Category Archives: Gene therapy

According to the recent GlobalData report Looking Ahead to 2022 The Future of Pharma, sales of biologics are forecast to significantly overtake those of innovative small molecules over the next five years, with biologic sales forecast to be $120bn greater than small molecule sales by 2027. This emphasises the continuing trend seen within the pharmaceutical industry of the continuing rise and dominance of biologics, and the whole sector pivot towards them was highlighted within the GlobalData webinar Trends in New Drug Approvals and Manufacturing.

As shown in Figure 1, nearly all of the subtypes of biologics GlobalData tracks are expected to grow significantly in sales revenue and will account for 55% of all innovative drug sales by 2027. Biologics sale are currently largely driven by monoclonal antibodies such as Ono Pharmaceuticals Opdivo, Regeneron Pharmaceuticals Dupixent and Mercks Keytruda, which are forecast to account for 46% of all biologic sales in 2027. Keytruda alone, which is marketed for oncology indications like non-small cell lung cancer, head and neck cancer and squamous cell carcinoma, is forecast to account for 4% of all biologics sales in 2027.

While monoclonal antibodies are projected to dominate biologic sales in 2027, gene therapies and gene-modified cell therapies are expected to display the greatest growth, with a forecast increase of more than 1,000% for both molecule types between now and 2027. Unlike monoclonal antibodies, however, gene therapy sales are being driven by currently unapproved pipeline therapies such as Rocket Pharmaceuticals RPA-501, which is currently only in Phase I and is indicated for glycogen storage disorders, a rare genetic disorder.

Despite the significant growth and predicted dominance of biologics in the future, small molecule sales are also forecast to continue to grow, with small molecule sales forecast to grow by 49% in 2027. This growth is driven by drugs for the central nervous system therapy area such as Bristol-Myers Squibbs Zeposia, which is indicated for multiple sclerosis and is forecast to become a blockbuster drug by 2027, with sales of more than $2bn.

Biologics have solidified their lead over small molecules and are now primary engines of value creation for large-cap pharma companies. As this trend continues, the increasing dominance of biologics within the sales of large-cap and mega-cap bio/pharma companies will be seen. This will have consequences not only for the sponsors, but also, as discussed in the webinar Trends in New Drug Approvals and Manufacturing, lead to an increase in contract manufacturing outsourcing for cell and gene therapies.

Process Innovation in Vacuum Drying and Vacuum Pumps

Contract Manufacturing and Packaging Services

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Biologic sales forecast to pass innovative small molecule sales in next five years - Pharmaceutical Technology

The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight.

Cellular therapy products include cellular immunotherapies, cancer vaccines, and other types of both autologous and allogeneic cells for certain therapeutic indications, including hematopoetic stem cells and adult and embryonic stem cells. Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. CBER has approved both cellular and gene therapy products a list of these products may be found here.

Cellular and gene therapy-related research and development in the United States continue to grow at a fast rate, with a number of products advancing in clinical development. In addition to regulatory oversight of clinical studies, CBER provides proactive scientific and regulatory advice to medical researchers and manufacturers in the area of novel product development.

07/09/2021

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Cellular & Gene Therapy Products | FDA

...FLOOD WATCH REMAINS IN EFFECT UNTIL 6 AM EDT EARLY THIS MORNING...* WHAT...Flooding caused by excessive rainfall continues to bepossible.* WHERE...Portions of New Jersey...and Pennsylvania...including thefollowing areas...in New Jersey...Hunterdon, Mercer, Middlesex,Morris, Somerset, and Warren. In Pennsylvania...EasternMontgomery, Lehigh, Lower Bucks, Northampton, Philadelphia, UpperBucks, and Western Montgomery.* WHEN...Until 6 AM EDT early this morning.* IMPACTS...Excessive runoff may result in flooding of rivers,creeks, streams, and other low-lying and flood-prone locations.* ADDITIONAL DETAILS...- Rainfall amounts of one half to an inch and a half of rainhave already fallen across the watch area. Heavy rainfallrates of 1/4 to 1/2 inch per hour are possible throughdaybreak, which may result in additional rises of creeks andstreams.- http://www.weather.gov/safety/flood

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Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. - 69News...

