Archive for October, 2020

This article was originally published here

J Racial Ethn Health Disparities. 2020 Oct 6. doi: 10.1007/s40615-020-00878-5. Online ahead of print.

ABSTRACT

Prior to embarking on a large descriptive evaluation of genetic/racial variations in symptom phenotype, we sought foundational information to determine racial differences in (1) feasibility (consent) and acceptability of collecting genomic samples, (2) genetic literacy, and (3) concerns of genomic research during breast cancer (BC) chemotherapy. Women with early-stage BC undergoing chemotherapy were recruited from an academic, urban breast care center. Information was collected for consent to participate, genetic literacy, and concerns about genetic testing in Black and White women with BC. Fifty-six women were eligible, and 48 were consented (24 Black, 24 White). All participants consented to blood testing. This highly educated samples mean age was 52.5 + 12.05 (years). Education (years) and genetic knowledge were positively correlated (p = .038). Genetic scores were high, and only one question significantly differed by race. On interview, most participants thought conducting genetic research helped to better understand hereditary disease and/or identify genes that cause disease and stated that they participated in the research to help other people. The majority of participants responded that friends/family would participate in genetic research without concerns, though three Black participants cited mistrust as a possible concern. Overall, there were high levels of genetic knowledge, slightly different between Black and White women. There were no high levels of personal concern regarding genetic testing. Black women reported more concern than White women that friends/family would have hesitations about participating in genetic research. There was general acceptability of blood collection for genetic testing among women with early-stage BC without racial difference.

PMID:33025420 | DOI:10.1007/s40615-020-00878-5

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Knowledge and Attitudes About Genetic Testing Among Black and White Women with Breast Cancer - DocWire News

When Dalyss Tomayer found out three of her relatives had had breast cancer, it was enough to qualify her for the genetic testing which showed she had a high risk of breast cancer herself.

Tomayer was able to discover the gene early due to getting tested at HCA Houston Healthcare North Cypress High Risk Breast Clinic, where they offer genetic testing for those who qualify to see if they may have the BRCA1 or BRCA2 gene which puts people at higher risk for breast cancer.

On HoustonChronicle.com: Willowbrook breast cancer support group still meeting virtually

I had known for years that two of my dads sisters had breast cancer, and it was in speaking with one of my aunts that I then found out also one of my cousins had breast cancer, Tomayer said. So, it was at that time that I realize that I did qualified for the genetic testing and I decided to go ahead and do it.

Janet Pollard, the coordinator for the High Risk Breast Clinic, said the breast center began as a full program under HCA Houston North Cypress CEO Jim Brown, who she said was passionate about having this resource.

On HoustonChronicle.com: Memorial Hermann rolls out breast cancer prevention program as screening rates plummet

Most of the time people think about genetic testing after you get cancer, Pollard said. It can drive the type of surgeries, it can change treatment regime, but basically its getting it done prior. Its screening people when they come in for their annual mammograms, talking with primary care doctors, educating them on what makes patients meet criteria.

Criteria established by the American Cancer Society for genetic testing includes people who were diagnosed with breast cancer at a younger age, those diagnosed with breast cancer a second time, people of Ashkenazi Jewish descent, people with a family history of breast cancer, ovarian cancer, pancreatic cancer or prostate cancer, and people with a known family history of BRCA mutation.

Before the pandemic, Pollard said the center was testing around 30 people a month, men and women, with about 10 percent of people testing coming back positive.

BRCA1 and 2 genes are huge red flags, Pollard said. Theyre the original genes and theyve been testing those genes for two decades.

The process Tomayer chose to go through after being tested was to have a double mastectomy to virtually remove all chance of breast cancer, as well as a hysterectomy to eliminate her chances of ovarian cancer.

Even though you do a double mastectomy, theres no way to get 100 percent of every breast cell in my body, so there is still a slight chance, but it is incredibly slight, she said.

Tomayer said she would continue to have ultrasounds for what remaining breast tissue she has left.

Im so very thankful they have the test, Tomayer said. It saved my life.

paul.wedding@hcnonline.com

Continued here:
HCA Houston Healthcare North Cypress offers preventative gene testing for breast cancer - Chron

SAN DIEGO, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) announced today that its Saphyr system played a key role in a research study by Marcin Imielinski, M.D., Ph.D., Assistant Professor of Pathology and Laboratory Medicine at Weill Cornell Medical School, which was published in the peer-reviewed journal Cell. The study identified three new distinct classes of structural variations (SVs) in the DNA of thousands of cancer samples across multiple cancer types, and used Saphyr to characterize the precise structure and genomic location of these variations. These previously unknown types of complex rearrangements help explain different mechanisms that enable cancer cells to expand and grow rapidly while simultaneously evading natural defense mechanisms and treatment. With the addition of Bionanos Saphyr data, the study demonstrated that these structural variants generate a large number of fusion proteins and represent therapeutic targets and/or prognostic biomarkers of disease progression.

While cancer genomes traditionally have been studied with a combination of low-resolution cytogenetic methods or with next-generation sequencing (NGS), Bionanos Saphyr instrument provides long-range data that enables a high-resolution, long-range view into the cancer genome and cancer biology. At a presentation providing an update on the study given at the March 31, 2020 Advances in Genome Biology and Technology Conference, Dr. Imielinski explained how short-read data alone is unable to resolve the structure of these newly discovered structural variants because short reads provide a very local view of the genome, and further explained how integrating long-range data generated by Bionanos optical mapping technology was essential to obtaining a complete understanding of these structures, including their biological significance.

As previously announced, a high-profile paper published last month by UCSD Professor Vineet Bafna in Nature Communications similarly used a combination of NGS and Bionanos Saphyr data to resolve the structure of extra-chromosomal circular DNA, another type of complex rearrangement of the cancer genome which similarly allows for tumor cells to increase their growth rate while evading natural defenses. In both studies, only the combination of short-read sequencing technology with optical mapping data generated by Saphyr was able to fully resolve these complex structures and provide a comprehensive picture of the cancer genome.

We are impressed by this important body of work and the ingenuity from Dr. Imielinskis team," said Erik Holmlin, Ph.D., chief executive officer of Bionano Genomics. By combining NGS with Bionanos genome imaging data, they were able to discover, analyze and fully characterize these novel variants that were so large and complex that they were hidden in plain sight from traditional sequencing methods. We believe these studies help generate a rapid increase in understanding of the complexity of cancer genomes driven by the long-range data that only Saphyr can provide, with a cost and throughput that makes the analysis of large sets of cancer samples possible. Since many of the most effective anti-cancer drugs target specific fusion proteins, we believe the discovery of these novel structural variants and the many fusions they generate has the potential to guide the future development of new therapeutics, better cancer diagnostics and a more efficient use of existing cancer drugs.

The publication is available at https://doi.org/10.1016/j.cell.2020.08.006

About Bionano GenomicsBionano is a genome analysis company providing tools and services based on its Saphyr system to scientists and clinicians conducting genetic research and patient testing, and providing diagnostic testing for those with autism spectrum disorder (ASD) and other neurodevelopmental disabilities through its Lineagen business. Bionanos Saphyr system is a platform for ultra-sensitive and ultra-specific structural variation detection that enables researchers and clinicians to accelerate the search for new diagnostics and therapeutic targets and to streamline the study of changes in chromosomes, which is known as cytogenetics. The Saphyr system is comprised of an instrument, chip consumables, reagents and a suite of data analysis tools, and genome analysis services to provide access to data generated by the Saphyr system for researchers who prefer not to adopt the Saphyr system in their labs. Lineagen has been providing genetic testing services to families and their healthcare providers for over nine years and has performed over 65,000 tests for those with neurodevelopmental concerns. For more information, visitwww.bionanogenomics.com or http://www.lineagen.com.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as may, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Bionanos contribution to understandings of extra-chromosomal circular DNA and structural variations in cancer, including its ability to influence improved use of existing cancer drugs or future development of better diagnostics and new therapeutics; Saphyrs capabilities in comparison to and in conjunction with traditional sequencing methods and NGS-based methods; and Saphyrs potential as an essential tool for personalized medicine and furthering understanding of cancer in the medical community. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the risks and uncertainties associated with: the impact of the COVID-19 pandemic on our business and the global economy; general market conditions; changes in the competitive landscape and the introduction of competitive products; changes in our strategic and commercial plans; our ability to obtain sufficient financing to fund our strategic plans and commercialization efforts; the ability of medical and research institutions to obtain funding to support adoption or continued use of our technologies; the loss of key members of management and our commercial team; and the risks and uncertainties associated withour business and financial condition in general, including the risks and uncertainties described in our filings with the Securities and Exchange Commission, including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2019 and in other filings subsequently made by us with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise.

CONTACTSCompany Contact:Erik Holmlin, CEOBionano Genomics, Inc.+1 (858) 888-7610eholmlin@bionanogenomics.com

Investor Relations Contact:Ashley R. RobinsonLifeSci Advisors, LLC+1 (617) 430-7577arr@lifesciadvisors.com

Media Contact:Darren Opland, PhDLifeSci Communications+1 (617) 733-7668darren@lifescicomms.com

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Bionano's Saphyr Plays Essential Role in Identifying Three Previously Unknown Genetic Mutation Types in Cancer in Study from Weill Cornell -...

by Liz Bonis & Merby Curtis, WKRC

Experts at St. Elizabeth Healthcare are expanding programs to treat and prevent cancer. Its all part of a personal prescription for your future health. (WKRC)

EDGEWOOD, Ky. (WKRC) Experts at St. Elizabeth Healthcare are expanding programs to treat and prevent cancer. Its all part of a personal prescription for your future health.

For years, we have focused on early detection of cancer, which is important. But now, the new St. Elizabeth Cancer Center has an important focus on preventing cancer with the help of screening that also detects risk: a field commonly called precision medicine.

The new Cancer Center has several teams dedicated to making this happen. Medical oncologist Dr. Brooke Phillips explains how it all works together:

One of the first things in cancer is trying to prevent it. We already have a robust genetic counseling program, but were going to have a whole floor dedicated to that. Were seeing not only patients that have cancer, but patients that have a family history or concerns related to that and will provide them care, really, in regards of prevention. If that involves screening tests, prophylactic surgeries, we will also have that in there. We will also have precision medicine as related to a specific tumor and a lot of genetic related to that tumor and a lot of clinical trials surrounding that.

If you know you have a family history of cancer and would like to find out more information, you can start your prevention journey by calling the genetic testing area at 859-301-5396.

Keep in mind, as part of this they also have nutrition programs and much more to focus on prevention at the new Cancer Center in Edgewood.

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Ask the Expert: How is precision medicine used in cancer prevention? - WKRC TV Cincinnati

Detect Lysosomal Storage Diseases program reduces barriers to genetic diagnosis through sponsored testing for lysosomal storage disorders, including GM2 gangliosidosis

Behind the Seizure program can help accelerate genetic epilepsy diagnosis in children experiencing unprovoked seizures

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced a partnership with Invitae, a leading medical genetics company, to support Invitaes Detect Lysosomal Storage Diseases (Detect LSDs) and Behind the Seizure programs. The Detect LSDs program enables the rapid diagnosis of lysosomal storage disorders (LSDs), including GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff disease). The Behind the Seizure program is an innovative, cross-company collaboration that supports faster diagnosis for children with epilepsy. The Behind the Seizure program will also support patient identification across Tayshas broad pipeline of gene therapies for which a number of indications have an underlying seizure phenotype.

