Archive for the ‘Gene Therapy Research’ Category

I favor running analogies even the name of this column pays tribute to the sport. But Im not the first patient advocate to evoke that ancient sport of quicker-than-average terrestrial locomotion. After all, we are racing to treatments because time is critical.

But in the fight against a rare condition like Batten disease, success involves much more than speed. You may be fast, but have you studied the course? Do you know its twists and turns? Do you have a team and support crew, or are you competing alone? Are you ready to take on unexpected obstacles?

Do you have what it takes?

Therapy development isnt for the faint of heart, but patients can and absolutely do make an impact on potentially life-saving treatments and the science and regulatory issues involved. Last month, I joined my mom, Taylors Tale President Sharon King, and Steven Gray, PhD, associate professor at University of Texas Southwestern Medical Center, onstage at the Global Genes RARE Patient Advocacy Summit to discuss that topic.

If you didnt make it to San Diego for the summit this year, I hope you had a chance to tune in to the livestream. If you didnt, or if youd like a recap, following are nine things that we shared about pushing promising work toward the finish line.

Like a marathon, the race to find treatments and cures isnt easy. Few people see the long hours in the lab or the thousands of experiments that dont produce a single breakthrough or correct answer. Miracles happen in science, but results come with a lot of hard work and some luck.

If you want to see a treatment for you or someone you love, you have to accept that it wont be easy. This race is hard. Youll feel winded. Youll get injured. Youll crave water stops. Youll get passed by someone running faster than you. Youll pass someone who doesnt want it as much as you do.

In his new book Chasing My Cure, my friend, David Fajgenbaum, writes, Fear disintegrates. Doubt disorganizes. Hope clears the way, pushes out the horizon, and gives us space to build structures.

Heres what I think David meant by that:

Being invincible in hope means having the courage and the will to take action. You cant trust and wait. You cant assume that someone else will swoop in to save the day. Thats not what hope means.

Instead, you have to look inward. You have to take an active role in transforming hope into reality. Thats hope in action.

Thats how you change the world.

Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has. Margaret Mead

***

Note: Batten Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Batten Disease News or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to Batten disease.

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9 Things We Should Know About the Race to New Treatments - Batten Disease News

STONY BROOK, N.Y.--(BUSINESS WIRE)--LineaRx, Inc., the majority-owned subsidiary of Applied DNA Sciences, Inc. (NASDAQ: APDN) focused on next-generation biotherapeutics, announced today that its invasive Circulating Tumor Cells (iCTCs) platform demonstrated superior correlation over Prostate Specific Antigen (PSA) in an ongoing Phase II trial in recurrent prostate cancer. Most notably, the concentration of rare iCTCs identified per milliliter of whole blood correlated more directly with disease status than did the levels of PSA. These iCTCs were captured and identified utilizing LineaRxs technology platform recently acquired from Vitatex Inc.

LineaRxs collaborator Tyme Technologies, Inc. (NASDAQ: TYME), reported yesterday results of this recently completed Phase II trial using SM-88 to treat recurrent prostate cancer at the Congress of the European Society of Medical Oncology (ESMO), one of the preeminent meetings for highlighting new cancer therapies and diagnostics. The study updated the safety and antitumor effects of SM-88 in men with non-castrate non-metastatic prostate cancer. Antitumor effects were assessed by post-therapy changes in PSA and the number of iCTCs. Results showed that patients treated with this oral non-hormonal therapy maintained metastases-free survival without testosterone deprivation or typical hormonal therapy side effects.

Dr. Wen-Tien Chen, Principal Investigator of CTC Programs at LineaRx stated: This use of iCTC quantification for providing earlier and more accurate identification of prostate cancer shows the importance of running trials using the iCTC functional capture and identification system from LineaRx. Published studies have shown that the concentration of iCTCs in blood correlates with disease status across other hard tumors, including metastatic sarcoma, pancreatic, ovarian, breast, lung and colorectal cancers. We believe our proprietary platform can help diagnose these difficult cancers earlier and with superior predictive capability.

The current reported SM-88 Phase II study examines iCTCs in prostate cancer for what we believe is the first time in a clinical trial. While PSA has historically been the recognized biomarker for prostate cancer, many studies over the last 20 years have shown it is an inadequate biomarker, since it may become elevated in patients with no apparent disease, or less serious pathology than cancer. Intensive efforts have been directed towards alternative prostate cancer biomarkers, particularly those that can predict disease aggressiveness and help to drive better treatment decisions. However, biomarker research has centered on disease diagnostics, rather than prognosis and prediction, which could work toward disease preventionan important focus moving forward.

The authors concluded: "Reductions in iCTC number may be a more informative indicator of benefit than changes in PSA. Dr. Chen added, New prostate cancer biomarkers should be targeted to addressing unmet clinical needs in prostate cancer management, including indicators for disease with low PSA values (<10ng/mL), prognostic markers to distinguish indolent from aggressive disease, and biomarkers for metastatic cancer.

LineaRxs iCTC technology is unique in that it isolates iCTCs on a functional basis (the invasion of a model tissue for the extracellular matrix, or the tissue between cells in an organ) and allows the cells to be cultured for deeper genomic analysis. iCTCs are a model for metastasis, since the tumor cells have left the primary tumor and entered the circulation. The LineaRx platform may serve as a standalone device or as a front-end to increase the sensitivity of approved diagnostics.

iCTCs have the potential for metastasis, and may be very useful in the diagnosis of cancer, even before tumors may be imaged, said Dr. James Hayward, CEO of LineaRx. The present study is exciting because we believe it shows that iCTCs may be used to accurately follow therapy success in this very prevalent cancer while also providing new tools for cell and gene therapy design and production.

