 months to two years after treatment. Reductions in cerebrospinal fluid (CSF) heparan sulfate (HS), denoting enzyme activity in the central nervous system, and liver volume reductions remain stable two years after treatment. Results from the Transpher B study showed that ABO-101 also improved multiple disease biomarkers providing clear evidence of a biologic effect in patients with MPS IIIB. Dosing in cohort 2 is complete and the first patient in cohort 3 was treated in late January, with a total of 8 patients treated to date. Both therapies have been well-tolerated to date. Abeona licensed the AAV9-based gene therapy technology underpinning ABO-102 and ABO-101 from AWRI at Nationwide Childrens where it was developed.

Todays presentations are available on abeonatherapeutics.com by following this link:https://investors.abeonatherapeutics.com/news-events

In total, the new results continue to show that early treatment with ABO-102 can help preserve neurodevelopment in children with MPS IIIA. These data will inform our ongoing discussions with the FDA and EMA, as we work towards providing a regulatory update in the second quarter, said Joo Siffert, M.D., Chief Executive Officer. For ABO-101, the reductions in disease-specific biomarkers are encouraging and demonstrate a clear biologic effect, which parallels that seen in the MPS IIIA study. We look forward to enrolling the Transpher B study as expeditiously as possible.

Results from the Transpher A study, an ongoing Phase I/II clinical trial with ABO-102 showed that:

The interim results presented today add to evidence suggesting a single intravenous dose of ABO-102 AAV9-based gene therapy has the potential to help MPS IIIA patients sustain neurocognitive development when they are treated at a young age, said Kevin Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study investigator. These data showed that ABO-102 can deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs, as evidenced by the clinical benefits in neurocognition and biophysical measures and improvements in disease-specific biomarkers.

Sites in the U.S., Spain, and Australia continue to enroll eligible patients into the Transpher A study. Additional information about the trial is available at AbeonaTrials.com and ClinicalTrials.gov.

Results from cohorts 1 and 2 (n=7) of the Transpher B study, an ongoing Phase I/II clinical trial showed that ABO-101 treatment demonstrated biologic effect in patients with MPS IIIB, as evidenced by initial improvements in multiple disease biomarkers associated with abnormal accumulation of glycosaminoglycans (GAGs) in the brain and throughout the body:

The Transpher B study provides hope that we may one day alter the course of this devastating disease, said Kim McBride, M.D., Principal Investigator at AWRI at Nationwide Children's and co-investigator for the Transpher B study. The impact on disease biomarkers in the early stages of follow up suggest the potential of ABO-101 gene therapy to break down the accumulation of glycosaminoglycans that underlie MPS IIIB pathology. I look forward to working with fellow investigators to gather more data from the study, including results from high-dose cohort 3.

Dose cohort 2 has been completed and dosing is underway in cohort 3 (n=1). Sites in the U.S., Spain, and France continue to enroll eligible patients into the Transpher B study. Additional information about the trial is available at abeonatherapeutics.com/clinical-trials and ClinicalTrials.gov.

About The Transpher A StudyThe Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase I/II global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The study has enrolled 14 patients to date across three dose-escalating cohorts (N=3, N=3, N=8) and remains open for enrollment. The gene therapy ABO-102 is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About The Transpher B StudyThe Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase I/II global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients 6 months to 2 years of age, or patients older than 2 years with a cognitive Developmental Quotient of 60% or above. The study has enrolled 8 patients to date across three dose-escalating cohorts (N=2, N=5, N=1) and remains open for enrollment. The gene therapy ABO-101 is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About ABO-102ABO-102 is a novel gene therapy in Phase I/II development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

About ABO-101ABO-101 is a novel gene therapy in Phase I/II development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

About Sanfilippo Syndrome Type A (MPS IIIA)Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Sanfilippo syndrome type B (MPS IIIB)Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

About Abeona TherapeuticsAbeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Companys clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively. The Companys portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates, including Regenerative Medicine Advanced Therapy designation for two candidates (EB-101 and ABO-102). http://www.abeonatherapeutics.com

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Abeona Therapeutics Announces Positive Interim Data from MPS III Gene Therapy Programs Presented at WORLDSymposium - PharmiWeb.com

North Carolina is home to one of the most dynamic life sciences clusters in the United States, with more than 600 life science companies, 75 percent in the Research Triangle, according to the Life Sciences 2020 report from Cushman and Wakefield,

Cushman and Wakefields numbers differ from those cited by the NC Biotech Center, which says there are more than 735 life sciences companies in the state.

Among other metrics cited: life sciences companies employ 6.9 thousand employees, up 84 percent since 2010; a 10.7 percent vacancy for office/lab space; the $20.1 billion in National Institute of Health funding for UNC-CH, $2 billion at Duke; and $160 million for the Research Triangle Institute. NC State, which also lands significant NIH funding, is not listed.

The report notes that Research Triangle Park is centrally located between Raleigh and Durham and is the largest research park in North America. It is home to industry giants such as GlaxoSmithKline, IQVIA, PPD, and Biogen. It also includes university spinouts and startups among Fortune 100 international corporations on its sprawling 7,000 acre campus.

The report points out that Forbes ranked NC as number one among the best states for business in 2017 and 2018. Key factors, it says, are extremely low cost of doing business, minimal regulations, impressive growth statistics, and a high quality of life.

Most notably, the report states, is home to three separate research universities (Duke, the University of North Carolina at Chapel Hill, and NC State University) which graduate 53,000 students annually. This provides life science companies with a pool of top talent.

The report lists top life science companies, top venture-funded companies, inventory growth projections, notable lease transactions and notable real estate sales transactions.

Other biotech hubs examined in the report are: Baltimore, Boston, Chicago, Montreal, New York City, New Jersey, Oakland/East Bay, Philadelphia, San Diego, San Francisco, Seattle, Toronto, and Washington DC.

Doug Edgeton, president and CEO of the NC Biotech Center, had this response to the report:

Were always pleased to be among the nations leaders in the annual Cushman & Wakefield life science report. It is, of course, quite specific in its focus on real estate, its always useful to see how we compare with other major life science hubs.

It also serves as a reminder of the importance of some recently announced projects that arent reflected in this survey, including Eli Lillys January announcement of a $474 million, 462-employee pharma manufacturing facility its building in RTP Lillys first in the state.

NC Biotechnology Center CEO Doug Edgeton

That followed Mercks announcement of a $680 million, 425-employee vaccine production expansion thatll add 225,000 square feet to its existing and already impressive Durham campus.

Fresenius Kabi is adding a $100 million 445-employee expansion of its pharmaceutical factory in Wilson. And Seqirus is constantly expanding its huge cell-based flu vaccine factory in Holly Springs, where its about to occupy a $140 million, 120-employee addition to give it 475,000 square feet of space the size of eight football fields at its 185-acre site.

And Pfizer has just embarked on a build-out of a $600 million, 340-employee gene therapy research and manufacturing facility at its 230-acre campus in Sanford, plus a $19 million investment in a 60,000-square-foot building on a 16-acre site in Durham. Pfizer already employs about 3,600 people in North Carolina at sites in Chapel Hill, Sanford and Morrisville.

And there are more announcements on the near horizon, because North Carolina is a great place to do business, especially in the life sciences. In fact, Forbes named us the best state for business in both 2017 and 2018.

Those same years, Site Selection Magazine has named us the top competitive state. And just last month The Boyd Company released a study comparing the top 25 global biopharma hubs for cost of operating a biomanufacturing plant.

We came in at number three, beaten only by Bangalore, India and Tel Aviv, Israel. We beat Austin, Texas; Denver; Pittsburgh; St. Louis; Seattle; Montgomery County, Maryland; and of course, San Diego, Philadelphia and Chicago.

We can never become complacent, because the life science world is extremely competitive, very rewarding, and necessary for global health and well-being. And were glad to be among the leaders of the pack.

Excerpt from:
Report says: North Carolina home to one of the most dynamic life science clusters in the US - WRAL Tech Wire

An emergency room physician, initially unable to diagnose a disoriented patient, finds on the patient a wallet-sized card providing access to his genome, or all his DNA. The physician quickly searches the genome, diagnoses the problem and sends the patient off for a gene-therapy cure. Thats what a Pulitzer prize-winningjournalist imagined2020 would look like when she reported on the Human Genome Project back in 1996.

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

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

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

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

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

A silver bullet genetic fix is still elusive for most diseases. (Credit: drpnncpptak/Shutterstock)

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

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

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

This article is republished from The Conversation under a Creative Commons license. Read the original article. This opinions expressed in this article belong solely to the author.

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We've Sequenced the Human Genome. So Why Haven't We Cured More Diseases? - Discover Magazine

IRVINE, Calif., Feb. 14, 2020 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. (Nasdaq: CLPT) (the Company), a leading platform neurosurgery company, today announced a strategic agreement with PTC Therapeutics, Inc. (PTC). The scope of the agreement includes hardware, software, clinical case and market development services for gene therapy cases in the United States and Europe to support PTCs potential commercialization in gene therapy globally upon regulatory approval. In addition to the announcement of this agreement, ClearPoint Neuro today announces the following 2020 Outlook for the companys performance:

We have put a tremendous amount of thought and effort into redefining our Company over the past two years, commented Joe Burnett, President and CEO of ClearPoint Neuro, Inc. This has included the vision, the team, the partnerships, and even the name of our company which officially changed this week to ClearPoint Neuro from MRI Interventions. We are thrilled by the evolution our Company has already undergone and believe we have only scratched the surface of the significant potential ahead.

We have evolved to become two companies in one, continued Burnett. On one side, we are a platform medical device company, consistently delivering double digit growth, and continuing to expand our installed base of neurosurgery centers in the U.S. Every year more surgeons and more patients gain access to the ClearPoint system and compatible disposable devices. On the other side of the business, we are a gene therapy and biologics enabling company, providing navigation, drug delivery, and case support to more than 20 companies in this exciting and growing space. Here we currently support pre-clinical and clinical efforts, but we believe that we are on the precipice of potentially explosive growth as these therapies progress through the regulatory process toward commercial launch. We feel that our company represents both scale and purpose through a unique combination of predictable device growth and a potential biologics opportunity, all supported under a common team dedicated to treating the most debilitating neurological disorders.

ClearPoint Neuros revenue outlook for 2020 includes an expectation that approximately 33% of total revenue will be derived from biologics and drug delivery products and services, up from approximately 20% in 2019, and reflecting the growth of this part of the business.

When planning for the development and potential commercialization of gene therapy globally, safety, consistency and predictability are crucial constructs that all must be included, commented Marcio Souza, Chief Operating Officer of PTC Therapeutics and ClearPoint Neuro Board Member. Our agreement with ClearPoint Neuro is designed to provide standardization across all of our centers of excellence as we can maintain consistency in navigation, delivery and clinical support. The more variables we can control, the more successful we believe the outcomes for patients will be.

As part of our Companys duality in devices and biologics, we are thrilled to deepen our partnership with PTC, commented Jacqueline Keller, Vice President and Program Manager at ClearPoint Neuro for the PTC Partnership. This new agreement with PTC highlights a turn-key solution for our gene therapy partners, whereby our commercial organization, with deep relationships in the neurosurgery community, can take on the burden of sales and clinical activities in the surgical suite, and allow our partners who are commonly small-molecule focused companies to continue to prioritize their efforts with neurologists, patients and reimbursement administrators. With the capacity to support thousands of cases each year, our team plans to provide this service across multiple companies and indications in neuro.

