Huntington's disease research suffered a double whammy after Swiss pharmaceutical giant Roche Holding AG halted late-stage trials of an experimental medicine and Wave Life Sciences Ltd. abandoned two drug studies for futility.

Roche stopped dosing patients with tominersen after an independent data monitoring committee questioned whether the drug's benefits outweighed its risks.

"Obviously, it's a very sad situation when we have to stop a pivotal program in a disease like Huntington's where there's such a high unmet need and such a terrible impact on these patients and their families," Bill Anderson, head of Roche's pharmaceuticals business, said on a call with reporters following Roche's first-quarter results. "It's a major setback for the field. But we will do everything we can to learn from the work that's been done. And if there's a way forward, we would certainly pursue it."

The research stumbles, while not uncommon in the notoriously complex world of central nervous system drug development, leave only a single experimental compound in the last stage of trials usually required to seek marketing approval: pridopidine from Naarden, Netherlands-based Prilenia Therapeutics BV.

First developed at Israel's Teva Pharmaceutical Industries Ltd., where Prilenia CEO Michael Hayden was global head of R&D and chief scientific officer until 2017, pridopidine is being studied in phase 3 trials that will probably read out by the third quarter of 2022. That makes it the most advanced experimental compound in development for the hereditary disease, which affects about 70,000 people in the U.S. and Europe, the biggest potential markets for treatments. No medicines have been approved to either delay onset or slow the disease's progression.

Roche is following up with the patients on the trials of tominersen to thoroughly assess the effect of therapy. The Basel, Switzerland-based pharma company intends to continue research into Huntington's in its early-stage neurology research groups, as well as possibly via gene therapy, Anderson said.

Regulators have expressed more support for Huntington's drug developers after the influence and investment brought to bear by Roche, Europe's largest pharmaceutical company by revenue.

"They know all about Huntington's disease," Prilenia's Hayden said in an interview with S&P Global Market Intelligence. "I'm very grateful to Roche in particular, that they brought their muscle and their weight and their resources to raising awareness [of the disease] all over the world."

A number of early studies are still ongoing, including one by Roche's crosstown rival Novartis AG, which is investigating its experimental spinal muscular atrophy treatment, branaplam, as a treatment for Huntington's. The once-a-week oral therapy is in phase 1 trials after securing orphan-drug designation from the U.S. Food and Drug Administration, with the first regulatory submissions planned for 2025.

Gene therapy?

Other early-stage approaches to treating the neurodegenerative disease include one-time gene therapies in trials with companies including uniQure NV and Voyager Therapeutics Inc., which had a clinical hold lifted by the FDA on April 26.

But gene therapy may not be the approach to take, Roche's Anderson said. Huntington's disease is caused by a single mistake in a particular gene that triggers the formation of a toxic protein that kills nerve cells. Gene therapy introduces genetic material into cells to make up for abnormal genes or to produce a necessary protein. The therapy also needs to be distributed throughout the brain, and that requires intracranial surgery, injections into the brain or both.

"There are different types of genetic disorders," Anderson said. "Some of them, you have a case where you're missing a protein. And it's a relatively easier thing to add a protein when you're missing a protein with gene therapy, than if you have a genetic defect that is causing a toxic protein to form. In which case, you have to knock that down. That's a harder task for gene therapy. It doesn't mean it's not possible. It's just a trickier thing to do."

Anderson said he was not referring to any research at Spark Therapeutics, the gene therapy company that Roche acquired in 2019.

Hayden, who is also a professor of medical genetics at the University of British Columbia, hopes to show the safety and tolerability already seen in more than 1,300 people to date. The phase 3 trial of the oral compound known as a sigma-1 receptor agonist is focused on the early Huntington's patient population, with a regulatory accepted endpoint of total functional capacity.

"We've learned deeply from the past. And we think we have it at least as close to right as we can now," Hayden said. "We believe that the study is quite de-risked, because we're not trying to look for new findings."

Pridopidine, which has patent protection until 2038, is easy to produce and available in tablet form. Hayden is keen to make the therapy readily available, should it gain approval, noting that the highest prevalence of the disease in the world was discovered in a remote tribe in Venezuela. Hayden also witnessed Huntington's impact at the start of his medical career in South Africa.

"I travelled the country and recognized that Huntington's disease was alive and well in Africa. These were [many] people who were disenfranchised by apartheid and disenfranchised by this disease," Hayden said.

While not wanting to raise unrealistic hopes, both Hayden and Anderson signaled that Huntington's researchers are not undeterred by recent setbacks.

"There are lots of things that we're doing these days that we didn't think we can do a few years ago," Anderson said. "And so we remain hopeful that we will find a solution."

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Huntington's disease setback for Roche shifts the research spotlight to Prilenia - S&P Global

Recommendation and review posted by Bethany Smith

Boston, MA, May 13, 2021 (GLOBE NEWSWIRE) -- Gene and cell therapy (GCT), a transformative approach to develop treatments, and, potentially, cures for congenital and other diseases, is the focus of this years World Medical Innovation Forum May 19-21. Widely recognized as a transformational opportunity in medicine, GCT has the potential to stop or slow the effects of disease by targeting them at the genetic level. When the genetic driver for a disease is known, patients can be molecularly matched to therapies.

Featuring a unique combination of nearly 200 speakers and panelists from Mass General Brighams Harvard-affiliated faculty, industry, venture, manufacturing, and regulatory agencies, the World Medical Innovation Forum, in an all-virtual format, will explore interdependent aspects of advancing GCT for patients worldwide. Topics cover GCT strategy, clinical opportunities, patient access, economic and investment considerations, regulatory frameworks and manufacturing scalability. Nearly 1000 organizations will be represented, including attendees from 43 countries and 40 states of the U.S.

The Forum includes updates on:

GCT Potential for PatientsGene Therapies for Specific Diseases Large Incidence and Rare Trends in Vector Development, including AAV, Lentivirus and Nano MethodsClinical Opportunities, Technology and Market PotentialRegulatory Frameworks for GCTShaping GCT InnovationCancer, Gene Therapy and Oncolytic VirusesCAR-T TherapyGCT Manufacturing, Supply Chain and ScalabilityImpact of mRNA Vaccines

The Forum fosters critical discussions and debates on issues that will determine the pace of expansion of GCT therapies for patients in the coming years, says Chris Coburn, Chief Innovation Officer, Mass General Brigham. In the spirit of collaborative innovation, this gathering of leading investors, entrepreneurs, industry executives, Harvard clinicians and scientists is unique in convening top decision-makers in sharing ideas for advancing these game-changing technologies to the front lines of medicine.

We are honored to be the presenting sponsor of this years World Medical Innovation Forum, said Dave Lennon, President, Novartis Gene Therapies. Having brought one of the first gene therapies to market, we have seen the enormous potential these breakthrough medicines hold to transform lives. We look forward to joining other leaders at the forum to share learnings and our vision for the future one in which gene therapies drive scientific innovation and deliver on their promise for patients and societies.

Showcasing Mass General Brigham BreakthroughsThe Forum showcases GCT innovation breakthroughs from Mass General Brighams Harvard Medical School faculty investigators in 10-minute First Look presentations, and a research poster session being included in the Forum for the first time. More than 40 emerging GCT research projects will be part of the virtual showcase, with topics including gene therapy for large incidence and rare diseases including neurology, cardiology, blood disorders, and oncology, CAR-T breakthroughs, and oncolytic, AAV and non-viral delivery methods.

Recipients of Mass General Brighams Innovation Discovery Grants for GCT research will be announced. The Forum concludes with the announcement of the annual Disruptive Dozen, 12 GCT advances members of the Mass General Brigham faculty believe are the most likely to break through in the next few years.

This years Forum co-chairs are Nino Chiocca, MD, PhD, Chair, Neurosurgery, Brigham and Womens Hospital and Cushing Professor of Neurosurgery, Harvard Medical School (HMS); Sue Slaugenhaupt, PhD, Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute, and Professor, Neurology, HMS; Ravi Thadhani, MD, CAO, Mass General Brigham and Professor, Medicine and Faculty Dean, HMS; and Luk Vandenberghe, PhD, Grousbeck Family Chair, Gene Therapy, Mass Eye and Ear, and Associate Professor, Ophthalmology, HMS.

About the World Medical Innovation Forum

The World Medical Innovation Forum was established seven years ago in response to the intensifying transformation of health care and its impact on innovation. The Forum is rooted in the belief that no matter the magnitude of change, the center of health care needs to be a shared, fundamental commitment to collaborative innovation industry and academia working together to improve patient lives.

To find out more about the Forum, speakers and agenda, and to register, visit https://worldmedicalinnovation.org/###

About Mass General Brigham

Mass General Brigham is an integrated academic healthcare system, uniting great minds in medicine to make life-changing impact for patients in our communities and people around the world. Mass General Brigham connects a full continuum of care across a system of academic medical centers, community and specialty hospitals, a health insurance plan, physician networks, community health centers, home care, and long-term care services. Mass General Brigham is a non-profit organization that is committed to patient care, research, teaching, and service to the community. In addition, Mass General Brigham is one of the nations leading biomedical research organizations and a principal teaching affiliate of Harvard Medical School. For more information, please visit massgeneralbrigham.org.

About Mass General Brigham Innovation

Innovation is the 150-person business development unit of Mass General Brigham responsible for the worldwide commercial application of the unique capabilities and discoveries of Mass General Brigham's 74,000 employees. Innovation supports the research requirements of its 6,200 Harvard Medical School faculty and research hospitals. It has responsibility for industry collaborations, venture investing, international consulting, licensing, innovation management, company creation, technology marketing, open innovation alliances, and workforce development.

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Gene and Cell Therapy Breakthroughs Focus of World Medical - GlobeNewswire

Recommendation and review posted by Bethany Smith

Mary Newman, former SVP of Regulatory Affairs at Astellas Gene Therapies, joins Taysha with over 30 years of experience in regulatory affairs and research and development in the biotech industry

Extensive experience deeply rooted in gene therapy at Astellas Gene Therapies, Audentes Therapeutics and BioMarin

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the most recent addition to its leadership team with the appointment of industry veteran Mary Newman as Chief Development Officer. Ms. Newman will oversee program and portfolio management, as well as translational sciences, and will report to Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Tayshas Chief Medical Officer and Head of Research and Development.

We are pleased to welcome Mary to the Taysha leadership team, said RA Session II, President, Founder and CEO of Taysha. In the past few months, we have significantly expanded our extremely talented R&D team, and Mary is an important addition to support the achievement of our ambitious pipeline advancement goals. Her decades of experience in all key aspects of drug development, particularly in gene therapy and rare disease, will be invaluable as we continue to grow our business.

Ms. Newman joins Taysha with more than 30 years of experience in regulatory affairs and research and development within the biotechnology industry, focusing on rare diseases. Most recently, she served as Senior Vice President of Regulatory Affairs at Astellas Gene Therapies (formerly Audentes Therapeutics), where she oversaw global regulatory strategic development, all primary regulatory agency interactions, and regulatory compliance for Audentes development candidates. Prior to joining Audentes, Ms. Newman served as the Senior Vice President, Regulatory Affairs and Quality Assurance at SARcode Bioscience Inc., and was responsible for the development of Xiidra for the treatment of dry eye disease. The company was acquired by Shire Ltd. She previously held various management positions, with increasing responsibility, in Regulatory Affairs at BioMarin Pharmaceutical, Inc., Berlex Inc. (now Bayer HealthCare Pharmaceuticals Inc.), and Sequus Pharmaceuticals, Inc. (now Johnson & Johnson). While at BioMarin, Ms. Newman oversaw the development and approval of Kuvan for the treatment of phenylketonuria (PKU), Naglazyme for mucopolysaccharidosis (MPS) VI, and supported the final approval of Aldurazyme for MPS I. She has also held various research and development leadership roles in oncology, neurology, and antifungal therapeutic areas. Ms. Newman serves as a member of the board of directors of Vedere Bio and is an advisor to the board of directors of Chameleon Biosciences. Ms. Newman holds a B.S. in Physiology from Oregon State University.

I am excited to join Taysha at such a transformative time in the companys corporate lifecycle, said Ms. Newman. Taysha is well-positioned to be a leader in developing disease-modifying gene therapies for patients with monogenic CNS diseases. I look forward to contributing to the further progression of the companys robust pipeline of promising drug candidates.

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.

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Taysha Gene Therapies Expands Leadership Team with the Appointment of Chief Development Officer - BioSpace

Recommendation and review posted by Bethany Smith

EMERYVILLE, Calif., May 13, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced financial results for the first quarter of 2021, and provided operational highlights.

We continue to relentlessly execute and innovate as demonstrated by achievements in our first full quarter as a public company, said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. The company remains on track to announce initial clinical data from both our 4D-310 Fabry disease product candidate and our 4D-125 XLRP product candidate in the second half of this year. In addition, we remain on track to initiate clinical trials in the second half of this year for 4D-150, our wet AMD and DME product candidate, and for 4D-710, our cystic fibrosis lung disease product candidate. We also recently expanded our technology platform to include applications of machine learning, and yesterday, at the annual ASGCT conference, we presented preclinical non-human primate data from 4D-150.