Hemophilia A and B are congenital X-linked bleeding disorders that stem from a deficiency in factor VIII (FVIII), in type A, or factor IX (FIX) in type B. The standard of care for treating patients with hemophilia has consisted of replacement therapy since the 1950s.

In a paper written by Margareth C Ozelo, MD, PhD, and Gabriela G Yamaguti-Hayakawa, MD, Hemocentro UNICAMP, University of Campinas, the authors explored the Considerable progress thats been made in advancing replacement therapy and therefore improving patient care and quality of life. However, obstacles still arise that constrain access and adherence to appropriate treatment for individuals with hemophilia worldwide.

By taking plasma-derived, or recombinant products, and using them to replace the missing clotting factor, hemostasis is restored and the adoption of prophylactic therapy is facilitated. The half-life of these treatments has also recently been extended, offering a longer duration of viability.

Despite the progress achieved with these bioengineered clotting factors, this treatment comes with challenges. Aside from the burden of financial costs, the procedure entails frequent intravenous injections and patients can still potentially develop inhibitors, essentially negating the purpose of the treatment.

Non-replacement therapy can mitigate some of the hindrances of replacement therapy. This class of products was designed to go beyond the replacement strategy with the aim to attain hemostasis through the method of mimetic products, or inhibit the anticoagulant pathways and stabilize hemostasis.

Emicizumab is the first and only non-replacement agent licensed for prophylaxis. It can be a good candidate for children since its administered subcutaneously making it easier to adhere to treatment. This therapy is also more effective for patients with neutralizing anti-FVIII or anti-FIX antibodies, who experience frequent bleeding episodes.

Achieving lasting plasma factor levels with a one-time treatment has been an area of focus for decades, which also applies to the gene therapy clinical trials for hemophilia. The trials include intravenous administration of the liver-directed delivery of FVIII or FIX transgene through recombinant non-integrating AAV vectors.

The first AAV liver-directed gene therapy clinical trial for hemophilia B was critical to the current success of clinical trials for hemophilia. Essential issues related to the AAV vector immunogenicity were revealed when the vector was administered into the hepatic artery. In the last decade, AAV vector-mediated gene therapy clinical trials for hemophilia A and B have produced promising results. Some patients even presented a meaningful expression of FVIII or FIX, bringing their phenotype from severe to mild or normal factor levels after a single treatment.

Authors explained that these novel agents, like extended half-life concentrates and emicizumab, have reached resource-constrained countries through the continuous efforts of the World Federation of Haemophilia Humanitarian Aid Program. The main challenge continues to be the inequity of access across the globe.

The pharmaceutical companies Sanofi and Sobi announced the donation of 1 billion IU of EHL FVIII and FIX concentrates over 10 years, in 2014. Until then donations were limited for emergencies. This expanded the amount of people and countries who benefited from the program and the goals of the initiative.

Several pharmaceutical companies continue to help increase access to treatment through the Humanitarian Aid Program, authors wrote. Despite the challenges and logistical restrictions with the COVID-19 pandemic, the program was not interrupted and was crucial to guarantee access to treatment for people in 69 countries in 2020.

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A Look at Global Hemophilia Treatment: Replacement, Non-Factor and Gene Therapies - MD Magazine

For Immediate Release: December 18, 2017

Espaol

The U.S. Food and Drug Administration today approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

Todays approval marks another first in the field of gene therapy both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses, said FDA Commissioner Scott Gottlieb, M.D. Were at a turning point when it comes to this novel form of therapy and at the FDA, were focused on establishing the right policy framework to capitalize on this scientific opening. Next year, well begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters including new clinical measures for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted.Luxturna is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.

Hereditary retinal dystrophies are a broad group of genetic retinal disorders that are associated with progressive visual dysfunction and are caused by mutations in any one of more than 220 different genes. Biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in the U.S. Biallelic mutation carriers have a mutation (not necessarily the same mutation) in both copies of a particular gene (a paternal and a maternal mutation). The RPE65 gene provides instructions for making an enzyme (a protein that facilitates chemical reactions) that is essential for normal vision. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision. Individuals with biallelic RPE65 mutation-associated retinal dystrophy experience progressive deterioration of vision over time. This loss of vision, often during childhood or adolescence, ultimately progresses to complete blindness.

Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patients vision loss. Luxturna uses a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene to the retinal cells to restore vision.