About the Company

The Companys mission is to improve and restore quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of the Companys current product portfolio include deep-brain stimulation, laser ablation, biopsy, neuro-aspiration, and delivery of drugs, biologics and gene therapy to the brain. The ClearPoint Neuro Navigation System has FDA clearance, is CE-marked, and installed in 60 active clinical sites in the United States. The Companys SmartFlow cannula is being used in partnership or evaluation with more than 20 individual biologics and drug delivery companies in various stages from preclinical research to late stage regulatory trials. To date, more than 3,500 cases have been performed and supported by the Companys field-based clinical specialist team which offers support and services for our partners. For more information, please visit http://www.clearpointneuro.com.

Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 regarding the planned offering. Additionally, all statements relating to any closing(s) of, and the amount or use of any proceeds from, the transactions described in this press release are considered to be forward-looking statements. Other forward-looking statements may be identified by the words guidance, plan, anticipate, believe, estimate, expect, intend, may, target, potential, will, would, could, should, continue, and similar expressions. Forward-looking statements are subject to risks and uncertainties, and the Companys actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of such risks and uncertainties, which include, without limitation, risks and uncertainties associated with market conditions and the satisfaction of closing conditions related to the transactions described in this press release. There can be no assurance that the parties will be able to complete the transactions described in this press release on the terms described herein or in a timely manner, if at all. More detailed information on these and additional factors that could affect the Companys actual results are described in the Risk Factors section of the Companys Annual Report on Form 10-K for the year ended December 31, 2018, and the Companys Quarterly Reports on Form 10-Q for the periods ended March 31, 2019, June 30, 2019 and September 30, 2019, all of which have been filed with the SEC, as well as the Companys Annual Report on Form 10-K for the year ended December 31, 2019, which the Company intends to file with the Securities and Exchange Commission on or before March 30, 2020. You are urged to carefully consider all such factors. Copies of these and other documents are available from the Company. The forward-looking statements contained herein represent the Companys views only as of the date of this press release the Company does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

For More Information

ClearPoint Neuro, Inc.Matt KrepsDarrow Associates Investor Relations(214) 597-8200mkreps@darrowir.com

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ClearPoint Neuro, Inc. Announces 2020 Revenue Outlook Ahead of Nasdaq Investor Presentation TodayNew US and European Partnership Agreement with PTC...

In pulmonary arterial hypertension (PAH), high blood pressure in the lungs arteries causes the heart to work extra hard to pump blood to the lungs and around the rest of the body. The condition is rare but deadly, and current treatments are expensive and have side effects and inconvenient modes of delivery. There is no cure.

With a goal of developing a more effective, convenient, and affordable therapy, research led by Henry Daniell of Penns School of Dental Medicine produced a protein drug in lettuce leaves to treat PAH. He worked with other scientists, including Steven M. Kawut of Penns Perelman School of Medicine; Tim Lahm from the Indiana University School of Medicine; Maria Arolfo and Hanna Ng of the Stanford Research Institute, on toxicology and pharmacokinetic studies; and Cindy McClintock and Diana Severynse-Stevens of RTI International, on regulatory studies.

The protein drug, composed of the enzyme angiotensin converting enzyme-2 (ACE2) and its protein product angiotensin (1-7), can be taken orally and, in an animal model of PAH, reduced pulmonary artery pressure and remodeling. In addition, rigorous toxicology and dose-response studies suggested the drugs safety in animals. Further work will be necessary to develop this novel treatment approach for patients with PAH. The teams findings appear in the March issue of the journal Biomaterials.

We completed extensive investigations to highly express these proteins in lettuce plants and to ensure the product is safe and effective, says Daniell. Were ready to progress with further work to move this to the clinic.

Daniell has employed his innovative platform to grow biomedically important proteins of many kinds in the leaves of plants. The system works by physically bombarding plant tissue with the genes of interest, prompting chloroplasts into taking up genes and then stably expressing that protein. Propagating those plants then creates a kind of pharmaceutical farm from which the researchers can harvest, dry, and process the leaves, resulting in a powder that can be placed in a capsule or suspended in a liquid for use as an oral medication.

A 2014 publication in the journal Hypertension, on which the current study was based, earned Daniell a prize from the American Heart Association, and support from the National Institutes of Health through its Science Moving TowArds Research Translation and Therapy (SMARTT) program, which aims to efficiently translate promising basic science discoveries into therapies that can make a difference in peoples lives.

That earlier publication had shown that ACE2 and angiotensin (1-7) could be expressed in tobacco leaves and, when fed to rats with a condition that models pulmonary arterial hypertension, could significantly reduce the animals pulmonary artery pressure while also improving cardiac function.

To create a drug that humans could safely ingest, however, required moving from a tobacco to a lettuce-based platform. The new work takes advantage of other advancements the Daniell lab has made during the last several years. He and colleagues have successfully devised methods to enhance expression of human genes in the plants and to remove the antibiotic resistance gene that is used to select for angiotensin-producing plants. Theyve also worked with a partner to produce genetically engineered plants in a production facility that adheres to FDA standards.

In the current work, the researchers demonstrated that they could accurately evaluate the dose of the ACE2 and angiotensin (1-7) proteins in lettuce, and that the products could be dried and kept shelf stable for as long as two years.

Funding from the SMARTT program enabled animal studies evaluating toxicology, pharmacodynamic, and pharmacokinetic studies, which evaluate the safety of the drug, where it goes in the body, and how long it persists in the body at different doses, in work done at Stanford University.

And to confirm that the lettuce formulation of the product had a positive impact on experimental PAH, the team fed rats a solution containing the drug for four weeks. Their lung pressures went down 30-50%, and the structure of their arteries also improved.

This is an innovative approach to targeting the renin-angiotensin-aldosterone system in pulmonary arterial hypertension, says Penn Medicines Kawut, which may hold promise in this and other diseases.

We are very excited about this work that shows efficacy of bioencapsulated ACE2 and angiotensin (1-7) in our animal model of pulmonary arterial hypertension, says Indiana Universitys Lahm. We now need to confirm that the intervention also works in other animal models and when given later in the disease. Ultimately, our goal is to move this to the clinic for trials in patients, but we need to make sure we learn as much as possible from animal studies and from studies in healthy human subjects to make sure this intervention is safe and efficacious in patients.

In other future work, Daniell hopes to continue evaluating the effects of ACE2 and angiotensin (1-7) in treating different types of cardiovascular disease, such as heart failure.

There are some potentially broad applications of this drug that were hoping to investigate, says Daniell.

Daniell, Kawut, and Lahms coauthors on the paper were Penn Dental Medicines Venkata Mangu, Jiyoung Park, Peyman Habibi, Yao Shi, and Patricia A. Gonnella; and Indiana Universitys Bakhtiyor Yakubov, Amanda Fisher, Todd Cook, and Lily Zeng.

Henry Daniell is vice chair and W.D. Miller Professor in the Department of Basic & Translational Sciences in the University of Pennsylvania School of Dental Medicine.

Steven M. Kawut is professor of medicine and director of the Pulmonary Vascular Disease Program at the University of Pennsylvania Perelman School of Medicine.

Funding for the study came from the National Institutes of Health (NIH) (grants HL107904, HL109442, and HL133191) and through the NIHs Science Moving TowArds Research Translation and Therapy (SMARTT) program (contracts HHSN268201600011C and HHSN268201600014C).

Continue reading here:
Advancing an oral drug for pulmonary arterial hypertension - Penn: Office of University Communications

MUNICH--(BUSINESS WIRE)--SIRION Biotech GmbH (SIRION), a world leader in viral vector-based gene delivery technologies for gene and cell therapy, today announced preliminary financial results for 2019. SIRIONs annual service and licensing revenues in 2019 exceeded 10M (US$11.2M). With a growing staff of 35 employees, the company reports a record profit and plans to invest a portion of these proceeds into an additional Munich site with expanded lab and process development capacities. In addition, SIRIONs offices in Paris and Boston will continue to expand.

In addition, the companys intellectual property is included in more than 10 clinical programs by leading drug developers, one of which was granted market approval in 2019. SIRIONs core fee-for-service business, providing vector materials for discovery and pre-clinical applications, experienced historic growth of more than 50% over the previous year. The company expanded into a new avenue of growth by assigning its share in joint intellectual property rights to both a client in USA and the Danish start-up, InProTher Aps, in return for shares and milestone payments as well as royalties on product net sales.

SIRION Biotechs business model comes from service revenue and licensing fees. The company provides high-grade viral vector materials for preclinical use, offering up to 1015 GMP-ready AAV vector genomes and 1010 infectious units of Lentivirus, currently paving all the way to toxicology studies. In addition, the company is forecasting significant increased demand for its clinical and commercial grade vectors.

As SIRION continues its growth trajectory, it will focus on attracting funding to expand preclinical development with collaborators from its large client network. With more than 2,000 single projects for over 200 recurring clients worldwide, SIRION is committed to building significant intelligence and insights into latest gene therapy drug developments.

After only 12 years in business, SIRION has been instrumental in bringing new gene therapy treatments to patients. Assigning intellectual properties to collaborators validates our comprehensive viral vector technology platforms and the highly skilled and creative lab staff that stands behind them. We continue our dedication and commitment to developing gene therapy on a global scale with the opening of our second Munich site this year that will expand our capacity for research and process development, said Dieter Lingelbach, Chief Operating Officer of SIRION.

Mr. Lingelbach continued, In 2020, we look forward to continued expansion of our US presence through our wholly-owned subsidiary, SIRION Biotech International Inc., as well as continued growth in Paris.

Gene and cell therapies are among the hottest topics in modern drug development. Viral vector technologies are the most promising gene editing tools available today, with significant potential both as therapeutics for a growing number of indications, for tumor vaccines and for further technical improvements. Key challenges include quality, yields, and improved transduction in order to make novel gene therapies reliable and affordable. Costs for gene therapies per patient remain high, and can be prohibitive for larger patient populations, even in well-developed markets.

About SIRION Biotech GmbHSIRION Biotech was founded in 2005 to lead the next generation of viral vector technologies for gene and cell therapy as well as vaccine development. Now SIRION offers one of the worlds most comprehensive viral vector technology platforms based on lenti-, adeno-, and adeno-associated viruses which expedites gene therapy research and advances drug development. SIRION is becoming a partner of choice in this growing sector. LentiBOOST has been used in several clinical trials from early stage clinical Phase 1/2 through late stage clinical Phase 3 trials and demonstrated clinical success in improving transduction of the therapeutic vector.

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Gene and Cell Therapy Solutions Provider, SIRION Biotech Reports Record Growth for Year Ended 2019 Based on Preliminary Financial Results - Business...

Maybe were all just batteries. Thats the bad news for Keanu Reeves Neo, who wakes up from a chemically-induced slumber and discovers hes nothing more than a pack of double-As connected to a massive power station run by evil robot overlords in the dystopian sci-fi film The Matrix.

But what if the power potential of human electric current wasnt so post-apocalyptic? As the International Energy Agency notes, global demand for power rose 2.3% in 2018, which represents the biggest increase in the last decade. Ever since Ben Franklin first decided that flying kites in dangerous weather was solid scientific practice, humans have been finding new ways to use electricity and discovering that demand never stops.

The bad news? Youre no coppertop. The better news? Bioelectricity is essential to life and may drive the future of human development.

Harvesting electricity from human activity is nothing new. As Knowable Magazine notes, breathing can produce more than 0.80 watts, body heat can generate up to 4.8 watts, and the motion of your arms creates a stunning 60 watts of power.