About Tyme Technologies, Inc

Tyme Technologies, Inc., is an emerging biotechnology company developing cancer therapeutics that are intended to be broadly effective across tumor types and have low toxicity profiles. Unlike targeted therapies that attempt to regulate specific mutations within cancer, Tyme Technologies, Inc.s therapeutic approach is designed to take advantage of a cancer cells innate metabolic weaknesses to compromise its defenses, leading to cell death through oxidative stress and exposure to the bodys natural immune system. For more information, visit http://www.tymeinc.com. Follow Tyme Technologies, Inc. on social media: @tyme_Inc, LinkedIn, Instagram, Facebook and YouTube.

About LineaRx

LineaRx seeks to commercialize the biotherapeutic value of Applied DNAs deep expertise and experience in the design, manufacture and chemical modification of DNA by large scale polymerase chain reaction (PCR). Linear DNA is a form of DNA distinct from the circular form of DNA most commonly produced in plasmids and grown in bacteria. Plasmids are extrachromosomal DNA found in bacteria and are associated with the genes for antibiotic resistance which are often exchanged between bacteria and consequentially, are seen by many to embody a serious threat to global health. In addition, many nucleic acid-based therapies also rely on viral vectors for efficient transfection and expression of plasmid DNA. These viral vectors carry additional nontrivial risks and are extremely time consuming and expensive to manufacture. Go to http://www.adnas.com for more information on LineaRx and to learn more about how Applied DNA makes life real and safe. LineaRx is a majority-owned Applied DNA Sciences, Inc. (Nasdaq: APDN) company.

About Applied DNA Sciences

Applied DNA is a provider of molecular technologies that enable supply chain security, anti-counterfeiting and anti-theft technology, product genotyping and pre-clinical nucleic acid-based therapeutic drug candidates.

Applied DNA makes life real and safe by providing innovative, molecular-based technology solutions and services that can help protect products, brands, entire supply chains, and intellectual property of companies, governments and consumers from theft, counterfeiting, fraud and diversion.

Visit adnas.com for more information. Follow us on Twitter and LinkedIn. Join our mailing list.

Common stock listed on NASDAQ under the symbol APDN, and warrants are listed under the symbol APDNW.

Forward-Looking Statements

The statements made by Applied DNA in this press release may be forward-looking in nature within the meaning of the Private Securities Litigation Act of 1995. Forward-looking statements describe Applied DNAs future plans, projections, strategies and expectations, and are based on assumptions and involve a number of risks and uncertainties, many of which are beyond the control of Applied DNA. Actual results could differ materially from those projected due to the risk that the acquisition will not be successfully integrated with LineaRx or that the potential benefits of the acquisition will not be realized, the Companys history of net losses, limited financial resources, limited market acceptance , the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Applied DNAs product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies, uncertainties relating to its ability to maintain its NASDAQ listing in light of delisting notices received and various other factors detailed from time to time in Applied DNAs SEC reports and filings, including our Annual Report on Form 10-K filed on December 18, 2018, as amended, and our subsequent quarterly reports on Form 10-Q filed on February 7, 2019,May 9, 2019 and August 13, 2019, and other reports we file with the SEC, which are available at http://www.sec.gov. Applied DNA undertakes no obligation to update publicly any forward-looking statements to reflect new information, events or circumstances after the date hereof to reflect the occurrence of unanticipated events, unless otherwise required by law.

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LineaRx's iCTC Platform for Isolation of Circulating Tumor Cells Shows Superior Correlation over PSA as a Biomarker in Prostate Cancer Trial -...

The Universities of Bristol and Nottingham will share over 500k to lead a consortium of experts on research projects into bioengineered blood and soft tissue regeneration, respectively. The research could not only save lives, but could also improve the quality of lives of injured personnel.

The funding is awarded at Phase 2 of the Defence and Security Accelerator (DASA) Regenerative Medicine themed competition which is facilitated and managed by the Defence Science and Technology Laboratory (Dstl) on behalf of the MOD, in partnership with the Academic Department of Military Surgery and Trauma (ADMST).

Dealing with damage caused by blast or ballistic trauma, which may involve significant blood loss and multiple complex wounds, is a challenge for even the most sophisticated medical facility. Yet to do this in the austere and remote environments within which the military operate further complicates the delivery of medical care.

Approaches in tissue engineering and regenerative medicine hold great promise for the treatment of injured service personnel and the new Defence regenerative medicine research strategy is focussed on delivering such advanced therapies in a way suitable for use in the field early after injury.

The 500k funding will enable the University of Bristol to continue its research to engineer a multi-compatible blood type, with an improved storage profile, that could be used to treat military personnel regardless of their blood type. This could transform the logistics of transporting and storing blood supplies on the front line. In the longer term, first responders like paramedics could also benefit from the portability of a blood supply that is suitable for all.

The University of Nottingham will continue to research a novel approach to preserve and regenerate soft tissue after blast and ballistic trauma through transient gene therapy. Preserving living tissue after injury is critically important, and will significantly improve quality of life.