"Through both initiatives, we are supporting the rapid identification of patients with debilitating diseases, allowing them to gain access to earlier therapeutic interventions. For LSDs, there are more than 50 different disorders with overlapping symptoms, making misdiagnosis common," said RA Session II, Tayshas President, CEO and Founder. "Likewise, more than 50% of epilepsies have a genetic basis. When a patient presents with seizures, genetic testing may help identify more than 100 underlying, often rare conditions. We are proud to support these initiatives to help patients gain timely access to natural history studies, clinical trials, and ultimately disease-modifying therapies."

Eligible individuals suspected of having an LSD or epilepsy will gain access to genetic testing and counseling at no charge through these programs. The Detect LSDs program will help identify individuals who are eligible for Tayshas study evaluating TSHA-101 in patients with GM2 gangliosidosis, expected to enter the clinic later this year. The Behind the Seizure program will enable patient identification across Tayshas broad pipeline of indications, some of which have an underlying seizure phenotype, and rapid enrollment into natural history studies and clinical trials.

Story continues

"Increasing access to genetic testing can support earlier diagnosis of neurodegenerative diseases, which in turn enables clinicians to provide precision therapies sooner and better overall outcomes," said Robert Nussbaum, M.D., Chief Medical Officer of Invitae. "These unique, cross-company collaborations have been shown to help increase access to testing and reduce time to diagnosis. We are pleased Taysha has joined us in helping increase access to testing for children impacted by neurodegenerative conditions."

Additional details, as well as terms and conditions of the Detect LSDs program, can be found at https://www.invitae.com/en/detectLSDs/. To learn more about the Behind the Seizure program, please visit https://www.invitae.com/en/behindtheseizure/.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

About Invitae

Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipates," "believes," "expects," "intends," "projects," and "future" or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the conduct or timing of our partnership with Invitae and our planned clinical trial of TSHA-101 for the treatment of GM2 gangliosidosis, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our prospectus dated September 23, 2020, as filed with the Securities and Exchange Commission ("SEC") on September 24, 2020, pursuant to Rule 424(b) under the Securities Act of 1933, as amended, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201006005450/en/

Contacts

Company Contact: Niren Shah, PharmD, MBATaysha Gene TherapiesNshah@tayshagtx.com

Media Contact: Carolyn HawleyCanale Communicationscarolyn.hawley@canalecomm.com

Link:
Taysha Gene Therapies Partners with Invitae to Enable Rapid Access to Genetic Testing and Earlier Diagnosis of Patients with CNS Disease for Rare and...

Newark, NJ, Oct. 05, 2020 (GLOBE NEWSWIRE) -- As per the report published by Fior Markets, theglobal cannabis testing market is expected to grow from USD 1.71 billion in 2019 and to reach USD 4.87 billion by 2027, growing at a CAGR of 14% during the forecast period 2020-2027.

The global cannabis testing market is witnessing significant growth from recent years. This growth is attributed to the growing number of medical applications of cannabis, rising investment by various research institutes, increasing focus to meet the needs of hemp and cannabis cultivator, and legalization of medical cannabis associated with a rising number of cannabis analysis laboratories. Rising adoption of LIMS in cannabis testing laboratories, growing awareness among people through workshops and conferences will propel the market growth.

The methods of drug testing for detecting the use of cannabis in an individual in the field of medicine, sport, and the law is known as cannabis testing. The various methods in which cannabis can be detected are hair testing, saliva testing, urine testing and various others. An analysis of a single cannabis sample tested to determine elements, such as CBD, THC, terpene levels and impurities like mold, residual solvents, pesticide residual and diseases, is referred to as cannabis testing. The amount of concentration obtained from these analyses can be useful in distinguishing elapsed time since used, active use from passive exposure and duration of use. Certified chemical and laboratory technicians can only perform cannabis testing.

The global cannabis testing market is expected to witness significant growth, owing to the increase in the development of therapies derived from cannabis and components, rising investment, and legalization of cannabis in various emerging economies. Lack of standard testing process, absence of trained laboratory professionals, and high cost of analytical instruments will restrain the market growth. Revoking of the ban on cannabis in developing economies will provide market growth opportunities. However, the lack of industry standards for cannabis testing and high start-up cost for cannabis testing laboratories will challenge the market growth.

DOWNLOAD FREE SAMPLE REPORT AThttps://www.fiormarkets.com/report-detail/418923/request-sampleThe key players operating in the global cannabis testing market are CannaSafe Analytics, Steep Hill, Anandia Labs, Eirlab, Phytovista Laboratories, CW Analytical Laboratories, Pure Analytics, Anresco, EVIO, SC Labs, and Digipath. To gain a significant market share in the global cannabis testing market, the key players are now focusing on adopting strategies such as product innovations, mergers & acquisitions, recent developments, joint venture, collaborations, and partnership. Digipath and CannaSafe are some of the key manufacturers in cannabis testing.

Chromatography instruments segment dominated the market and held the largest share of 38.9% in the year 2019On the basis of product and software, the global cannabis testing market is segmented into analytical instruments, consumables, and software. The analytical instruments segment is further segmented into chromatography instruments, spectroscopy instruments, and other analytical instruments. The chromatography instruments are further classified into liquid chromatography, gas chromatography, and other chromatography instruments. The spectroscopy instruments segment is further classified into mass spectrometry, atomic spectroscopy, and atomic spectroscopy. The consumables segment is further segmented into chromatography columns, standards & CRMS, sample preparation products, and supplies and accessories. Chromatography instruments segments dominated the market and held the largest share of 38.9% in the year 2019. This growth is attributed to the application of chromatography techniques in potency testing of cannabis. The spectroscopy instrument segment is anticipated to witness significant growth due to the increasing legalization of cannabis testing and its derivative for adults' recreational and medical use.

Potency testing segment dominated the market and held the largest share of 21.4% in the year 2019On the basis of testing procedure, the global cannabis testing market is segmented into the residual solvent screening, terpene profiling, potency testing, genetic testing, pesticide screening, heavy metal testing, and microbial analysis. The potency testing segment dominated the market and held the largest share of 21.4% in the year 2019. This growth was attributed to the increasing applications of cannabinoids for medical purposes. It is also essential for the precise labeling of medical purposes.

Browse full report with TOC athttps://www.fiormarkets.com/report/cannabis-testing-market-by-product-software-analytical-418923.html

Laboratories segment dominated the market and held the largest share of 31.2% in the year 2019On the basis of end-user, the global cannabis testing market is segmented into laboratories, cannabis cultivators, research institutes, and drug manufacturers & dispensaries. Laboratories segment dominated the market and held the largest share of 31.2% in the year 2019. This growth was attributed to the increasing legalization of cannabis for medical as well as recreational use, and rising demand for cannabis testing software in labs.

Regional Segment of Cannabis Testing Market

On the basis of geography, the global cannabis testing market is classified into North America, Europe, Asia-Pacific, Middle East & Africa, and South America. North America held the major share of 28.71% in the year 2019. This growth was attributed to the growing legalization pertaining to the use of cannabis across various U.S. states, increasing cultivators of marijuana, and the rising number of testing laboratories. The growing demand for cannabis for personal use will further propel the market growth. The Chile cannabis testing market is anticipated to grow due to the growing awareness of the medical use of cannabis and the increase in cannabis use for medical purposes. The Germany market is expected to witness significant growth due to increased approval of production licenses and new medical cannabis legislation.

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About the report:The global cannabis testing market is analysed on the basis of value (USD billion). All the segments have been analysed on global, regional and country basis. The study includes the analysis of more than 30 countries for each segment. The report offers in-depth analysis of driving factors, opportunities, restraints, and challenges for gaining the key insights of the market. The study includes porters five forces model, attractiveness analysis, raw material analysis, and competitors position grid analysis.

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Global Cannabis Testing Market Is Expected to Reach USD 4.87 billion by 2027 : Fior Markets - GlobeNewswire

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 07 October 2020 13:48Hits: 333

NEW YORK, NY & BRISBANE, CA, USA I October 7, 2020 I Pfizer Inc. (NYSE: PFE) and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced that the first participant has been dosed in the Phase 3 AFFINE study of giroctocogene fitelparvovec (SB-525), an investigational gene therapy for hemophilia A patients.

AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on Factor VIII (FVIII) replacement therapy collected in the Phase 3 lead-in study period. Participants will be analyzed throughout the 5-year study period following the single infusion to further assess the durability and efficacy.

The initiation of the pivotal Phase 3 dosing study of giroctocogene fitelparvovec is a significant achievement for Pfizer as we continue our longstanding commitment to improving care for the hemophilia community, said Brenda Cooperstone, Chief Development Officer, Rare Disease, Pfizer Global Product Development. Enrollment in the lead-in study is progressing well and recruitment is on track for Phase 3. Given the Phase 1/2 study findings to date, we believe that giroctocogene fitelparvovec has the potential to sustain factor levels and reduce annual bleed rates, suggesting this one-time gene therapy could potentially transform the standard of care for eligible patients worldwide.

Data from the Phase 3 lead-in study will provide a baseline for patients evaluated in the Phase 3 study. Updated Phase 1/2 data announced at a Pfizer investor event on September 15, 2020 demonstrated that giroctocogene fitelparvovec was generally well tolerated. Each of the five patients in the high dose cohort sustained FVIII activity levels without bleeds or the need for prophylactic factor through up to 85 weeks. Factor VIII activity levels were sustained at a clinically meaningful level, with a geometric mean of ~71% when measured between the weeks of 9 and 52.

We are encouraged that findings from the Phase 1/2 Alta study met two critically important measures for the hemophilia A patient community, showing clinically meaningful factor levels and reduced bleeds, said Bettina M. Cockroft, M.D., M.B.A, Chief Medical Officer of Sangamo. The progress of this program, the most advanced of our gene therapy product candidates, into Phase 3 is an important milestone for Sangamo, as it represents our first asset in a registrational trial.

Per the terms of the collaboration agreement, Sangamo has now earned a $30 million milestone payment. The giroctocogene fitelparvovec collaboration was established in May 2017. Under the terms, Pfizer is now operationally and financially responsible for research, development, manufacturing and commercialization activities for giroctocogene fitelparvovec following the transfer of the Investigational New Drug (IND) from Sangamo to Pfizer in December 2019. Sangamo is eligible to receive total potential milestone payments of up to $300 million for the development and commercialization of giroctocogene fitelparvovec, and up to $175 million for additional hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will, additionally, receive tiered royalties starting in the low teens and up to 20% of annual net sales of giroctocogene fitelparvovec.

About the AFFINE study

The Phase 3 AFFINE (efficAcy and saFety Factor vIii geNe thErapy in hemophilia A patients; NCT04370054)study is an open-label, multicenter, single arm study to evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (ages 18-64 years) male participants with moderately severe to severe hemophilia A. Eligible study participants will have completed at least six months of routine FVIII prophylaxis therapy during the lead-in Phase 3 study (NCT03587116) in order to collect pretreatment data for efficacy and selected safety parameters.