But the notion of electricity as fundamental to biological life isnt quite so clear-cut. According to Quartz, while there were some experiments measuring human electrical currents in the mid-1920s, its wasnt until 1949 that Alan Hodgkin and Andrew Huxley identified the movement of ions across cell nerve membranes. The pair took home a Nobel Prize for their work, but this electric revolution was quickly outpaced by the double-helix discovery of DNA. For decades, genes became the best-fit scientific foundation for biology, while electricity research was short-circuited.

Then, in 1976, Erwin Neher and Bert Sakmann developed a tool capable of circumventing bioelectricitys biggest problem: studying ion movements without killing their cellular transport mediums. And later, in 2012, Richard Nuccitelli created a device sensitive and subtle enough to track human electric currents on skin, and discovered that, when skin cells are wounded, they emit an injury current that calls for help from other cells. The larger the wound, the bigger the current and the current decreases with age. Other work found that charges inside embryo cells significantly affected development. As NOVA states, Researchers overwhelmingly agree that bioelectric currents are essential to nerve and muscle function.

With the human nervous system constantly generating a fluctuating electric current, why cant we all just plug in and power up? It all comes down to the two halves of electric potential: positive and negative charges.

The electricity were most familiar with the kind Franklin flew kites for and that powers our smartphones, dishwashers and light bulbs depends on the flow of negatively-charged electrons to produce a current. Meanwhile, in our bodies, its the movement of positively-charged ions such as potassium, sodium and calcium passing through cell membranes that create electric potential. And while this variable voltage is essential to keep hearts beating, limbs moving and minds functioning, its not great for typical electrical applications. For example, when animal cells take in sodium and chloride ions and discharge potassium ions, the result is a voltage between -40 to -80 mV across membranes, significantly less than a single watch battery.

However, as it turns out, human electric current offers significant potential for internal applications.

The biggest potential for human-produced power? Improved healing. Studies published in the journal Advances in Wound Care have shown that supplementing the bodys electric current with outside electrical stimulation can help to reduce the recovery time needed for bedsores, which are some of the most difficult wounds to mitigate, let alone fully heal. Similar work has shown improvements in healing bone fractures.

Next on the list? Cancer. While research in the 1920s demonstrated a connection between changing electric gradients and cancerous tumors, cell mutations are the most commonly cited cause of cancer concerns. Now, theres speculation that misregulation of electric currents may lead to cellular communication challenges in effect, cells forget theyre part of a larger network and begin acting selfishly by hoarding resources and growing out of control. Research from the University of Nottingham found that biologically-generated currents underpin specific cancer cell behaviors, and new techniques using a combination of gene therapy and light-activated ion channels have seen success treating cancer in tadpoles.

However, despite steady progress, challenges remain. Human genomes are far more complex than those of rats or tadpoles, and gene therapies face significant regulatory challenges. Electric treatments for wound healing also struggle with standardization how long should currents be applied to wounds for maximum effect without causing secondary damage? At what voltage? Using what type of device? Is alternating current (AC) or direct current (DC) safer? More effective?

The result is a kind of cautious optimism. While bioelectric benefits are grounded in solid science, more testing and research is necessary to standardize and streamline medical processes.

Bioelectric research offers the tantalizing potential to tap the inherent power of the human nervous system, but were not there yet.

Still, theres good reason to be optimistic. Mike Levin of Tufts University, whose lab is on the leading edge of human electric current research, puts it simply: Understanding the bioelectricity, biomechanics, and transcriptional circuits that allow cells to cooperate toward large-scale goals is the key to regenerative medicine, birth defects, cancer reprogramming, aging, synthetic bioengineering, and even new AI.

Put simply? Were not batteries thanks to positive bioelectric potential, were even better.

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Batteries Included? The Power Potential of Human Electric Current Now. Powered by - Now. Powered by Northrop Grumman.

New research has identified gene variants found only in the brains of transgender individuals. These genes are primarily involved in estrogens critical sprinkling of the brain right before or after birth, which is essential to masculinization of the brain.

The study, which included 13 transgender males (female to male) and 17 transgender females (male to female), reveals some of the first biological evidence of the incongruence transgender people experience because their brain indicates they are one sex and their body another.

Twenty-one variants in 19 genes have been found in estrogen signaling pathways of the brain critical to establishing whether the brain is masculine or feminine, says Dr. J. Graham Theisen, obstetrician/gynecologist and National Institutes of Health Womens Reproductive Health Research Scholar at the Medical College of Georgia at Augusta University.

In natal males (people whose birth sex is male), this critical estrogen exposure doesnt happen, or the pathway is altered so the brain does not get masculinized. In natal females, it may mean that estrogen exposure happens when it normally wouldnt, leading to masculinization.

Both could result in an incongruence between a persons internal gender and their external sex. The negative emotional experience associated with this incongruence is called gender dysphoria.

They are experiencing dysphoria because the gender they feel on the inside does not match their external sex, Theisen says. Once someone has a male or female brain, they have it and you are not going to change it. The goal of treatments like hormone therapy and surgery is to help their body more closely match where their brain already is.

The findings are published in the journal Scientific Reports.

It doesnt matter which sex organs you have, its whether estrogen, or androgen, which is converted to estrogen in the brain, masculinizes the brain during this critical period, says Dr. Lawrence C. Layman, chief of the MCG Section of Reproductive Endocrinology, Infertility and Genetics in the Department of Obstetrics and Gynecology. We have found variants in genes that are important in some of these different areas of the brain.

While its too early to definitively say the gene variants in these pathways result in the brain-body incongruence called gender dysphoria, it is interesting that they are in pathways of hormone involvement in the brain and whether it gets exposed to estrogen or not, says Layman.

This is the first study to lay out this framework of sex-specific development as a means to better understand gender identity, Theisen says. We are saying that looking into these pathways is the approach we are going to be taking in the years ahead to explore the genetic contribution to gender dysphoria in humans.

For the study, the team looked at the DNA of 13 transgender males, individuals born female and transitioning to male, and 17 transgender females, born male and transitioning to female.

The extensive whole exome analysis was conducted at the Yale Center for Genome Analysis.The variants found were not present in a group of 88 control exome studies in nontransgender individuals also done at Yale. They also were rare or absent in large control DNA databases.

Reproductive endocrinologist/geneticist Layman says his 20 years of experience taking care of transgender patients made him think there was a biological basis. We certainly think that for the majority of people who are experiencing gender dysphoria there is a biologic component, says Theisen. We want to understand what the genetic component of gender identity is.

Although genetics have been suggested as a factor in gender dysphoria, proposed candidate genes to date have not been verified, the researchers say. Most gene or gene variants previously investigated have been linked to receptors for androgens, hormones more traditionally thought to play a role in male traits but, like estrogen in males, are also present in females.

The team decided instead to take what little is known about sex-specific brain development that estrogen bath needed in early life to ensure masculinization of the brain to hone in on potential sites for relevant genetic variances.

Extensive DNA testing initially revealed more than 120,000 variants, 21 of which were associated with these estrogen-associated pathways in the brain.

Theisen notes that we all are full of genetic variants, including ones that give us blue eyes versus brown or green, and the majority do not cause disease, but rather help make us individuals. I think gender is as unique and as varied as every other trait that we have, Theisen says.

The investigators suggest modification of the current system for classifying variants that would not imply that a variant means pathogenic (disease causing).

Last year, the World Health Organization said that gender incongruence is not a mental health disorder and six years before that, the Diagnostic and Statistical Manual of Mental Disorders replaced gender identity disorder with general dysphoria.

About 0.5 to 1.4% of individuals born male and 0.2 to 0.3% of individuals born female meet criteria for gender dysphoria. Identical twins are more likely than fraternal twins to both report gender dysphoria.

Gender affirming therapies, like hormone therapies and surgeries, along with mental health evaluation and support help these individuals better align their bodies and brains, the scientists say.

Source: Medical College of Georgia at Augusta University

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Study IDs Gene Variants Potentially Linked to Brain-Body Incongruence in Transgender - PsychCentral.com

Feb 14, 2020 (Thomson StreetEvents) -- Edited Transcript of Urovant Sciences Ltd earnings conference call or presentation Thursday, February 13, 2020 at 9:30:00pm GMT

Urovant Sciences Ltd. - CFO & Senior VP of Business Development

Urovant Sciences Ltd. - Chief Medical Officer of USI

* Keith A. Katkin

Urovant Sciences Ltd. - CEO & Director

Urovant Sciences Ltd. - Executive Director of IR

* Biren N. Amin

H.C. Wainwright & Co, LLC, Research Division - Equity Research Associate

Good day, ladies and gentlemen, and welcome to Urovant Fiscal Third Quarter 2019 Earnings Call.

(Operator Instructions) As a reminder, today's conference is being recorded. I would now like to turn the call over to Ryan Kubota. Sir, you may begin.

Ryan Kubota, Urovant Sciences Ltd. - Executive Director of IR [2]

Thank you, Fati. Good afternoon, and thank you all for joining Urovant's Fiscal 2019 Third Quarter Financial Results Conference Call.

With me today are 4 members of our leadership team. Keith Katkin, Chief Executive Officer; Ajay Bansal, Chief Financial Officer; Dr. Cornelia Haag-Molkenteller, Chief Medical Officer; and Christine Ocampo, Chief Accounting Officer.

Today, after market close, we issued a press release containing detailed information on our quarterly results. You may access our release on our company website, urovant.com.

During our call, we'll be making forward-looking statements, including statements regarding our plans and strategies for the clinical development of vibegron and other treatments for urologic diseases.

Listeners are cautioned that all of our forward-looking statements are based on our current expectations and assumptions, which are subject to numerous risk factors that could cause our actual results to differ materially. Accordingly, we advise listeners to review the forward-looking statements disclosure in today's press release and the Risk Factors section of our Form 10-K, as well as our Form 10-Q, which we filed later today.

With that said, I will now turn the call over to our CEO, Keith Katkin. Keith?

Keith A. Katkin, Urovant Sciences Ltd. - CEO & Director [3]

Thank you, Ryan, and my thanks for all of you for joining us this afternoon.

This has been a very exciting and transformational quarter for Urovant, marked by key milestones across all aspects of our business.

First, in December, we submitted our New Drug Application to the U.S. Food and Drug Administration for vibegron for the treatment of patients with overactive bladder. The submission comes one quarter earlier than we initially anticipated, and we are very excited to have submitted our application by the end of the calendar year and ahead of schedule.

Second, we initiated our Phase IIa study in URO-902, our novel gene therapy product for patients with overactive bladder, suffering with urge urinary incontinence that have failed oral pharmacologic therapy. The study is evaluating URO-902's efficacy, safety and tolerability of a single administration of the product, and we expect top line safety data towards the end of the year.

Third, pursuant to the transaction between Sumitomo Dainippon Pharma, also known as DSP, and Roivant Sciences, DSP is now Urovant's majority shareholder. As part of the transaction, Urovant entered into a loan agreement with DSP, whereby DSP provided Urovant with a $300 million low interest, interest-only 5-year term loan facility with no repayments due until the end of the term. This loan facility provides Urovant with capital well into 2021 and enabled us to pay back the outstanding amount on our previous loan agreement with Hercules Capital.

DSP also expects to support Urovant through profitability and provide support for the commercialization of vibegron, providing access to its U.S. commercial infrastructure, including drug distribution, operations and managed care support.

Finally, Urovant and DSP entered into an investor rights agreement that provides certain protections to Urovant minority shareholders for as long as DSP holds between 50% and 90% of Urovant's outstanding voting power.

We're excited about our new strategic relationship with DSP, the potential benefits that it brings to Urovant and the future of our company. With the submission of our New Drug Application and launch preparations for vibegron well underway, we look forward to the strategic support and proven commercial resources of DSP.