Recent Operational Highlights

Expected Upcoming Milestones

Financial Results for the First Quarter Ended March 31, 2021

Cash and Cash Equivalents: Cash and cash equivalents were $259.9 million as of March 31, 2021. We expect cash and cash equivalents to be sufficient to fund operations into mid-2023.

Revenue: Total revenue was $2.0 million for the quarter ended March 31, 2021, as compared to $3.5 million for the quarter ended March 31, 2020. The decrease was primarily driven by decreased revenue recognized under the Roche collaboration agreement.

R&D Expenses: Research and development expenses were $12.8 million for the quarter ended March 31, 2021, as compared to $13.2 million for the quarter ended March 31, 2020. This decrease was primarily driven by decreased external manufacturing expense, which was partially offset by higher payroll and stock-based compensation expense.

G&A Expenses: General and administrative expenses were $5.5 million for the quarter ended March 31, 2021, as compared to $3.7 million for the quarter ended March 31, 2020. This increase was primarily due to higher payroll and stock-based compensation expense and higher business insurance expense.

Net Loss: Net loss was $16.4 million for the quarter ended March 31, 2021, as compared to $13.2 million for the quarter ended March 31, 2020.

About 4DMT

4DMT is a clinical-stage company harnessing the power of directed evolution for targeted gene therapies. 4DMT seeks to unlock the full potential of gene therapy using its platform, Therapeutic Vector Evolution, which combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. The company is initially focused in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The 4DMT targeted and evolved vectors are invented with the goal of being delivered through clinically routine, well-tolerated and minimally invasive routes of administration, transducing diseased cells in target tissues efficiently, having reduced immunogenicity and, where relevant, having resistance to pre-existing antibodies. 4DMT is currently conducting three clinical trials: 4D-125 is in a Phase 1/2 clinical trial for XLRP patients, 4D-110 is in a Phase 1 clinical trial for choroideremia patients and 4D-310 is in a Phase 1/2 clinical trial for Fabry disease patients.

4D Molecular Therapeutics, 4DMT, Therapeutic Vector Evolution, and the 4DMT logo are trademarks of 4DMT.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding plans and timelines for the clinical development of 4D-310, 4D-125, 4D-110, 4D-150 and 4D-710, including the therapeutic potential and clinical benefits thereof; the estimated timing of clinical data being available for 4D-125s Phase 1/2 clinical trial and 4D-310s Phase 1/2 clinical trial; the estimated timing of initiating the clinical trials for 4D-150 and 4D-710; expectations on how long our cash and cash equivalents can fund operations; expectations regarding current and future interactions with the U.S. Food and Drug Administration (FDA); and 4D Molecular Therapeutics' strategy, business plans and focus. The words "may," might, "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," expect, "estimate," seek, "predict," future, "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks associated with: the impact of COVID-19 on countries or regions in which we have operations or do business, as well as on the timing and anticipated results of our clinical trials, strategy and future operations; the delay of any current or planned clinical trials for the development of 4D Molecular Therapeutics' drug candidates, the risk that the results of our clinical trials may not be predictive of future results in connection with future clinical trials; 4D Molecular Therapeutics' ability to successfully demonstrate the safety and efficacy of its drug candidates; the timing and outcome of our planned interactions with regulatory authorities; and obtaining, maintaining and protecting our intellectual property. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics most recent Quarterly Report on Form 10-Q to be filed on or about the date hereof, as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

4D-310, 4D-125 and 4D-110 are our product candidates in clinical trials and have not yet been approved for marketing by the US FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of 4D-310, 4D-125, or 4D-110 for the therapeutic use for which they are being studied.

4D Molecular Therapeutics, Inc.Condensed Statements of Operations and Comprehensive Loss(Unaudited)(in thousands, except share and per share amounts)

4D Molecular Therapeutics, Inc.Condensed Balance Sheet Data(Unaudited)(inthousands)

Contacts:

Media:

Theresa Janketjanke@4dmt.com

Investors:

Mike ZanoniEndurance Advisorsmzanoni@4dmt.com

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4D Molecular Therapeutics Reports Financial Results for the First Quarter of 2021 and Provides Operational Highlights - GlobeNewswire

Recommendation and review posted by Bethany Smith

DUBLIN, May 10, 2021 /PRNewswire/ -- The "The Market for Cell and Gene Therapy Contract Manufacturing Organizations (CMOs)" report has been added to ResearchAndMarkets.com's offering.

Contract manufacturing is an increasing part of the pharmaceutical business. In cell therapy, this need is magnified because of the demand for cell and gene therapy products.

The report the Market for Cell and Gene Therapy Contract Manufacturing Organizations (CMOs) details the following:

There are many companies in this market, and the analyst profiles a select group of companies that are representative of the market or handle a large volume.

These companies include:

Key Topics Covered:

Chapter 1: Executive Summary

Overview

Scope and Methodology

Covid 19

Market Overview and Potential

Chapter 2: Introduction to Cell and Gene Therapy

Introduction

Chapter 3: Cell and Gene Therapy Industry - Product Pipeline, Production & Key Players

Overview

Chapter 4: Cell and Gene Therapy Markets

Overview

Chapter 5: Biopharma Contract Manufacturing

Introduction

Chapter 6: Biopharma Contract Manufacturing Markets

Overview

Chapter 7: Cell & Gene Therapy Contract Manufacturing

Introduction

Chapter 8: The Market for Cell & Gene Therapy Contract Manufacturing

Overview

Chapter 9: Key Players in the Cell & Gene Therapy CMO Market

For more information about this report visit https://www.researchandmarkets.com/r/jih3bw

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Global Cell and Gene Therapy Contract Manufacturing Organizations (CMOs) Market Report 2021: Increasing Need for CMOs to Cope with Demand for Cell and...

Recommendation and review posted by Bethany Smith

The Cancer Gene Therapy Market is expected to grow at a CAGR of 9.36% and is poised to reach US$XX Billion by 2027 as compared to US$XX Billion in 2020. The factors leading to this extraordinary growth is attributed to various market dynamics discussed in the report. Our experts have examined the market from a 360 degree perspective thereby producing a report which is definitely going to impact your business decisions.

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Market by TypeGene Induced ImmunotherapyOncolytic VirotherapyGene Transfer

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North America US, Mexico, Canada Europe Russia, Ukraine, France, Spain, Sweden, Norway, Germany, Finland, Poland, Italy, United Kingdom, Greece, Austria, Denmark, Switzerland, Netherlands, Belgium, Turkey, Luxembourg Asia-Pacific China, Japan, India, Australia, South Korea, Taiwan, Malaysia, Philippines, Thailand, Singapore South America- Brazil, Argentina, Peru, ChileMiddle East and Africa Bahrain, Egypt, Israel, Kuwait, Qatar, Saudi Arabia, United Arab Emirates, South Africa

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Cancer Gene Therapy Market Supply Chain Analysis, Trends and Insights | Key Participants Arimex, Hines Nut Company, Archer Daniels Midland, Olam...

Recommendation and review posted by Bethany Smith

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the publication of new preclinical data for TSHA-102 in Rett syndrome. The data were published online and will be included in the May edition of Brain, a highly esteemed neurological science peer-reviewed journal.

Effective gene therapy targeting MECP2 for the treatment of Rett syndrome has been elusive due to the inability to properly regulate transgene expression, said Steven Gray, Ph.D., Chief Scientific Advisor at Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. The built-in self-regulatory feedback loop mechanism in TSHA-102, work that was initiated in my laboratory in 2007, is a completely novel approach that allows for regulated expression of MECP2 on a cell-by-cell basis. These results published today are highly encouraging and allow us to conceive additional novel approaches using miRARE in conditions with dose-sensitive genes.

The complexities of developing an efficacious and well-tolerated gene therapy for the treatment of Rett syndrome are highlighted by phenotypic variability, mosaicism and the need to regulate MECP2 such that it does not cause overexpression-related toxicity. Todays data give us confidence that we can achieve appropriate MECP2 expression in all cells in a genotype-dependent manner, which we believe significantly de-risks the developmental program in its translation to humans, said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. Historically, unregulated gene replacement of MECP2 resulted in overt adverse events, including death in wild type mice due to overexpression of the MECP2 protein. With the built-in regulatory element, miRARE, TSHA-102 provided a statistically significant survival extension by 56% in 4- to 5-week-old knockout Rett mice. We see the potential for broadening the miRARE platform to other CNS diseases requiring regulated gene expression. These positive data support our intent to file an IND/CTA for TSHA-102 in the second half of this year, followed by initiation of a Phase 1/2 trial by year-end 2021. TSHA-102 has the potential to address a significant unmet need for an estimated 25,000 patient with Rett syndrome across the United States and in Europe.

The preclinical study was conducted by the UT Southwestern Medical Center (UT Southwestern) laboratory of Sarah Sinnett, Ph.D., and evaluated the safety and efficacy of regulated miniMECP2 gene transfer, TSHA-102 (AAV9/miniMECP2-miRARE), via intrathecal (IT) administration in adolescent mice between four and five weeks of age. TSHA-102 was compared to unregulated full length MECP2 (AAV9/MECP2) and unregulated miniMECP2 (AAV9/miniMECP2).

TSHA-102 extended knockout survival by 56% via IT delivery. In contrast, the unregulated miniMECP2 gene transfer failed to significantly extend knockout survival at either dose tested. Additionally, the unregulated full-length MECP2 construct did not demonstrate a significant extension in survival and was associated with an unacceptable toxicity profile in wild type mice.

In addition to survival, behavioral side effects were explored. Mice were subjected to phenotypic scoring and a battery of tests including gait, hindlimb clasping, tremor and others to comprise an aggregate behavioral score. miRARE attenuated MECP2-mediated aggravation in wild type aggregate phenotype severity scores. Mice were scored on an aggregate severity scale using an established protocol. AAV9/MECP2- and AAV9/miniMECP2-treated wild type mice had a significantly higher mean (worse) aggregate behavioral severity score versus that observed for saline-treated mice (p <0.05; at 630 and 727 weeks of age, respectively). TSHA-102-treated wild type mice had a significantly lower (better) mean aggregate severity score versus those of AAV9/MECP2- and AAV9/miniMECP2-treated mice at most timepoints from 1119 and 920 weeks of age, respectively. No significant difference was observed between saline- and TSHA-102-treated wild type mice.

Of note, miRARE-mediated genotype-dependent gene regulation was demonstrated by analyzing tissue sections from wild type and knockout mice treated with AAV9 vectors given intrathecally. TSHA-102 demonstrated regulated MECP2 expression in different regions of the brain. In the pons and midbrain, miRARE inhibited mean MECP2 gene expression in a genotype-dependent manner as indicated by significantly fewer myc(+) cells observed in wild type mice compared to knockout mice (p<0.05), thereby demonstrating that TSHA-102 achieved MECP2 expression levels similar to normal physiological parameters.

It has been a challenge finding an approach that can appropriately regulate MECP2 expression in Rett syndrome but the preclinical data for TSHA-102 published today support the miRARE approach, said Sarah Sinnett, Ph.D., Assistant Professor in the Department of Pediatrics at UT Southwestern. It is clear that the disease is reversible, and I am encouraged that this novel strategy may enable us to make a difference in the management of this disease.

The publication is available by clicking on the following link: https://academic.oup.com/brain/advance-article-abstract/doi/10.1093/brain/awab182/6265600.

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.

Drs. Gray and Sinnett have intellectual property interest in Taysha and UTSW has a financial interest in the company.

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 the potential of our product candidates, including TSHA-102, 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, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements 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 (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, 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.

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Taysha Gene Therapies Announces Publication of Preclinical Data for TSHA-102 in Rett Syndrome in Brain, a Highly Esteemed Neurological Science...

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Global Stem Cell Therapy Market by Type (Allogeneic, Autologous), Therapeutic Application (Musculoskeletal, Wound & Injury, CVD, Autoimmune & Inflammatory), Cell Source (Adipose tissue, Bone Marrow, Placenta/Umbilical Cord) - Forecasts to 2026" report has been added to ResearchAndMarkets.com's offering.

The global stem cell therapy market is projected to reach USD 401 million by 2026 from USD 187 million in 2021, at a CAGR of 16.5% during the forecast period.

Growth in this market is majorly driven by the increasing investment in stem cell research and the rising number of GMP-certified stem cell manufacturing plants. However, factors such as ethical concerns and the high cost of stem cell research and manufacturing process likely to hinder the growth of this market.

The allogeneic stem cell therapy segment accounted for the highest growth rate in the stem cell therapy market, by type, during the forecast period

The stem cell therapy market is segmented into allogeneic and autologous stem cell therapy. Allogeneic stem therapy segment accounted for the largest share of the stem cell therapy market. The large share of this segment can be attributed to the lesser complexities involved in manufacturing allogeneic-based therapies.

This segment is also expected to grow at the highest growth rate due to the increasing number of clinical trials in manufacturing allogeneic-based products.

Bone Marrow-derived MSCs segment accounted for the highest CAGR

Based on the cell source from which stem cells are obtained, the global stem cell therapy market is segmented into four sources. These include adipose tissue-derived MSCs (mesenchymal stem cells), bone marrow-derived MSCs, placenta/umbilical cord-derived MSCs, and other cell sources (which include human corneal epithelium stem cells, peripheral arterial-derived stem cells, and induced pluripotent stem cell lines).