Dave Henson, co-founder of the CASEVAC club, a support network for individuals that were severely wounded in combat, said:

Understanding that saving a life on the battlefield without due consideration for the future quality of that life is nonsensical. Significant progress has been made in the medical arena throughout the duration of recent conflicts. The development of technologies such as were seeing in this latest of the DASA competition, with the reduction in burden associated with blood supplies, and the immediate improvement of wound management techniques, provides strong assurances that the functional outcome from battlefield trauma will continue to improve.

Dr Abi Spear, Technical Lead for the regenerative medicine project at Dstl said:

Im delighted that the Universities of Bristol and Nottingham have won this Phase 2 competition. Their work represents innovative, discovery science thats high risk but with potentially huge clinical benefit.

Dr Adam Staines, Themed Competition Lead, DASA:

We are pleased that this competition has harnessed cutting edge bio-medical research that seeks to make a real difference on the front line and could also have positive implications for the civilian market in future too.

The Dstl regenerative medicine research strategy looks to support research in four areas, as defined by an evidence-based scoping study, through a variety of activities, including funding and collaboration. If you would like more information on the project as a whole please email DSTLRegenDefenceAccelerator@dstl.gov.uk.

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Defence investment set to transform recovery from combat trauma - GOV.UK

People with spinal muscular atrophy (SMA) type 1 may have an increased risk of impaired kidney function, a small study suggests.

The studys findings highlight the need to further evaluate non-muscular conditions associated with SMA type 1 and to identify co-adjuvant therapies to manage them.

The research was published in the article, Impaired kidney structure and function in spinal muscular atrophy, in the journal Neurology Genetics.

SMA is caused by mutations in theSMN1 gene, which leads to reduced production of the SMN protein. While motor nerve cells are those most vulnerable to SMN deficiency, decreased levels of this protein occur throughout the body, which may contribute to the development of the disease.

With new therapies to treat all types of SMA, including type 1 (the most common and one of the most severe types), patients are living longer, and thus the overall impact of SMN deficiency beyond motor function needs to be evaluated, scientists say.

In addition, an increased risk of renal toxicity is listed on the insert for Spinraza, the first therapy approved by the U.S. Food and Drug Administration to treat SMA, and similar therapies have been associated with acute tubular injury, the researchers wrote.

With this in mind, they evaluated whether kidney function was impaired in 13 children with SMA type 1 who died due to SMA complications.

Analysis of blood data showed that most of the children had values outside of the expected range for several molecules associated with kidney function, including creatinine and cystatin C. Most of them also had potentially harmful aggregates, called granular casts and amorphous crystals, and high levels of amino acids (the building blocks of proteins) in their urine, which are all linked with kidney damage.

Molecular and morphological analyses of kidney tissues of 12 patients as well as age- and sex-matched children without SMA showed that two-thirds of the SMA patients had several abnormalities highly associated with kidney disease.

Kidney tissues of children with SMA type 1 showed varying degrees of tubular damage, with scarring and abnormal calcium deposits, and significant changes in the levels of genes associated with kidney toxicity or kidney cystic disease.

Also, they had low levels of CaSR and CALB1 (proteins involved in the maintenance of their calcium balance, thus preventing accumulation of calcium deposits) and high levels of IGFBP1 and IGFBP3 which has been associated with kidney damage and dysfunction compared with the non-SMA children.

The researchers noted all these features were consistent with kidney dysfunction more specifically kidney tubular dysfunction which strongly suggests that most of the children with SMA type 1 had impaired kidney function before death.

Therefore, the study recommends that physicians consider this increased risk of impaired kidney function when treating their patients with SMA type 1.

The researchers also noted that since these patients are now living longer due to recently approved treatments, kidney disease may become a significant comorbidity, and future research should focus on identifying therapies to manage kidney dysfunction.

Future studies will be important to determine whether impaired kidney function is a primary consequence of reduced SMN protein, a secondary consequence of muscle atrophy and bone disease, or more likely a combination of both, the researchers said.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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SMA Type 1 Kids May be at Higher Risk of Kidney Dysfunction, Study Says - SMA News Today

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Longevity And Anti-senescence Therapy Market Research on Trend, Demand and Precise Outlook 2019-2025 - The Ukiah Post

Gene Therapy Market look into report is a in-depth analysis of current situation of the market, which covers a few market dynamics. A isolate area with Gene Therapy industry key players is incorporated into the report, which gives a far reaching investigation of sales and revenue, value, cost, gross, product picture, details, organization profile, and contact data. The Market Report additionally gives a diagnostic appraisal of the prime difficulties looked by Gene Therapy Market at present and in the coming years, which helps Market members in understanding the issues they may face while working in this Market over a more extended timeframe.

Market Segmentation: Key Players

Avalanche BioAdvantageneDimension TherapeuticsBluebird BioSangamoCelladonVical Inc.Spark Therapeutics

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The report gives the concise examination report of the Gene Therapy Market showcase around the United States. The Gene Therapy think about contributes tasteful answer for the client. The examination think about incorporates investigation, Gene Therapy market forecast and income from 2019 to 2026. The Gene Therapy report unveils the near outcome between various players spread in the United States. It likewise gives the different kinds of sections of the Gene Therapy market seeing to the item, for example, type, Regions/Countries, application and players. Current and refreshed Gene Therapy trends likewise included to the report.

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Global Gene Therapy Market Analysis By Product Types:

In VivoEx Vivo

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Cardiovascular DiseasesInfectious DiseasesMonogenic DiseasesCancer DiseasesOthers

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The fundamental regions analyzed in this report include North America, Europe, Asia-Pacific, Middle East& Africa and South America. The growth rate and production value are analyzed for these regions. Further, the above regions are bifurcated to provide country-level Gene Therapy industry statistics for the below countries.