Their drug distribution, operational and managed care support capabilities should greatly optimize Urovant's commercial approach to the U.S. market.

Now to comment briefly on some of our other important business highlights. We continue to enroll patients into Part 2 of the Phase III COURAGE trial, which will assess both efficacy and safety of vibegron in men with OAB and BPH, a patient population for which no product is currently approved.

Enrollment also continues for patients into our IBS-associated abdominal pain trial with top line results expected later this year.

With that, I'll now turn the call over to our Chief Medical Officer, Dr. Cornelia Haag-Molkenteller, who'll provide more detail on our clinical programs.

--------------------------------------------------------------------------------

Cornelia Haag-Molkenteller, Urovant Sciences Ltd. - Chief Medical Officer of USI [4]

--------------------------------------------------------------------------------

Thank you, Keith. As mentioned before, we are pleased to have submitted our new drug application for vibegron for the treatment of OAB to the FDA at the end of last year.

Let me briefly outline what to expect regarding the OAB filing and the program, then provide an update on our other clinical development programs.

In OAB, first, the submission of a New Drug Application to the FDA at the end of December 2019, we'll be waiting for the notification by the FDA of the acceptance of our file in March of 2020.

Second, we're expecting a 12-month review cycle at the FDA and potential approval could occur at the end of this year. We are looking forward to working with the FDA during the review process.

Third, in May, at the upcoming AUA, American Urological Association's 2020 Annual Meeting in Washington D.C., we will have 2 presentations: the first presentation would be our 52-week EMPOWUR extension study data; and the second presentation will be that of the EMPOWUR data by age.

Regarding the EMPOWUR extension study, we performed a post hoc analysis to test the difference in week -- at week 52 between vibegron 75 milligram and the active comparator tolterodine on the change of baseline in the urge urinary incontinence and total incontinence episodes between vibegron and tolterodine.

Notably, for both parameters, vibegron demonstrated a statistically significant reduction over tolterodine. The symptoms of urinary incontinence are key symptoms OAB and the main reason for patients to seek treatment. We're very excited by this data and believe this further supports the strong efficacy profile of vibegron.

Let me now turn to our other clinical programs. I'm pleased to report that we continue to make excellent progress across all of our development programs. Regarding our international, Phase III COURAGE development program for vibegron in men with OAB and benign prostatic hyperplasia, or BPH, we continue to enroll patients into Part 2 of the trial, which over 1,000 patients will be enrolled.

The trial is running in North America, and we will soon be initiating studies in Europe.

Our 2 of the Phase III current trail, which will assess both the efficacy and safety of vibegron in men with OAB and BPH. The co-primary endpoints are reduction in micturition frequency and urgency episodes per 24 hours. Key secondary endpoints are reduction in nocturia episodes, which means the awakening at night avoid, prostate symptom scores and safety. In addition, the first patients from Part I have enrolled into the long-term extension study, which will follow patients for a total exposure of 52 weeks.

We're excited about this program as there is currently no FDA-approved product specifically indicated for overactive bladder in men with BPH.

Let me now turn to our Phase IIa clinical program for IBS-associated abdominal pain. The study continues to enroll female patients with IBS-associated pain. Patients are randomized to either 75-milligram of vibegron or placebo. The primary endpoint is a 30% reduction in abdominal pain intensity on an 11-point rating scale over 12 weeks for IBS-D, which is the IBS with diarrhea. A responder is defined with the subject with at least 30% decrease in worst abdominal pain compared to the weekly baseline average.

Secondary endpoints include a global rating scale and safety, in particular, a lack of negative effects of stool frequency or consistency. We expect to report top line data from this study in the third quarter of 2020.

Finally, as reported recently, we initiated a Phase IIa trial for URO-902 in December 2019, our novel injectable gene therapy for patients with OAB, who've failed oral pharmacologic therapy.

This is a randomized, double-blind, placebo-controlled trial that will evaluate the efficacy, safety and tolerability of a single administration of URO-902. The therapy is administered by direct intradetrusor injections into the bladder wall under local anesthesia.

The trial is expected to enroll approximately 80 female patients in the U.S., 2 cohorts. The first cohort will receive either single administration of 24 milligram of URO-902 or matching placebo. The second cohort will receive 48 milligrams of URO-902 or matching placebo. Patients will be then followed for 48 weeks.

The primary outcome measure is the change in the average daily number of urge urinary incontinence for baseline, 2 week trial as well as assessment of safety and tolerability for this potential new therapy.

I am pleased with the progress we made over the last quarter and look forward to providing you with further updates in the coming quarters.

Now I'll pass to Ajay for a financial update.

--------------------------------------------------------------------------------

Ajay Bansal, Urovant Sciences Ltd. - CFO & Senior VP of Business Development [5]

--------------------------------------------------------------------------------

Thank you, Cornelia.

In addition to the financial results summarized in our press release, you can find additional information in our upcoming Form 10-Q, which will be filed later today.

R&D expenses were $23.1 million for the third quarter of 2019 compared with $21.3 million for the same period in the prior year. In the third quarter of 2019, R&D expenses were comprised of costs related to the submission of a new drug application for vibegron to the U.S. FDA.

Clinical development. Specifically, our ongoing studies in OAB plus BPH and abdominal pain associated with IBS. When comparing the 2 quarters, the increase in R&D expenses is primarily due to: a $2.9 million PDUFA fee for our NDA submission of vibegron for the treatment of OAB; a $2.5 million milestone payment as part of a collaboration agreement to Kyorin Pharmaceuticals in connection with our FDA submission of vibegron; and an increase of $2.6 million in share-based compensation due to the acceleration of vesting of certain stock options and RSUs as a result of the sale of Roivant's interest in the company to DSP.

These increased expenses were partially offset by lower clinical research organization costs, primarily due to the completion of Phase III EMPOWUR study earlier this fiscal year.

G&A expenses were $16.7 million for the third quarter of 2019 compared with $4.9 million for the same period in the prior year. The increase in G&A expense is primarily due to an increase of $8.7 million in share-based compensation from acceleration of vesting of certain stock options and RSUs and an increase in personnel costs as well as other general and corporate expenses.

Total operating expenses were $39.8 million for the third quarter of 2019 compared with $26.2 million for the same period in the prior year. As mentioned before, the increase is primarily driven by the increase in share-based compensation expense, PDUFA fee and Kyorin milestone payment.

Cash used in operations was $23.6 million for the quarter ended December 31, 2019, a decrease of $0.9 million as compared to the immediate prior quarter ended September 30, 2019.

Net loss was $41.3 million or $1.36 per share for the third quarter of 2019 compared with a net loss of $26.4 million or $0.87 per share for the same period in the prior year.

At December 31, 2019, total cash and cash equivalents were $131.9 million. As you're aware, in December of last year, we entered into a $300 million low-interest, interest-only 5-year term loan facility with DSP with no repayments due until the end of the term.

At closing, we drew down $87.5 million and still have $212.5 million available to us under this facility, eliminating the need for any short-term equity financing.

In addition, DSP also expects to continue to support Urovant through profitability. Looking ahead, for the fourth quarter of fiscal 2019, ending on March 31, we expect our total operating expenses to be in the range of $41 million to $43 million, which includes a $10 million milestone payment that will become due upon acceptance of our NDA submission by the FDA.

Cash used in the quarter ending March 31, 2020, will include the $48.2 million that we used to repay the Hercules Capital loan and the $10 million milestone payment due upon acceptance of our NDA. We expect to end fiscal 2019 with a cash balance of $44 million to $46 million.

Turning now to fiscal 2020. We expect fiscal 2020 to be an exciting year for us as we prepare for the launch of vibegron during the first 3 quarters and then launch vibegron if approved by the FDA in the fourth quarter.

For fiscal 2020, which begins April 1, we expect our quarterly operating expenses to be higher than the first 3 quarters -- to be higher in the first 3 quarters as compared to approximately $30 million of quarterly operating expenses, excluding stock-based compensation for fiscal 2019.

Operating expenses will increase further in the fourth quarter of fiscal 2020 as we bring our sales force onboard and launch vibegron if approved by the FDA.

With that financial update, let me turn the call back over to Keith.

--------------------------------------------------------------------------------

Keith A. Katkin, Urovant Sciences Ltd. - CEO & Director [6]

--------------------------------------------------------------------------------

Thanks, Ajay.

I'd like to reiterate our excitement for what we have accomplished during this past quarter, especially, the progress we have made with the submission of our New Drug Application for vibegron and the initiation of our Phase IIa study for URO-902.

Our new relationship with DSP provides us with a stronger and more stable financial profile, while also providing access to a global platform, enhanced commercialization resources and other significant advantages as we prepare for the launch of vibegron.

We remain very excited about the market opportunity for vibegron in OAB. Our belief in the differentiation of vibegron is further supported by what we believe has been a very strong launch in Japan.

While the market dynamics in Japan are very different than the United States, current data suggests that vibegron was able to take meaningful share from mirabegron in a short period of time.

In closing, the third fiscal quarter of 2019 was transformational for our company, marked by key milestones across all aspects of our business.

We look forward to several upcoming milestones that will continue to drive towards the goal of developing Urovant into a leading specialty urology company. These upcoming milestones include: the FDA acceptance of our New Drug Application for vibegron in OAB, which we anticipate by the end of Q1 2020; our 2 upcoming presentations in May at AUA in Washington, D.C., that includes data from our recently completed post hoc study, demonstrating the statistically significant benefit of vibegron over tolterodine at week 52; Phase IIa top line safety data for IBS-associated pain in the second half of 2020; the continued and the patient of cohort 1 enrollment in Phase IIa of our novel injectable gene therapy for OAB URO-902, followed by the initiation of cohort 2 enrollment later this year; and the ongoing evaluation of Part 2 of the Phase III COURAGE trial, which will assess both efficacy and safety of vibegron in men with OAB and BPH.

With that, I'll now open the call to questions. Operator, can you open the line?

================================================================================

Questions and Answers

--------------------------------------------------------------------------------

Operator [1]

--------------------------------------------------------------------------------

(Operator Instructions)

Your first question comes from the line of Mr. Eric Joseph from JP Morgan.

--------------------------------------------------------------------------------

Eric William Joseph, JP Morgan Chase & Co, Research Division - VP & Senior Analyst [2]

--------------------------------------------------------------------------------

You kind of anticipated one of them here. I'm just curious to get a better of a sense of what you're seeing from Kyorin and their launch of Biova in Japan? And -- understanding there's going to be differences in dosing in the label here in the U.S., but where are they seeing patient demand come from? And what's the dynamic been with mirabegron there?

--------------------------------------------------------------------------------

Keith A. Katkin, Urovant Sciences Ltd. - CEO & Director [3]

--------------------------------------------------------------------------------

Yes. Thanks for the question, Eric.

We don't have a ton of information from Japan, particularly, we don't have insight into exactly what patients are going after with their campaign. What we do have is that we've got 2 sources of information: we've got essentially the Japanese IQVIA data; and also and maybe more importantly, we've got the sales numbers that have been reported by Kyorin as they're a publicly traded company.

Both the IQVIA-like data in Japan and also the sales numbers that we're seeing from Kyorin suggest they're taking a really nice percent share. We don't want to go into the specifics, but even if we got that share within the first 12 months of our launch, we would be extraordinarily happy with the uptake of our launch as we think everybody else would be as well.