The bone marrow-derived MSCs segment is expected to witness the highest growth rate during the forecast period, owing to an increasing number of clinical trials focused on bone marrow-derived cell therapies and the rising demand for these cells in blood-related disorders.

Asia Pacific: The fastest-growing country in the stem cell therapy market

The stem cell therapy market is segmented into North America, Europe, Asia Pacific, RoW. The stem cell therapy market in the Asia Pacific region is expected to grow at the highest CAGR during the forecast period.

Factors such as the growing adoption of stem cell-based treatment in the region and the growing approval & commercialization of stem cell-based products for degenerative disorders drive the growth of the stem cell therapy market in the region.

Market Dynamics

Drivers

Restraints

Opportunities

Challenges

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/qiagh1

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Stem Cell Therapy Market by Type, Therapeutic Application and Cell Source - Global Forecasts to 2026 - ResearchAndMarkets.com - Business Wire

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When he called the Olivia Hospital and Clinic to postpone his physical, he was urged to keep it. Physicals are important, he was reminded.

Keeping that date proved to be a lifesaving decision.

The physical went well, and shortly after he told his daughter that he was as fit as a horse.

But Dr. Jon Kemp, his primary physician who had urged him to keep the date for the physical, noticed a slight abnormality in a standard blood test. He recommended further testing.

On Dec. 20 Kramer was diagnosed with multiple myeloma.

Thanks to the early diagnosis, Kramer, age 62, has the means of keeping this disease at bay. Its a cancer of the plasma cells in bone marrow, and is the second most common blood cancer.

He is about to undergo a stem cell transplant this week as part of his treatment.

He learned that hes not alone on the journey ahead.

At Tuesdays meeting of the Renville County Board of Commissioners, fellow board members came wearing T-shirts proclaiming: In this county, nobody fights alone.

Organizers of the surprise sold 76 of the T-shirts to show support for Kramer and raise funds for the Renville County Walk in the Park campaign. More than 40 T-shirt wearing supporters joined the meeting via Zoom. Staff in the health department sang a song to express their support, and staff members told him they would keep him in their thoughts and prayers.

Thank you, said Kramer. He told the West Central Tribune that he was totally surprised by the display of support.

He has lots of support from family and friends, and its all-important. Kramer farms in eastern Renville County. He has lined up plenty of helping hands while he undergoes the stem cell transplant, which will sideline him for at least six weeks.

He said doctors are confident the stem cell transplant can knock the cancer into remission. They will be harvesting bone marrow cells and freezing a portion of them to make it possible to perform at least two more transplants in future years as well.

The decision to keep the date of that routine physical made all the difference. Absolutely, said Kramer.

Health providers told him that in too many cases, multiple myeloma is not diagnosed until a patient comes in with a broken leg or other bone, and wondering why. The cancer carves holes and weakens bones as it progresses unbeknownst to the person.

Thanks to the early diagnosis, Kramer said they found only pinholes in his bones, having caught the disease in the first of its three stages. He began chemotherapy in early January, and it has proven effective, he added.

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Keeping the physical appointment was critical, the show of support appreciated by Renville County Commissioner - West Central Tribune

Recommendation and review posted by Bethany Smith

Multiple myeloma is a type of cancer that originates from white blood cells called plasma cells. Many factors affect the outlook for a person with this disease, including their age, overall health, and kidney function, as well as the stage of cancer at diagnosis.

Multiple myeloma is a cancer of the plasma cells, which are a type of white blood cell. Over time, myeloma cells multiply and accumulate in the bone marrow and solid parts of the bones.

Multiple myeloma can lead to organ damage that affects the kidneys, the bones, and the overall immune system.

In this article, we look at the outlook for people with different stages of multiple myeloma. We also look at the symptoms and treatment of multiple myeloma and what can affect a persons outlook.

The American Cancer Society (ACS) estimates that doctors will diagnose 34,920 new cases of multiple myeloma in 2021 and that there may be 12,410 deaths from the disease.

When a person receives a multiple myeloma diagnosis, the doctor will use the Revised International Staging System (RISS) to determine the stage of the cancer. This staging system is based on:

A person will receive a diagnosis of either stage 1, 2, or 3 multiple myeloma. There is also a stage 0, a slow-growing type of multiple myeloma that is called smoldering myeloma.

However, survival rates are based on summary staging, which the Surveillance, Epidemiology and End Results (SEER) program developed. This staging system groups cancers into:

As multiple myeloma does not spread to the lymph nodes, the regionalized stage is not relevant to this cancer.

The 5-year relative survival rate for multiple myeloma is as follows:

These statistics mean that a person with localized multiple myeloma is 75% as likely as someone without multiple myeloma to live for 5 years after receiving the diagnosis.

People who receive a smoldering myeloma diagnosis can live for years without any treatment. Additionally, beginning treatment early does not appear to affect the outlook.

The stage of multiple myeloma is among the factors that can affect a persons outlook.

Other factors include:

A small 2014 study involving 82 people with an average age of 61 years found that those with damaged kidneys had a median survival rate of 13 months, whereas those without kidney damage lived for an average of 41 months.

Additionally, changes in chromosomes and genetic abnormalities can affect a persons outlook. The specific chromosomal abnormalities that doctors consider high risk affect chromosomes 4, 14, 16, and 17.

The treatment for smoldering myeloma typically consists of watchful waiting, as this stage is slow growing.

Drug therapy for multiple myeloma consists of:

Other treatment options include:

Multiple myeloma can cause:

A doctor may recommend supportive therapies to help manage these side effects. These may include surgery to help support weakened bones and prevent fractures.

Learn more about the treatment options and how to manage the symptoms.

A person should contact a healthcare professional if they notice any symptoms of multiple myeloma.

After receiving a multiple myeloma diagnosis, a person may want to ask the following questions:

Multiple myeloma is a type of cancer that affects the blood. The outlook for people with multiple myeloma depends on the stage of the cancer at the time of diagnosis. It also depends on how well a persons kidneys are functioning and their age and overall health.

However, different treatment options are available. A person should talk with a healthcare professional about which treatment options would best suit them.

Excerpt from:
Outlook for multiple myeloma: Figures and factors that affect it - Medical News Today

Recommendation and review posted by Bethany Smith

On the day of his Year 10 school prom, as other students excitedly prepared for the big occasion, then 15-year-old Rian Harvey was sat in a ward of Royal Marsden Hospital, awaiting the stem cell transplant that would save his life after a leukaemia relapse.

Despite the hot weather on that day back in July 2015, his hospital room windows had to remain sealed shut, as even the smallest bug bite could have killed him due to his compromised immune system.

Six years on, he finds himself grateful that he relapsed when he did, with five years to build his immunity before the Covid-19 pandemic hit.

Blood cancer patients are one of the most vulnerable groups of people at risk of Covid-19, according to research, being 57 per cent more likely to suffer severe disease compared to other cancer patients.

Recalling his own experience, Rian, now 22, says: Its scary, you look at everything that person has gone through, they had blood cancer and then had a stem cell transplant, they have gone through all the stress of only to be taken by a pandemic that came out of nowhere.

I know the vulnerability that you are in for stem cell transplants, Ive been there myself. Your immune system cant take anything.

Despite the high risk these patients face, charities such as Anthony Nolan, which assist blood cancer patients with finding a stem cell match, were left out of the allocated government budget that was announced in March.

The cancellation of face-to-face fundraising and events, despite the increase in demand for services, have led their gross income to be down by an estimated 5.5m for 2021.

Henny Braund, chief executive of the charity, said people with blood cancer and blood disorders were heavily impacted by the pandemic and everyone who needs treatment and support must be able to access it without delay.

This budget does not address the pressure currently facing cancer services across the UK, he adds.

Stem cell transplants are carried out to treat conditions such as blood cancer. The process involves removing the healthy stem cells of one person and transferring them to another, provided they have a similar or identical special genetic marker called the HLA.

While this match is sometimes present between family members, it is not always the case, leaving patients in the UK reliant on the British Bone Marrow Registry to find a suitable match. The odds of a match are one in 1000.

One of Anthony Nolans primary roles is to encourage more people to put themselves on the registry so patients have an increased chance to find a match. This can be done via a simple cheek swap, which provides sufficient HLA data for the initial matching process.

Will Briant, 24, from London, donated stem cells in 2015 after signing up to be on the registry at university. I think it ultimately is a huge part of who I am now, he says. Its something that I look to in my darker moments and find great inner strength from.

The identities of donors and recipients remain anonymous to one another, but they are allowed to exchange letters after the transplant.

I was incredibly emotional when I got the letter, he adds. He made it clear that not only was I giving him the chance of time for himself, but it was also for all of his family and friends, he told me he had a very big family. Looking back now, at a time where we cant all be with our families, it just highlights just how important and valuable that must have been for him.

Apart from encouraging people to sign up to the registry, the money Anthony Nolan raises go towards funding research, offering support and information to patients and families as well as providing post-transplant-care. They have helped 18,000 people find a match.

Unfortunately, they are part of the 35 per cent of charities who used the furlough scheme offered by the government to curb the loss of income. To ensure their survival, 24 per cent of surveyed charities said they were letting furloughed employees return as volunteers.

Terence Lovell, chief engagement and marketing officer at Anthony Nolan, says: We still desperately need funds to continue our life-saving work through providing stem cells transplants and co-ordinating efforts across the NHS to ensure patients receive the care and support they need.

Despite the circumstances, Rian has decided to make the most of his time in lockdown. He regularly shares his experience fighting cancer on his social media platforms and is currently in the process of writing a book and producing a podcast to further share his message.

The cancer mill is still very much open for business and I am trying to push people, that have not necessarily been through what Ive been through, to be more positive and see the world the way that I do, he says, I wake up in the morning, open my front door, take a deep breath of fresh air and I think this is amazing because five years ago I couldnt even open a window in the hospital.

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How Covid-19 has disrupted efforts to care for blood cancer patients - The Independent

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Gene therapy has shown promise in recent years for treating a range of diseases, including sickle-cell anemia, hemophilia, various forms of inherited blindness, mesothelioma, and Duchenne muscular dystrophy. A new success story may soon be added to this list, with the publication yesterday of the outcomes of a clinical trial that used gene therapy to cure a rare immune system disorder in infants.

The study, described in the New England Journal of Medicine, was carried out by researchers from UCLA and Great Ormond Street Hospital in London over the course of five years, beginning in 2012.

Adenosine deaminase (ADA) is an enzyme found in a type of white blood cell called lymphocytes, which are primarily active in the brain, GI tract, and thymus gland. Lymphocytes make antibodies and attack infected cells, so theyre pretty crucial to the immune system.

ADAs job is to convert a molecule thats harmful to lymphocytes into a non-harmful version of itself. If ADA cant work its magic, that molecule starts to build up in lymphocytes, becoming toxic and ultimately killing the cellsand leaving the immune system virtually defenseless, highly vulnerable to invaders like viruses and bacteria.

Mutations in the ADA gene mean the body doesnt make enough of the enzyme to successfully do its job. This deficiency of ADA leads to a condition called severe combined immunodeficiency (SCID). Those suffering from SCID can not only get sick very easily, but conditions that would be neutralized by a normal immune system quickly become deadly for them.

SCID was more commonly known as bubble boy disease after David Vetter, a boy born in Texas in 1971, spent 12 of his 13 years of life enclosed in a plastic bubble to protect him from germs.

About 20 different genetic mutations can cause SCID; ADA-SCID refers to immunodeficiency caused by lack of the ADA enzyme: severe combined immunodeficiency due to adenosine deaminase deficiencya bit of a mouthful. The worst part of ADA-SCID is that it occurs in babies; most are diagnosed with the condition before theyre even six months old, and without treatment they typically dont live past age two.

ADA is rare, estimated to occur in about 1 in 200,000 to 1,000,000 newborns worldwide; both the mothers and the fathers ADA gene must have mutations for the child to end up with this condition.

The first step in the gene therapy treatment was to collect hematopoietic stem cells, which are those that manufacture blood cells, from the patients. The researchers then inserted an intact copy of the ADA gene into the stem cells using an RNA virus called a lentivirus (the most well-known lentivirus is HIV).

The altered cells were re-injected into the patients, where they started producing ADA normally, yielding healthy immune cells.

Out of 50 total patients30 in the US and 20 in the UKwith ADA-SCID, 48 appear to have been rid of their condition thanks to the gene therapy, with no complications reported. The two patients who didnt have success with the therapy went back to traditional treatment methods, and didnt experience any adverse effects as a result of having tried the therapy.

If, or hopefully when, gene therapy becomes the go-to treatment for ADA-SCID, it will be a welcome reprieve from traditional options, which are neither pleasant nor cheap: patients need weekly injections of ADA until a bone marrow transplant can be done, and absent a donor, they must consistently receive injections, take antibiotics, and undergo antibody infusions for life.