The approval of Luxturna further opens the door to the potential of gene therapies, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research (CBER). Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed.

Luxturna should be given only to patients who have viable retinal cells as determined by the treating physician(s). Treatment with Luxturna must be done separately in each eye on separate days, with at least six days between surgical procedures. It is administered via subretinal injection by a surgeon experienced in performing intraocular surgery. Patients should be treated with a short course of oral prednisone to limit the potential immune reaction to Luxturna.

The safety and efficacy of Luxturna were established in a clinical development program with a total of 41 patients between the ages of 4 and 44 years. All participants had confirmed biallelic RPE65 mutations. The primary evidence of efficacy of Luxturna was based on a Phase 3 study with 31 participants by measuring the change from baseline to one year in a subjects ability to navigate an obstacle course at various light levels. The group of patients that received Luxturna demonstrated significant improvements in their ability to complete the obstacle course at low light levels as compared to the control group.

The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear.

The FDA granted this application Priority Review and Breakthrough Therapy designations. Luxturna also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The sponsor is receiving a Rare Pediatric Disease Priority Review Voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive Priority Review of a subsequent marketing application for a different product. This is the 13th rare pediatric disease priority review voucher issued by the FDA since the program began.

To further evaluate the long-term safety, the manufacturer plans to conduct a post-marketing observational study involving patients treated with Luxturna.

The FDA granted approval of Luxturna to Spark Therapeutics Inc. The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Luxturna is the first gene therapy approved in the U.S. to target a disease caused by mutations in a specific gene

Andrea Fischer301-796-0393

888-INFO-FDAOCOD@fda.hhs.gov

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FDA approves novel gene therapy to treat patients with a ...

BERKELEY, Calif., April 18, 2022 /PRNewswire/ -- Bakar Labs, the incubator at UC Berkeley's Bakar BioEnginuity Hub (BBH), announced today a unique collaboration with the Cystic Fibrosis Foundation to accelerate the application of new technologies for the treatment of cystic fibrosis. The CF Foundation is sponsoring a "Golden Ticket" competition at the new incubator and is encouraging companies with emerging technology in gene editing, gene delivery, and gene therapy/gene insertion that may work in CF to apply. Applications open Monday, May 2.

Up to three potential winners will be provided lab space and facilities at Bakar Labs. In addition to lab and office space, companies will have access to the extensive resources of the CF Foundation, including scientific experts and advice, lab/research tools and techniques, an extensive patient registry, clinical trial design support, and a Therapeutics Development Network of over 90 clinical trial sites in the US.

"Working with the CF Foundation entirely fits with the spirit of entrepreneurship to benefit society," said Regis Kelly, PhD, OBE, director of Bakar Labs and executive director at QB3, the University of California entrepreneurship institute that partnered with Berkeley to launch BBH. "Our double bottom line at Bakar Labs emphasizes public good on an equal basis with the potential for profit."

"This collaboration is inspiring," said David Schaffer, PhD, executive director of BBH. "Students and professors will see the potential of entrepreneurship fused with the resources and mission of a major patient advocacy organization. It will be a tremendous example that could well spur partnerships in a broad range of areas." Schaffer is Hubbard Howe Distinguished Professor at UC Berkeley, with appointments in the departments of chemical and biomolecular engineering, bioengineering, and molecular and cell biology.

"Collaborating with the CF Foundation creates an extraordinary opportunity," said Gino Segr, PhD, managing director of Bakar Labs. "We will draw attention to a devastating condition and make available special resources that will inspire and support a community of researchers and entrepreneurs to apply their breakthrough ideas to the development of a cure. We're delighted that Bakar Labs is now a proving ground for major advances."

A focus on entrepreneurship for the public good is now widely seen by many philanthropies and investors as a valuable and effective complement to their support of academic scholarship. The CF Foundation has achieved global admiration for its support of disease-related research efforts, not only by scientists in academic labs but by entrepreneurs in startup companies.

For more information, visit the CF Foundation Golden Ticket page on the Bakar Labs website or contact [emailprotected].