North America region covers the United States, Canada and the rest of the countries

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Gene Therapy Market Overview. Global Gene Therapy Market Competition by Manufacturer.

Global Gene Therapy Capacity, Production, Revenue(value) by region(2014-2019).

Global Gene Therapy Supply(Production), Consumption, Export, Import By Region(2014-2019)

Global Gene Therapy Capacity, Production, Revenue(value), Price Trend by Type.

Global Gene Therapy Market Analysis By Application.

Global Gene Therapy Manufacturers profiles/Analysis

Global Gene Therapy Manufacturing Cost Analysis

Industrial chain, sourcing strategy and downstream buyers.

Marketing stratergy analysis, Distributors/Traders.

Global Gene Therapy Market Forecast Analysis(2019-2026).

Research Finding and Conclusions.

Appendix.

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Global Gene Therapy Market- Industry Growth, Trends, New Tech Developments, Revenue Predicted to 2026 - Space Market Research

Three new animal-model tests of a potential gene therapyfor oculopharyngeal muscular dystrophy (OPMD), known as BB-301, are planned, Benitec Biopharmaannounced. Results are expected tosupport a request for a Phase 1 clinical trial in patients.

OPMD results from a faulty PABPN1 gene, leading to a protein that can form insoluble clumps linked with muscle weakness.

BB-301 uses a DNA-directedRNA interference(ddRNAi) strategy. Delivered via a modified, harmless adeno-associated virus (AAV), the therapy is intended to silence and replace the mutant protein. It suppresses its production and provides a source of normal, or wild-type, protein to target cells.

A 2017 study showedthat this approach significantly reduced the amount of PABPN1 aggregates, restored muscle strength and eased muscle fibrosis (scarring) in mice. Subsequent experiments in cells from OPMD patients confirmed these results.

The preclinical research, to be conducted in a canine disease model, will help to optimize methods of administration, confirm the efficiency of the AAV vector in introducing a healthy gene into key muscle cells, assess optimal dosing, and further characterize toxicological data necessary for regulatory filings and clinical trial design.

According to the company, these three planned dog studies will support the submission of an Investigational New Drug (IND) application, an essential step in getting regulatory approval to start the trial. Pre-IND application meetings with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Health Canada took place in 2017.

Through our continued focus on BB-301 for the treatment of [OPMD], our team has an unprecedented opportunity to develop a novel genetic medicine that could facilitate clinically meaningful patient benefit, Jerel A. Banks, MD, PhD, Benitecs executive chairman and CEO, said in a press release.

The preclinical studies will be done in partnership with a medicine and surgery team with several decades of experience in OPMD treatment, Benitec added.

The FDA granted orphan drug status to BB-301 as a potential OPMD treatment in January 2018. The EMA awarded it similar status in early 2017.

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Final Tests Planned into Potential OCMD Gene Therapy, Could Support Trial in Patients - Muscular Dystrophy News

RESEARCH TRIANGLE PARK Pfizer is investing $19 million to create a new gene therapy production facility in RTP and could add up to 120 scientists and staff to operate it.

Information provided by Pfizer on Thursday says the company will move operations from a related facility in Chapel Hill that it acquired as part of a deal to buy Bamboo Therapeutics in 2016.

The new location is near the intersection of I-40 and I-540 in RTP.

According to the drug giant, an existing building will be retrofitted to accommodate production. It will provide facilities for drug substances, and related products as well as testing labs.

The news follows the recent announcement that Pfizer would invest $500 million and add some 300 jobs related to gene therapy at its campus in Sanford.

Construction will begin in 2020 with completion expected in 2022.

Pfizer adding 300 jobs, investing $500M at Sanford campus

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Pfizer to build gene therapy facility in RTP, could add up to 120 scientists, staff - WRAL Tech Wire

Until the day she dies, Sandy Morris is hunting for a comma and a clause.

In May 2018, the 53-year-old mother of three learned she has ALS, a diagnosis made even more devastating by a somber directive to get her affairs in order period. The finality speaks to the absence of an effective treatment, yet alone a cure, for the neurodegenerative disease also known as Lou Gehrig's disease.

Since the diagnosis, Morris has sought to give future ALS patients some measure of optimism, amending the 'get your affairs in order' instruction to add 'but, there's something worth looking into.' While medical advances in areas like gene therapy show potential to change the course of other potentially fatal diseases, a disease-modifying treatment in ALS has proved elusive.

"In cancer, you have a shot," Morris said in an interview. "Not always a great shot, but you have one. In this land, everybody is just like, 'We don't have anything.'"

But now, Morris said she has reason for hope, stemming from a first-of-its-kind study in ALS that will test five experimental drugs simultaneously in a platform trial. Five drugmakers selected for the trial were announced last week.

After years of work, the broader promise of these adaptive trials, which allow more flexibility to make changes throughout, is beginning to be put into action. This trial aims to accelerate the pace of ALS research and may prove to be a template for other rare diseases as well, experts said.

The design offers advantages for each key stakeholder in drug development: patients have a lower probability of receiving placebo due to the study's shared control arm; companies get quicker topline results at lower cost; and researchers talk of the study as an "endpoint engine" that could advance their scientific understanding of the disease.

Formidable challenges remain in the way of broader adoption, particularly among leaders of the drug industry. The ALS study uses experimental treatments from small biotechs instead of industry giants, which can afford to prioritize control and pay for their own studies instead.