If approved in the future, this treatment could be standard for ADA-SCID, and potentially many other genetic conditions, removing the need to find a matched donor for a bone marrow transplant and the toxic side effects often associated with that treatment, said Dr. Claire Booth, co-author of the study and a consultant in pediatric immunology and gene therapy at Londons Great Ormond Street Hospital.

Theres no mention of the cost of the therapy, nor whether this could be a prohibitive factor to making it a viable option. Nonetheless, the study is encouraging not just for its potential to revolutionize treatment of ADA-SCID, but as a harbinger for the promise of gene therapy for a multitude of genetic conditions.

People ask us, is it a cure? Who knows long term, but at least up to three years, these children are doing well, said Dr. Stephen Gottschalk, who was not involved in this study but performed a similar gene therapy on kids with SCID at St. Jude Childrens Research Hospital in Memphis. The immune function seems stable over time so I think it looks very, very encouraging.

Image Credit: liyuanalison from Pixabay

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How One Round of Gene Therapy Fixed 48 Kids' Immune Systems - Singularity Hub

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Dr Pria Suchak, 31, initially registered with blood cancer charity DKMS last July, when she was inspired by a message on social media.

Every 20 minutes someone in the UK is diagnosed with a blood cancer those that affect the body's bone marrow, blood or lymphatic system - such as leukaemia, myeloma or lymphoma.

Yet, only two per cent of the UK population are registered as potential blood stem cell donors.

Pria said: My friends nephew had leukaemia, so she was using her Facebook page to encourage strangers to sign up him.

"Her nephew is of mixed heritage - half Chinese and half Caucasian. So she was trying to encourage more people for minority ethnic communities to sign up.

"I wanted to help give someone a second chance of life, so I signed up with DKMS, and my husband registered at the same time.

Patients from black, Asian or other minority backgrounds have a 20 per cent chance of finding the best possible blood stem cell match from an unrelated donor, compared to 69 per cent for northern European backgrounds.

Pria ordered a home swab kit in July 2020 and was contacted by DKMS just five months later, informing her that she was a potential match for a stranger in need of a lifesaving blood stem cell transplant.

The mum-of-two said: I received a call from a lady at DKMS. She said I was extremely close to being a match, but there were also eight other people who were identified as possible matches too.

"A few weeks later, I received another call from DKMS saying that I was the best match out of the nine potential donors.

"I didnt expect that. As it was nine of us in total, you never expect you'll be chosen.

Following further tests and a medical examination, a date was set for Pria to donate her blood stem cells by peripheral blood stem cell collection (PBSC).

In the run-up to the procedure, donors are given a drug with the growth factor G-CSF to increase the number of stem cells in the blood.

Pria said: At the time I had so many things going on. We had just gotten past Christmas, both of my children had birthdays in January, and I was about to sit a final GP exam.

"DKMS were excellent and did their best to schedule my G-CSF injections the day after I sat the exam. Of course, they checked that this wouldnt impact the patient.

My actual donation was really nice, especially as there were other donors in the room at the same time donating for other patients.

"We all got on really well and chatted loads. The clinicians told us that we were the chattiest group they had ever had. Ive remained terrific friends with one of my fellow donors.

Because of the minimum two-year anonymity period in the UK, donors can only contact the patient anonymously, by letter or email.

Pria said: I dont know anything about my patient other than she is a woman. She really is a stranger, but I hope my blood stem cells help her to live a long life.

I strongly encourage people in Crawley to register with DKMS. By donating their blood stem cells, not only will you potentially help a stranger in desperate need, but you'll also help their family and friends by giving them more time together.

Crawley has a population of around 114,000 with 14 neighbourhoods, the largest inland town in West Sussex. Yet, just 865 residents have registered with DKMS.

On May 28, DKMS celebrates their day of awareness - World Blood Cancer Day. This May, the charity aims to register 2,000 new registrations (roughly one for every donor in the UK waiting) by the end of May 28.

If you are called upon to donate your blood stem cells it is because you are likely the patients best match.

There are two donation methods. Around 90 per cent of all donations are made through a method called peripheral blood stem cell (PBSC) collection.

This method is very similar to giving blood. It involves being connected to an apheresis machine. Apheresis means 'to separate'.

This machine separates blood being taken from one of the donor's arms, and separates the blood stem cells from it. The donor's blood is then returned to them through their other arm. This is an outpatient procedure that is usually completed in four-to-six hours.

In just ten per cent of cases, donations are made through bone marrow collection. Bone marrow is taken from the pelvic bone under general anaesthetic, and this lasts around an hour.

DKMS need blood stem cell donors from all backgrounds. If you are aged between 17-55 and in good general health, you can support Gareth and the other 2,000 people in need of a lifesaving blood stem cell transplant by registering online at http://www.dkms.org.uk/register-now for your home swab kit.

By registering, you'll join a group of over 840,000 other DKMS lifesavers-in-waiting, ready to make a difference by giving someone a much-needed second chance of life.

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Crawley GP urges residents to save the life of a stranger - Crawley Observer

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CLEVELAND When 61-year-old Ken Anderson was diagnosed with Multiple Myeloma 3 years ago, he didnt know what to expect.

It kind of hits you. It hits you hard, he said. Its a blood cancer, and its in your bone marrow, and it degenerates your bones is what it does.

The cancer is incurable, but treatable.

You live with it and you have to have many rounds of chemotherapy to keep the myeloma at bay, said Dr. Ted Teknos, the president of University Hospitals Seidman Cancer Center.

With so many unknowns, the dad of 4 girls and grandfather of 2 knew one thing, he was going to fight.

You just have to look to the road ahead, he said.

For the past 3 years, that road has been filled with ups and downs and countless rounds of chemotherapy treatments and even a bone marrow transplant.

They give you your stem cells back and those regenerate and lasted for about 6 months, and then there was a relapse, said Anderson.

Through it all, he remained hopeful for a medical breakthrough. He read about the research and followed up on the results of clinical trials in something called CAR T therapy.

I didn't know how far out that would be. It didn't say how far out it was. It sounded, to me, something like 10 or 20 years.

But it wasnt 20 years, the FDA approved CAR T therapy for Multiple Myeloma patients, and University Hospitals is the first in Ohio to treat patients with it. Anderson, who is from Kirtland, is the first patient in Ohio to receive it.

These treatments, now, are available for those that have run out of options, said Dr. Teknos.

Dr. Teknos compared the treatment to something straight out of a science fiction movie.

In essence, its like a heat-seeking missile for the cells to go find the cancer and eradicate it, he said.

It works by taking a patients own white blood cells, genetically modifying them in a lab and then infusing them back into their body so the patients cells can fight off the cancer cells.

They will engineer them to attack my cancer cells, said Anderson.

Dr. Teknos calls it living therapy.

You're taking living cells out of a patient, you're modifying them, and then you're growing them up in the lab and then re-infusing them back into the patient, he said. It's their own cells that have been modified and fight the cancer.

Dr. Teknos said in clinical trials, about 75% of Multiple Myeloma patients had a response to therapy, and in 1/3 of patients, their cancer went away.

Its really a game changer, said Dr. Teknos. There are patients who literally had weeks to live and then a year and a half later, have no cancer at all.

Andersons cells are currently in the lab. He will receive his infusion next month. He is cautiously optimistic that the next stop on his journey will have him feeling better.

I won't have to be on the chemo anymore, so I'm just back to feeling like myself would be would be really exciting, he said. People who are out there and diagnosed with this, with this disease, know that we are on the cusp of some big things here in the treatment of it, and this is a huge advance.

While Anderson is currently fighting Multiple Myeloma, University Hospitals is also offering a new CAR T cell therapy treatment for patients diagnosed with Diffuse Large B-Cell Lymphoma.

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University Hospitals treats first cancer patient in Ohio with "game changing" CAR T therapy - News 5 Cleveland

Recommendation and review posted by Bethany Smith

Genetic factors appear to affect the risk posed to female athletesfor getting certain kinds of sport-related injuries, a new study suggests.

Scientists found that female athletes fortunate enough to be resistant to muscle dislocation are more prone to getting fatigue fractures.

The researchers said they discovered through their analysis that the genetic factors that help build tolerance against muscle fragmentation and fatigue fractures do not work at the same time.

The finding is expected to lead to the development of new methods for injury prevention based on patients genetic conditions.

However, that correlative relationship does not appear to exist in men.

Women have fewer proteins in their bones and muscles, and the differences come to the surface more easily, said Eri Miyamoto, an assistant professor of genetics at Juntendo University.

Miyamoto and her colleagues examined the genes of 1,667 male and female athletes primarily in their 20s and 30s. They compared their genetic makeup with their records of fatigue fractures, muscle dislocation and other kinds of myopathy, or muscle tissue problems.

The results revealed no fewer than 17.8 percent of women marked by a certain genetic composition had developed stress fractures, while the ratio of myopathy was as low as 9.9 percent for the group.

About 80 percent of the athletes had that genetic feature, which is associated with proteins that constitute bones and muscles.

Researchers found those athletes had lower bone density and more flexible muscles.

Women with another genetic element developed myopathy and fatigue fractures with a probability of 18.6 percent and 9.0 percent, respectively. This marked a sharp contrast to the results for the other group.

However, no significant differences in the development risks from the disorders were found among the male athletes, according to the study.

The teams findings were published in March in the online edition of the U.S. sports medical journal Medicine & Science in Sports & Exercise, available at: https://journals.lww.com/acsm-msse/Abstract/9000/Female_Athletes_Genetically_Susceptible_to_Fatigue.96070.aspx

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Female athletes at risk of bone fracture resistant to muscle injury : The Asahi Shimbun - Asahi Shimbun

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Refinery29

If there's one thing quarantine has shown us, it's that at-home bang trims, root touch-ups, and DIY blowouts aren't as daunting as they seemed in those blissful pre-COVID days. And, now that you're a total hair pro, it only makes sense to keep the new at-home grooming going even if you're fully vaccinated. An easy and affordable way to do that is by stocking up on the essentials during Ulta Beauty's most hair-raising sale of the year, the Gorgeous Hair Event.For the uninitiated, the sale is similar to the iconic 21 Days Of Beauty promo instead, this time around the deal spotlight is shining on your locks. From now through May 29, you can expect half-off daily deals on hair tools, intensive treatments, styling products, and much more. Each promo only lasts for 24 hours, so shop quickly and confidently! To assist, we've corralled every major hair deal happening this month ahead. At Refinery29, were here to help you navigate this overwhelming world of stuff. All of our market picks are independently selected and curated by the editorial team. If you buy something we link to on our site, Refinery29 may earn commission.Living Proof Full Thickening MousseDate: May 11Also On Sale: InStyler Airless Blowout Revolving Styler, NioxinLiving Proof Full Thickening Mousse, $, available at Ulta BeautyKitsch Satin PillowcaseDate: May 12Also On Sale: Viviscal, KeracolorKitsch Satin Pillowcase, $, available at Ulta BeautyMadison Reed Radiant Hair Color KitDate: May 13Also On Sale: Color Wow Root Cover Up, Voir Select Care and Treatment, Manic PanicMadison Reed Radiant Hair Color Kit, $, available at Ulta BeautyCHI for Ulta Beauty Titanium Temperature Control Hairstyling IronDate: May 14Also On Sale: Curlsmith, Matrix Biolage ColorBalm Color Depositing Conditioner, Gold 'N Hot, BiosilkChi CHI for Ulta Beauty Pink Titanium Temperature Control Hairstyling Iron, $, available at Ulta BeautyPureology Hydrate Sheer Shampoo & ConditionerDate: May 15Also On Sale: Joico Masks, American CrewPureology Hydrate Sheer Shampoo, $, available at Ulta BeautyPureology Hydrate Sheer Conditioner, $, available at Ulta BeautyDrybar The 3-Day Bender Digital Curling IronDate: May 16Also On Sale: Living Proof Restore Perfecting Spray, Alterna My Hair My Canvas Styling, Pravana Intense Therapy and Color Protect Shampoo & ConditionerDryBar The 3-Day Bender Digital Curling Iron, $, available at Ulta BeautyRevlon XL Hair Straightening Heated Styling BrushDate: May 17Also On Sale: Invisibobble, AG HairRevlon XL Hair Straightening Heated Styling Brush, $, available at Ulta BeautydpHUE ACV ProductsDate: May 18Also On Sale: Paul Mitchell 1" Neuro Halo Styling Iron with Neuro Protect, SebastiandpHUE Apple Cider Vinegar Hair Rinse, $, available at Ulta BeautyOuidad (Select Products)Date: May 19Also On Sale: Klorane, WellaOuidad VitalCurl+ Tress Effect Styling Gel, $, available at Ulta BeautyFekkai (Select Technician Color Products)Date: May 20Also On Sale: Lime Crime Hair Color, Rusk Puremix MasksFekkai Technician Color Treatment Masque, $, available at Ulta BeautyTgin (Entire Brand)Date: May 21Also On Sale: Matrix Total Results Toning Shampoos, Conditioners & Masks, Select Biolage Jumbos, Bed Head Mess Behave Deep Hair Wavertgin Honey Miracle Hair Mask Deep Conditioner, $, available at Ulta BeautyAlterna (Select Caviar Anti-Aging)Date: May 22Also On Sale: Bio Ionic 10X Pro Straightening & Styling Iron, Kenra Professionals Leave-InsAlterna Caviar Anti-Aging Replenishing Moisture CC Cream, $, available at Ulta BeautyWet Brush Pro Shine Enhancer BrushDate: May 23Also On Sale: Bumble and bumble Thickening Full Form Soft Mousse, Bondi Boost, Paul Mitchell, Captain Blankenship Shampoo & ConditionerWet Brush Pro Shine Enhancer Brush, $, available at Ulta BeautyT3 Convertible CollectionDate: May 24Also On Sale: IGK Treatment Masks, Tula Balanced Beauty Gummy Vitamins,T3 Convertible Base, $, available at Ulta BeautyChi (Entire Brand)Date: May 25Also On Sale: Chi G2 Ceramic & Titanium Hairstyling Irons, Matrix Biolage Hydrasource TreatmentsChi Silk Infusion Silk Reconstructing Complex, $, available at Ulta BeautyNatureLab Tokyo Shampoos and ConditionersDate: May 26Also On Sale: Curlsmith Curl Quenching Conditioning Wash, Sexy HairNatureLab Tokyo Perfect Smooth Shampoo, $, available at Ulta BeautyNatureLab Tokyo Perfect Smooth Conditioner, $, available at Ulta BeautyTangle Teezer The Scalp Exfoliator & MassagerDate: May 27Also On Sale: Punky Colors, Toppik Root Touch-Up Spray, Eva NYC Spectrum Far-Infrared Hot ToolsTangle Teezer The Scalp Exfoliator & Massager - Pink, $, available at Ulta BeautyFlora & Curl (Entire Brand)Date: May 28Also On Sale: DevaCurl, Keratin Complex, Elchim Healthy Ionic Hair Dryer, Design Essentials Natural Almond & Avocado Curl Enhancing MousseFlora Curl Organic Rose & Honey Leave-In Detangler, $, available at Ulta BeautyHot Tools 24K Gold Charcoal Infused One-Step BlowoutDate: May 29Also On Sale: It's a 10, Bosley, We Are Paradoxx (Select Products), Olivia Garden Ceramic+Ion Speed XL Round BrushesHot Tools 24K Gold Charcoal Infused One-Step Blowout, $, available at Ulta BeautyLike what you see? 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Female Receding Hairlines, Explained: Here's What Experts Say About the Condition, Including How to Reverse It - Yahoo Lifestyle