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Edited Transcript of UROV.OQ earnings conference call or presentation 13-Feb-20 9:30pm GMT - Yahoo Finance

The latest research analysis titledGlobalGene TherapyMarketgives a detailed assessment of the market where each factor, components, segments, and other sections of the market are comprehensively described. The report forecasts theGene Therapymarket to portray prominent growth during the forthcoming years from 2019 to2025. The report delivers geological study into several regions with market growth, production, consumption, and revenue. The research report focuses on critical data that makes it a very important tool for research, analysts, experts, and managers. It examines data and estimates on the market structure, dynamics, and trends.

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Excerpt from:
Global Gene Therapy Market 2019 Research Strategies and Forecasts till 2025 - Galus Australis

Novel therapies developed in the past several decades may have improved long-term survival for patients with multiple myeloma (MM) undergoing autologous stem cell transplantation (ASCT), with more patients in the past several years having what approaches a normal life expectancy, according to research published in Blood Advances. Outcomes differ, however, depending on patient age, disease risk group, and cytogenetic characteristics.

Previously published data suggest that patients with MM had the best survival outcomes in the period between 2002 and 2004, with a 5-year relative survival of 34.7%. However, inevitable disease relapses have been found to occur at increasingly short intervals in this patient population.

ASCT is a further mainstay of MM management, and there are limited data regarding outcomes among patients who undergo the total therapy approach, which involves induction therapy, ASCT, maintenance, and novel therapies as they become available for use. For this study, researchers evaluated outcomes among patients with MM who underwent total therapy and for whom long-term data were available.

Overall, 4329 patients were included in the analysis, with a median age of 58.9 years (range, 17.4-84.9) and 72.1% of patients being younger than 65 years; 2646 patients (61.1%) were male, 3723 (86%) were Caucasian, and 1861 (43%) were enrolled in a total therapy clinical trial. Patients were also categorized by the year in which they underwent ASCT: 1997 or earlier (661 patients), 1998 to 2003 (1002 patients), 2004 to 2008 (1294 patients), 2009 to 2013 (837 patients), and 2014 or later (535 patients).

The overall median follow-up was 10.5 years (range, 0.01-26.4), with patients who received ASCT in 1997 or earlier having the longest median follow-up (21.5 years) and those who received ASCT in 2014 or later having the shortest (2.5 years).

Overall survival improved over the evaluated period, with patients who received ASCT in 2014 or later having a hazard ratio for mortality of 0.35 compared with patients in the 1997 or earlier group (early mortality, 32.3% vs 36.4%). Other independent predictors of early mortality across the patient sample included age of 65 years or greater (37.2% vs 27.6% in patients younger than 65 years), high Gene Expressing Profile 70 risk status (61.4% vs 17.5% for patients with low risk), and presence of a chromosomal abnormality (41.7% vs 22.8% for patients with no abnormalities).

Being enrolled in a total therapy clinical trial was also associated with better early mortality (20.9% vs 37.9% for patients not on a total therapy protocol).

Going forward, it is clear that continued efforts to control and eradicate residual disease will be important strategies to increase the likelihood of achieving MM cure, the researchers wrote.

Reference

1. Nishimura KK, Barlogie B, van Rhee F, et al. Long-term outcomes after autologous stem cell transplantation for multiple myeloma. Blood Adv. 2020;4:422-431. doi:10.1182/bloodadvances.2019000524

Read more here:
Total Therapy Approach May Improve Transplant Outcomes in Patients With Myeloma - Hematology Advisor

This research study on Gene Therapy for Rare Disease market reports offers the comparative assessment of Gene Therapy for Rare Disease market and consist of Historical data, Significance, statistical data, size & share, Market Price & Demand, Business overview, Market Analysis By Product and Market Trends by Key Players. This Gene Therapy for Rare Disease Market is Segmented in two type on the basis of type of materials and end-users. It has global market covered in all the regions, ranging to that fundamental market, key trends and segmentation analysis are coated throughout Gene Therapy for Rare Disease market report.

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Gene Therapy for Rare Disease Market With Four Main Geographies And Their Countries - Redhill Local Councillors

Gene Therapy for Inherited Genetic Disorders Market report is designed by detailed investigation procedure to collect all the necessary data. This report contains the brief profile of leading players in the industry along with their future plans and current developments. Further, report considers the revenue generated from the market analysis and opportunity analysis to estimate the market size. The report initiates with the basic market outlook and structure along with forecast of the various segments and sub-segments.

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Gene Therapy for Inherited Genetic Disorders Market research report involves emphasis on historic along with forecast revenue of the market segments and anticipated growth rates. The chief elements driving and impacting growth market data and analytics are derived from a combination of primary and secondary sources.

The key players covered in this study, BioMarin Pharmaceutical Inc., bluebird bio Inc., Novartis AG, Orchard Therapeutics Plc, Spark Therapeutics Inc.,

No of Pages: 95

The scope of the Global Gene Therapy for Inherited Genetic Disorders Report:

Market segment by Type, the product can be split intoEye DisordersHematological DisordersCentral Nervous System DisordersMuscular DisordersOthersMarket segment by Application, split intoHospitalClinicResearch InstituteOthers

Important Aspects of Gene Therapy for Inherited Genetic Disorders Report:

Why To Select This Report:

Complete analysis on market dynamics, market status and competitive Gene Therapy for Inherited Genetic Disorders view is offered.

Forecast Global Gene Therapy for Inherited Genetic Disorders Industry trends will present the market drivers, constraints and growth opportunities.

The five-year forecast view shows how the market is expected to grow in coming years.

All vital Global Gene Therapy for Inherited Genetic Disorders Industry verticals are presented in this study like Product Type, Applications and Geographical Regions.

Table of Contents

1 Study Coverage

2 Executive Summary

3 Breakdown Data by Manufacturers

4 Breakdown Data by Type

4.1 Global Gene Therapy for Inherited Genetic Disorders Sales by Type

4.2 Global Gene Therapy for Inherited Genetic Disorders Revenue by Type

4.3 Gene Therapy for Inherited Genetic Disorders Price by Type

5 Breakdown Data by Application

5.1 Overview

5.2 Global Gene Therapy for Inherited Genetic Disorders Breakdown Data by Application

6 North America

7 Europe

8 Asia Pacific

9 Central & South America

10 Middle East and Africa

11 Company Profiles

12 Future Forecast

13 Market Opportunities, Challenges, Risks and Influences Factors Analysis

14 Value Chain and Sales Channels Analysis

15 Research Findings and Conclusion

16 Appendix

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Gene Therapy for Inherited Genetic Disorders Market by Key Players, Deployment Type, Applications, Vertical, and Region-Global 2026 Forecast -...

The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases.Now, a team of scientists from Purdue University and other research institutions around the world have come together to better understand the growing number of worldwide patented innovations available for gene therapy treatment. They specifically focus on non-viral methods, which use synthetic or natural compounds or physical forces to deliver materials generally less toxic than their viral counterparts into the therapy treatments.

The possibility of using non-viral vectors for gene therapy represents one of the most interesting and intriguing fields of gene therapy research, said Marxa Figueiredo, an associate professor of basic medical sciences in Purdues College of Veterinary Medicine, who helped lead the research team and works with the Purdue Research Foundation Office of Technology Commercialization to patent her technologies related to health. This is an innovative method for identifying the technological routes used by universities and companies across the world and uncovering emerging trends for different gene therapy sectors.

The scientists used big data, patent and clinical data mining to identify technological trends for the gene therapy field. They envision that their analysis will help guide future developments for gene therapy.

This work brought together investigators from across the globe in a joint effort to use new databases and methods to better understand the trends of the gene therapy field in respect to non-viral vectors. Dimas Covas, coordinator of the Center for Cell-based Therapy, affiliated with the University of So Paulo in Brazil, lent his extensive experience in cell therapy. Aglaia Athanassiadou, Virginia Picano-Castro and Figueiredo contributed their extensive experience on non-viral vectors for gene therapy. Cristiano Pereira and Geciane Porto brought their expertise in economics and business administration to the analyses. Each contribution was fundamental to achieving a new way to identify technological trends in this field.

This work brought together investigators from very diverse disciplines to create a different perspective of the gene therapy field, Figueiredo said. Our groups continue to work individually or in collaboration to generate and patent new vectors to help fill the needs of this re-emerging field of non-viral gene therapy.ReferencePicano-Castro et al. (2020) Emerging patent landscape for non-viral vectors used for gene therapy. Nature Biotechnology. DOI: https://doi.org/10.1038/s41587-019-0402-x

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Technological Trends in Gene Therapy - Technology Networks

Have you ever slipped when trying to avoid sugar, quit smoking, or break another habit or addiction? Usually that one piece of cake or one cigarette wont ruin your whole plan, but for people struggling with cocaine addiction, one slip can undo months of hard work.

Cocaine consumption is increasing, with 2.2 million people in the U.S. admitting to recent cocaine use in 2017. In 2014, the National Survey on Drug Use and Health estimated that nearly 1 million Americans were addicted to cocaine. The effect of cocaine on the brain and body is so powerful that, even after state-of-the-art treatments, many people trying to quit cocaine relapse within a year.

What if cocaine could be made less euphoric, so that a single use by a recovering addict doesnt result in a full-blown relapse? Scientists at the Mayo Clinic recently published progress toward making this idea a reality a gene therapy that would treat cocaine addiction by making cocaine less rewarding.

We are a molecular biologist and a neurobiologist who are interested in understanding and treating human disease, including neurological disorders such as cocaine addiction. As University of Tennessee faculty members leading basic biomedical research, we have worked for years on how genes are turned on and off in people and the effects of cocaine on mice, respectively. So, we were excited to see a promising convergence of novel gene therapy and cocaine addiction therapy.

Beginning more than 20 years ago, scientists have worked to engineer a new version of a human protein that could break down cocaine so quickly that it doesnt produce an addictive high. We all have the normal human protein BChE that helps regulate neurotransmitters, and which can slowly break down cocaine. Targeted mutations in BChE can turn it into a super-CocH a protein that can quickly break down cocaine. When this CocH is injected into the bloodstream, it breaks down cocaine very fast before the user can experience the pleasurable effects so a dose of cocaine is less rewarding. Being less rewarding means it is easier to stop using cocaine.

Previous research has shown that injections of the super-CocH protein drastically decrease addictive behavior in cocaine-addicted rats. Thats great. But the problem is that daily CocH injections would be too expensive and difficult to maintain for the years needed to prevent cocaine relapse for human users. It would be much more practical to provide a single treatment that could provide enough CocH to last for years.

One way to do that is gene therapy: Give patients the DNA sequence (the gene) that contains the instructions for making super-CocH so their bodies can keep making it for months or potentially years. Fortunately, over the past decade, this type of gene therapy has been moving from science fiction to hopeful reality. Clinical trials have demonstrated the potential of gene therapy to treat diseases from hemophilia to neurodegenerative disorders, and a handful of these are FDA-approved. The new Mayo Clinic study takes an important step toward making CocH gene therapy a reality.

How exactly does a scientist give a person a gene? You cant just swallow DNA the way you would a pill. The Mayo Clinic scientists had to find a way to deliver the gene to every cell in the liver. The way they did this was to insert the gene for super-CocH into a virus called adeno-associated virus (AAV). AAV has been modified so that when it infects cells it cannot reproduce in the body or make someone sick. It is just a delivery vehicle. The virus works by delivering the CocH gene to liver cells, where it remains for months or years. The cells read the super-CocH gene and use it to manufacture many copies of the CocH protein, which then breaks down cocaine.