The primary endpoint is impact on ABR through 12 months following treatment with giroctocogene fitelparvovec, as compared to ABR on prior FVIII prophylaxis replacement therapy. The secondary endpoint is FVIII activity level after the onset of steady state and through 12 months following infusion of giroctocogene fitelparvovec.

About giroctocogene fitelparvovec

Giroctocogene fitelparvovec (SB-525 or PF-07055480) comprises a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII. The giroctocogene fitelparvovec expression cassette was designed for optimal liver-specific expression of FVIII protein and supports production of high yields of the vector. The giroctocogene fitelparvovec transcriptional cassette incorporates multi-factorial modifications to the liver-specific promoter module, FVIII transgene, synthetic polyadenylation signal and vector backbone sequence.

The U.S. Food and Drug Administration has granted Orphan Drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations to giroctocogene fitelparvovec, which also received Orphan Medicinal Product designation from the European Medicines Agency. Giroctocogene fitelparvovec is being developed as part of a global collaboration between Sangamo and Pfizer.

About Hemophilia A

Hemophilia is a genetic hematological rare disease that results in a deficiency of a protein that is required for normal blood clottingclotting factor VIII in hemophilia A. The severity of hemophilia that a person has is determined by the amount of factor in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur which can lead to serious health problems.

Hemophilia A occurs in approximately one in every 5,000-10,000 male births worldwide. For people who live with hemophilia A, there is an increased risk of spontaneous bleeding as well as bleeding following injuries or surgery. It is a lifelong disease that requires constant monitoring and therapy.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

About Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

Pfizer Inc.: Breakthroughs that change patients lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

SOURCE: Sangamo Biotherapeutics

Read more:
Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment | DNA RNA and Cells | News Channels -...

It will also offer GMP manufacturing services in that Billingham location.

In terms of the driver behind the companys decision to establish such services at its UK site,Andy Ross, spokesperson for Fujifilm Europe, told BioPharma-Reporter:In the gene therapy viral vector CDMO market, Europe is the second largest after North America. Therefore, in order to expand the gene therapy drug CDMO business, Fujifilm will make an initial investment in the UK base this time and [later] expand the gene therapy drug CDMO business to Europe.

The FDB viral vector process development laboratories in the UK are due to be online from spring 2021; the company outlined how the site will be outfitted with equipment to support upstream - suspension and adherent - processes, downstream processing and analytical development.

The GMP manufacturing capabilities of that CDMO are expected to be available at the UK base from autumn 2021; they will support bulk drug substance production of investigational drugs used in early-stage clinical trials.

The company previously announced it was constructing a research center at the UK site in March this year.

Fujifilm has positioned its CDMObusiness for the gene therapy drug of viral vector process development and manufacturing as a priority field, said Ross. It is an area where the companyhas a strong track record, he added.

The company first established a viral vector and vaccine development and manufacturing offer in 2014 at its College Station, Texas site, in the US.

Flagging up Fujifilms USP in relation to viral vector and GMP manufacturing, Ross said the company is able to consistently provide flexible service from process development, formulation development, bulk drug substance production, fill finish and final packaging.We have state of the art facilities and equipment, world class staff, and proprietary expression technologies to quickly develop and provide the optimum production process.

Fujifilm, in another effort to grow this CDMO business, is making a capital investment in FDBs Texas operations. Earlier this year,it announced that this site would be expanded with US$35m (31m) earmarked for the upgrade.

Moreover, as part of the US governmentsOperation Warp Speed, the Biomedical Advanced Research and Development Authority (BARDA), back in August, enlisted the support of FDB for the production of COVID-19 vaccine candidates.

The agreement will see Fujifilm Diosynths Texas site contracted to provide manufacturing capacity through the end of 2021.

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Fujifilm expands its gene therapy drug CDMO business - BioPharma-Reporter.com

Cleveland Clinic released its list of the top 10 medical innovations it believes will change health care next year, including gene therapy for blood disorders, a new class of medications for cystic fibrosis and increased access to telemedicine, according to a news release.

The annual list, which is selected by a panel of Clinic researchers and clinicians, was announced in conjunction with the system's 18th annual Medical Innovation Summit, which was held virtually on Tuesday, Oct. 6.

The committee of experts that selected the breakthrough technologies was led by Dr. Will Morris, executive medical director for Cleveland Clinic Innovations (the system's development and commercialization arm), and Dr. Akhil Saklecha, managing director of Cleveland Clinic Ventures, which was formed three years ago to invest in emerging health care companies that can deliver financial returns to the Clinic.

The Clinic also announced that it will present a new award at next year's summit to a team, organization or individual who has made a significant contribution to health care delivery with a focus on one or more defined areas, according to the release, which notes that details will be released early next year.

Below are the Clinic's top 10 medical innovations for 2021, in order of importance, according to the release:

Read more:
Cleveland Clinic unveils top 10 medical innovations for 2021 - Crain's Cleveland Business

Application of ImmTORplus IGANs IgA protease to targetimmunoglobulin A (IgA) deposition, the critical event leading to IgA Nephropathy (IgAN)

Selectas IgA nephropathy program builds upon SEL-212, which has demonstrated ImmTOReffective at tolerizing a highly immunogenic therapeutic enzyme

Investigational New Drug (IND) application expected in Q4 2021

WATERTOWN, Mass., and BOSTON, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc.(NASDAQ: SELB) and IGAN Biosciences, Inc. today announced that they have entered into a Research License and Option agreement to study Selectas ImmTORimmune tolerance platform in combination with IGANs immunoglobulin A (IgA) protease for the treatment of IgA Nephropathy (IgAN). In advance of exercising its option to enter into an exclusive license agreement, Selecta will conduct preclinical research to evaluate the utility of the combination of ImmTOR and IGANs IgA protease.

Previous studies in animal models conducted at independent laboratories established the ability of IGANs IgA protease to remove injurious IgA from kidneys and improve markers of renal dysfunction. These results suggest that it is an excellent candidate to decrease the rate of disease progression and possibly even reverse the disease. The barrier to IgA protease commercialization is the bacterial origin of the protease, which makes it immunogenic. Selectas proprietary immune tolerance platform, ImmTOR, has shown in clinical studies the ability to mitigate the formation of anti-drug antibodies (ADAs) to immunogenic enzymes. This has been demonstrated with its phase 3 lead product candidate, SEL-212, which is a combination of ImmTOR and pegadricase, an immunogenic proprietary pegylated uricase for the treatment of chronic refractory gout. Selecta and IGAN intend to combine IGANs IgA protease with Selectas ImmTOR platform to develop a novel combination product to treat IgA nephropathy, a disease with significant unmet medical need.

We are excited to build on our learnings from SEL-212 in combining an immunogenic enzyme with our ImmTOR platform to develop a product candidate for IgA nephropathy, said Carsten Brunn, Ph.D., President and Chief Executive Officer of Selecta Biosciences. IGANs IgA protease has shown tremendous promise in preclinical studies and combining it with ImmTOR to prevent ADAs could lead to a transformational product for patients with this debilitating disease.

IGAN is pleased to enter into this agreement with Selecta Biosciences to use their ImmTOR technology with IgA proteases as biological therapy for IgA nephropathy, said Andrew G. Plaut, M.D., Co-Founder and Chief Medical Officer of IGAN Biosciences Inc. In combination, these products offer the prospect of realizing the potential value of IgA proteases to remove kidney IgA deposits, thus reducing progression of renal injury to renal failure.

IgA Nephropathy is characterized by deposition of galactose-deficient IgA1 immunoglobulin in the glomerular mesangium and is a leading contributor to development of chronic kidney disease and renal failure. Genetic or environmental causes that form this abnormal IgA1 and its accumulation in the kidney can result in the development of IgAN. Hypertension, proteinuria and decreased eGFR at the time of diagnosis are associated with poor prognosis. It can result in incremental loss of renal function and results in end stage renal disease (ESRD) in approximately 30 - 40% of patients. There are no approved therapies for the treatment of IgAN.

Under the terms of the research license and option agreement, Selecta will make an initial payment to IGAN. If Selecta exercises its option to enter into an exclusive license agreement, IGAN will receive an additional payment and will be eligible for additional development, regulatory, and commercial milestone payments, as well as tiered royalties on net product sales.

AboutSelecta Biosciences, Inc.Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the bodys natural self-tolerance in autoimmune diseases. The companys first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visit http://www.selectabio.com.

About IGAN BiosciencesIGAN Biosciencesis a private biotechnology company advancing IgA protease treatment for IgA deposition diseases. Its founders discovered and characterized IgA proteases during more than forty years of research conducted at Tufts University School of Medicine and Tufts Medical Center, Boston, MA.

Selecta Forward-Looking Statements

This press release, contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Selectas plan to participate in upcoming investor conferences. All such forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Such risks and uncertainties include but are not limited to the companys plans to exercise its option to enter into a licensing agreement, preclinical research and development activities related to IGANs IgA Protease, the ability of the IgA Protease to remove injurious IgA from kidneys and improve markers of renal dysfunction, the companys plans to develop a product candidate to treat IgA Nephropathy or other autoimmune indications, the material impact, if any, of the COVID-19 outbreak on the Companys operations, including supply chain and clinical trials, other COVID-19 related risks and those set forth in the Risk Factors section of Selectas Quarterly Report on Form 10-Q filed with theSecurities and Exchange Commission, orSEC, for the quarter endedMarch 31, 2020, and in other filings thatSelectamakes with theSEC. In addition, any forward-looking statements included in this press release represent Selectas views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date.Selectaspecifically disclaims any obligation to update any forward-looking statements included in this press release.

For more information please contact:

Selecta:

For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

For Media: Meredith Sosulski, Ph.D.LifeSci Communications, LLC+1-929-469-3851msosulski@lifescicomms.com

IGAN Biosciences:Donald CollinsIronwood Investment Management+1-617-757-7620dcollins@ironwoodfunds.com

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Selecta Biosciences and IGAN Biosciences Enter into Research License and Option Agreement to Study Selecta's ImmTOR Immune Tolerance Platform in...

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy BMN 307 (BioMarin Pharmaceutical) for the treatment of patients with phenylketonuria (PKU).

BMN 307 is an adeno-associated virus type 5 (AAV5)-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells.

The Company recently initiated an open-label phase 1/2 study (PHEARLESS) to evaluate the safety, efficacy and tolerability of a single intravenous administration of BMN 307 in adults with PKU with PAH deficiency. The study will assess whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.

Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy, said Hank Fuchs, MD, President, Worldwide Research and Development at BioMarin. We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit biomarin.com.

BioMarin, pioneer in phenylketonuria (PKU) and gene therapy, receives FDA Fast Track designation for PKU investigational gene therapy, BMN 307. https://www.prnewswire.com/news-releases/biomarin-pioneer-in-phenylketonuria-pku-and-gene-therapy-receives-fda-fast-track-designation-for-pku-investigational-gene-therapy-bmn-307-301144678.html. Accessed October 5, 2020.