Even so, it's an exciting advancement in ALS research. Morris, who is also a leader of the patient group I Am ALS, said the trial can bring hope to future patients, even if it comes too late to help her.

"It's not going to save me. I'm not in time for any of this," Morris said. "But, dammit, we have to make it better for the rest."

Merit Cudkowicz understands speed. After 25 years specializing in ALS research, Cudkowicz is fully aware of how ALS changes a patient's life, shortening their life expectancy from decades to just years. The disease has a roughly three-year median survival.

Merit Cudkowicz

Massachusetts General Hospital

That life-changing diagnosis came in May 2018 for Sean Healey, then the CEO of an asset management firm worth more than $8 billion. After resigning from that post, Healey connected with Cudkowicz as he searched for treatment options and realized there was an opportunity to accelerate the pace of research.

Just six months from his diagnosis, Healey raised $40 million to launch a research center at Massachusetts General Hospital. Now, slightly less than a year after being established, the Sean M. Healey & AMG Center for ALS Research is aiming to start the platform trial in the first few months of 2020.

And earlier this month, the Healey Center announced the first five therapies to be tested in the study, including drugs from Biohaven Pharmaceutical and Ra Pharmaceuticals.

Cudkowicz, who leads the study, as well as the Healey Center and Mass General's neurology department, said in an interview the trial aims to have 160 patients for each of five treatments. The primary outcome will be whether or not the drug boosted a functional rating score for ALS after six months. One placebo group will be shared for all treatment arms, and more drugs can be added as the study continues. (Cudkowicz cautioned the plan is nearing finalization with the Food and Drug Administration, so specifics could still change.)

Moving quickly is the trial's aim. Cudkowicz called the study "an endpoint engine" that can help create better outcome measurements, including future surrogate markets, pushing ALS research forward. She estimated topline results to come 12 to 18 months after the study begins, depending on enrollment speed.

But those goals are steps toward the actual mission: developing a cure, Healey wrote in an email to BioPharma Dive.

"Of course, we all understand that the most meaningful measure of success will be the development of effective treatments and ultimately a cure," Healey wrote. "I am convinced that the Platform Trial, along with other initiatives we are supporting, will substantially accelerate the achievement of this ultimate goal."

For the first five biotechs, the decision to participate was a no-brainer. The center and other groups are footing the majority of the cost, leaving the companies' primary expense to simply provide their drug.

Out of about 30 applicants, the Healey Center selected five drugs for the study, most notably Biohaven's verdiperstat and Ra Pharma's zilucoplan. The trial also will test therapies from Implicit Bioscience, Prilenia Therapeutics and Clene Nanomedicine.

Cudkowicz said the typical applicants were small biotechs that "have great ideas but not deep pockets."

The expense of clinical trials limits the ability for biotechs to run multiple studies simultaneously across a range of indications. Instead, companies typically focus on a lead indication, with others following in succession.

"As a small company, we live and die by being able to run efficiently and test our hypotheses," said Irfan Qureshi, Biohaven's vice president of neurology.

For instance, Biohaven's verdiperstat is in Phase 3 testing for multiple system atrophy, and Ra Pharma's zilucoplan is focused on a different neuromuscular disorder called generalized myasthenia gravis.

"We wanted to do the study anyway, but to be honest, we probably wouldn't have gotten to it for years if this had not come along," Ra Pharma CEO Doug Treco said in an interview.

While smaller biotechs have bought into the platform trial and its efficiencies, missing from the list of initial participants are industry leaders. With a market value of about $2 billion, Biohaven is the largest company involved.

Cudkowicz noted larger drugmakers have shown interest in roundtable discussions that shaped the trial's design, and there's always potential to add additional therapies after the trial starts.

"We're talking to the other ones like Biogen and Sanofi," she said. "They are interested and came to these meetings, but they have the money to do it on their own."

But the main hang-up for these companies is ceding control, said Scott Berry, a senior statistical scientist and co-founder of Berry Consultants, which worked on the ALS platform trial.

"All these companies have people that this is what they do for a living they run trials, and they know how to do it," Berry said. "To hand your drug to somebody else and you don't have control over making sure that happens is uncomfortable for companies, and it's different than what they usually do."

Additionally, with lengthy protocols and many moving parts, the trials are complex and typically require extensive consulting to get off the ground. Running a multi-arm study also brings statistical pitfalls that can make it harder to interpret results.

Beyond ALS, adaptive trials have been started in breast cancer, Alzheimer's disease and glioblastoma. Experts say they anticipate other rare diseases as logical future targets for these studies.

"As these start to get developed and people see them, in most rare diseases there will be people jumping on board," predicted Berry.

Biohaven's Qureshi added that once companies are willing to experiment beyond typical drug development, these trials could be particularly attractive for rare diseases by easing enrollment concerns where there are "not patients growing on trees," he said.

But the ultimate test to get the industry's full attention, Qureshi said, would be such a study yielding an approved drug.

In the meantime, platform trials appear here to stay. Janet Woodcock, the long-time leader of the FDA's drug review center, has been an influential supporter for these study designs. Just this month, the agency finalized guidance on ALS research that advised companies to consider adaptive trial designs.

Earlier this year, Woodcock told BioPharma Dive she believes these types of studies will gain ground as patients become more vocal about how trials are conducted.

Morris said the platform trial has struck her with its compassion. While no patient wants to be put on placebo, it's of particular importance in a disease that progresses as rapidly and severely as ALS, leaving most patients the time to try one study in their life.