About the Bakar BioEnginuity Hub

The Bakar BioEnginuity Hub empowers fearless founders and founders in the making to realize bold solutions to our world's most pressing problems. Focused on people working at the convergence of the life sciences with the physical, engineering, and data sciences, the Bakar BioEnginuity Hub provides the intellectual, entrepreneurial, and community resources needed to learn and then to launch their own ventures. The Bakar BioEnginuity Hub is located on the UC Berkeley campus in the stunning Woo Hon Fai Hall. Visit bioenginuityhub.berkeley.edu.

About Bakar Labs

Bakar Labs is the flagship life science-focused incubator at UC Berkeley's Bakar BioEnginuity Hub. Operated by QB3, Bakar Labs provides extensive equipment, lab and office facilities, and a community of like-minded entrepreneurs to helps startups grow. Bakar Labs can support as many as 50 early-stage companies from around the world focused on translating life science based innovations that promise to improve human health. No UC affiliation is required to join. For information about how to join or form a partnership, visit bakarlabs.berkeley.edu.

Contact:

Kaspar Mossman(415) 514-9790[emailprotected]

SOURCE Bakar Labs

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Bakar Labs and the Cystic Fibrosis Foundation Support Entrepreneurs to Develop New Genetic Therapies for CF - PR Newswire

Clinical Neurosciences

Location: Southampton General HospitalSalary: 31,406 to 37,467 Per annumFull Time Fixed Term until 30 June 2023Closing Date: Wednesday 11 May 2022Interview Date: To be confirmedReference: 1784522FC

HAVE YOU GOT THE VISION TO CHANGE THE FUTURE?

The University of Southampton is committed to excellence in all we do, applying our insights and inventiveness to solve the most complex societal and environmental challenges. As a world-leading, research-intensive university, with a strong and high-quality educational offering, we are renowned for our innovation and enterprise and are within the top 1% of universities worldwide.

The Vision Sciences Research Group, based in the School of Clinical and Experimental Sciences have an exciting opportunity for a Post-doctoral Research Fellow to support an industry funded project: a proof of concept study to assess the efficacy of a gene therapy product in a laser-induced choroidal neovascularisation (CNV) mouse model.

You will join an established interdisciplinary team of research staff, students and senior academics studying retinal disease therapies. You will have prior molecular and cell biology laboratory experience, preferably involving animal (mouse) tissue. Your direct responsibilities will involve the downstream processing and analysis of mouse tissue from gene therapy experiments specifically: ELISA, qPCR, immunohistochemistry and confocal microscopy.

Additionally, you will lead in the statistical analysis of experimental results, the preparation of figures for peer-reviewed publications and the presentation of results at appropriate events. You will have a strong background in the skills involved in the day-to-day running of a research laboratory, such as preparing/updating risk assessments and standard operating procedures, maintaining laboratory consumables and equipment as well as supervising technical & junior research staff, as required.

Practical laboratory and statistical analysis skills are essential for this role, as well as an attention to detail and an ability to work pro-actively with colleagues. Prior experience of working with animal tissue and a history of publication in scientific journals would be a distinct advantage.

This post-doctoral position is full-time and fixed term until 30 June 2023. Informal inquiries should be directed to Prof Andrew Lotery, Chair of Ophthalmology, (A.J.Lotery@soton.ac.uk) in the first instance.

Applications for Research Fellow positions will be considered from candidates who are working towards or nearing completion of a relevant PhD qualification. The title of Research Fellow will be applied upon successful completion of the PhD. Prior to the qualification being awarded the title of Senior Research Assistant will be given.

Application Procedure:

You should submit your completed online application form at https://jobs.soton.ac.uk. The application deadline will be midnight on the closing date stated above. If you need any assistance, please call Jane Sturgeon (HR Recruitment Team) on +44 (0) 23 8059 4043 or email recruitment@soton.ac.uk Please quote reference 1784522FC on all correspondence.

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Research Fellow in Retinal Gene Therapy job with UNIVERSITY OF SOUTHAMPTON | 290122 - Times Higher Education

IRVINE, Calif. & BASEL, Switzerland--(BUSINESS WIRE)--Urovant Sciences, a wholly-owned subsidiary of Sumitovant Biopharma Ltd., announced that data from a Phase 2a trial of the investigational, novel gene therapy, URO-902, will feature as a late-breaker at the 2022 annual meeting of the American Urological Association (AUA2022), May 13-16, in New Orleans, Louisiana. The plenary presentation will include interim efficacy and safety data on URO-902 from the ongoing Phase 2a trial.