Read more here:
Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked - Monthly Prescribing Reference

The Global Oncolytic Virus Immunotherapy Market Will Be Driven By Increasing Number Of Clinical Trials Along With New Product Launches Over Next 5 Years Says Kuick Research

NEW DELHI, Oct. 8, 2020 /PRNewswire/ -- "Global Oncolytic Virus Immunotherapy Market, Dosage, Price & Clinical Trials Outlook 2026" Report Highlights:

Download Report:

https://www.kuickresearch.com/report-oncolytic-virus-immunotherapy-market-size-sales-clinical-trials-cancer-oncology-melanoma-report-talimogene-laherparepvec-imlygic--oncorine

or contact: [emailprotected]

Increase in prevalence and mortality rate associated with cancer have always remained a challenge to the modern medicine with respect to the development of more reliable therapies. In the past few years, one of the greatest breakthrough novel cancer therapy that has been developed by the researchers is the oncolytic virus immunotherapy. The overall procedure of the therapy is based on the ability of the oncolytic virus to infect and further lyse the tumor cells by boosting the immune system of the patient. Some of the virus strains that have been tested and proved to be adding to the various applications of the therapy are canine distemper virus, adenovirus, vaccinia virus and reovirus in the preclinical and clinical studies.

The viral toxicity and the in-vitro manipulation of the virus is estimated to be providing successful clinical outcomes, leading to the enhancement and the expansion of the market linked with the therapy. All the clinical trials associated with the therapy are underway but all are estimated to look forward by bringing numerous opportunities for the cancer patients. The market concerned with the growth of the oncolytic virus therapy is focused on increasing the research and development sector, which is apparently considered as an important asset for the therapy in order to change the stringent cancer treatment paradigm available in the industry for a long period of time. The current clinical platform is estimated to be developing a competitive landscape for the other cancer markets as the therapy of interest is getting fragmented at a high speed for different cancer types.

The blockbuster model held by the therapy is also believed to be driving the large shareholder value of the market in the present as well as in the upcoming years. The increase in the perception of highly expensive drugs available under cell and gene therapy are expected to be one of the most regulated drivers for the global expansion of the oncolytic therapy. As a result of strong clinical background and healthy research and development sector, oncolytic virus therapy is becoming more prominent in the cancer landscape. With the primary goal of revolutionizing the cancer therapeutics market, it is estimated that the therapy in a short period of time will be successful in re-defining the unstructured cancer paradigm for the patients with advanced cancers.

Being not only restricted to clinical benefits, the therapy is moving forward to provide better post-treatment lives to the cancer patients. The therapy in the clinical studies have been able to put forward the value of the patients in terms of clinical outcomes as well as patient-related outcome measures, thus causing a wider acceptance to the therapy in the cancer therapeutics market. Some of the primary aim associated with the current clinical pipeline of the therapy involves limited cost expansion and increased success rate. Prominent factors like these are estimated to enhance access of the patient population to effective oncolytic virus therapy.

The future view of the oncolytic virus therapy is estimated to be enabling such innovative mechanisms that would be responsible for providing tremendous amount of opportunities to the cancer patients. Different gene-editing technologies, advancement in genomics and many others will map the therapy among the best-cancer regimens ever discovered. The leverage provided to the therapy in terms of opportunities depicts the possibility of the therapy to get recognized as a real cure to the cancer patients and eventually a dominating market. With the advent of the therapy for the cancer platform, there has been a significant transformation in the current cancer therapeutics market. In financial services, the market is estimated to perform with accuracy and speed in the future years.

As per " Global Oncolytic Virus Immunotherapy Market, Dosage, Price & Clinical Trials Outlook 2026" report findings, it is estimated that the total inflation of the market with respect to clinical platform, wider acceptance in a short period of time will drive the market in the upcoming years. Gradual increase in the interest of the researchers towards oncolytic virus therapy will develop the market which will eventually bring complete revolution in terms of applications to the cancer patients. The future market insight for the therapy is analyzed to provide unlimited number of trends and opportunities, leading to aggregation of all the cancer related bio-pharmaceutical companies and research centers towards the further development and commercialization of the therapy.

Contact:

Neeraj Chawla[emailprotected]+91-9810410366

Logo: https://mma.prnewswire.com/media/1245952/Kuick_Research_Logo.jpg

SOURCE Kuick Research

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Global Oncolytic Virus Immunotherapy Market To Reach US$ 750 Million By 2026 With 500% Absolute Growth - PR Newswire India

DetailsCategory: More NewsPublished on Thursday, 08 October 2020 10:17Hits: 238

Establishment of Social Cooperation Program "Protein Degradation Drug Discovery"

TOKYO, Japan I October 8, 2020 I The University of Tokyo and Eisai Co., Ltd. announced today a collaboration aiming for the development and drug discovery of targeted protein degradation technology has been created, with the establishment of a social cooperation program, "Protein Degradation Drug Discovery". The research time span will last five years from October 1, 2020 to September 30, 2025.

The social cooperation program is established and operated based on funds of private organizations dedicated to conducting research in collaboration with the University of Tokyo regarding shared issues of high common concern.

The "Protein Degradation Drug Discovery" course is to be established within the Graduate School of Pharmaceutical Sciences, the University of Tokyo. Dr. Mikihiko Naito, Former Director of the Division of Molecular Target and Gene Therapy Products, National Institute of Health Sciences, has been inaugurated as a project professor for this program and will lead research with protein degradation technology including SNIPER. This research will combine the world's most advanced ubiquitin-proteasome research as conducted in the graduate school with drug discovery knowledge fostered by Eisai, for the development of new protein degradation technology towards proteins targeted by drugs and the promotion of drug discovery research based on this technology. In addition, through this research, the course will educate and train the next generation of leaders in this research field.

Targeted protein degradation is a series of technologies in which precisely designed compounds force target proteins into proximity with E3 ubiquitin ligase and apply the ubiquitin-proteasome system to induce degradation of the target proteins. The technology provides a means of creating medicines for not only conventional targets such as specific enzymes and receptors, but also disease-related proteins for which drug discovery up to this point has been difficult. Through the development of this technology and drug discovery, the University of Tokyo and Eisai aim to provide new treatment options to patients for which treatment options were previously limited.

About SNIPER

SNIPER (Specific and Nongenetic IAP-dependent Protein Eraser) is a compound which utilizes the ubiquitin-proteasome system to degrade target proteins. This compound is a "hybrid compound", and consists of a moiety that binds to the target protein and a moiety that binds to E3 ubiquitin ligase (IAPs) with an appropriate linker. Designing this compound requires advanced medicinal chemistry and cutting-edge structural biology. When the SNIPER compound brings the target protein and E3 ubiquitin ligase (IAPs) into proximity (Step 1 in the chart below), ubiquitin as a protein degradation tag transfers from the E2 ubiquitin conjugating enzyme to the target protein (2) for recognition of the protein by the proteasome and subsequent degradation (3).

Conventional small-molecule inhibitors bind to the active moiety of target enzymes, and express pharmacological activity by inhibiting the activity thereof. On the other hand, because SNIPER exhibits pharmacological properties by target protein degradation as described above, it is not only expected to exhibit different pharmacological activity from small-molecule inhibitors; it is also predicted to target proteins that do not have enzymatic activity. Similar technologies include PROTAC (PROteolysis TArgeting Chimeras) and Degronimid.

About the Ubiquitin-Proteasome System

The ubiquitin-proteasome system is one of the naturally occurring mechanisms for controlling the degradation of unneeded proteins and is in control of critical movements relating to the preservation of life including cell cycle, transcription regulation, and signal transmission. When ubiquitin as a protein degradation tag connects to unneeded proteins through agents such as the E3 ubiquitin ligase, it marks the protein for recognition by the proteasome for subsequent degradation. In recent years, the relation between the ubiquitin-proteasome system and major human diseases including cancer and neurodegeneration is becoming evident.

About Eisai

Eisai Co., Ltd. is a leading global research and development-based pharmaceutical company headquartered in Japan. We define our corporate mission as "giving first thought to patients and their families and to increasing the benefits health care provides," which we call our human health care (hhc) philosophy. With approximately 10,000 employees working across our global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to realize our hhc philosophy by delivering innovative products to address unmet medical needs, with a particular focus in our strategic areas of Neurology and Oncology. As a global pharmaceutical company, our mission extends to patients around the world through our investment and participation in partnership-based initiatives to improve access to medicines in developing and emerging countries.

For more information about Eisai Co., Ltd., please visit https://www.eisai.com

SOURCE: Eisai

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The University of Tokyo and Eisai Announce Research Collaboration for the Development and Drug Discovery of Targeted Protein Degradation Technology |...

Facts & Factors (FnF),a leading market research company recently published a research report onResearch Report on Cell and Gene Therapy Consumables Market: By Product Types, Segments, Size, Share, Applications, Analysis and Forecast, 2020-2026to its research database. This[190+] PDF pages Cell and Gene Therapy Consumables Marketreport analyzes the comprehensive overview of the market comprising an executive summary that covers core trends evolving in the market.

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Cell and Gene Therapy Consumables Market Excessive Growth Opportunities and Trends Estimated in Report Forecast Forecast, 2020-2026 - re:Jerusalem

New York, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Regenerative Medicine Market by Product, Application, Geography - Global Forecast to 2025" - https://www.reportlinker.com/p04700208/?utm_source=GNW However, the high cost of cell and gene therapies and ethical concerns related to the use of embryonic stem cells in research and development are expected to restrain the growth of this market during the forecast period.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market, by product, during the forecast periodBased on products, the regenerative medicine market is segmented into tissue-engineered products, cell therapies, gene therapies, and progenitor and stem cell therapies.The cell therapies segment accounted for the highest growth rate in the regenerative medicine market in 2019.

The increasing adoption of tissue-engineered products for the treatment of chronic wounds and musculoskeletal disorders and the rising funding for the R&D of regenerative medicine products and therapies are the major factors driving the growth of this segment.

Oncology segment accounted for highest CAGRBased on applications, the regenerative medicine market is segmented into musculoskeletal disorders, wound care, oncology, ocular disorders, dental, and other applications.In 2019, the oncology segment accounted for the highest growth rate.

This can be attributed to the rising prevalence of orthopedic diseases, growing geriatric population, increasing number of stem cell research projects, growing number of clinical researches/trials, and the rich pipeline of stem cell products for the treatment of musculoskeletal disorders.

Europe: The fastest-growing region regenerative medicine marketThe global regenerative medicine market is segmented into North America, Europe, the Asia Pacific, and Rest of the World.The North America region is projected to grow at the highest CAGR during the forecast period in 2019.