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Side effects can be a powerful deterrent stopping people from getting vaccinated. To address this issue, in 1991, a group of scientists in Minnesotaat the Department of Veterans Affairs and the Mayo Clinicdevised an experiment to see just how frequent these unpleasant reactions were.

The study involved more than 300 veterans over the age of 65 who were given either a flu shot followed two weeks later by a placebo injection of salt water, or a placebo shot followed two weeks later by the real vaccine.

When the researchers unblinded the study to see who received the vaccine versus the placebo, the side effects were split equally between the two groups, says Robert Jacobson, medical director for the population health science program at the Mayo Clinic. About five percent said they got sicker than they ever had been in their entire life, says Jacobson. Half of these people had received the placebo and yet they complained of the worst headaches, or worst fever, of their lives. The take-home message here, says Jacobson: Its easy to confuse an allergic reaction with nervousness or emotions or even stomach upsets from anxiety.

Recent studies show some side effects, even ones from the COVID-19 vaccines, arent due to the shots at all, but to our own fears. Weve seen this in the military, when young recruits, who think they can tolerate anything, faint when they get the injections, because their body overreacts, says Jacobson.

Its a lesson that may be useful to medical professionals, who can reassure patients that most side effects are normal and predictableand may not even be caused by the shot. Case in point, in studies of the Pfizer/BioNTech vaccine, 23 percent of people aged 16 to 55 who received the placebo complained of fatigue after their second jab, and 24 percent noted headaches.

Studies do suggest that up to seven out of ten people getting their second shot have some type of reaction. Some feel a soreness at the injection site on their arm. They may experience itching or hives, or a range of flu-like symptoms, such as chills and fever, headaches, or debilitating fatigue, that can leave them bedridden for a day or two. Still, its important to put these side effects in perspective, says Jacobson, because these are mild, temporary, and transient reactions that disappear within a few days.

In the case of the authorized COVID-19 vaccinesPfizer, Moderna, and Johnson & Johnsonall contain a genetic blueprint for manufacturing spike proteins, which sit on the surface of the coronavirus and enable it to infect human cells. When human cells receive these instructions, they churn out copies of spike protein. But since the cells make only a piece of the virus, and not the whole pathogen itself, we dont get sick. But while the foreign spike cant cause disease, it can activate a two-step immune responseexactly as it is supposed to do.

The immediate physical reaction to the COVID-19 vaccine is caused by the innate immune system. When a person receives a shot, a flurry of white blood cells called macrophages and neutrophils arrive at the injection site and begin producing chemicals called cytokines. This response triggers a wide range of symptoms, from inflammation and swelling at the injection site to fever, fatigue, and chills.

As a result, side effects are a natural reaction to vaccination. This responsecalled reactogenicitymeans the vaccines instigate a strong, initial immune response and trigger a wide range of symptoms. Out of about 3,600,000 vaccinated people who participated in a survey in February, approximately 70 percent reported pain at the injection site, 33 percent felt fatigued, 29 percent suffered headache, 22 percent had muscle pain, and 11 percent experienced chills and fever after their first shot of a COVID-19 vaccine. The symptoms were even more pronounced after the second dose. Still, the innate immune response is short-lived, lasting only a few days.

But not everyone experiences side effects after a COVID-19 vaccine. Some feel fine after both doses. Scientists dont really know why, says Sujan Shresta, an immunologist at the Center for Infectious Disease and Vaccine Research at the La Jolla Institute for Immunology, in California. But its not a surprise that each person mounts the immune response differently.

Several factors can contribute to this wide variation. Women, for example, typically have stronger immune reactions than men, which may be part of what makes them more prone to suffering from side effects from the shots.

We all have our own individualized immune system, says John Wherry, director of the institute for immunology at the University of Pennsylvania, in Philadelphia. Its almost like our own immune fingerprint thats driven by genetics, gender, diet, our environment, and even our life history, which are the things our immune system has been exposed to in the past and has been trained to respond to over the years.

Even if you dont have an unpleasant reaction, the vaccines are still doing their job, because the real work of the immune systemand of the vaccinestakes place during the second, or adaptive, phase of the immune response. During this phase, the spike protein generated via the vaccine trains the B-cells to produce antibodies that match the virus, and the T-cells to seek-and-destroy infected cells. But it takes days to weeks to provide this long-lasting protection against the virus.

This is also the reason why people often have more rigorous reactions to the second shot. Three weeks after the first shot, the immune system has already been primed, and the B-cells and T-cells are ready to fight. When the second shot is delivered, both the innate and adaptive systems respond.

Still, we dont really know if having a serious response to the vaccines is a measure of the strength of the immune system. We also dont know if it means that someone who doesnt have a strong innate response will be more vulnerable to COVID or more resistant. We really dont have any data in the field on thiswhether a person with strong side effects would have a more severe COVID infection and vice versa, says Wherry.

In a February study that looked at the data from the first 13.7 million COVID-19 vaccine recipients, the Centers for Disease Control and Prevention found that nearly 80 percent of the people reporting reactions were female, even though only 61.2 percent of the injections had been given to women. In a similar vein, the CDC reported that all anaphylactic reactions to the Moderna shot have been in women; 44 of the 47 people whove had these reactions to the Pfizer injection were female.

The majority of people who have experienced the severe blood clotting issues with the J & J vaccine, and also the AstraZeneca vaccine in Europe and the United Kingdom, have been women. There has been speculation about hormones playing a rolewhich is always the first culprit thats looked at when you see a major sex difference, says Penns Wherry.

Several other factors may also contribute to this gender imbalance. Women also seem to have a more robust immune system, both in their innate responses and in their adaptive immune reactions. Females mount a stronger antibody response than males but its a double-edged sword because this is why women have more auto immune disease than men, says Shresta of the La Jolla Institute for Immunology.

Other studies have shown that a womans response to half a dose of the influenza vaccine was the same as mens full dose, so females might not need full doses of the COVID-19 vaccines. We have this idea that one size fits all, but this may be part of whats contributing to the higher rate of reactions among women, says Rosemary Morgan, a scientist specializing in gender research at the Johns Hopkins Bloomberg School of Public Health. There is also a behavioral componentwomen are more likely to visit the doctor and to be more proactive about reporting unpleasant symptoms.

But side effects and adverse eventswhich often get conflatedare not the same, says Wherry. Side effects are pretty commonoccurring maybe 50 to 70 percent of the time. But adverse events are rare and unexpected, like the clotting disorders.

Immediately after injection, about two to five people per million experience anaphylaxis, a severe allergic reaction that causes a dramatic drop in blood pressure and difficulty breathing. But even this is easily treatable with an EpiPen and antihistamines, which is why everyone is asked to stick around for 15 minutes after their COVID-19 shots.

The blood clots associated with the Johnson & Johnson vaccines, that have occurred within six to 13 days of receiving the shot, can be dangerous and even life threatening. But the incidence is quite low; there are only 23 confirmed cases out of 8.4 million doses of the vaccine.

This is very rare, says Ofer Levy, director of the precision vaccines program at Boston Childrens Hospital and a professor of pediatrics at Harvard Medical School. The risk of getting COVID and possibly dying is much higher than getting blood clots from the vaccines.

There is some worry that there may be other adverse effects that have gone largely unreported.

The three COVID-19 vaccines that have been authorized in the United States have been tested on tens of thousands of people in clinical trials, and manufacturers were required to follow up with at least half the vaccine recipients for two months or more after they received both shots. But now that more than 116 million Americans have been fully vaccinated, rare side effects that dont show up in smaller human clinical trials can emergewhich is why surveillance systems are important.

Here in the U.S., we have a patchwork of systems: the Vaccine Adverse Event Reporting System (VAERS), the Vaccine Safety Datalink, and the CDCs new phone-based tracking program, v-safe.

All of these have limitations, including that someone has to suspect these health outcomes are related to vaccination and go to the trouble of filling out the form, says Katherine Yih, a biologist and epidemiologist at Harvard Medical School, specializing in infectious diseases, immunization, and vaccine safety monitoring. We have a vigorous surveillance system in place. But we cant be sure its picking up everything.

Whats more, these incidents only show correlation. In other words, if someone died or had a stroke after getting vaccinated, physicians dont know if it was triggered by the shot. Only further study can reveal that.

The swift identification of the rare blood clotting disorder related to the J & J vaccine was reassuring. Initially, six cases were reported, prompting the FDA and the CDC to temporarily halt its use. When the CDCs Advisory Committee on Immunization Practices met in late April to determine the vaccines fate, 15 cases had been detected out of seven million people who had the shot. The discovery of that association with the J & J vaccinewhich is very rareis a real demonstration of how good our safety program is, says the Mayo Clinics Jacobson. At this point in the pandemic, a risk of less than three per million should not enter into our calculus of how to proceed.

Continue reading here:
Why vaccine side effects really happen, and when you should worry - National Geographic

Recommendation and review posted by Bethany Smith

Lupus is unpredictable, triggering different symptoms in different patients. A chronic illness, it can even attack many different parts of the body.

The condition is an autoimmune disease, which means that a persons immune systemthe body system that usually fights infectionsattacks healthy tissue instead. It can cause inflammation and pain anywhere in a patients body.

Because the illness can look different in every patient, it can go undetected and undiagnosed for years.

The Lupus Foundation of America estimates that 1.5 million Americans and at least 5 million people worldwide have some form of lupus. There are four forms in all: systemic lupus erythematosus (SLE), lupus confined to the skin, lupus caused by some prescription drugs and a rare lupus that affects infants of women with the disease.

Despite ongoing research into how the disease manifests in patients and what causes it, scientists and other experts sometimes describe the disease as cruel mystery which is to say, they still have a lot to learn about it.

There is a lot about lupus that is still a conundrum to us, said Dr. Karen H. Costenbader, a professor of medicine at Harvard Medical School and director of the Lupus Program at Brigham and Womens Hospital in Boston.

Its treatable, but it can still be very painful and challenging to overcome, said Costenbader, who is also chair of the Lupus Foundation of Americas Medical-Scientific Advisory Council.

May 10 is World Lupus Day, and we recently caught up with Costenbader to learn more about the disease.

This conversation has been edited and condensed for clarity.

CNN: What does it mean that lupus is an autoimmune disease?

Dr. Karen Costenbader: It means the immune system is attacking healthy tissue inside the body instead of infections. With lupus, patients make antibodies against any different number of organs, cells, internuclear proteins and intercellular particles.

Any organ system can be involved. As a result, causes a wide range of different symptoms that can come and go over time and vary from person to person. These include extreme fatigue, pain or swelling in the joints, low-grade fevers and damage to internal organs like the kidneys, heart and lungs. Thats what makes it so difficult to diagnose and treat.

CNN: If lupus presents so differently in every patient, how do experts diagnose it?