In the new study, the team tested this approach in mice. The results are very promising and suggest that this gene therapy is safe and effective. Mice receiving the gene therapy alone were healthy. Mice given cocaine became hyperactive and showed signs of liver damage. When the mice were given cocaine plus gene therapy they behaved normally, as if they had not been given the drug. The cocaine was quickly broken down by their new super-CocH proteins, and their livers showed no signs of damage.

The results are promising enough that the FDA has approved plans to proceed with human clinical trials.

Keep in mind, this treatment wont hit the market anytime soon. It took six years from initial tests of AAV-CocH therapy in mice to reach the point where the technique is safe enough for human trials. There are many aspects of the treatment that need to be evaluated and modified to make sure it is both safe and effective in humans.

For example, AAV gene therapy can produce unwanted immune responses in people that will need to be carefully monitored. Issues such as discomfort caused by the therapy, different responses based on an individuals genetic makeup and interactions with other medications or medical conditions will also need to be addressed.

Because this study only monitored mice for two months, longer-term effects of the gene therapy will need to be investigated. Also, how well this therapy works to treat cocaine addiction in mice is not really known, and treating addiction in humans is certain to be even more complicated.

This gene therapy could someday make a dose of cocaine less rewarding, but a full recovery from addiction will likely require a combination of treatments administered over many years.

Like many, the two us have family members or friends who struggle with addictions that cannot be cured simply by trying harder. This recent work combines careful scientific progress with a creative new idea, giving hope to those trying to overcome cocaine addiction.

See original here:
Potential gene therapy to combat cocaine addiction - The Conversation US

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

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

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

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

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

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

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

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

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

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market by Vectors [Non-viral (Oligonucleotides), Viral (Retroviral (Gammaretroviral, Lentiviral)), Adeno-associated], Indication (Cancer, Neurological Diseases), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2024" report has been added to ResearchAndMarkets.com's offering.

High incidence of cancer & other target diseases is a major factor driving the growth of the gene therapy market

The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of this market. In addition, the strong product pipeline of market players is expected to offer significant growth opportunities in the coming years. However, the high cost of treatment is expected to hamper the market growth to a certain extent in the coming years.

Neurological diseases segment accounted for the largest share of the gene therapy market, by indication, in 2018

Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications. The neurological diseases segment accounted for the largest share of the market in 2018. This can be attributed to the increasing number of gene therapy products being approved for the treatment of neurological diseases and the high market penetration of oligonucleotide-based gene therapies.

Viral vectors segment to register the highest growth in the gene therapy market during the forecast period

The gene therapy market, by vector, has been segmented into viral and non-viral vectors. In 2018, the non-viral vectors segment accounted for the largest share of this market. However, the viral vectors segment is estimated to grow at the highest CAGR during the forecast period, primarily due to the increasing demand for CAR T-based gene therapies and the rising incidence of cancer.

North America will continue to dominate the gene therapy market during the forecast period

Geographically, the market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the gene therapy market, followed by Europe. Factors such as the rising prevalence of chronic diseases, high healthcare expenditure, presence of advanced healthcare infrastructure, favorable reimbursement scenario, and the presence of major market players in the region are driving market growth in North America.

Key Benefits of Buying the Report:

This report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall gene therapy market and its subsegments. It will also help stakeholders better understand the competitive landscape and gain more insights to position their business better and make suitable go-to-market strategies. Also, this report will enable stakeholders to understand the pulse of the market and provide them with information on the key market drivers, challenges, and opportunities.

Key Topics Covered:

1 Introduction

1.1 Objectives of the Study

1.2 Market Definition

1.3 Market Scope

1.4 Currency

1.5 Limitation

1.6 Stakeholders

2 Research Methodology

2.1 Research Data

2.2 Secondary Data

2.3 Primary Data

2.4 Market Size Estimation

2.5 Market Breakdown and Data Triangulation

2.6 Assumptions for the Study

3 Executive Summary

4 Premium Insights

4.1 Gene Therapy Market Overview

4.2 North America: Market, By Vector (2018)

4.3 Geographical Snapshot of the Market

5 Market Overview

5.1 Introduction

5.2 Market Dynamics

5.2.1 Drivers

5.2.1.1 High Incidence of Cancer and Other Target Diseases

5.2.1.2 Product Approvals

5.2.1.3 Funding for Gene Therapy Research

5.2.2 Opportunities

5.2.2.1 Strong Product Pipeline

5.2.3 Challenges

5.2.3.1 High Cost of Treatments

6 Gene Therapy Market, By Vector

6.1 Introduction

6.2 Non-Viral Vectors

6.3 Viral Vectors

7 Gene Therapy Market, By Indication

7.1 Introduction

7.2 Neurological Diseases

7.3 Cancer

7.4 Hepatological Diseases

7.5 Duchenne Muscular Dystrophy

7.6 Other Indications

8 Gene Therapy Market, By Delivery Method

8.1 Introduction

8.2 In Vivo Gene Therapy

8.3 Ex Vivo Gene Therapy

9 Gene Therapy Market, By Region

9.1 Introduction

9.2 North America

9.3 Europe

9.4 Asia Pacific

9.5 Rest of the World

10 Competitive Landscape

10.1 Overview

10.2 Market Share Analysis, 2018

10.3 Key Strategies

10.4 Competitive Leadership Mapping (2018)

10.4.1 Visionary Leaders

10.4.2 Innovators

10.4.3 Dynamic Differentiators

10.4.4 Emerging Companies

11 Company Profiles

11.1 Biogen

11.2 Gilead Sciences, Inc.

11.3 Amgen, Inc.

11.4 Novartis AG

11.5 Orchard Therapeutics Plc

11.6 Spark Therapeutics, Inc. (A Part of Hoffmann-La Roche)

11.7 Molmed S.P.A.

11.8 Anges, Inc.

11.9 Bluebird Bio, Inc.

11.10 Human Stem Cells Institute (HSCI)

11.11 SIBIONO Genetech Co., Ltd.

11.12 Shanghai Sunway Biotech Co., Ltd

11.13 Uniqure N.V.

11.14 Gensight Biologics S.A.

11.15 Celgene Corporation (A Bristol-Myers Squibb Company)

11.16 Cellectis

11.17 Sangamo Therapeutics

11.18 Mustang Bio

11.19 AGTC (Applied Genetic Technologies Corporation)

11.20 Poseida Therapeutics, Inc.

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

Read more:
Global Gene Therapy Market is Projected to Reach USD 13.0 Billion by 2024 from USD 3.8 Billion in 2019, at a CAGR of 27.8% - ResearchAndMarkets.com -...

Surge in investments in R&D activities, rise in prevalence of cancer, and increase in awareness about benefits drive the growth of the global gene therapy market. However, expensive costs related to gene therapies and undesirable responses from immune system restrain the market growth. On the other hand, unlocked potential from the emerging markets would offer new opportunities in the near future.

Based on vector type, the viral vector segment accounted for the highest market share in 2018, holding more than half of the global gene therapy market, owing to ease in modifications offered by many viruses including Lentivirus, RetroVirus & Gamma RetroVirus, and Adeno-Associated Virus (AAV) for developing gene therapy drugs. However, the non-viral vector segment is estimated to grow at the fastest CAGR of 38.8% from 2019 to 2026. This is due to technological advancements taking place in physicochemical approaches of non-viral vectors.

Based on gene type, the tumor suppressor segment is estimated to portray the highest CAGR of 52.9% during the forecast period, owing to surge in number of methodology and clinical trials adopted for the gene therapy treatment. However, the antigen segment accounted for the highest share in 2018, accounting for more than one-fifth of the market, owing to the presence of different genetic mutations and dysregulated gene expression presented by tumor cells.

Based on region, North America accounted for the highest share in 2018, contributing for nearly half of the global gene therapy market, owing to rise in prevalence of rate of cancer and increase in investments for R&D activities. However, Asia-Pacific region is expected to grow at the fastest CAGR of 45.4% from 2019 to 2026, owing to increase in number of people getting affected by chronic diseases and launches of new gene therapy products.

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Global Gene Therapy Market to Reach $6.21 Billion by 2026: AMR - PRNewswire

BOSTON and LONDON, Feb. 10, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (ORTX), a global gene therapy leader, today announced upcoming presentations from its neurometabolic franchise at the 16th Annual WORLD Symposium on February 10-13 in Orlando, FL. Accepted abstracts include encore clinical presentations for OTL-200 and emerging data quantifying metachromatic leukodystrophy (MLD) caregiver-reported quality of life experiences, as well as clinical data for investigational treatments in mucopolysaccharidosis type I (MPS-I) and mucopolysaccharidosis type IIIA (MPS-IIIA).

Neurometabolic disorders such as MLD can have a devastating, lifelong impact, not only on children but on their caregivers, support systems and the broader community, said Mark Rothera, president and chief executive officer of Orchard. We look forward to showcasing both real-world and clinical study data from our neurometabolic portfolio at the upcoming WORLD Symposium as we strive to bring about a brighter future for all those affected by rare disease.

The presentations are listed below and the full preliminary program is available online at the conference website.

Oral presentation details:

Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIA*Presenter: Jane Kinsella, Royal Manchester Childrens HospitalSession: Translational Research IIDate: Wednesday, February 12Time: 9:15-9:30 a.m. ET

Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patientsPresenter: Francesca Fumagalli, San Raffaele Telethon Institute for Gene TherapySession: Clinical Trials II: Clinical OutcomesDate: Thursday, February 13Time: 8:15-8:30 a.m. ET

Poster presentation details:

Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patientsPoster abstract #: P126Presenter: Francesca Fumagalli, San Raffaele Telethon Institute for Gene TherapySession: Poster Reception (Exhibit Hall)Date: Monday, February 10Time: 4:30-6:30 p.m. ET

Caregiver-reported impact on quality of life and disease burden in patients diagnosed with metachromatic leukodystrophy: Results of an online survey and a qualitative interviewPoster abstract #: P320Presenter: Francis Pang, Orchard TherapeuticsSession: Poster Reception (Exhibit Hall)Date: Tuesday, February 11Time: 4:30-6:30 p.m. ET

Extensive metabolic correction of mucopolysaccharidosis type I (MPS IH, Hurler syndrome) by hematopoietic stem and progenitor cell (HSPC) based gene therapy (GT): Preliminary results from a phase I/II trialPoster abstract #: LB-15Presenter: Francesca Tucci, San Raffaele Telethon Institute for Gene TherapySession: Poster Reception (Exhibit Hall)Date: Wednesday, February 12Time: 4:30-6:30 p.m. ET

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

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Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018, as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Source: Orchard Therapeutics (Europe) Limited

*Patient was treated by the Royal Manchester Childrens Hospital (RMCH) under a Specials license, granted by the UK government for the use of an unlicensed pharmaceutical product in situations of high unmet need when there is no other treatment option available. Orchard holds the license to the MPS-IIIA investigational gene therapy product (OTL-201) and is funding the proof-of-concept clinical trial being conducted at RMCH, which utilizes the same technology and procedures that were used to treat this first MPS-IIIA patient.

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Orchard Therapeutics Announces Presentation of Clinical Data from Neurometabolic Franchise at 16th Annual WORLD Symposium - Yahoo Finance

NEW YORK, Feb. 4, 2020 /PRNewswire/ --Hoth Therapeutics, Inc. (NASDAQ:HOTH), a biopharmaceutical company focused on unique targeted therapeutics for patients suffering from dermatological indications ranging from atopic dermatitis, psoriasis and acne along with gene therapy treatment for asthmatics, is pleased to announce the initiation of a preclinical study for the treatment of asthma and allergic inflammation in collaboration withNorth Carolina State University(NC State).