The growth in the North American regenerative medicine market can be attributed to rising stem cell banking, tissue engineering, and drug discovery in the region; expansion of the healthcare sector; and the high adoption of stem cell therapy and cell immunotherapies for the treatment of cancer and chronic diseases.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1 - 20%, Tier 2 - 45%, and Tier 3 - 35% By Designation: C-level - 30%, D-level - 20%, and Others - 50% By Region: North America - 36%, Europe - 25%, Asia Pacific - 27%, and Rest of the World 12%

Lits of companies Profiled in the Report: 3M (US) Allergan plc (Ireland) Amgen, Inc. (US) Aspect Biosystems (Canada) bluebird bio (US) Kite Pharma (US) Integra LifeSciences Holdings Corporation (US) MEDIPOST Co., Ltd. (South Korea) Medtronic plc (Ireland) Anterogen Co., Ltd. (South Korea) MiMedx Group (US) Misonix (US) Novartis AG (Switzerland) Organogenesis Inc. (US) Orthocell Limited (Australia) Corestem, Inc. (South Korea) Spark Therapeutics (US) APAC Biotech (India) Shenzhen Sibiono GeneTech Co., Ltd. (China) Smith & Nephew plc (UK) Stryker Corporation (US) Takeda Pharmaceutical Company Limited (Japan) Tego Science (South Korea) Vericel Corporation (US) Zimmer Biomet (US)

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The report also includes an in-depth competitive analysis of the key market players, along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report:The report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall regenerative medicine 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.

This report will enable stakeholders to understand the pulse of the market and provide them with information on the key market drivers, restraints, opportunities, and trends.

Read the full report: https://www.reportlinker.com/p04700208/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global regenerative medicine market is projected to reach USD 17.9 billion by 2025 from USD 8.5 billion in 2020, at a CAGR of 15.9% -...

The label expansion includes the addition of longer-term efficacy data demonstrating sustained phenylalanine (Phe) lowering out to three years and over six years of data further supporting a well-characterized safety profile. The labeling was updated to allow individualized maintenance dosage to achieve blood Phe control (blood Phe concentrations less than or equal to 600 mol/L), taking into account patient tolerability to Palynziq and dietary protein intake.

Palynziq is indicated to reduce blood Phe concentrations in adults with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 mol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first and only approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe.

Palynziqis BioMarin's second approved treatment for this serious condition. BioMarin also recently announced that the Company had dosed the first participant in the global Phearless Phase 1/2 study of BMN 307, an investigational gene therapy for people with PKU, and potentially its third approved treatment. BMN 307 has Fast Track Designation in the US, as well as Orphan Drug Designation in the U.S. and E.U.

This labeling long term data update is based on efficacy data from an open-label extension study out to three years demonstrating that two-thirds (66%) of the 86 participants treated for at least two years, had a blood Phe level 360 mol/L consistent with the Phe target in American College of Medical Genetics and Genomics (ACMG) recommended guidelines, and 50% of participants had blood Phe levels 120 mol/L at two years.Of the 77 participants treated, 58 (75%), 51 (66%), and 37 (48%) had a blood Phe concentration less than or equal to 600, 360, and 120 mol /L, respectively, at three years of treatment. Additional safety data with over six years of follow up remains consistent with the previous safety profile of Palynziq, irrespective of dose.

PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. PKU affects approximately 1 in 12,500 live births in the United States each year. PKU is marked by an inability to break down Phe, an amino acid that is found in all forms of protein. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological impairment, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, infants are screened at birth to ensure that they are diagnosed early and treated to avoid intellectual disability and other complications. Individuals living with PKU require life-long management, including adherence to a challenging and severely restrictive daily diet of medical foods and formula that avoids the ingestion of Phe that is present in most foods.

"BioMarin is pleased that the FDA has recognized the importance of including an additional dosing option to individuals with PKU. Consistent with the label in Europe, Palynziq is now available in the U.S. at doses of up to 60 mg," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer of BioMarin. "BioMarin remains committed to the PKU community and continues to build on our 15 plus years of research and development, which enabled us to deliver the first two PKU drug therapies. We continue our commitment to further understand the safety and efficacy of our treatments for PKU. We are grateful to the study participants, investigators, study staff and BioMarin employees who were essential to enable this label expansion."

"Adding a maximum dose of 60 mg allows more patients to optimize and achieve treatment goals to keep blood Phe levels within the range set in the medical guidelines or within normal range," said Cary Harding, M.D., professor at Oregon Health & Science University and Steering Committee Chair for the Palynziq program. "More than six years of long-term safety data further supports a well-characterized safety profile similar to the initial data and is important information to physicians considering a chronic therapy."

"It is essential to the PKU community to have treatment options that can control Phe levels and provide long term data on safety and efficacy as it becomes available. The additional information added to the Palynziq label, particularly the long-term safety follow-up demonstrates BioMarin's ongoing commitment to people with PKU," said Christine Brown, MS, executive director of the National PKU Alliance. "BioMarin's 15-year focus on innovative medical research to advance the standard of care in PKU with an existing treatment and for new treatments is transforming the way that the community thinks about this rare genetic disease."

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and an investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About ACMG Guidelines

Practice guidelines issued by ACMG support the need for lifelong management of Phe levels in patients with PKU. The diagnosis and management guidelines were published online in Genetics In Medicine's Advance Online Publication (AOP) service.

The guidelines state that treatment of PKU should be initiated as early as possible and must be continued throughout adulthood and "lifelong," with a goal of maintaining blood levels of Phe for all patients between 120 to 360 micromol/L. Patients treated from the early weeks of life with initial good metabolic control, but who lose that control in later childhood or adult life, may experience both reversible and irreversible neurocognitive and neuropsychiatric consequences.

According to the guidelines "the primary goal of therapy is to lower blood Phe, and any interventions, including medications, or combination of therapies that help to achieve that goal in an individual, without other negative consequences, should be considered appropriate therapy."

Evidence for the guidelines are drawn from two previous independent review processes from the National Institutes of Health (2001) and the Agency for Health Research and Quality (2012). The guidelines can be accessed here.

About Palynziq

Palynziqsubstitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with the PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe. Palynziqis administered using a dosing regimen designed to facilitate tolerability; Palynziq's safety profile consists primarily of immune-mediated responses, including anaphylaxis, for which robust risk management measures effective in clinical trials are in place.

The dosing and administration of Palynziq follows an induction, titration, and maintenance paradigm. Treatment is individualized to the lowest effective and tolerated dosage. Prescribers may consider increasing from 20 mg to 40 mg in patients who have been on 20 mg once daily continuously for at least 24 weeks and who have not achieved blood Phe control. Prescribers may increase dosage to a maximum of 60 mg once daily in patients who have been on 40 mg once daily continuously for at least 16 weeks and who have not achieved blood Phe control. Prescribers are instructed to discontinue treatment in patients who have not responded after 16 weeks of continuous treatment with the maximum dosage of 60 mg once daily. Periodic blood Phe monitoring is recommended, and patients should be counseled on how to adjust their dietary intake, as needed, based on blood Phe concentrations.

To reach a BioMarin RareConnections case manager, please call, toll-free, 1-866-906-6100 or e-mail[emailprotected]. For more information about Palynziq, please visitwww.palynziq.com. For additional information regarding this product, please contact BioMarin Medical Information at [emailprotected].

INDICATION

PALYNZIQ(pegvaliase-pqpz) is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe levels in adult patients with phenylketonuria who have uncontrolled blood Phe levels greater than 600 micromol/L on existing management.

BOXED WARNING: RISK OF ANAPHYLAXIS

WARNINGS AND PRECAUTIONS

Anaphylaxis

Other Hypersensitivity Reactions

ADVERSE REACTIONS

Blood Phenylalanine Monitoring and Diet

DRUG INTERACTIONS

Effect of PALYNZIQ on Other PEGylated Products

USE IN SPECIFIC POPULATIONS

Pregnancy and Lactation

Pediatric Use

Geriatric Use

You are encouraged to report suspected adverse reactions to BioMarin at1-866-906-6100, or to FDA at1-800-FDA-1088orwww.fda.gov/medwatch.

Please see accompanying full Prescribing Information, including Boxed Warning.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

For additional information, please visit http://www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: expectations regarding the potential impact of Palynziq in the PKU community; the updated Palynziq label allowing individualized maintenance dosage to achieve blood Phe control (blood Phe concentrations less than or equal to 600 micromol/L), taking into account patient tolerability to Palynziq and dietary protein intake; new data supporting Phe lowering and a well-characterized safety profile; the Company's Phase 1/2 Phearless study of BMN 307, including BMN 307 potentially becoming BioMarin's third approved treatment for PKU; and BioMarin's development programs for PKU and BioMarin's ongoing commitment to patients with PKU generally. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: actions by regulatory agencies, results and timing of current and planned clinical trials of BioMarin's products, the risks related to the commercialization of Palynziq, our ability to manufacture sufficient quantities of Palynziq; and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in the Company's Securities and Exchange Commission (SEC) filings including the Annual Report on Form10-Q for the quarter ended June 30, 2020, and future filings and reports by the Company. Stockholders are urged not to place undue reliance on forward-looking statements contained in this press release, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation, to update or alter any forward-looking statement, whether as a result of new information, future events, changes in its expectations or otherwise.

BioMarin and Palynziq are registered trademarks of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

https://www.biomarin.com/

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BioMarin, Pioneer in Rare Disease Treatments for Phenylketonuria (PKU), Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for...

WORCESTER, Mass., Oct. 08, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that initial Phase 1 data on MB-105, a PSCA-targeted CAR T administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC), will be presented at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat, which is being held October 20 23, 2020.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are pleased that City of Hope will be presenting initial data from the Phase 1 trial of MB-105 at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat. MB-105 is our CAR T cell therapy that is being studied for the potential treatment of prostate cancer. We look forward to continuing to progress this PSCA-targeted CAR T program.

Details of the presentation are as follows:

Title: Clinical Development of PSCA-targeted CAR T cell therapy for mCRPCSession Date and Time:Friday, October 23, 2020, 11:50 a.m. PTPresenter: Tanya Dorff, M.D., Associate Professor, Department of Medical Oncology & Experimental Therapeutics,Head, Genitourinary Cancer Program, City of Hope, Duarte, CA

For more information, please visit the 27th Annual Prostate Cancer Foundation Scientific Retreat website at https://www.pcf.org/scientific-retreat/27th-annual/.

About MB-105 (PSCA CAR T technology)MB-105 was developed in the laboratory of Saul Priceman, Ph.D., assistant professor in City of Hopes Department of Hematology & Hematopoietic Cell Transplantation and a scientist in the T Cell Therapeutics Research Laboratory led by Stephen Forman, M.D., leader of City of Hopes Hematologic Malignancies and Stem Cell Transplantation Institute and the laboratorys director.

The Phase 1 clinical trial of MB-105, one of the first chimeric antigen receptor T cell (CAR T) trials for prostate cancer in the nation, can enroll up to 33 patients. Its primary endpoints are to define the side effects and the best dose of the prostate stem cell antigen (PSCA) CAR T cells in treating patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC). Secondary endpoints include assessing the expansion and persistence of PSCA CAR T cells, the clinical response based on Prostate Cancer Working Group 3 (PCWG3) criteria, the survival outcomes and serum cytokine profiles in peripheral blood pre- and post-therapy, as well as describing the PSCA expression level on tumor cells prior to CAR T cell infusion and the relationship it may have with disease response and toxicities. For more information on this Phase 1 trial, please visit http://www.clinicaltrials.gov using identifier NCT03873805.