In addition, two podium presentations at AUA2022 will feature new analyses of data from the EMPOWUR 40-week extension trial of GEMTESA (vibegron) 75 mg, a Phase 3, randomized, double blind, active-comparator controlled multicenter study to evaluate long-term safety and efficacy in patients with symptoms of OAB. GEMTESA is approved by the U.S. Food and Drug Administration (FDA) for the treatment of OAB in adults with symptoms of UUI, urgency, and urinary frequency.

Overactive bladder remains a condition in need of additional treatment options. We look forward to sharing new data related to the use of GEMTESA in the OAB patient population as well as providing an initial read-out on the progress of our investigational gene therapy, URO-902, said Sef Kurstjens, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Urovant Sciences. We believe that URO-902 could potentially offer a new treatment option for patients with overactive bladder who have been inadequately managed by oral pharmacologic therapy, if approved by the FDA. The two podium presentations on GEMTESA will also add to the scientific and medical communitys understanding of this important therapy.

Data on the potential novel gene therapy, URO-902, will be presented during Friday mornings plenary session:

Late-Breaking Abstract PLLBA-03, presented by Kenneth M. Peters, M.D., principal investigator, and Chief of the Department of Urology at Beaumont Hospital, Royal Oak; Medical Director of the Beaumont Womens Urology and Pelvic Health Center and professor and Chair of Urology of the Oakland University William Beaumont School of Medicine in Rochester, Michigan., titled, Efficacy and Safety of a Novel Gene Therapy (URO-902; pVAX/hSlo) in Female Patients with Overactive Bladder and Urge Urinary Incontinence: Results from a Phase 2a Trial. This presentation will take place on Friday, May 13, at 11:21 to 11:29 a.m. CDT during the plenary session in the Ernest N. Morial Convention Center, Great Hall A.

Data on GEMTESA will also be featured in two podium presentations at the conference on May 15, 2022:

Abstracts are available in the Journal of Urology at the following links:

URO-902: https://www.auajournals.org/doi/10.1097/JU.0000000000002671.03

EMPOWUR-EXT older adults: https://www.auajournals.org/doi/10.1097/JU.0000000000002596.11

EMPOWUR-EXT PRO: https://www.auajournals.org/doi/10.1097/JU.0000000000002596.12

About the Phase 2a Study of URO-902

This 48-week multicenter, randomized, double blind, placebo-controlled, dose-escalation study will evaluate the efficacy, safety, and tolerability of a single administration of URO-902, a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. URO-902 is administered via direct intradetrusor injections via cystoscopy into the bladder wall under local anesthesia in patients who are experiencing OAB symptoms and UUI.

The Phase 2a trial includes 80 female patients in two cohorts. The first cohort received either a single administration of 24 mg of URO-902 or matching placebo into the bladder wall, and the second cohort received 48 mg of URO-902 or matching placebo into the bladder wall. Patients will be followed for up to 48 weeks after initial administration. Exploratory endpoints included change from baseline to week 12 in mean daily micturitions, urgency episodes, UUI episodes, and quality of life measures, as well as assessing the safety and tolerability of this investigational gene therapy for OAB.

About URO-902

URO-902 has the potential to be the first gene therapy for patients with OAB. If approved, this innovative treatment has the potential to address an unmet need for patients who have failed oral pharmacologic therapies.

About the EMPOWUR Trial

The EMPOWUR trial was an international, randomized, double-blind, placebo and active comparator-controlled Phase 3 clinical trial evaluating the safety and efficacy of investigational vibegron in men and women with symptoms of overactive bladder, including frequent micturition, urgency, and UUI. A total of 1,518 patients were randomized across 215 study sites into one of three groups for a 12-week treatment period with a four-week safety follow-up period: vibegron 75 mg administered orally once daily; placebo administered orally once daily; or tolterodine ER 4 mg administered orally once daily.

About the 40-Week EMPOWUR Extension

The EMPOWUR 40-week extension trial was a Phase 3, randomized, double blind, active-comparator controlled multicenter study to evaluate the long-term safety and efficacy of vibegron in patients with symptoms of overactive bladder. The extension study enrolled approximately 500 EMPOWUR completers. The primary endpoint was safety, measured by incidence of adverse events. Secondary endpoints were changes from EMPOWUR baseline at week 52 in average daily micturitions, UUI, urgency, and total urinary incontinence.