She already took her shot, enrolling in a clinical trial that required a three-month observation period before receiving treatment, which then carried a 50% chance of being placebo. "We are humans," she said. "We aren't zebrafish."

Now, she wants to take the baton from "the voices in the graveyard" and pass it onto the next generation. Platform trials may allow her to cover a bit more ground before that hand-off, eventually reaching a day when people might be able to live with ALS, like HIV.

"ALS's day is coming," Morris said.

See the article here:
First-of-its-kind trial in ALS spurs hope for brutal disease - BioPharma Dive

Modern retroviral therapies have done a good job of turning HIV/AIDS into a treatable chronic illness, but except in a few rare experimental cases using stem cell transplants, there is no cure. Cure, in this case, meaning that the HIV virus is completely eradicated in the patient. Retroviral therapies cause the virus to become inactive, but they still remain in the body and may potentially reactivate if treatment ends.

Researchers at the University of California, San Diego (UCSD) School of Medicine have now identified a key switch that has the potential to eliminate dormant HIV reservoirs. They published their research in the journal mBio.

This is one of the key switches that the HIV field has been searching for three decades to find, said Tariq Rana, professor of pediatrics and genetics at UCSF. The most exciting part of this discovery has not been seen before. By genetically modifying a long noncoding RNA, we prevent HIV recurrence in T-cells and microglia upon cessation of antiretroviral treatment, suggesting that we have a potential therapeutic target to eradicate HIV and AIDS.

Rana and fellow researchers utilized genome-wide expression analysis of long noncoding RNA (lncRNA) in specialized immune cells called macrophages that had been infected with HIV. Macrophages promote inflammation, stimulate the immune system and remove foreign matter. Usually, lncRNAs dont behave the way other RNAs do, which is to say, RNAs deliver DNA codes for proteins. The lncRNAs are involved in controlling which genes are switched on or off in a cell.

The researchers focused on a single lncRNA called HIV-1 Enhanced LncRNA (HEAL) that is found in higher amounts in HIV patients. The researchers believe this is a recently emerged gene that regulates HIV replication in immune cells, including macrophages, microglia and T-cells.

They ran experiments that silenced HEAL or removed it using CRISPR-Cas9 gene editing, which resulted in HIV no longer recurring when antiretroviral treatment was halted. The researchers expect to continue to confirm the data in animal models.

Our results suggest that HEAL plays a critical role in HIV pathogenesis, Rana said. Further studies are needed to explain the mechanism that leads to HEAL expression after an individual is infected by HIV, but this finding could be exploited as a therapeutic target.

This appears to be a major finding, but there have been others recently as well. In August, researchers from the University of Texas Medical Branch at Galveston discovered that the protein BRD4 has a significant role in regulating the production of new copies of the HIV gene. The research group, led by Haitao Hu, assistant professor of microbiology and immunology, designed, synthesized and studied several small molecules to selectively program BRD4 to suppress HIV. They then picked a lead compound, ZL-580, which significantly delayed the reactivation of dormant HIV after antiretroviral therapy was halted.

Like the research published by the UCSD team, this appears to have the potential to be a drug therapy to eradicate the virus, or at least better deal with resistant strains of the disease.

HIV still affects about 37 million people worldwide and approximately 1 million people die annually from HIV-related causes. Treatment typically involves a cocktail of antiretroviral therapy which HIV patients take their entire lives.

Read the original here:
New Research Suggests a Cure for HIV Could be on the Horizon - BioSpace

By Murielle Gonzalez 26-Sep-2019

Design and Build | Pharmaceuticals

Expansion follows the success of first six cleanrooms built at the large-scale manufacturing centre in Stevenage

The centre has been fully operational since April 2019 and achieved MHRA licensure in August 2019

The UK's Cell and Gene Therapy Catapult (CGT Catapult) has completed six additional modules of cleanroom space at its manufacturing centre in Stevenage. The first collaborators are expected to start benefitting from this new space at the beginning of 2020.

CGT Catapult said the centre has seen increasing demand, which accelerated the planned build-out of the expansion phase with an additional six cleanrooms in the already constructed space on the second floor of the building.

The centre has been fully operational since April 2019 and achieved MHRA licence in August 2019. Depending on the process, each module can accommodate 20 parallel autologous cell processes or a bioreactor process up to 1,000L bioreactors for allogeneic cell or viral vector manufacture.

Dr Ian Campbell, Interim Executive-Chair, Innovate UK, for UK Research and Innovation, commented: In its first year and a half, the Cell and Gene Therapy Catapult manufacturing centre has gone from strength to strength and has established itself as a crucial part of the UKs medicines infrastructure.

Each module can accommodate 20 parallel autologous cell processes or a bioreactor process up to 1,000L bioreactors for allogeneic cell or viral vector manufacture

"This world-leading centre is bringing manufacturing of pioneering treatments closer to patients and will complement the new Advanced Therapy Treatment Centres. By bridging the gap between scientific research and commercialisation of the medicines of the future, this centre, which we are proud to support, will play an ever-greater role in this vital sector.

At the centre, CGT Catapult provides the infrastructure and expertise to enable companies to develop their manufacturing capabilities and systems for large scale, commercial cell and gene therapy supply.

Companies currently collaborating at the centre are Adaptimmune, Autolus, Cell Medica, Freeline Therapeutics and TCR2 Therapeutics.