About Mustang BioMustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Mustang Bio Announces MB-105 Data Selected for Presentation at the Virtual 27th Annual Prostate Cancer Foundation Scientific Retreat - GlobeNewswire

New York, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Soft Tissue Repair Market 2020-2024" - https://www.reportlinker.com/p04796687/?utm_source=GNW Our reports on soft tissue repair market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the rising incidence of accidental injuries, rise in strategic partnerships for distribution of products and reimbursement for soft tissue repair products. In addition, rising incidence of accidental injuries is anticipated to boost the growth of the market as well. The soft tissue repair market analysis includes product segment and geographical landscapes.

The soft tissue repair market is segmented as below: By Product Soft tissue fixation devices and accessories Cell therapy Tissue scaffold

By Geographical Landscapes North America Europe Asia ROW

This study identifies the growing demand for gene therapy as one of the prime reasons driving the soft tissue repair market growth during the next few years. Also, increasing strategic alliances and the development of novel products will lead to sizable demand in the market.

The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our soft tissue repair market covers the following areas: Soft tissue repair market sizing Soft tissue repair market forecast Soft tissue repair market industry analysis

Read the full report: https://www.reportlinker.com/p04796687/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Global Soft Tissue Repair Market is expected to grow by USD 10.44 bn during 2020-2024, progressing at a CAGR of 11% during the forecast period -...

- Kuick Research Report Analyzes Clinical & Commercial Opportunity In Global Bispecific Antibodies Market

NEW DELHI, Oct. 7, 2020 /PRNewswire/ --

"Global Bispecific Antibody Market Opportunity, Drug Sales & Clinical Trials Insight 2026" Report Highlights:

Download Report:

https://www.kuickresearch.com/report-global-usa-europe-japan-bispecific-antibody-antibodies-therapeutics-therapy-market-sales-size--trends-clinical-trials-emicizumab-hemlibra-blincyto--blinatumomab-sales

Despite having several hundred clinical success stories of different immunotherapies against solid and advanced cancers, the overall response rate of the patients has reached approximately 50% even when the patients fit completely under all the criteria of treatment. To pave with the clinical failures, researchers have developed and evaluated bispecific therapeutic antibodies, that are combination of two binding specificities leading to improve selectivity as well as efficacy of antibody-based treatment for cancer. The arrival of bispecific antibody for cancer treatment has estimated to be of great potential for wide range of applications in cancer treatment. The current biotherapeutic market for cancer treatment and prevention is estimated to be dominated by antibody drugs, where bispecific antibody represents a novel ally for the next-generation of cancer therapy.

In the past few years, the remarkable progress that has been made with respect to understanding the important hallmarks of the cancer progression and main epitopes involved in it have led to paradigmatic development in the bispecific antibody capable of offering novel promises to the patients who haven't respond to any medical therapy for cancer. The practice of bispecific antibody is not new i.e. this version of cancer therapeutics is a modified version of monoclonal antibody. By observing the increasing incidence of cancer, the management associated with the bispecific antibody has started to develop several strategies important for clinical management of the cancer in the present and in the future.

As per the analysis conducted, it is estimated that the bispecific antibody therapeutics market is growing at a double-digit rate, thus boosting the overall cancer therapeutics market to extreme, especially when compared with other immunotherapies. Further, trends and opportunities coupled within the market i.e. rise in the number of cancer cases, government favorable policies and many others are tending to drive the growth of the market. In our viewpoint, novel alliances developed by major key players of the market and local manufacturers are estimated to be playing important role for delivering tremendous opportunities for the researchers, patients, stakeholders and the payers.

The analysis of the Global bispecific antibody drug market represents that the market is completely focused on providing a better structure to the overall therapeutics market and offer the pharmaceutical industry a broad perspective of an efficient therapy for every cancer type. The research report provides analysis about the assess growth potential of the market by verifying various driving forces, in combination withall the important trends, opportunities and challenges. In addition to it, the research report also provides a long-term outlook for the industry and various other important measures that have helped the market to attract thousands of investors and local drug manufacturers. Lastly, the report also profiles various pharmaceutical companies of the dominating markets that have been boosting the R&D sector of the market.

Table of Contents

1. Introduction to Bispecific Monoclonal Antibody

1.1 Overview of Monoclonal Antibody

1.2 Overview of Bispecific Monoclonal Antibody

1.3 Advantage of Bispecific Antibodies upon Monospecific Monoclonal Antibodies

2. The Engineering & Development of Bispecific Antibodies

2.1 Bispecific Antibodies Similar to IgG

2.1.1 Quadroma (Hybrid Hydromas) Approach

2.1.2 "Knobs Into Holes" Approach

2.1.3 CrossMab Approach

2.1.4 Dual-Variable-Domain Immunoglobulin Approach

2.2 Small Bispecific Antibodies

2.2.1 Bispecific Diabodies

2.2.2 Bispecific T-Cell Engager Antibodies (BiTEs)

3. Mechanism Of Action Of Bispecific Antibody

3.1 Trifunctional Antibody: Catumaxomab (Removab)

3.2 Blinatumomab

4. Applications of Bispecific Antibodies in Diagnostic & Treatment

4.1 BsMAb for Diagnosis of Bacterial & Viral Infectious Diseases

4.2 BsMAb for Cancer Diagnostic

4.3 BsAbs Blocking Signaling Pathways

4.4 BsAbs Targeting Tumor Angiogenesis

4.5 Specific Delivery of Effector Compounds to Targets

4.6 Bispecific Antibodies & Gene Therapy

5. Global Bispecific Antibody Market Outlook

5.1 Current Market Scenario

5.2 Availability of Bispecific Antibodies

5.2.1 Catumaxomab (Removab)

5.2.2 Blinatumomab

5.2.3 Emicizumab

5.2.4 Duligotumab

5.2.5 SAR 156597

6. Approved Bispecific Antibody Dosage & Price Analysis

6.1 Blincyto

6.2 Hemlibra

7. Approved Bispecific Antibody Sales Analysis

7.1 Blincyto

7.2 Hemlibra

8. Global Bispecific Antibodies Clinical Pipeline Overview

8.1 By Phase

8.2 By Country/Region

8.3 By Company

8.4 By Indication

8.5 Orphan Designated Bispecific Antibodies

9. Global Bispecific Antibodies Clinical Pipeline By Company, Indication & Phase

9.1 Research

9.2 Preclinical

9.3 Phase-I

9.4 Phase-I/II

9.5 Phase-II

9.6 Phase-II/III

9.7 Phase-III

10. Marketed Bispecific Antibodies Clinical Insight

10.1 Blinatumomab - Amgen

10.2 Emicizumab - Chugai Pharmaceutical

11. Global Bispecific Antibody Market Dynamics

11.1 Favorable Market Parameters

11.2 Commercialization Challenges

12. Global Bispecific Antibody Market Future Prospects

13. Competitive Landscape

13.1 Ablynx

13.2 Adimab

13.3 Affimed Therapeutics

13.4 Amgen

13.5 AstraZeneca (MedImmune)

13.6 Chugai Pharmaceutical

13.7 Eli Lilly

13.8 EMD Serono

13.9 Emergent BioSolutions

13.10 Genentech

13.11 Genmab

13.12 Immunomedics

13.13 Jounce Therapeutics

13.14 MacroGenics

13.15 Merus

13.16 Neovii Biotech

13.17 NovImmune SA

13.18 OncoMed Pharmaceuticals

13.19 Pieris

13.20 Regeneron Pharmaceuticals

13.21 Roche

13.22 Sanofi

Contact:Neeraj Chawla[emailprotected]+91-9810410366

Logo: https://mma.prnewswire.com/media/1245952/Kuick_Research_Logo.jpg

SOURCE Kuick Research

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Global Bispecific Antibodies Therapy Blincyto Hemlibra Therapeutics Market Size To Witness 118% Growth To Reach US$ 10 Billion By 2026 - PR Newswire...

Newswise PHILADELPHIA Picture a future where embedded medical devices not only treat and teach patients, but learn from them. Imagine chronic pain being managed without the negative side effects of opioids. Penn Medicine researchers are working to make those scenarios a reality. These initiatives are among innovative new National Institutes of Health (NIH) funded efforts that are mapping the future of medicine.

The NIH has selected two researchers from the Perelman School of Medicine at the University of Pennsylvania to receive its prestigious Directors Awards, part of the NIH Common Funds High-Risk, High-Reward Research Program honoring exceptionally creative scientists. Brian Litt, MD, a professor of Neurology, Neurosurgery, and Bioengineering, was honored with a Pioneer Award for $5.6 million, supporting novel neurodevice research. Gregory Corder, PhD, an assistant professor of Psychiatry and Neuroscience, was selected as a New Innovator Award winner, receiving $2.4 million for research investigating the mechanisms of chronic pain.

This NIH initiative, designed to fuel research endeavors that are more opened-ended and have a potentially broader effect on scientific understanding compared to more traditional research, awards these to scientists to support research over a five-year period. The 2020 Penn recipients are among 85 awardees nationally:

Pioneer Award Ghost in the Machine: Melding Brain, Computer and Behavior

The Pioneer Award challenges investigators to pursue new research directions and develop groundbreaking, high-impact approaches to a broad area of biomedical or behavioral science. This award supports Litts work to develop a new generation of autonomous neurodevicesimplanted machines that can question, record, and combine learning algorithms based on neurological signals and feedback to act and alter human behavior on the fly.

In epilepsy, for example, these devices would predict and prevent seizures; in Parkinsons patients, implants will measure and communicate with patients to improve mobility, reduce tremor and enhance responsiveness. Other implants might improve hearing or psychiatric symptoms by querying patient perceptions, feelings, and altering stimulation patterns algorithmically to improve them. The loop is closed in real time, so the host can change their behavior based upon device feedback to improve their health.

Imagine this: A 30 year-old veteran walks into a bar. None of the patrons are aware of the anti-seizure device in his brain, or the traumatic brain injury that requires it. After he drinks a beer, his phone vibrates with a text from the implantable device asking what hes doing and sharing his probability of a seizure has increased. And with a quick explanation from the veteran, the device stimulates and suggests avoiding a second beverage, Litt explains. Were working towards this future, with the help of the Pioneer Award.

This is a paradigm shift from todays simple devices, which rely on physicians to give device feedback to patients and change simple parameters by hand during occasional office visits. Litts goal is to build a foundation for responsive implants that can collaborate with hosts, linking human experience and perception to machine algorithms, actions, and therapy, predicting and preventing events before they start. Not only will the patient teach the device, but the device will teach the patient.

Grant ID: DP1 NS122038-01.

New Innovator Award Harnessing Cortical Neuromodulation to Disrupt Pain Perception

The New Innovator Award supports unusually innovative research from early career investigators who have not yet received a research project grant or equivalent NIH grant. The award will support Corders efforts to research the mechanisms of chronic paina major health crisis in the United States, affecting millions, and a driver of the opioid epidemic.

Corders goal is to identify which parts of the brain are important for pain perception and which circuits impact pain relief from opioids. He hopes to decode how this neural activity evolves during chronic pain. Once the brain circuits and pathways that contribute to the suffering and perception of pain are identified, they can be targeted for potential therapeutics which could be more effective at reducing pain and without the addictive elements of prescription opioids.