About Overactive Bladder

Overactive bladder (OAB) is a clinical condition that occurs when the bladder muscle contracts involuntarily. Symptoms may include urinary urgency (the sudden urge to urinate that is difficult to control), urgency incontinence (unintentional loss of urine immediately after an urgent need to urinate), frequent urination (usually eight or more times in 24 hours), and nocturia (waking up more than two times in the night to urinate).1

Approximately 30 million Americans suffer from bothersome symptoms of OAB, which can have a significant impairment on a patients day-to-day activities.1, 2

About GEMTESA

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

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

IMPORTANT SAFETY INFORMATION

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

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

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

What are the possible side effects of GEMTESA?

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

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

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

Please click here for full Product Information for GEMTESA.

About Urovant Sciences

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

About Sumitovant Biopharma

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

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Urovant Sciences to Present Interim Data from Phase 2a Study of Potential Novel Gene Therapy, URO-902, and New Analyses of Data from Phase 3 EMPOWUR...

BOSTON Hydrocephalus, or water on the brain, occurs when the cerebral ventriclesfour interconnected cavities of the brain that are filled with cerebrospinal fluidbecome enlarged, but its cause is unknown in many cases. A better understanding could lead to improved treatments for hydrocephalus, which is the leading reason for brain surgery in children and is associated with neurodevelopmental disability. To provide insights, a team led by investigators at Massachusetts General Hospital (MGH) and Yale University analyzed variations in genetic sequences and gene expression patterns in the brains of patients with congenital hydrocephalus. The research, which is published in Nature Neuroscience, indicates that hydrocephalus does not result from a defect of cerebrospinal fluid plumbing but rather arises because primitive cells in the brain do not behave properly during development.

In patients with hydrocephalus, continued accumulation of fluid dilates the cerebral ventricles, increases pressure in the skull, and compresses the surrounding brain structure. This compression can cause acute symptoms such as vomiting and headache, and even coma or even death. In the long-term, brain compression can lead to neurocognitive issues and neurodevelopmental disabilities in children, even when a medical device called a shunt is surgically placed in the brain.

Neurosurgical shunting of cerebrospinal fluid addresses some consequences of the disease but does not target the underlying mechanisms, says senior author Kristopher T. Kahle, MD, PhD, director of Pediatric Neurosurgery at MGH and director of the Harvard Center for Hydrocephalus and Neurodevelopmental. Knowing the molecular cause of disease could be very helpful towards clinical decision making.

To provide insights, Kahle and his colleagues genetically sequenced cells from 483 children with hydrocephalus and their unaffected parents, using a profiling technology that uncovers gene mutations in patients across the entire genome. By combining the genetic sequence data with gene expression data, the team found that many hydrocephalus-associated genes converge not in fluid circulation components but instead in neuroepithelial cells, which are the earliest stem cells of the brain that arise during the first several weeks of development. These cells go on to generate all of the neurons and support cells of the brain.

This began to hint to us that rather than affecting fluid circulation, hydrocephalus gene mutations may be disrupting the earliest processes of human brain development to cause hydrocephalus, says colead author Phan Q. Duy, an MD/PhD student at Yale University School of Medicine.

The most frequently mutated gene in the studys patientscalled TRIM71codes for a protein that is part of a pathway that regulates the timing of stem cell development. When the investigators bred mice to express TRIM71mutations, the mice developed fetal-onset hydrocephalus similar to human patients. Mechanistically, stem cells in the brains of the Trim71-mutated mice prematurely generated neurons, leading to a deficient pool of stem cells to support brain growth and development. This caused deficient expansion of brain tissue and underdevelopment of the cerebral cortex.

The scientists note that the resulting altered structure of the brain is not capable of holding the pressure exerted by cerebrospinal fluid, and thus the brain deforms and its ventricles passively expand. The site of pathology is therefore not happening in the fluid itself, but rather the vesselor the brain tissuethats holding the fluid, says Duy.