Costenbader: The one thing all lupus patients have in common is that they have dealt with chronic pain and discomfort for a while. When lupus first presents, symptoms can include fever, joint pain and rashes. These symptoms are similar to symptoms of infections and other autoimmune diseases such as rheumatoid arthritis, so a diagnosis can be tricky. It takes an astute clinician and expert. Sometimes it also takes time(recent) research indicates it can take as long as (almost seven) years. As soon as symptoms involve three or more different organ systems, clinicians should start thinking about lupus.

CNN: What is the treatment? Does hydroxychloroquine, the drug that we learned doesnt work for Covid-19, really work on lupus?

Costenbader: Thankfully, today we have more and more treatments through clinical trials. Historically, though, the treatment has been hydroxychloroquine, the same drug that was touted for a while as a potential treatment for Covid-19. It turns out the drug doesnt work for Covid, but it does work well for lupus.

Last year during the pandemic, when people thought it worked for Covid, we had huge shortages of hydroxychloroquine, which was a problem for our patients. Anti-malaria drugs also help stabilize the disease, and steroids work, too. Steroids have a lot of side effects such as weight gain and glaucoma. The goal of some of the new treatmentstheyre all immunosuppressantsis to keep organ involvement under control.

CNN: Are men or women more likely to get lupus?

Costenbader: Lupus mainly strikes femalesnine of 10 people with lupus are female. It also commonly starts between ages 15 to 44 years. We dont know why this happens; it is one of the conundrums. A lot of autoimmune diseases have sex predilections. With lupus, incidence picks up during menstruation, decreases after menopause. Men who have two X chromosomes also have a high prevalence of lupus, so perhaps X chromosomes have something to do with it. This is one of the aspects of the disease were still studying.

CNN: What are some of the other conundrums experts have identified about lupus?

Costenbader: Lupus is two to three time more common among people of colorAfrican Americans, Hispanics/Latinos, Asians, Native Americans, Alaska Natives, Native Hawaiians and other Pacific Islandersthan among people of European ancestry. The real burden in this country is in young African American women. Lupus is one of the top 5 causes of death of young African American women. Were just starting to have large enough populations involved in the studies to see that the genetics involved with patients may be different.

CNN: Is lupus contagious?

Costenbader: Its not contagious. But it does run with other autoimmune diseasespeople who have lupus usually have family members with rheumatoid arthritis and other autoimmune diseases. For people who have lupus, relatives have a 5 to 20% chance of also developing it. Experts think it may develop in response to certain hormones (like estrogen) or environmental triggers.

An environmental trigger is something outside the body that can bring on symptoms of lupus or make them worse. For instance, we know smoking increases the risk of lupus and oral contraceptives might trigger it as well. Obesity, stress and post-traumatic stress disorder can be triggers. We know that if people have a genetic predisposition and they are in the wrong environment, their inflammatory pathways get triggered, and they are at risk.

CNN: What is the outlook for someone with lupus?

Costenbader: Lupus is not a death sentence. With the right meds, and the right physicians and caregivers involved, lupus patients can have kids, hold jobs, and lead productive lives like anybody else.

Visit link:
5 things to know about the 'conundrum' of lupus - ABC17News.com

Recommendation and review posted by Bethany Smith

Beef Central publishes an occasional summary of appointments, departures and achievements occurring across the red meat and livestock supply chain. Send details for entries to admin@beefcentral.com

After six years at the helm of Stockyards Kerwee feedlot on Queenslands Darling Downs, Steve Martin recently stepped down from his role as general manager of feedlot operations.

Steven Martin

While he has been active in industry circles in recent weeks, attending both the Australian Wagyu Association conference and Beef 2021, Mr Martin is yet to make a decision on his future path, short of telling Beef Central he is keen to remain within the beef industry.

Suffice to say he was observed having lengthy discussions with some large Wagyu supply chain players during the recent Wagyu conference.

Mr Martin joined Stockyard in 2015 and steered the company through a major expansion in 2017 that doubled the capacity of the Kerwee feedlot; and more recently the establishment of Stockyards Fullblood and purebred Wagyu breeding and backgrounding business.

Following his departure, the Stockyard Group has promoted George Lubbe as general manager of feedlot operations. Mr Lubbe has previously worked since 2017 as Kerwees assistant manager.

George Lubbe

A South African by birth, Mr Lubbe previously worked with large-scale Queensland egg producer Mclean Farms, and civil engineers Ostwald Brothers, as well as his familys cattle trading business based near Texas on the NSW/QLD border.

Another long-term Kerwee staffmember, Stevie-Lee Wayman, has been appointed assistant manager of Kerwee feedlot, moving up from her livestock supervisor role. Both she and George Lubbe bring a wealth of experience to their new positions.

Good feedlot managers and grainfed supply chain managers were in high demand and short supply across the industry at present, one of Australias most prominent ag recruiters told Beef Central recently.

Northern Territory Livestock Exporters Association chairman David Warriner has confirmed the commencement of the organisations new CEO, Tom Dawkins, in Darwin last week. Mr Dawkins has previously held a number of senior industry affairs roles with national agricultural bodies the Australian Livestock Exporters Council, the Cattle Council of Australia and most recently Australian Pork Ltd.

Tom Dawkins

He began his career as a livestock markets reporter with Rural Press/Fairfax newspaper Stock Journal in his home state of South Australia in 2006. He was appointed editor of the masthead in 2008, before transferring to the editors desk at Victorian stablemate Stock & Land in 2010.

Mr Dawkins follows in the footsteps of numerous well-known export industry figures who have served terms heading-up the NTLEA, including regular Beef Central contributor Dr Ross Ainsworth, Patrick Underwood, Lach Mackinnon, Kevin Mulvahil and Bernie Brosnan. Mr Dawkins immediate predecessor Will Evans, who is now the CEO of the NTCA, followed on in the NTLEA position after Stuart Kemp, who is now based in Queensland as Livestock Manager North with Australian Country Choice.

Tracey Hayes, the first female chief executive officer of the Northern Territory Cattlemens Association and the facilitator of the successful class action against the 2011 suspension of the live cattle export trade to Indonesia, was awarded the Beef Achiever Award at a Beef 2021 dinner hosted by Rabobank and Queensland Country Life in Rockhampton last week.

NTCA executive director Tracey Hayes addresses a function at the Australian Embassy in Jakarta.

Tracey is regarded as a senior business and industry leader, having also previously served as chair of the Darwin Waterfront Corporation and as a Member of the Order of Australia Honours Council. She is a director of the Cooperative Research Centre for Northern Australia, sits on the board of the Australia Indonesia Institute and is a member of the advisory board of the National Drought and North Queensland Flood Response and Recovery Agency.

Tracey is also small business proprietor, a long-term former Territory pastoralist, pilot and Justice of the Peace, and ran against NT chief minister Michael Gunner as a candidate for the Country Liberal Party for the seat of Fannie Bay in the NT election last year.

Asked to reveal something about herself in an interview with the NT Independent last year, she noted that at one point in time she was licenced to operate and use aeroplanes, trucks, cars, motorbikes, boats, firearms, forklifts, and S7 pharmaceuticals and chemicals. Not unusual for bush women she added. Keep an eye on Beef Central for an upcoming interview with Tracey Hayes.

The Beef Industry Achiever award was initiated by Beef Central publisher Jon Condon back in 1997.

Two new councillors elected to the Australian Lot Feeders Association board in November have been out and about around the industry, since the lifting of the COVID curtain.

Attending Beef 2021 last week, coinciding with the launch of ALFAs new community-facing website, grainfedbeef.com.au was newly-minted councillor Daryle Belford, from Whyalla feedlot, near Texas Qld

Pictured on ALFAs stand during Beef 2021 were new ALFA councillor Daryle Belford, chief executive Christian Mulders, CARS Road Safety Ambassador Judy Lindsay, and ALFAs Elise Mizzi. Click on image for a larger view

Mr Belford managed cattle properties in Central Queensland between 1990 and 2004 before moving into an agribusiness and financial advisory role based in Emerald up to 2009. He entered the feedlot industry in 2010 with NH Foods Whyalla yard, having worked in numerous parts of the business up to his current position of assistant general manager. He replaces Whyalla general manager Tony Fitzgerald on the ALFA board.

Also joining the ALFA council lineup this year is was Tom Green, from Thomas Foods Internationals Iranda Beef feedlot in South Australia.

Tom worked in North America on large-scale bull breeding properties before entering the feedlot industry in 2012 when he joined Teys Australia at Jindalee feedlot as a Graduate Manager before becoming the Livestock Manager followed by the Operations Manager.

In 2016 he joined TFI as operations manager at Iranda Beef before being appointed General Manager of Feedlot and Farming in 2018. Tom was awarded Young Lot Feeder of The Year in 2017 and a Nuffield Scholarship in 2019. Tom has been an external representative on ALFAs Feedlot Management committee during 2019 and ALFAs R&D committee during 2020.

Angus Australia has appointed Jake Phillips as the breed societys new Breed Development Officer.

Jake Phillips

Mr Phillips grew up in South Australia and has spent considerable time working across the beef supply chain in various positions throughout Eastern Australia. After uni he joined ABRI as a BreedPlan consultant, followed by four years with Meat Standards Australia based in Queensland in the areas of the integrity and adoption. More recently he has spent the past seven years working for Teys Australia at Naracoorte in South Australia in a variety of roles including livestock procurement, branded beef, QA management and livestock strategic operations. His most recent Teys role was as livestock strategic operations manager.

Jake and his wife run a small seedstock operation near Naracoorte, recently added Angus genetics to their herd.

In his role with Angus Australia, he will work alongside existing Breed Development Officer, Matt Reynolds, in the delivery of education and extension programs that assist Angus breeders with the utilisation of genetic improvement technologies to enhance the profitability of Angus cattle and beef across the beef supply chain. He will be regionally based, working from Naracoorte in South Australia.

Meat & Livestock Australias Man in Singapore, Dr Michael Patching, has moved on to establish his own industry consultancy.

Michael Patching

Dr Patching has worked as MLAs livestock services manager and more recently market development manager for the Asia Pacific region for the past two and a half years, after earlier acting as market development manager for in-country operations in Vietnam for three years.

A Murdoch University and University of Edinburgh trained veterinarian with masters degree qualifications in international animal welfare, ethics and law, Dr Patching has worked closely with Australian live exporters and Vietnamese importers over the past six years, during the period of dramatic volume growth, more recently adding Indonesia and other southeast Asian customer countries to his responsibility. Click here to read Beef Centrals report from an interview.

Dr Patching is now moving on to start his own consultancy business, Beanstalk Agtech, based out of Singapore, where he will focus on filling a gap in trade facilitation work for companies seeking to develop commercial agricultural and food market opportunities in the ASEAN region.

His replacement in the MLA Singapore role has not yet been announced.

Roger Desailly

The Australian Meat Industry Council has appointed Roger Desailly as the organisations new state manager for Queensland. He replaces Peter Talbot, who filled the role for the past two years.

In his role, Mr Desailly works principally with independent retail butchers across the state, but also has a watching brief across AMICs country processor, wholesaler and smallgoods manufacturer member network.

He is a former chief executive of the industrys major Beef Australia expos in Rockhampton, and most recently has worked in his own agribusiness consultancy after a period as state education and training manager for the Queensland Agricultural and Training Colleges organisation.

Mr Desailly is operating from AMICs Brisbane office in Red Hill, but travels widely across the state.

Laura Penrose

The formal launch of the Australian Wagyu Associations major ten-year Progeny Test Program (click here for earlier story) at the 2021 WagyuEdge annual conference recently has coincided with the appointment of AWAs first genetics improvement program manager.

Laura Penrose took up her new position in March. A Bachelor of Science graduate from the University of New England, Laura previously worked as a project assistant at UNE, and as a technical officer with the NSW Department of Planning, Industry and Environment.

She will be based out of AWAs Armidale office.

Queensland agribusiness and agtech pioneer, William Harrington was recently awarded a prestigious Fulbright academic scholarship, sponsored by the Food Agility CRC.

Mr Harrington is studying for his Food Agility PhD at James Cook University in Townsville. His thesis topic is measuring and quantifying the benefits of improved internet connectivity in regional and remote Australia and its effect on adoption of technology.

William Harrington

My PhD project aims to understand how farmers use the internet, including how often, what a farmers typical data usage is, and what types of websites and applications they use, he said. At this point in time there is very little, if any research in this area and I am excited to be able to contribute.

Growing up on a remote cattle station in the north west of Queensland, Mr Harrington was an early agtech pioneer, developing animal ID and iSee remote monitoring and W-Sky connectivity technologies, despite his remote location.

I have always loved being able to bring together my two passions, technology and agriculture, he said, after being awarded his scholarship. Improved connectivity can make a tangible difference to the lives of people in regional Australia and enables the adoption of new technologies that improve efficiency, production and environmental outcomes. There has been a lot of talk in the media about connectivity in regional Australia, and I decided to do a PhD to try and produce scientific data that can be used to guide decision and policy makers.

Mr Harringtons Fulbright scholarship will involve study in the US, where he will be based at Ohio State University for four months, studying the US approach to rural broadband.

The US is a world leader in this area and I would like to use the opportunity to learn from institutions such as the Federal Communications Commission, US Department of Agriculture, and academic organisations such as the Perdue Centre for Regional Development, he said.