The study has begun thedelivery and distribution of nebulized particleswhich willenable the therapeutic oligonucleotide (oligo), short DNA and RNA molecules that have a wide range of applications in gene testing.Hoth has appointed Dr. Glenn Cruse to its Scientific Advisory Board and will oversee the Company's gene therapy programs advancements.

Mr.Robb Knie, Chief Executive Officer of Hoth Therapeutics, Inc. commented,"We are extremely pleased that our gene therapy program with NC State has officially begun and that Dr. Cruse who is overseeing the advancement ofexperimentshas joined our Scientific Advisory Board. Commencement of this initiative is an important step in the development and growth of our company. Dr. Cruse's expertise asa leading mast cell biologist in allergic and inflammatory diseases will be invaluable for the preclinical development of Splice-switching oligonucleotides (SSOs) for asthma."

In November 2019 Hoth entered into a licensing agreement with North Carolina State University (NC State) to study NC State's Exon Skipping Approach for Treating Allergic Diseases. This Exon Skipping Approach was developed by Dr. Glenn Cruse, Principal Investigator and Assistant Professor in the Department of Molecular Biomedical Sciences at the NC State College of Veterinary Medicine. During Dr. Cruse's research, a new approach for the technique of antisense oligonucleotide-mediated exon skipping to specifically target and down-regulate IgE receptor expression in mast cells was identified. These findings set a breakthrough for allergic diseases as they are driven by the activation of mast cells and the release of mediators in response to IgE-directed antigens.

Glenn Cruse completed his Ph.D. at Glenfield Hospital, The University of Leicester, UK in 2009. He then moved to the National Institutes of Health in Bethesda, Maryland in January 2010 to start a visiting postdoctoral fellowship in the Laboratory of Allergic Diseases, NIAID, In January 2015, Dr. Cruse was appointed as a Research Fellow in the same laboratory. Dr. Cruse joined the Department of Molecular Biomedical Sciences at NC State in January 2016 as an Assistant Professor.

Dr. Cruse is a mast cell biologist that has authored and co-authored over 30 publications including articles in top journals such as the New England Journal of Medicine, Proceedings of the National Academy of Sciences USA and Immunity. The Cruse lab is interested in the role that mast cells play in allergic and inflammatory diseases and identifying novel therapeutics that target mast cells. Since mast cells act as sentinel cells that participate in both innate and acquired immunity, particularly at biological barriers, emphasis on diseases in tissues at the interface with the environment such as the lung, skin, gastrointestinal tract and even the neuro-immune axis are the main focus of the lab.

About Hoth Therapeutics, Inc.Hoth Therapeutics, Inc. isa clinical-stage biopharmaceutical company focused on developing new generation therapies for dermatological disorders. HOTH's pipeline has the potential to improve the quality of life for patients suffering from indications including atopic dermatitis, chronic wounds, psoriasis, asthma and acne. To learn more, please visitwww.hoththerapeutics.com.

Forward Looking StatementsThis press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the BioLexa Platform, the commencement of clinical trials, the availability of data from clinical trials and other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Hoth's current expectations and various assumptions. Hoth believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Hoth may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Hoth's Form 10K for the period endingDecember 31, 2018, and Hoth's other filings made with the Securities and Exchange Commission. Consequently, forward-looking statements should be regarded solely as Hoth's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. Hoth cannot guarantee future results, events, levels of activity, performance or achievements. Hoth does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

ContactsInvestor Relations Contact:Phone: (646) 756-2997Email:investorrelations@hoththerapeutics.comwww.hoththerapeutics.com

KCSA Strategic CommunicationsValter Pinto (212) 896-1254Hoth@kcsa.com

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Hoth Initiates Preclinical Gene Therapy Program with NC State for the Treatment of Asthma and Allergic Inflammation - P&T Community

Biotech stocks recovered last week along with the broader markets, which bounced back from a China coronavirus-induced sell-off.

Big pharma earnings took the spotlight, with Merck & Co., Inc. (NYSE: MRK), Bristol-Myers Squibb Co (NYSE: BMY), Gilead Sciences, Inc. (NASDAQ: GILD) and AbbVie Inc (NYSE: ABBV) among the notable ones reporting in the week. Merck surprised the markets by announcing plans to spin-off its slow-growing Women's Health, Legacy Brands and Biosimilars business.

Here're the key biotech catalysts for the unfolding week.

Bio CEO & Investor Conference: Feb. 10-11 in New York16th Annual WORLDSymposium: Feb. 10-13 in Orlando, FloridaGuggenheim Healthcare Talks/Oncology Day: Feb. 13 in New YorkASCO 2020 Genitourinary Cancers Symposium: Feb. 13-15 in San Francisco, California

FDA's Tobacco Products Scientific Advisory Committee is scheduled to meet Friday to discuss the modified risk tobacco product applications submitted by 22nd Century Group Inc (NYSE: XXII) for VLN King and VLN Menthol King combusted, filtered cigarettes. The company projectsthese tobacco products to be "very low nicotine content" cigarettes.

Avrobio Inc (NASDAQ: AVRO) will present at the 2020 WORLDSymposium updates for its investigational programs in Fabry disease and cystinosis as well as data from the first clinical use of its plato gene therapy platform. At an analyst and investor event scheduled for Monday, the company will present updates on Phase 1/2 clinical trial of AVR-RD-04, an investigational gene therapy for cystinosis, and Phase 2 study of AVR-RD-01 in Fabry disease.

Regenxbio Inc (NASDAQ: RGNX) will make poster presentation of interim data from the Phase 1/2 trial of its investigational gene therapy RGX-121 for the treatment of Mucopolysaccharidosis Type II. The presentation is scheduled for Wednesday.

ASCO 2020 Genitourinary Cancers Symposium Presentations

Exelixis, Inc. (NASDAQ: EXEL): Phase 1b data for cabozantinib and Roche Holdings AG Basel ADR Common Stock's (OTC: RHHBY) Tecentriq in solid tumors (Thursday)

Advaxis, Inc. (NASDAQ: ADXS): Phase 1/2 data for ADXS-PSA and Merck's Keytruda from the KEYNOTE-046 study in castrate-resistant prostate cancer (Thursday)

TrovaGene Inc (NASDAQ: TROV): new Phase 2 data for onvansertib and zytiga in prostate cancer (Thursday)

Corvus Pharmaceuticals Inc (NASDAQ: CRVS): updated Phase 1b/2 data for CPI-444 in solid tumors

See Also: 13 Key NASH Drug Candidates To Watch For In A Potential $30-Billion Market

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Revolution Medicines, a biotech developing targeted cancer therapies using RAS pathway inhibitors, has filed to offer 10 million shares in an IPO, which is expected to be priced between $14 and $16. The company expects its shares to be listed on the Nasdaq under the ticker symbol "RVMD."

IPO Quiet Period Expiry

I-Mab (NASDAQ: IMAB)

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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The Week Ahead In Biotech: SMID-Cap Earnings In The Spotlight - Benzinga

PLYMOUTH MEETING, Pa., Feb. 10, 2020 /PRNewswire/ -- Inovio Pharmaceuticals, Inc. (NASDAQ: INO) today announced the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application to evaluate its DNA medicine INO-3107 in a Phase 1/2 trial for treatment of Recurrent Respiratory Papillomatosis or RRP. RRP is a rare disease caused by the human papillomavirus (HPV) types 6 and 11 infections, a condition that causes noncancerous tumor growths leading to life-threatening airway obstructions, and occasionally can progress to cancer. Currently, the disease is incurable and is mostly treated by surgery, which temporarily restores the airway. The tumor almost always recurs and the surgery must be repeated, often multiple times a year. RRP can severely impact the quality of life for those living with the disease.

The open-label, multicenter Phase 1/2 trial will enroll approximately 63 subjects in the U.S. and will evaluate the efficacy, safety, tolerability, and immunogenicity of INO-3107 in subjects with HPV 6 and/or 11-associated RRP who have required at least two surgical interventions per year for the past three years for the removal of associated papilloma(s). For this study, adult subjects will first undergo surgical removal of their papilloma(s) and then receive four doses of INO-3107, one every three weeks. The primary efficacy endpoint will be a doubling or more in the time between surgical interventions following the first dose of INO-3107 relative to the frequency prior to study therapy. Upon obtaining sufficient safety and potential efficacy data in adults, Inovio plans to expand the trial to include pediatric patients as well as a potential booster regimen.

"Inovio's investigational DNA medicine INO-3107 is designed to destroy and clear tumors caused by HPV 6 and 11 infections from the body exactly where they are hiding," said Jeffrey Skolnik, M.D., Inovio's Vice President of Clinical Development. "We believe this DNA medicine has the potential to provide people living with RRP a long-term, if not life-long, improvement in their disease, especially as an alternative to often successive and debilitating surgeries that may temporarily remove HPV growths from the airways but do not address the underlying recurring virus."

J. Joseph Kim, Ph.D., Inovio's President and Chief Executive Officer, said "Our mission at Inovio is to rapidly provide patients with urgent health needs access to our novel DNA medicines. We are pleased the FDA has authorized our INO-3107 clinical trial, and look forward to working closely with the RRP patient and medical community to drive recruitment as quickly as possible."

In addition to initiating this efficacy trial, Inovio also plans to attain Orphan Disease designation with the FDA's Office of Orphan Products Development (OOPD). The FDA grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. OOPD provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.

Inovio recently published data from its pilot clinical study of INO-3106 (DNA medicine candidate targeting HPV6 caused RRP) in the scientific journal Vaccines (MDPI). Study results demonstrated that INO-3106 generated immunogenicity and engagement and expansion of an HPV 6-specific cellular response, including cytotoxic T cells. The paper also showed that Inovio's immunotherapy allowed two out of two patients who previously required approximately two surgeries per year for several years to manage this disease to delay the need for surgery to a robust degree; with one patient able to delay surgery for over a year and a half (584 days surgery-free) and a second that remained surgery-free for over two and a half years (over 915 days surgery-free).

About Inovio's DNA Medicines

Inovio has 15 DNA medicine clinical programs currently in development focused on HPV-associated diseases, cancer, and infectious diseases, including the novel coronavirus (2019-nCoV) under a grant from the Coalition for Epidemic Preparedness Innovations (CEPI). DNA medicines are medicines composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

Inovio's DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using Inovio's proprietary hand-held smart device called CELLECTRA. CELLECTRA uses a brief electrical pulse to open small pores in the cell reversibly to allow the plasmids to enter. Once inside the cell, the plasmids begin replicating, thereby strengthening the body's own natural response mechanisms. Administration with the CELLECTRA device ensures that the DNA medicine is delivered directly into the body's cells, where it can go to work immediately mounting an immune response. Inovio's DNA medicines are not interfering with or changing in any way an individual's own DNA, which is the case with gene therapy or gene editing.

With more than 2,000 patients receiving Inovio investigational DNA medicines in more than 6,000 applications across a range of clinical trials, Inovio's DNA medicines have consistently activated safe, robust, and fully functional T cell and antibody responses against targeted pathogens and cancers.

About RRP

Recurrent respiratory papillomatosis (RRP) is a rare disease (estimated at 15,000 active cases in the U.S.) that is characterized by the growth of tumors in the respiratory tract caused by the human papillomavirus. Although benign, papillomas can cause severe, even life-threatening airway obstruction and respiratory complications. A distinguishing aspect of this disease is the tendency for the papilloma to recur after surgical procedures to remove them. Left untreated, if RRP develops in the lungs, affected individuals can potentially experience recurrent pneumonia, chronic lung disease (bronchiectasis) and, ultimately, progressive pulmonary failure. In extremely rare cases (less than 1%), papillomas can become cancerous (malignant transformation) developing into squamous cell carcinoma. Additional symptoms of RRP can include hoarse voice, difficulty in sleeping and swallowing, and chronic coughing. RRP symptoms are usually more severe in children than in adults. In children, the disorder is most often diagnosed at or around the age of four years. In adults, the disorder occurs most often in the third or fourth decade.