Located within the rapidly growing European cell and gene therapy cluster in Stevenage, the manufacturing centre, alongside wider CGT Catapult initiatives, reinforces the UK Governments ambition to support the rapidly growing global cell and gene therapy industry in the United Kingdom.

The centre is backed by over 75 million of funding, including investment from the UK Governments Industrial Strategy Challenge Fund; the department of Business Energy and Industrial Strategy, from Innovate UK, the UKs innovation agency, and from the European Regional Development Fund.

CGT Catapult was awarded an additional 3.36m in funding from the European Regional Development Fund (ERDF) and 12m Medicines Manufacturing Industrial Strategy Challenge Fund to further support this expansion.

We are very pleased to be able to double the existing capacity of the CGT Catapult manufacturing centre with help from our partners Innovate UK and the European Regional Development Fund," said Keith Thompson, CEO, Cell and Gene Therapy Catapult.

Thompson pointed out that the growth of this centre is integral to the success of the industry in the UK. "We can now collaborate potentially with up to a further six companies to support them in developing their GMP manufacturing processes, preparing for large scale and commercial supply," he said.

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Cell and Gene Therapy Catapult doubles capacity at UK site - Cleanroom Technology

WASHINGTON--(BUSINESS WIRE)--Greenleaf Health, Inc., a leading Food and Drug Administration (FDA) regulatory consulting firm, today announces that the firm has expanded its portfolio of services to guide companies developing cell and gene therapy products. Greenleafs team of regulatory experts will be led by Karen Midthun, M.D., former Director of the FDAs Center for Biologics Evaluation and Research (CBER), and John Taylor, former FDA Counselor to the Commissioner and Principal Deputy Commissioner.

REGULATORY LANDSCAPE: Cell & Gene Therapy

The rapidly evolving fields of cell and gene therapy offer the possibility of novel treatments, and perhaps ultimately cures, for devastating and intractable illnesses. In response to what the FDA has called a "turning point in the development of these technologies and their application to human health, new policies have been introduced to address the development of safe and effective cell and gene therapies.

With innovation often comes uncertainty. In the case of cell and gene therapy products, the FDA has raised concerns about developers operating outside of the existing regulatory paradigm. To prevent this, the FDA has clarified the regulatory framework for regenerative medicine products and announced near-term enforcement actions aimed at ensuring compliance by companies developing and manufacturing cell and gene therapies.

Greenleafs expanded services support companies striving to introduce cell and gene therapy products to patients. The firms team of experts has a robust blend of technical skill and FDA institutional knowledge that spans all therapeutic areas and quality, manufacturing, and compliance systems. By working cross-functionally, Greenleaf ensures that clients have the comprehensive, specialized support needed to understand and navigate the complex regulatory landscape for cell and gene therapies.

FULL-SERVICE SUPPORT

Members of Greenleafs Drug and Biological Products Team work together with the firms Product Quality, Manufacturing, and Compliance Team to deliver guidance on cell and gene therapy products.

Product Development & Review

With the expert direction of Karen Midthun, M.D., Greenleafs team of advisors assists sponsors of cell and gene therapies by optimizing FDA interactions and submissions to support development and regulatory review. Greenleaf also helps sponsors understand and respond to the FDA requirements applicable to various cellular products, and provides guidance to sponsors of cell and gene therapies to treat rare and ultra-rare diseases on ways to maximize trial design using appropriate clinical endpoints and natural history study data to aid efficient product development.

Quality, Manufacturing & Compliance

Greenleafs Product Quality, Manufacturing, and Compliance Team, led by John Taylor and supported by the firms network of independent compliance experts, offers credible, informed guidance to help manufacturers of cell and gene therapies comply with the FDAs multiple current GXP regulations. Greenleaf experts provide strategic and technical support for establishing manufacturing and quality controls; pre- and postapproval inspection readiness; compliance assessments; evaluating and responding to FDA regulatory correspondence; and engaging with CBERs Advanced Technologies Team.

UNMATCHED EXPERTISE

Greenleaf is comprised of experts with a combined total of more than 250 years of FDA experience. The firms team of advisors demonstrates unmatched levels of skill in its specialties of drug and biological products and product quality, manufacturing, and compliance. Greenleafs Cell and Gene Therapy Team, led by Dr. Karen Midthun and John Taylor, is guided by decades of regulatory experience in senior FDA positions, global public health organizations, academia, and industry.

Karen Midthun, M.D.Principal, Drug & Biological Products

Dr. Midthun contributes specialized insight informed by her regulatory, research, and clinical experience to FDA-regulated entities developing cell and gene therapies. Dr. Midthun joined Greenleaf following a distinguished 28-year career in public health, of which 22 years were dedicated to the FDA.

An infectious disease physician by training, Dr. Midthun most recently served as the Director of the FDAs Center for Biologics Evaluation and Research (CBER). During her FDA tenure, Dr. Midthun played a critical role in facilitating policy and technology development in the areas of cell, tissue, and gene therapies, blood products, and vaccines.

John Taylor, J.D.President, Greenleaf Health, and Principal, Compliance & Regulatory Affairs

Taylor has held many high-profile positions at the FDA, as well as senior leadership roles within industry. Taylors wealth of regulatory experience, robust technical skills, and unique strategic perspective are unmatched. Clients working with Greenleafs Product Quality, Manufacturing, and Compliance Team benefit from Taylors vast FDA institutional knowledge.