On completing his PhD, Mr Harrington plans to remain in regional Australia and continue to research topics that are related to farmers, connectivity and technology adoption.

Dr Robyn Cleland was recently appointed as the new chief environmental biosecurity officer with the Department of Agriculture, Water and the Environment in Canberra.

Deputy secretary of biosecurity and compliance, Andrew Tongue, said Dr Cleland brought a wealth of experience and expertise to the role, having held senior leadership roles in the Australian Public Service for more than a decade.

Dr Cleland has worked across the portfolios of agriculture, health and environment, working in policy, compliance and regulation.Her scientific expertise spans biosecurity, plant health, biotechnology, food, ecology and agriculture.

The role of the chief environmental biosecurity officer is to liaise between government, industry and the community to raise awareness and build Australias capacity to manage biosecurity risks.

This is a hugely important role, charged with protecting Australias unique wildlife, our way of life, and our status as a clean, green exporter of high-quality food, Mr Tongue said.

Dr Clelandhas worked with State and Territory jurisdictions, NGOs and the community across a number of national regulatory schemes and has extensive experience in engaging with diverse stakeholders across contentious issues. Before she joined the APS, she was a research scientist at the University of Cambridge, the University of Sheffield and the Australian National University.

Agtech developer Jock Lawrence last month received the Victorian Young Achiever Award in Small Business.

Mobble founder, Jock Lawrence

The 28-year-old co-founder of the Mobble farm management software platform, Mr Lawrence received the award at a gala dinner in Melbourne. The Young Achiever awards began in 2012 with the purpose of promoting, acknowledging and encouraging the positive achievements of young people in industry up to 30 years of age.

In his acceptance speech, he thanked the farmers who had supported his business through the years, as well as his team at Mobble for their commitment to the companys vision of making life easier for farmers and simplifying farm management.

He noted that farmers are the reason we get out of bed every day, to make their lives easier and more successful. Mobble is a centralised livestock farm management software platform, used by more than 500 farms across Australia and New Zealand, currently managing more than two million stock units across those properties. The software puts a focus on connection and simplicity by farmers for farmers.

Beau North from Sarina, Queensland was recently announced as the Lachlan Hughes Foundation 2021 Foundation Scholar. An independent selection panel identified Beau from a strong field of applicants from across Australia.

With his partner Katherine Fausett, Beau has recently purchased a grazing property in the Sarina area, which is the focus of his project for the scholarship. As a new landholder, Beau recognises that he has a duty of care to protect and enhance water quality but also mitigate erosion on his property.

He is embarking on a journey to retire areas on the property, which were farmed for sugar cane for 80 years, to a mixed species of perennial pastures and beneficial vegetation for grazing, diversity and ecological value. Using regenerative agricultural principles where possible and practical, Beau aspires to enhance water cycles on farm, slowing the runoff and returning it to the soils to support fertility and drought resilience.

The scholarship mentoring will provide access to expertise and training to refine his current property plan and support him on the ground as he furthers his long term dream of building a beef grazing business.

Beau knows first-hand how hard it is to get a start in the beef industry as grazing land has been expensive in his region. He and his partner have been leasing country for his small cattle herd for the past eight years. Learning a trade as an electrician has helped him build up the capital to help pursue his passion for grazing and allowed him to finally purchase his first property.

Beau also hopes that the scholarship will benefit other farmers, through growing the regen community in the wider region.

Beau follows in the footsteps of inaugural scholar Jack Groat, who shared his experiences at the MLA BeefUp event in Roma earlier this year. Jack spoke on how his journey to learn more about regenerative agriculture has been rapidly enhanced through the scholarship.

The opportunity provided by the Lachlan Hughes Scholarship has allowed me to give back to the regenerative agriculture movement as well. It has improved my focus on hydrating our landscapes through a multi lever approach, implementing rotational grazing, contours and leaky weirs to reduce run-off, soil erosion and keep ground cover better for the environment and better for business, Jack said.

The Darling Downs lost one of its greatest export superstars in April. Geraldine Doumany, or Gerry as she was known, spent her career assisting businesses experience international trade success and she was one of the regions strongest advocates for creating a thriving export community.

Raised on a farm near Cunnamulla in Western Queensland, and always maintaining a connection to regional and rural communities, Gerry held senior roles with government agencies Austrade and Trade and Investment Queensland. She also worked within the private sector, and most recently was leading Toowoomba and Surat Basin Enterprises (TSBE) export initiatives.

Gerry was adept at helping businesses understand how to export and how to navigate the many challenges that every export journey entails.

I remember many times hearing phone calls from panicked local exporters with Gerry calmly stepping in to locate products stuck on a wharf in China or fixing export documentation or using her many international contacts to smooth the way for Darling Downs products to reach overseas markets, TSBE CEO, Ali Davenport said.

Export is incredibly complicated, but Gerry was brilliant and could solve just about any export problem.

Last year, the Toowoomba Chamber of Commerces Business Excellence Awards introduced the Gerry Doumany Export Award, which was won by local beef export supply chain Mort & Co.

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People on the Move: Appointments, retirements, achievements - Beef Central

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SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that new data from clinical trials of 19 approved and investigational medicines across 20 cancer types will be presented at the 2021 ASCO Annual Meeting, which will be held June 4-8, 2021. A total of 132 abstracts that include a Genentech medicine will be presented at this year's meeting. These data advance oncology by showing the importance of making patient-centric treatment decisions and providing tailored medical care based on specific cancer types.

We will be presenting data from across our diverse oncology portfolio that has the potential to help more people living with many types of cancers, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. We are particularly excited about our compelling immunotherapy data in lung cancer, which may provide new hope for patients with earlier stage disease.

Focusing on earlier treatment and targeted lung cancer care

Positive results from the Phase III IMpower010 study will be presented that show Tecentriq (atezolizumab) improved disease-free survival (DFS) in people with resected early-stage non-small cell lung cancer (NSCLC) compared to best supportive care - a first in cancer immunotherapy. This advance is significant, as half of all people with early-stage lung cancer today still experience a recurrence following surgery; therefore, treating lung cancer early, before it has spread, can provide the best opportunity for a cure. Additionally, updated data for GavretoTM (pralsetinib) in patients with advanced RET fusion-positive NSCLC, including in patients who are treatment nave, will be reported. These data highlight the need for early RET fusion-positive testing to identify candidates who may benefit from treatment with Gavreto.

Exploring personalized cancer care for more patients

Genentech will present several studies that take tumor-agnostic approaches to clinical development, and in breast cancer, that may benefit people with rare and common tumors alike. These studies bring together next-generation sequencing, targeted therapies and patient-centric clinical trial design that show how personalized treatment plans are helping to evolve the way people are treated. The Phase II ALPHA-T study, made possible through a collaboration with Foundation Medicine and Science37, is pioneering a decentralized approach to clinical trial design which enables patients to participate from their own homes while remaining under the care of their oncologist. The Phase II TAPISTRY study, a platform umbrella trial, will pair patients with immunotherapy, targeted therapy or treatment combinations based on distinct tumor biology characteristics. The similarly designed Phase II MyTACTIC study is enrolling a diverse population of patients to direct them to appropriately targeted treatments based on the results of comprehensive genomic profiling.

With our research we are contributing to the body of evidence in hormone receptor (HR)-positive breast cancer, the most prevalent type of all breast cancers. For giredestrant, a third-generation oral selective estrogen receptor degrader (SERD), we will present data further supporting the tolerable safety profile and single agent clinical activity, as well as pharmacodynamics data from studies in HR-positive early and metastatic breast cancer.

Defining new solutions for patients with difficult-to-treat blood cancer

New and updated data in non-Hodgkin lymphoma (NHL) will be shared, including data from the T-cell engaging CD20xCD3 bispecific antibody development program. Glofitamab and mosunetuzumab are both T-cell engaging CD20xCD3 bispecific antibodies that are being studied as single agents or in combination with other Genentech therapies. Together, they may offer a new immunotherapy-based approach to tackle a range of blood cancers. In addition, data exploring novel combinations with mosunetuzumab and Polivy (polatuzumab vedotin), an antibody-drug conjugate, will also be featured. These data demonstrate how Genentech continues to seek new solutions for people living with a range of malignant blood disorders, where treatment options are still limited and both relapse and treatment resistance are common.

Furthermore, Genentechs data showcase a commitment to health equity through medicine delivery approaches that reduce treatment time and cost, trial designs that help remove barriers to clinical trial participation, pioneering cancer immunotherapy to improve outcomes for earlier disease stages, and a focus on inclusivity through developing tumor-specific therapies and therapy combinations based on the specific characteristics of each persons disease.

Keep up to date with ASCO news and updates by using the hashtag #ASCO21 and follow Genentech on Twitter via @Genentech and on LinkedIn.

Overview of key presentations featuring Genentech medicines

Medicine

Abstract title

Abstract number

Lung cancer

Alecensa

Final OS analysis from the phase III j-alex study of alectinib (ALC) versus crizotinib (CRZ) in Japanese ALK-inhibitor nave ALK-positive non-small cell lung cancer (ALK+ NSCLC).

9022

Gavreto

Safety and efficacy of pralsetinib in patients with advanced RET fusion-positive non-small cell lung cancer: Update from the ARROW trial.

9089

Tecentriq

IMpower010: Primary results of a phase III global study of atezolizumab versus best supportive care after adjuvant chemotherapy in resected stage IB-IIIA non-small cell lung cancer (NSCLC).

8500

Tecentriq

Artificial intelligence (AI)powered pathologic response (PathR) assessment of resection specimens after neoadjuvant atezolizumab in patients with non-small cell lung cancer: Results from the LCMC3 study.

106

Tecentriq

Pooled analyses of immune-related adverse events (irAEs) and efficacy from the phase 3 trials IMpower130, IMpower132, and IMpower150.

9002

Tecentriq

CONTACT-01: A phase III, randomized study of atezolizumab plus cabozantinib versus docetaxel in patients with metastatic non-small cell lung cancer (mNSCLC) previously treated with PD-L1/PD-1 inhibitors and platinum-containing chemotherapy.

TPS9134

Tecentriq

Clinicogenomic real-world data analysis of patients (pts) with KRAS G12C-mutant advanced non-small cell lung cancer (aNSCLC) from the natural history cohort of the Blood First Assay Screening Trial (BFAST).

9023

Tecentriq

Real-world treatment patterns in stages IA-IIIB non-small cell lung cancer.

e20528

Blood cancer

Gazyva

Obinutuzumab short-duration infusion (SDI) in previously untreated advanced follicular lymphoma: Results from the end of induction analysis of the phase IV GAZELLE study.

7545

Glofitamab

Glofitamab step-up dosing (SUD): Complete response rates in updated efficacy data in heavily pretreated relapsed/refractory (R/R) non-Hodgkin lymphoma (NHL) patients (pts).

7519

Mosunetuzumab

Promising tolerability and efficacy results from dose-escalation in an ongoing phase Ib/II study of mosunetuzumab (M) with polatuzumab vedotin (Pola) in patients (pts) with relapsed/refractory (R/R) B-cell non-Hodgkins lymphoma (B-NHL).

7520

Polivy

Polatuzumab vedotin (Pola) + rituximab (R) + lenalidomide (Len) in patients (pts) with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL): Primary analysis of a phase 1b/2 trial.

7512

Venclexta

Measurable residual disease response in acute myeloid leukemia treated with venetoclax and azacitidine.

7018

Breast cancer

Giredestrant

acelERA Breast Cancer (BC): Phase II study evaluating efficacy and safety of giredestrant (GDC-9545) versus physicians choice of endocrine monotherapy in patients (pts) with estrogen receptor-positive, HER2-negative (ER+/HER2-) locally advanced or metastatic breast cancer (LA/mBC).

TPS1100

Giredestrant

persevERA Breast Cancer (BC): Phase III study evaluating the efficacy and safety of giredestrant (GDC-9545) + palbociclib versus letrozole + palbociclib in patients (pts) with estrogen-receptor-positive, HER2-negative locally advanced or metastatic BC (ER+/HER2 LA/mBC).

TPS1103

Giredestrant

Safety and activity of single-agent giredestrant (GDC-9545) from a phase Ia/b study in patients (pts) with estrogen receptor-positive (ER+), HER2-negative locally advanced/metastatic breast cancer (LA/mBC).

1017

Giredestrant

Evaluation of pharmacodynamic (PD) and biologic activity in a preoperative window-of-opportunity (WOO) study of giredestrant (GDC-9545) in postmenopausal patients (pts) with estrogen receptor-positive, HER2-negative (ER+/HER2) operable breast cancer (BC).

577

Kadcyla

Safety of trastuzumab emtansine (T-DM1) in patients (pts) with HER2-positive locally advanced or metastatic breast cancer (mBC): Final results from KAMILLA Cohorts 1 (global) and 2 (Asia).

1039

Phesgo

Potential non-drug cost differences associated with the use of the fixed-dose combination of pertuzumab and trastuzumab for subcutaneous injection (PH FDC SC) in the treatment of HER2-positive early breast cancer patients in Western Europe and the United States.