Corder envisions these next generation pain therapeutics leveraging viral-delivered cell-specific gene therapies to disrupt the pain-processing circuits in the cortex. This would will allow patients to sense pain but without the unpleasant aversion.

We currently have a limited understanding of the neural pathways in the brain that contribute to pain, which has been a significant barrier for treating pain efficiently, without negative side effects. But, if we can identify and understand these circuits, we can then try to rewrite the neural code of pain, Corder said. Picture being able to specifically target the one desired brain region or circuit that processes pain in order to provide pain relief. This research will help us lay the groundwork for new classes of therapeutics, which could have a profound and broad impact for treating patients with chronic pain, while reducing the burden of the national opioid crisis.

Grant ID: DP2 GM140923-01.

###

Penn Medicineis one of the worlds leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of theRaymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nations first medical school) and theUniversity of Pennsylvania Health System, which together form a $8.6 billion enterprise.

The Perelman School of Medicine has been ranked among the top medical schools in the United States for more than 20 years, according toU.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $494 million awarded in the 2019 fiscal year.

The University of Pennsylvania Health Systems patient care facilities include: the Hospital of the University of Pennsylvania and Penn Presbyterian Medical Centerwhich are recognized as one of the nations top Honor Roll hospitals byU.S. News & World ReportChester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; and Pennsylvania Hospital, the nations first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is powered by a talented and dedicated workforce of more than 43,900 people. The organization also has alliances with top community health systems across both Southeastern Pennsylvania and Southern New Jersey, creating more options for patients no matter where they live.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2019, Penn Medicine provided more than $583 million to benefit our community.

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Penn Medicine Researchers Receive Prestigious National Institutes of Health Directors Awards - Newswise

LONDON, Oct. 06, 2020 (GLOBE NEWSWIRE) -- (Oncology Drugs Companies Included: F. Hoffmann-La Roche AG, Novartis AG, Bristol-Myers Squibb Company, Merck & Co., Inc., Johnson & Johnson)

The global oncology/cancer drugs market size reached a value of nearly $167.9 billion in 2019, having increased at a compound annual growth rate (CAGR) of 9.8% since 2015. The oncology market size 2020 is expected to decline from $167.9 billion in 2019 to $149.9 billion in 2020 at a rate of -11%. The decline is mainly due to lockdown and social distancing norms imposed by various countries and economic slowdown across countries owing to the COVID-19 outbreak and the measures to contain it. The market is then expected to grow slightly from $201.1 billion in 2021 to $284.5 billion in 2023 at a CAGR of 9.7%. North America has the largest cancer drug market share, accounting for 41.2% of the total market.

The Business Research Companys report titled Oncology Drugs Market - Opportunities And Strategies - Global Forecast To 2030 covers major oncology drugs companies, oncology drugs market share by company, oncology drugs manufacturers, oncology drugs infrastructure market size, and oncology drugs market forecasts. The report also covers the global oncology drugs market and its segments. The cancer market by type is segmented into drugs for lung cancer, pancreatic cancer, breast cancer, prostate cancer, ovarian cancer, colorectal cancer, gastric cancer, kidney cancer, brain tumor, thyroid cancer, skin cancer, bladder cancer, cervical cancer, blood cancer and others, by drug class type into targeted therapy, immunotherapy (biologic therapy), chemotherapy and hormonal therapy, by distribution channel into hospital pharmacies, retail pharmacies/drug stores and others, by route of administration into oral, parental and others, by drug classification into branded drugs and generic drugs.

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Companies in the oncology drugs market are increasing their product innovation through strategic collaborations. To sustain in the increasingly competitive market, organizations are developing innovative products as well as sharing skills and expertise with other such enterprises. While oncology drug companies have long collaborated with each other as well as with academic and research institutions in this market by way of partnerships, in or out licensing deals, this trend has been increasing over the recent years.

New technologies are being implemented in the cancer drug market, such as artificial intelligence in the research and development process, as well as 3D printing devices to mimic the human body for trials and testing of the drugs developed. Companies are also investing in technologies to develop next generation biologics such as antibody drug conjugates, bispecifics, fusion proteins, cell and gene therapy that will be more effective as they are expected to have better potency against the target disease as well as have the ability to treat more than one aspect of the disease. Over the last few years, there has been a significant rise in accelerated approval of cell and gene therapies for cancer treatment by regulatory bodies across the globe. CRISPR technology can be used to discover the non-coding cancer genome. CRISPR-Cas9 genome editing reduces the processing associated with the generation of cell line and animal models of cancer and complex generations. Thus, it generates a better cancer model for target validation and drug evaluation. CRISPRCas9 is accelerating the different stages of oncology drug discovery including target identification, validation and deconvolution, drug synthesis, assessment of drug sensitivity and resistance.

With innovation through technology and rising focus on development of new drugs, the oncology drugs market trends ought to bring advances in cancer therapies in the forecasted period.

Oncology Drugs Market - Opportunities And Strategies - Global Forecast To 2030 is one of a series of new reports from The Business Research Company that provide oncology drugs market overviews, oncology drugs market analyze and forecast oncology drugs market size and growth for the whole market, oncology drugs market segments and oncology drugs market geographies, oncology drugs market trends, oncology drugs market drivers, oncology drugs market restraints, oncology drugs market leading competitors revenues, profiles and market shares in over 1,000 industry reports, covering over 2,500 market segments and 60 geographies.

The report also gives in-depth analysis of the impact of COVID-19 on the market. The reports draw on 150,000 datasets, extensive secondary research, and exclusive insights from interviews with industry leaders. A highly experienced and expert team of analysts and modelers provides market analysis and forecasts. The reports identify top countries and segments for opportunities and strategies based on market trends and leading competitors approaches.

Here Is A List Of Similar Reports By The Business Research Company:

Clinical Oncology Next Generation Sequencing Global Market Report 2020-30: COVID 19 Growth and Change

Interventional Oncology Devices Global Market Report 2020-30: COVID 19 Growth and Change

Cancer Diagnostics Global Market Report 2020-30: COVID 19 Growth And Change

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Oncology Market Size 2020 Particularly Prone To Disruption During Pandemic In The Global Oncology Market - GlobeNewswire

DELHI, India, Oct. 07, 2020 (GLOBE NEWSWIRE) -- "Global Bispecific Antibody Market Opportunity, Drug Sales & Clinical Trials Insight 2026" Report Highlights:

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https://www.kuickresearch.com/report-global-usa-europe-japan-bispecific-antibody-antibodies-therapeutics-therapy-market-sales-size--trends-clinical-trials-emicizumab-hemlibra-blincyto--blinatumomab-sales

Despite having several hundred clinical success stories of different immunotherapies against solid and advanced cancers, the overall response rate of the patients has reached approximately 50% even when the patients who fit completely under all the criteria of treatment. To pave with the clinical failures, researchers have developed and evaluated bispecific therapeutic antibodies, that are combination of two binding specificities leading to improve selectivity as well as efficacy of antibody-based treatment for cancer. The arrival of bispecific antibody for cancer treatment has estimated to be of great potential for wide range of applications in cancer treatment. The current biotherapeutic market for cancer treatment and prevention is estimated to be dominated by antibody drugs, where bispecific antibody represents a novel ally for the next-generation of cancer therapy.

In the past few years, the remarkable progress that has been made with respect to understanding the important hallmarks of the cancer progression and main epitopes involved in it have led to paradigmatic development in the bispecific antibody capable of offering novel promises to the patients who havent respond to any medical therapy for cancer. The practice of bispecific antibody is not new i.e. this version of cancer therapeutics is a modified version of monoclonal antibody. By observing the increasing incidence of cancer, the management associated with the bispecific antibody has started to develop several strategies important for clinical management of the cancer in the present and in the future.

As per the analysis conducted, it is estimated that the bispecific antibody therapeutics market is growing at a double-digit rate, thus boosting the overall cancer therapeutics market to extreme, especially when compared with other immunotherapies. Further, trends and opportunities coupled within the market i.e. rise in the number of cancer cases, government favorable policies and many others are tending to drive the growth of the market. In our viewpoint, novel alliances developed by major key players of the market and local manufacturers are estimated to be playing important role for delivering tremendous opportunities for the researchers, patients, stakeholders and the payers.

The analysis of the Global bispecific antibody drug market represents that the market is completely focused on providing a better structure to the overall therapeutics market and offer the pharmaceutical industry a broad perspective of an efficient therapy for every cancer type. The research report provides analysis about the assess growth potential of the market by verifying various driving forces, in combination with the all the important trends, opportunities and challenges. In addition to it, the research report also provides a long-term outlook for the industry and various other important measures that have helped the market to attract thousands of investors and local drug manufacturers. Lastly, the report also profiles various pharmaceutical companies of the dominating markets that have been boosting the R&D sector of the market.

Neeraj Chawla neeraj@kuickresearch.com +91-9810410366

Table of Contents

1. Introduction to Bispecific Monoclonal Antibody

1.1 Overview of Monoclonal Antibody 1.2 Overview of Bispecific Monoclonal Antibody 1.3 Advantage of Bispecific Antibodies upon Monospecific Monoclonal Antibodies

2. The Engineering & Development of Bispecific Antibodies

2.1 Bispecific Antibodies Similar to IgG

2.1.1 Quadroma (Hybrid Hydromas) Approach 2.1.2 Knobs Into Holes Approach 2.1.3 CrossMab Approach 2.1.4 Dual-Variable-Domain Immunoglobulin Approach

2.2 Small Bispecific Antibodies

2.2.1 Bispecific Diabodies 2.2.2 Bispecific T-Cell Engager Antibodies (BiTEs)

3. Mechanism Of Action Of Bispecific Antibody

3.1 Trifunctional Antibody: Catumaxomab (Removab) 3.2 Blinatumomab

4. Applications of Bispecific Antibodies in Diagnostic & Treatment

4.1 BsMAb for Diagnosis of Bacterial & Viral Infectious Diseases 4.2 BsMAb for Cancer Diagnostic 4.3 BsAbs Blocking Signaling Pathways 4.4 BsAbs Targeting Tumor Angiogenesis 4.5 Specific Delivery of Effector Compounds to Targets 4.6 Bispecific Antibodies & Gene Therapy

5. Global Bispecific Antibody Market Outlook

5.1 Current Market Scenario

5.2 Availability of Bispecific Antibodies 5.2.1 Catumaxomab (Removab) 5.2.2 Blinatumomab 5.2.3 Emicizumab 5.2.4 Duligotumab 5.2.5 SAR 156597

6. Approved Bispecific Antibody Dosage & Price Analysis

6.1 Blincyto 6.2 Hemlibra

7. Approved Bispecific Antibody Sales Analysis

7.1 Blincyto 7.2 Hemlibra

8. Global Bispecific Antibodies Clinical Pipeline Overview

8.1 By Phase 8.2 By Country/Region 8.3 By Company 8.4 By Indication 8.5 Orphan Designated Bispecific Antibodies

9. Global Bispecific Antibodies Clinical Pipeline By Company, Indication & Phase

9.1 Research 9.2 Preclinical 9.3 Phase-I 9.4 Phase-I/II 9.5 Phase-II 9.6 Phase-II/III 9.7 Phase-III

10. Marketed Bispecific Antibodies Clinical Insight

10.1 Blinatumomab - Amgen 10.2 Emicizumab - Chugai Pharmaceutical

11. Global Bispecific Antibody Market Dynamics

11.1 Favorable Market Parameters 11.2 Commercialization Challenges

12. Global Bispecific Antibody Market Future Prospects

13. Competitive Landscape

13.1 Ablynx 13.2 Adimab 13.3 Affimed Therapeutics 13.4 Amgen 13.5 AstraZeneca (MedImmune) 13.6 Chugai Pharmaceutical 13.7 Eli Lilly 13.8 EMD Serono 13.9 Emergent BioSolutions 13.10 Genentech 13.11 Genmab 13.12 Immunomedics 13.13 Jounce Therapeutics 13.14 MacroGenics 13.15 Merus 13.16 Neovii Biotech 13.17 NovImmune SA 13.18 OncoMed Pharmaceuticals 13.19 Pieris 13.20 Regeneron Pharmaceuticals 13.21 Roche 13.22 Sanofi

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Global Bispecific Antibodies Therapy Blincyto Hemlibra Antibody Therapeutics Market Size To Witness 118% Growth To Reach US$ 10 Billion By 2026 -...