The findings suggest that treatment strategies for hydrocephalus should go beyond draining fluid in the brain. A more nuanced treatment approach may include not only cerebrospinal fluid diversion but also other approaches more tailored towards improving neurodevelopmental function, says Kahle. In the long-term, with continued gene discovery and better understanding of how other gene mutations disrupt brain development to cause hydrocephalus, we may be able to develop drug treatments or even gene therapy to correct the gene mutations months before the birth of patients.

Beyond providing a better understanding hydrocephalus, this work may offer additional insights into other pediatric brain disorders. In fact, ventricular dilation is a common feature in developmental neuropsychiatric diseases such as autism and schizophrenia, and many of the processes involved with hydrocephalus may also be relevant for other structural brain malformations.

This work was supported by the National Institutes of Health, Rudi Schulte Institute, and the Hydrocephalus Association.

About the Massachusetts General Hospital

Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The Mass General Research Instituteconducts the largest hospital-based research program in the nation, with annual research operations of more than $1 billion and comprises more than 9,500 researchers working across more than 30 institutes, centers and departments. In August 2021, Mass General was named #5 in theU.S. News & World Reportlist of "Americas Best Hospitals." MGH is a founding member of the Mass General Brigham health care system.

Nature Neuroscience

Impaired neurogenesis alters brain biomechanics in a neuroprogenitor-based genetic subtype of congenital hydrocephalus

4-Apr-2022

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Genetic analysis provides insights into the cause of hydrocephalus, or water on the brain - EurekAlert

The cessation of operations does not come as a complete surprise.The biotech, in an earnings call in November last year, had flagged that it would run out of cash at some point in the second quarter.

According to Fridays SEC filing, a strategic process to find a solution, with Kaleido having engaged professional advisors, did not result in the identification of any viable transactions.

The company had already shrunk its workforce, back in January this year, following its decision to halt a planned phase 2 trial in chronic obstructive pulmonary disease (COPD) and to end its agreement with the COPD Foundation.

Last year, Kaleido received a warning letter from the US Food and Drug Administration (FDA) over a program it was running with its COPD drug in COVID-19 studies. The FDA said the company failed to seek an Investigational New Drug (IND) application for the study using its COPD therapy, KB109. Kaleido argued that it investigated KB109 as a food rather than a drug and did not need to be authorized under an IND.

Kaleidos closure comes as several other biotechs are experiencing a cash crunch and as investor sentiment in the sector would seem to be waning, for now. Biogen, bluebird bio, and Taysha, among others, have announced layoffs of late.

Last Tuesday, gene therapy player, bluebird Bio, reported it was cutting staffing numbers by nearly 30%, with it targeting up to US$160m in cost savings over the next two years. The restructuring drive is expected to lower the companys 2022 cash burn to less than $340m, with a 35 to 40% reduction in operating costs anticipated by year-end 2022.

In March, the biotech had warned that its financial position raised substantial doubt about its ability to continue as a going concern.

Bluebird bio has faced a number of unexpected hurdles recently in its bid to get approval for its investigational therapies.

In December 2021, the FDA paused a trial of its gene therapy candidate - lovo-cel - for sickle cell disease patients under the age of 18, while, in January this year, the US regulatory body extended the review period for the biologics licensing applications (BLA) for bluebird's lentiviral vector gene therapies betibeglogene autotemcel (beti-cel) for beta-thalassemia and elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD).

CEO Andrew Obenshain, on a call last week, noted the combination of those setbacks plus a tough biotech market has taken some traditional financing off the table in the near term. Were optimistic that these options may be viable sources of funding in the future, but we recognize the need for action today.

Bluebird said it now intends to sharpen its focus on near-term catalysts, including anticipated FDA approvals for both of its gene therapies and that it also expects to submit a BLA for lovo-cel in Q1 2023.

The company outlined how it is intending to maintain targeted research efforts focused on in vivo lentiviral vector (LVV) gene therapies and that it will deprioritize direct investments in reduced toxicity conditioning and cryopreserved apheresis.

Texas based Taysha, a biotech also focused on gene therapies, announced a 35% reduction in employees at the end of March. It has narrowed its R&D pipeline to two programs: giant axonal neuropathy (GAN) and Rett syndrome.

Activities for other ongoing clinical programs will be minimized and all additional research and development will be paused to increase operational focus and efficiency, it added.

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Kaleido Biosciences is winding up operations as bluebird bio and other biotech firms announce layoffs - BioPharma-Reporter.com