544

Tecentriq

The tumor microenvironment (TME) and atezolizumab + nab-paclitaxel (A+nP) activity in metastatic triple-negative breast cancer (mTNBC): IMpassion130.

1006

Colon cancer

Tecentriq

Phase Ib/II open-label, randomized evaluation of atezolizumab (atezo) + Imprime PGG (Imprime) + bevacizumab (bev) vs regorafenib (rego) in MORPHEUS: Microsatellite-stable (MSS) metastatic colorectal cancer (mCRC).

3559

Liver cancer

Tecentriq

IMbrave150: Exploratory analysis to examine the association between treatment response and overall survival (OS) in patients (pts) with unresectable hepatocellular carcinoma (HCC) treated with atezolizumab (atezo) + bevacizumab (bev) versus sorafenib (sor).

4071

Tecentriq

IMbrave150: Exploratory efficacy and safety results of hepatocellular carcinoma (HCC) patients (pts) with main trunk and/or contralateral portal vein invasion (Vp4) treated with atezolizumab (atezo) + bevacizumab (bev) versus sorafenib (sor) in a global Ph III study.

4073

Personalized healthcare and health equity

Association of electronic-health record (EHR)-derived race with BRCA testing in patients (pts) with breast cancer (BC) with similar genetic ancestry (GA) in a clinicogenomic database (CGDB).

6524

Racial, ethnic, and socioeconomic disparities in treatment outcomes in patients (pts) with diffuse large B-cell lymphoma (DLBCL): A U.S. real-world study using a de-identified electronic health record (EHR)-derived database.

e18514

Tumor agnostic

Alecensa

Alpha-T: An innovative decentralized (home-based) phase 2 trial of alectinib in ALK-positive (ALK+) solid tumors in a histology-agnostic setting.

TPS3155

Gavreto

Clinical activity and safety of the RET inhibitor pralsetinib in patients with RET fusion-positive solid tumors: Update from the ARROW trial.

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Databridgemarketresearch.com Present Global Induced Pluripotent Stem Cells Market Industry Trends and Forecast to 2027 new report to its research database. The report spread No of pages : 350 No of Figures: 60 No of Tables: 220 in it.

Induced pluripotent stem cells (iPSCs) marketis expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 2,442.97 million by 2027 growing at a CAGR of 7.5% in the above-mentioned forecast period. Increasing R&D investment activities is expected to create new opportunity for the market.

Induced pluripotent stem cells market report helps businesses to look for a better solution to refine their business strategies with which they can succeed in this competitive market place. This report underlines the moves of key market players like product launches, joint ventures, developments, mergers and acquisitions which is affecting the market and healthcare Industry as a whole and also affecting the sales, import, export, revenue and CAGR values. All the statistical and numerical data that has been forecasted in the winning Induced pluripotent stem cells report is represented with the help of graphs, charts, or tables which makes this report more user friendly.

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Few of the major competitors currently working in global induced pluripotent stem cells market areFUJIFILM Holdings Corporation, Astellas Pharma Inc, Fate Therapeutics, Bristol-Myers Squibb Company, ViaCyte, Inc., CELGENE CORPORATION, Vericel Corporation, KCI Licensing, Inc, STEMCELL Technologies Inc., Japan Tissue Engineering Co., Ltd., Organogenesis Holdings Inc, Lonza, Takara Bio Inc., Horizon Discovery Group plc, Thermo Fisher Scientific.

Study Objectives Of Induced pluripotent stem cells Market

Global Induced Pluripotent Stem Cells (iPSCs) Market Scope and Market Size

Induced pluripotent stem cells (iPSCs) market is segmented of the basis of derived cell type, application and end- user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

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Global Induced Pluripotent Stem Cells Market Drivers:

Increasing R&D investment activities is expected to create new opportunity for the market.

Increasing demand for personalized regenerative cell therapies among medical researchers & healthcare is expected to enhance the market growth. Some of the other factors such as increasing cases of chronic diseases, growing awareness among patient, rising funding by government & private sectors and rising number ofclinical trialsis expected to drive the induced pluripotent stem cells (iPSCs) market in the forecast period of 2020 to 2027.

High cost of the induced pluripotent stem cells (iPSCs) and increasing ethical issues & lengthy processes is expected to hamper the market growth in the mentioned forecast period.

Key Developments in the Market:

TOC of Induced Pluripotent Stem Cells Market Report Contains:

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Data bridge is an aftermath of sheer wisdom and experience which was formulated and framed in the year 2015 in Pune. We ponder into the heterogeneous markets in accord with our clients needs and scoop out the best possible solutions and detailed information about the market trends. Data Bridge delve into the markets across Asia, North America, South America, Africa to name few.

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Global CAR T-cell Therapy Market 2021-2026 by Type, by Applications, and Region: Trend Outlook and Growth Opportunity is based on comprehensive research of the global CAR T-cell Therapy market with all its key segments through extensively detailed classifications. this report study consists of profound analysis and assessment generated from premium primary and secondary information sources with inputs derived from industry professionals across the value chain.

This report provides a detailed historical analysis of the global market for CAR T-cell Therapy from 2016-2020 and provides extensive market forecasts from 2021-2026 by region or country level. It covers the sales volume, price, revenue, gross margin, historical growth, and future perspectives in the CAR T-cell Therapy market.

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In-depth qualitative analyses include identification and investigation of the following aspects:

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CAR T-cell Therapy Market offers industry standpoint with development, Size, Share, Key Players procedures examination and memorable and cutting-edge pattern. This CAR T-cell Therapy Market report concedes the serious and quickly advancing industry, promoting guidance to follow execution and settle on choices such as both continuance and development.

Market split by Type can be divided into:

Market split by Application can be divided into:

CAR T-cell Therapy Market segment by Region/Country including:

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NEW YORK, and RESEARCH TRIANGLE PARK, N.C., April 28, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced four upcoming oral presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held virtually between May 11th to May 14th, 2021.

The AXO-AAV-GM1 presentation will include a review of patient-level data on safety and efficacy at 6 months follow up from the low-dose cohort of the Companys ongoing clinical study. Additionally, Dr. Cynthia Tifft, the lead investigator for the study, will present 6-month biomarker data from cerebrospinal fluid (CSF) in the 5 children who received intravenous AAV9 gene therapy.

Oral Presentation Details:

Presentation Title: AXO-AAV-GM1 for the Treatment of GM1 Gangliosidosis: Preliminary Results from a Phase I-II trialAbstract Number: 162Session: Clinical Trials and Advanced Preclinical Studies for Neurologic DiseasesPresenting Author: Cynthia Tifft, MD, PhD, Deputy Clinical Director, National Human Genome Research InstitutePresentation Date and Time: Thursday, May 13, 2021 6:15 PM 6:30 PM EDT

Presentation Title: AXO-Lenti-PD gene therapy for Parkinsons disease: efficacy, safety, and tolerability data from the second cohort in open-label dose evaluation study SUNRISE-PD at 6 months post administrationAbstract Number: 163Session: Clinical Trials and Advanced Preclinical Studies for Neurologic DiseasesPresenting Author: Gavin Corcoran, MD, Chief R&D Officer Presentation Date and Time: Thursday, May 13, 2021 6:30 PM 6:45 PM EDT

Presentation Title: Immune Modulation Preceding AAV9-GLB1 Gene Therapy Preserves the Possibility for Re-Dosing in Children with GM1 GangliosidosisAbstract Number: 179Session: Immunotherapy and VaccinesPresenting Author: Precilla DSouza, DNP, MSN, CRNP, National Human Genome Research InstitutePresentation Date and Time: Thursday, May 13, 2021 7:00 PM 7:15 PM EDT

Presentation Title: A GLP Safety and Biodistribution Study of AXO-Lenti-PD Manufactured via Two Processes Delivered at a Higher Volume and Flow RateAbstract Number: 256Session: Pharmacology/Toxicology Studies or Assay DevelopmentPresenting Author: Thomas Pack, PhD, Sio Gene TherapiesPresentation Date and Time: Friday, May 14, 2021 from 1:45 PM 2:00 PM EDT

About AXO-AAV-GM1

AXO-AAV-GM1 delivers a functional copy of theGLB1gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from theFood and Drug Administrationand is the only gene therapy in clinical development for both Type I and Type II GM1 gangliosidosis.

In 2018, Sio licensed exclusive worldwide rights from theUniversity of Massachusetts Medical Schoolfor the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About AXO-Lenti-PDAXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinsons disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration.Axovantexpects to dose the first patient in EXPLORE-PD, a randomized, sham controlled study in 2021.

AboutSio Gene TherapiesSio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visitwww.siogtx.com.

Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as believe, "estimate," may be and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities, funding requirements and/or runway to meet its upcoming clinical milestones, and timing of its upcoming clinical milestones are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations; the actual funds and/or runway required for our clinical and product development activities and anticipated upcoming milestones; actual costs related to our clinical and product development activities and our need to access additional capital resources prior to achieving any upcoming milestones; the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for Axo-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononFebruary 9, 2021, as updated by its subsequent filings with theSecurities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officer investors@siogtx.com

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Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy -...

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BOSTON--(BUSINESS WIRE)--SOLA Biosciences, LLC, today announced positive preclinical data in mice from a study designed to evaluate the efficacy of SOL-257, an innovative gene therapy candidate selectively targeting pathogenic TDP-43, which is found in approximately 97% of ALS patients. The data was presented during the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular Disease on April 29, 2021, by Akinori Hishiya, Ph.D., Founder/CSO of SOLA.

SOLA has developed an innovative chaperone technology (JUMP70) to selectively eliminate disease-causing misfolded proteins using the patients' own chaperones. SOLA has developed nine gene therapy candidates incorporating JUMP70 to address conformational diseases such as Amyotrophic Lateral Sclerosis (ALS), Huntington's disease, Parkinson's disease, and Alzheimer's disease. These candidates demonstrated preclinical activities and safety to prove the mechanism of the JUMP70 technology. SOL-257 is a flagship compound to selectively target misfolded and neurotoxic TDP-43 proteins without affecting healthy TDP-43.

At the conference, SOLA presented unprecedented in vivo efficacy data using the NEFH-hTDP-43NLS mouse model, which expresses a doxycycline-repressible form of human TDP-43 lacking a nuclear localization signal (NLS). The transgenic mice accumulate cytoplasmic insoluble TDP-43 in neurons of the brain and spinal cord soon after pathogenic TDP-43 proteins are expressed. The mice develop many features reminiscent of ALS, including motor deficits, denervation of neuromuscular junctions, motor neuron loss, and rapid loss in viability. The mice treated with control gene therapy (placebo) started dying 3 weeks after pathogenic TDP-43 expression, and by 5 weeks, 100% of male mice died. In contrast, all male mice treated with SOL-257 gene therapy survived.

"Now we know many diseases such as ALS are caused with protein folding issues. Repairing or removing the misfolded disease-causing proteins can be a fundamental treatment for the patients, although the treatment has not been realized yet. We learned the natural protein quality control system and carefully designed our novel therapeutics," says Dr. Akinori Hishiya. "Our designed therapeutic enables highly specific intervention, only misfolded disease-causing proteins can be targeted and removed."

"SOL-257 targeting only pathogenic TDP-43 to repair the specific problem has a significant potential for a broadly applicable ALS treatment," says Keizo Koya, Ph.D., Founder/CEO of SOLA. "I hope this groundbreaking animal data of SOL-257 will bring new hope for patients with ALS.

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SOLA Biosciences Presented Positive ALS Animal Efficacy Data of SOL-257 at the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular...

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DUBLIN--(BUSINESS WIRE)--The "Cell & Gene Therapy Technologies and Supplies" report has been added to ResearchAndMarkets.com's offering.

Cell and gene therapy (CGT) is a rapidly evolving field producing powerful new treatments for cancers and genetic diseases, and expanding into autoimmune disease, cardiovascular disease, musculoskeletal disease, and many others. The CGT field has generated great interest and hope among researchers, patient groups, and regulators, which has led to huge investments in R&D.

Globally, many governments and regulatory bodies have adopted policy and patent environments supportive of CGT development. Since CGTs often target rare and underserved disease areas for which few other therapeutics exist, there is a sense of urgency within the pharma/bio space to develop CGTs.

As development and market entries of CGTs accelerate, the market for suppliers of laboratory and clinical tools within the CGT R&D and manufacturing spaces will see very rapid growth. The Cell & Gene Therapy Technologies and Supplies examines the global market for analytical technologies and products used throughout the various stages of CGT development and manufacturing, evaluating 21 technologies grouped into six categories.

The goal of this report is to provide demand growth projections by technique, region, and function, while also providing comprehensive views of the competitive landscape for each technology.

Report Overview

Participants include the following:

Key Topics Covered:

1. Introduction

2. Market Insights

3. Market Demand

3.1 Overall Demand

3.2 General Techniques

3.3 Gene Therapy Development

3.4 Transduction and Transfection

3.5 Cell Enrichment

3.6 Cell Culture and Cell Expansion

3.7 Market Demand by Technique (2020)

3.8 Cryopreservation

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Cell & Gene Therapy Technologies and Supplies Report 2021: Market Demand and Five-year Forecasts, Segmented by Technique, Region and Function -...

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