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Pittsburgh, PA (KDKA) A West Virginia teen is among the first in the country to get gene therapy treatment for a rare condition.

Kendra Goins has Hurler Syndrome, meaning her body cannot break down certain types of sugar. In addition to damaging her organs, the condition also makes it difficult for her to grow.

Ive spoken with experts all across the country, all the way from the chemist that made this down to the doctor who has used it. This looks like her golden ticket, said Sheryl Goins, Kendras mother.

The goal of the treatment is to force Kendras body to produce the enzymes to break down sugars.

Please note: This content carries a strict local market embargo. If you share the same market as the contributor of this article, you may not use it on any platform.

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Teenager among first to receive gene therapy treatment for rare condition - KRDO

A Yes vote authorizes the state to sell $5.5 billion in general obligation bonds primarily for stem cell research and the development of new medical treatments in California. A No vote would mean the state's stem cell research agency will probably shut down by 2023.

In the ramp-up to the 2004 election, a California TV viewer may have come across the popular actor Michael J. Fox urging her to vote Yes on a state proposition. His voice slurred faintly by Parkinsons disease, he still sounded wry, boyish and familiar.

My most important role lately is as an advocate for patients and for finding new cures for diseases, said Fox, eyes level with the camera. Californias Stem Cell Research Initiative 71 will support research to find cures for diseases that affect millions of people, including cancer, diabetes, Alzheimer's and Parkinson's.

Within that 30-second spot, Fox, diagnosed at age 29 with a neurodegenerative disorder that typically does not strike until after 60, used the word "cures" three times.

Proposition 71, which passed with 59% of the vote, authorized the sale of $3 billion in bonds to create an agency that funded stem cell research. The successful campaign grew out of a time, in the early 2000s, when the promise of stem cell and regenerative medicine excited both scientists and the public.

Whether the project has lived up to that promise is a matter of opinion. How voters view the record of the agency may go a long way in their decision whether or not to replenish the fund, which is fast running out of money, with an additional $5.5 billion to be raised with new bonds authorized by Proposition 14, now on the ballot.

President Bush A Demon to Attack

Scientists since the1800s have known about stem cells, which are not yet dedicated to any particular anatomical function and have the potential to become nerve cells, blood cells, skin cells or any other type. They are found in blastocysts, which are human embryos four to five days after fertilization, and in a few areas, such as bone marrow and gonads, in adults.

In the late 1990s, researchers developed ways to steer the development of these cells, and the possibilities for improving medicine seemed endless. If malfunctioning cells were at the root of a particular disease, could new healthy cells tailored to the job fix what was wrong? Scientists and many members of the public were eager to find out.

Anti-abortion groups, however, a key constituency of President George W. Bush, opposed the research, and in 2001 he limited federal funding to a few existing lines of embryonic stem cells, severely curtailing research.

Some in the state of California wanted to get around Bushs restrictions, and Proposition 71 was born.

"(T)hey had this demon they could attack in the campaign the Bush administration," said David Jensen, author of "California's Great Stem Cell Experiment," who also writes the blog California Stem Cell Report. "They could say, 'This is a great opportunity, and the only way we're going to get it done is to do it here in California.'"

The measure created the California Institute for Regenerative Medicine. The stem cell research agency is unique in the U.S.

"No other state has done this kind of level of funding and focus on this kind of thing, said Jensen. It's a really cutting-edge area of science."

A Few Successes

The pace of innovation has been slower than many hoped. As it turned out, grand discoveries were not around the corner, and to date there is no widespread stem cell treatment approved for the public. To date, CIRM has funded more than 64 trials directly and aided in 31 more. Not all have or will result in treatments.

But despite the lack of a marquee cure like one for Alzheimers or Parkinsons, the agency has seen some notable triumphs.

"Probably one of the most spectacular successes they have certainly so far," said Jensen, "is clinical trials that have saved the lives of what they say are 40 children."

Those children were born with severe combined immunodeficiency (SCID), commonly known as "bubble baby syndrome," a rare, generally fatal condition in which a child is born without a working immune system. An FDA-approved gene therapy that grew out of CIRM-funded research can now cure the disease by taking a patients own blood stem cells and modifying them to correct the SCID mutation. The altered cells generate new, healthy blood cells and repair the immune system.

The FDA has also approved two drugs for rare blood cancers that were developed with CIRM funds.

Sandra Dillon, a graphic designer in San Diego, credits one of the drugs with saving her life. She was diagnosed when she was just 28, in 2006. Her doctors told her they would try to manage her symptoms, but that she was going to get progressively sicker.

"Even just the idea of a cure or getting better wasn't even on the table back then," said Dillon, who is featured in ads for the Yes on 14 campaign.

"I remember just praying and begging into the universe, please, someone just look at my disease, please someone help, who is going to look at this thing.

By 2010, Dillon was extremely ill. She connected with a doctor at UC San Diego who received early-stage funding from CIRM and told her she could take part in clinical trials.

"For the first time, there was this moment of, 'Oh, my gosh! There are researchers doing something. And it could help me and I can get access to it.' It was amazing."

The drug received FDA approval in 2019, and today Dillons cancer has retreated to the point where she can live a normal life.

"I love that I am not tethered to a hospital anymore. I can go out on long backpacking trips and hiking and surfing," she said. "I am a completely different person with this drug. And I have a whole future ahead of me."

The original funding raised by Proposition 71 is running out. Proposition 14 would authorize the sale of a new bond to refill the agency piggy bank. Gov. Gavin Newsom, the UC Board of Regents, and scores of patient advocacy groups also support the measure.

Many newspaper editorial boards, however, oppose the proposition, including the San Francisco Chronicle, Mercury News and Los Angeles Times.

Right now the state still owes about $1 billion toward the debt created by Proposition 71. If Proposition 14 passes, the yearly price tag to pay off the new bond would be about $260 million per year for about 30 years.

One of the selling points of the original proposition was the potential for the state to earn big money in royalties from the treatments it helped develop, says Jeff Sheehy, an HIV patient advocate and the only CIRM board member to oppose Proposition 14.

"The promises were made that this would pay for itself. We would be able to pay back the bonds with the money we would get from royalties, etc., etc.

That has not worked out as envisioned: CIRM estimates it has received less than $500,000 in royalties. Early this year, Forty Seven, a company whose therapies were heavily funded by CIRM, sold to Gilead for $4.9 billion. While millions went to various researchers, neither CIRM nor the state of California received anything.

One of the flaws in the original measure is that we [the agency] cannot hold stock in the products that we develop," says Sheehy. "And that's because the California Constitution says that the state of California cannot, as a government entity, hold equity.

Proposition 14 makes it impossible for the state to use profits from its investment on, say, schools or other funding priorities. Instead, any royalties earned must be fed back into programs to make CIRM-funded treatments more affordable.

"What it does is it basically takes all of our returns that we get from this and gives it back to the pharmaceutical and biotech companies," said Sheehy. "It becomes just a blatant giveaway to these companies when we should be requiring access and requiring fair pricing."

Sheehy says he supports medical research, but doesn't like the state going into more debt to pay for it. The greater the state's obligations in bond money, which has to be paid back with interest, the less there is in the general fund, and Sheehy says the state has more pressing needs than stem cell research things like housing, education and transportation.

"The biggest and perhaps the most compelling reason why I feel so strongly that this is not a good idea is that we simply cannot afford it, he said. "If we think this is so important," asks Sheehy, "why don't we just don't pay for [this research] out of the general fund? It would be cheaper.

Opponents of Proposition 14 also point to longstanding complaints of conflicts of interest among the agency board. Most of the $3 billion distributed by the agency has gone to institutions with connections to board members. Critics say the structural conflicts of interest between the board and agency are not addressed in the new measure. Proposition 14 would balloon an already huge board of 29 members to 35.

Funding needs for stem cell research also are not as acute as they were back in 2004. The federal National Institutes of Health now funds some basic stem cell research, spending about $2 billion a year, with $321 million of that going toward human embryonic stem cell research. And private ventures, like nonprofits started by tech billionaires, are pouring more money into biotech.

The problem with assuming that, says Melissa King, executive director of Americans for Cures, the stem cell advocacy group behind the Yes on 14 campaign, is that CIRM fills a neglected funding need.

The NIH does not fund clinical trials at nearly the rate that CIRM can and has been, King said.

She says that's important because of what she calls the "Valley of Death," where promising early-stage research frequently fails to translate into promising treatments that can be tested in clinical-stage research. (What works well in a test tube often does not work well in an organism.) This weeding-out process is costly but necessary. And its where CIRM focused a lot of its effort.

The first- and maybe even second-phase clinical trials, its very difficult to get those funded, King said. It is too much of a risk for business to take on on its own. Venture [capital] isnt going there. Angel [funding] isnt going there.

What voters have to ask themselves, says writer Jensen, is whether stem cell funding is "a high priority for the state of California? Different people make different judgments about that."

CIRM supporters say if Prop. 14 doesn't pass, critical research will stall. Others say federal and private funding will step in and fill the gap.

Absent new funding, the institute expects it will wind down operations leading to a complete sundown in 2023.

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Proposition 14: With Just Handful of Cures, California Stem Cell Agency's Fate Is In Hands of Voters - KQED

National/World

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Pittsburgh, PA (KDKA) A West Virginia teen is among the first in the country to get gene therapy treatment for a rare condition.

Kendra Goins has Hurler Syndrome, meaning her body cannot break down certain types of sugar. In addition to damaging her organs, the condition also makes it difficult for her to grow.

Ive spoken with experts all across the country, all the way from the chemist that made this down to the doctor who has used it. This looks like her golden ticket, said Sheryl Goins, Kendras mother.

The goal of the treatment is to force Kendras body to produce the enzymes to break down sugars.

Please note: This content carries a strict local market embargo. If you share the same market as the contributor of this article, you may not use it on any platform.

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Teenager among first to receive gene therapy treatment for rare condition - NewsChannel 3-12 - KEYT