About Inovio Pharmaceuticals, Inc.

Inovio is a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to treat, cure, and protect people from diseases associated with HPV, cancer, and infectious diseases. Inovio is the first and only company to have clinically demonstrated that a DNA medicine can be delivered directly into cells in the body via a proprietary smart device to safely produce a robust immune response to destroy and clear high-risk HPV 16 and 18, which are responsible for 70% of cervical cancer, 90% of anal cancer and 69% of vulvar cancer. In addition to HPV, Inovio's optimized plasmid design and delivery technology have been demonstrated to consistently activate robust and fully functional T cell and antibody responses against targeted cancers and pathogens. Inovio's most advanced clinical program, VGX-3100, is in Phase 3 development for the treatment of HPV-related cervical pre-cancer. Also in development are Phase 2 immuno-oncology programs targeting HPV-related cancers and GBM, as well as externally funded vaccine development programs in Zika, MERS, Lassa, HIV, and the novel coronavirus (2019-nCoV). Partners and collaborators include ApolloBio Corporation, AstraZeneca, The Bill & Melinda Gates Foundation, Coalition for Epidemic Preparedness Innovations (CEPI), Defense Advanced Research Projects Agency, GeneOne Life Science, HIV Vaccines Trial Network, Medical CBRN Defense Consortium (MCDC), National Cancer Institute, National Institutes of Health, National Institute of Allergy and Infectious Diseases, Regeneron, Roche/Genentech, University of Pennsylvania, Walter Reed Army Institute of Research, and The Wistar Institute. For more information, visit http://www.inovio.com.

This press release contains certain forward-looking statements relating to our business, including our plans to develop DNA medicines, our expectations regarding our research and development programs, as well as commercialization activities, including the planned initiation and conduct of clinical trials, the availability and timing of data from those trials and our commercialization strategy and tactics. Actual events or results may differ from the expectations set forth herein as a result of a number of factors, including uncertainties inherent in pre-clinical studies, clinical trials, product development programs and commercialization activities and outcomes, the availability of funding to support continuing research and studies in an effort to prove safety and efficacy of electroporation technology as a delivery mechanism or develop viable DNA vaccines, our ability to support our pipeline of SynCon active immunotherapy and vaccine products, the ability of our collaborators to attain development and commercial milestones for products we license and product sales that will enable us to receive future payments and royalties, the adequacy of our capital resources, the availability or potential availability of alternative therapies or treatments for the conditions targeted by us or our collaborators, including alternatives that may be more efficacious or cost effective than any therapy or treatment that we and our collaborators hope to develop, issues involving product liability, issues involving patents and whether they or licenses to them will provide us with meaningful protection from others using the covered technologies, whether such proprietary rights are enforceable or defensible or infringe or allegedly infringe on rights of others or can withstand claims of invalidity and whether we can finance or devote other significant resources that may be necessary to prosecute, protect or defend them, the level of corporate expenditures, assessments of our technology by potential corporate or other partners or collaborators, capital market conditions, the impact of government healthcare proposals and other factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2018, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, and other filings we make from time to time with the Securities and Exchange Commission. There can be no assurance that any product candidate in our pipeline will be successfully developed, manufactured or commercialized, that final results of clinical trials will be supportive of regulatory approvals required to market products, or that any of the forward-looking information provided herein will be proven accurate. Forward-looking statements speak only as of the date of this release, and we undertake no obligation to update or revise these statements, except as may be required by law.

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SOURCE Inovio Pharmaceuticals, Inc.

Company Codes: NASDAQ-NMS:INO

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Inovio Receives Authorization from the US FDA To Begin Phase 1/2 Clinical Trial for INO-3107, a DNA Medicine To Treat a Rare Disease -- Recurrent...

Although vaping-related lung illnesses are declining, more than 2,700 have been reported across the U.S. and its territories since last March, according to the CDC. A total of 60 have died. Public health news is on a rare neuromuscular disease, cervical cancer, obesity, preparing for civilization's end, longevity, vocal disorders, postpartum depression, organ transplants, a sweet substitute for kids' cough medicines, Alzheimer's disease, mental health, and medical errors, as well.

The Wall Street Journal:Vaping-Related Deaths Fall, But Families Still Look For AnswersKimberly Boyd keeps a stack of her sons medical files on her dining-room table, in neatly organized folders. In a Ziploc bag, there are some of the nicotine vaping cartridges he used at their Orlando, Fla., home before the 28-year-old died in November. Across the country, in Seattle, Robin Hurt is waiting for a response to a public-record request she filed with the states medical examiner in Oregon, asking for the autopsy report on her 23-year-old grandson, who unexpectedly died in October after having recently taken up vaping. (Ansari, 2/9)

Stat:Lottery Like No Other Offers A Cutting-Edge Medicine With Lives On The LineThe treatment, a gene therapy called Zolgensma, is designed for children like Wynter who have a neuromuscular disease called spinal muscular atrophy, or SMA. Without it or other treatments, those with the most serious type are likely to die as babies. It was first approved by U.S. regulators only last year, and is not yet available in other countries. The lottery was devised by the drugs manufacturer, Novartis, to give families in those places a chance to get it through a novel form of compassionate use a way to get medications that have not been approved while they wait. Fifty doses are slotted to be given away for free in the first half of the year, with up to 100 total. The first drawing occurred Monday. (Joseph and Silverman, 2/7)

The Washington Post:WHOs Aggressive, Three-Part Strategy Aims To Make Cervical Cancer A Thing Of The PastIn just 35 years, the United States managed to reduce cervical cancer rates by 54 percent with the help of Pap smears. Now, human papillomavirus vaccination, double screening and more effective treatment might be able do away with the cancer. In two new studies in the Lancet, the World Health Organization lays out how. The studies model what might happen if the United Nations health agency commits to a three-part strategy to wipe out cervical cancer. (Blakemore, 2/8)

The New York Times:Half Of Us Face Obesity, Dire Projections ShowClimate change is not the only source of dire projections for the coming decade. Perhaps just as terrifying from both a health and an economic perspective is a predicted continued rise in obesity, including severe obesity, among American adults. A prestigious team of medical scientists has projected that by 2030, nearly one in two adults will be obese, and nearly one in four will be severely obese. (Brody, 2/10)

WBUR:Preparing For The End Of The World, On A BudgetA Harvard Ph.D. and former military intelligence officer with 30 years of experience, Miller would know a good defensible spot when he sees it. Miller is a self-described "prepper," someone who makes active preparations to survive the fall of human civilization. The nationwide prepper community is often painted as composed of conspiracy-crazed eccentrics, he said, thanks in large part to television shows such as the National Geographic Channel's Doomsday Preppers. (Boyce, 2/10)

The Washington Post:For Aging, Strength Training Is Vital In Avoiding Injuries And Staying IndependentWhen an intruder broke into the Rochester, N.Y., home of 82-year-old Willie Murphy a few months ago, he was met with a big surprise. Murphy, a diminutive but powerlifting woman, quickly jumped into action, using her strength to pummel the intruder with a broom and send him running for the door. Not surprisingly, the story went viral as people embraced the images of the elderly Murphy flexing her muscles for the cameras. (Loudin, 2/9)

Stat:The Vodka Trial: In Search Of A Treatment For Vocal Disorders, A Researcher Puts Patient Anecdotes To The TestPharmacists had prepared the therapy specifically for her, in little reddish bottles that reminded her of liquid penicillin. A research assistant gave her careful instructions. But Feeley already knew exactly what do to do. What do you do with a shot of vodka? Basically, you pour it down your neck. So I drank it and then banged the bottle on the table, Feeley said. Usually, shes more of a wine or daiquiri sort of person, but shed put aside her taste and a few days of her time to help answer a question that had been rattling around Simonyans mind. It had first appeared about 15 years ago, when Simonyan was doing neuroscience research in New York City, trying to understand exactly how the brain choreographs the intricate dance of muscle and air that gives rise to speech. (Boodman, 2/10)

CNN:Women Who Have General Anesthesia During C-Sections Are More Likely To Experience Postpartum Depression, Study FindsWomen who have general anesthesia during C-sections are significantly more likely to experience severe post-partum depression resulting in hospitalization, suicidal thoughts or self-harm, according to a study published last week. That might be because general anesthesia can delay breastfeeding and skin-to-skin interaction between the mother and infant, and often results in more acute and persistent pain after childbirth, researchers from Columbia University explained. "These situations are often coupled with a new mother's dissatisfaction with anesthesia in general, and can lead to negative mental health outcomes," said Jean Guglielminotti, lead author and an assistant professor of anesthesiology at Columbia, in a news release. (Kaur, 2/8)

Kaiser Health News:How Lifesaving Organs For Transplant Go Missing In TransitWhen a human heart was left behind by mistake on a Southwest Airlines plane in 2018, transplant officials downplayed the incident. They emphasized that the organ was used for valves and tissues, not to save the life of a waiting patient, so the delay was inconsequential. It got to us on time, so that was the most important thing, said Doug Wilson, an executive vice president for LifeNet Health, which runs the Seattle-area operation that processed the tissue. (Aleccia, 2/10)

NPR:A Cough Cure For Kids? Try HoneyIf you don't have little kids, or it's been a while, let me just break down for you why kids' coughs can be a truly miserable problem that can drive you to madness. Imagine this: Your kid's coughing it's almost always worse at night then they start crying because they're tired and can't sleep with all the coughing. The coughing and crying means that not only do they not sleep, but you also don't sleep no one in the house sleeps and this can go on for weeks. (Simmons-Duffin, 2/8)

Kaiser Health News:Finding Connections And Comfort At The Local CafeDoug and Connie Moore met at seminary. He was a student and pastor of an inner-city congregation, and she was a student and a public health nurse. Shes the one who drew me to the needs of the poor, Doug says. The pair wed in 1974, and Doug became a pastor at the First Evangelical Free Church of Los Angeles in 1983. They became deeply involved in their community and dedicated much of their free time to teaching English as a second language, creating tutoring programs and mentoring students in poor communities here and abroad. (De Marco, 2/10)

The Washington Post:How Anxiety And Midlife Crisis Are Playing Out Among Gen-X WomenThree years ago, Ada Calhoun couldnt sleep. The 41-year-old writer stared at her ceiling wondering why her hard-won accomplishments had left her feeling exhausted, anxious and wanting. She had written two well-regarded books and a long list of magazine articles. She was married and her son had just been accepted to a great public middle school. From the outside, she knew, her life looked good. (Schaaff, 2/8)

USA Today:Mom Of HBO's Bleed Out Filmmaker Steve Burrows Dies 11 Years After Hip Surgery, Brain DamageThe producer and director of an HBO documentary about his mother's medical errors at a Milwaukeehospital went to Capitol Hill lastweek to show clips of the film.Three nights later,Steve Burrows' mother, Judie, diedat 79 after an 11-year struggle to survive. Friday, the Milwaukee medical examiner's office saidher death was due to complications of repeat hip surgeries.(O'Donnell, 2/8)

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Investigation Into Vaping-Related Deaths Continues: With No Known Cause, Relatives Struggle To Find Out What Killed Family Members - Kaiser Health...