Taylor joined Greenleaf following a distinguished 20-year career at the FDA, where he served in multiple leadership positions, including as the FDAs Acting Deputy Principal Commissioner, FDA Counselor to the Commissioner, Acting Deputy Commissioner for Global Regulatory Operations and Policy, and Associate Commissioner for Regulatory Affairs.

ABOUT GREENLEAF

Greenleaf Health provides strategic and technical guidance to pharmaceutical, biotechnology, and medical device companies researching, developing, and manufacturing innovative solutions to pressing global public health challenges.

The firm includes former leaders and regulatory professionals from the FDA, Capitol Hill, top global pharmaceutical and medical device companies, leading law firms, and the top U.S. biotechnology trade organization. Greenleafs blend of former FDA officials and industry experts provides a unique set of capabilities when advising entities regulated by the FDA.

For more information on Greenleafs cell and gene therapy services and Greenleaf Health, visit greenleafhealth.com.

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Greenleaf Health Expands Services to Support Cell & Gene Therapy - Business Wire

RESEARCH TRIANGLE PARK Fennec Pharmaceuticals Inc. has appointed Jodi Cook to its board of directors the company announced in a statement Tuesday.

Cook is currently the head of gene therapy for PTC Therapeutics Inc., a global biopharmaceutical company. Before beginning her work at PTC, she helped found Agilis Biotherapeutics, a clinical-stage company focused on gene therapies, where she also served as chief operating officer.

Jodi Cook

On behalf of the board of directors, we welcome theaddition of Jodi Cook, saidKhalid Islam, Fennecs chairman, in a press release. She brings extensive scientific, clinical and executive business experience to the Company. Her background and track record of success will enhance our team as we further advance the commercialization and development strategy of PEDMARK.

PEDMARK is a therapy intended to help children with hearing loss as a result of platinum-based chemotherapy. There is currently no preventative measure for this type of hearing loss. Fennec aims to complete its NDA submission for the treatment in early 2020 with a potential commercial launch of PEDMARK in the second half of 2020.

I am delighted to be joining Fennecs board of directors at this key point of development for PEDMARK, said Cook in the press release. Cisplatin induced hearing loss is a significant unmet medical need, a solution to which has evaded medicine for decades.

This story is from the North Carolina Business News Wire, a service of UNC-Chapel Hills School of Media and Journalism.

Excerpt from:
Fennec Pharma names gene therapy exec to its board - WRAL Tech Wire

WEST CHESTER Nathanael Ogden is your average 13-year-old. He has a younger brother, Jackson. He likes to read and play Dungeons & Dragons, enjoys board games and logic puzzles, and is running for vice president of his eighth-grade class at Peirce Middle School.

He also has something the majority of his peers dont have: a white cane. Nathanael has lost 95 percent of his vision over the last 10 years. But theres one more important thing he has: the spirit of a fighter.

Before he was born, Nathanael was diagnosed with Bardet Biedl Syndrome (BBS,) a rare genetic disorder.

Never heard of Bardet Biedl Syndrome? You're not alone. It is a rare syndrome that affects about 1 in 250,000 people in North America. According to bardetbiedl.org, people with BBS have a defect in the way their cells communicate with each other. It's a complex syndrome with a wide range of symptoms and a lot of variation from person-to-person. However, usually, people with BBS have low muscle tone, impaired kidney function, hampered senses, and vision loss.

Nathanael's parents, Tim and Catherine Ogden, first became aware that something was wrong with their baby during a 26-week ultrasound. The scan revealed that his kidneys were three times larger than they should have been, which Tim says is also indicative of a fatal kidney disease.

"We were told he wasn't going to live," said Tim Ogden.

The couple went to Children's Hospital of Philadelphia for a follow-up and doctors concluded that the baby most likely had BBS.

"They couldn't say definitively, but they were like 'Yeah, we think we know what this is.' Honestly, it was a huge blessing because most people will spend years trying to get a diagnosis for their child. It made a big difference."

Enlarged kidneys weren't the only symptom of BBS for Nathanael.

"Cilia are essentially the radio antennas of our cells. They are responsible for sending and receiving signals from cell to cell," explained Tim.

"When they break down, messages get garbled, or they don't get passed and, his kidneys didn't stop developing. He was also born with 24 digits for the same reason. His body made a pinky but didn't receive the message to stop, so it made another one. He was born with six toes on each foot, and seven fingers on his left hand."

Nathanael also had to deal with poor muscle development, speech problems, and dietary issues. All things he works on with pure grit and determination, and the aid of occupational, physical, and speech therapists. Nathanael has also learned to read braille and how to use a cane to help him navigate while walking.

"You never find him feeling sorry for himself or discouraged. It's always just 'This is what I have to do. This is my next task'" Tim says of Nathanael.

And, preparing for the next task he is. On Sunday, Sept. 29, Nathanael and Tim will tackle the third annual Rocky Ride for Bardet Biedl Syndrome. The trek begins at Uptown Worthington in Malvern, and winds through the Chester Valley Trail to the Schuylkill River Trail concluding at the Philadelphia Museum of Art. And, of course, the journey would not be complete without a triumphant run up the famous art museum steps.

The money raised from the Rocky Ride goes to support the Clinical Registry Investigating Bardet Biedl Syndrome (CRIBBS.) The registry consists of slightly over 500 people in the world diagnosed with BBS and helps researchers to develop effective and targeted treatment for those with the syndrome.

In the first year, the Rocky Ride raised $25,000. In year two, it raised nearly $35,000.

Funds raised from the Rocky Ride also support gene therapy research at the University of Iowa.

"Researchers have identified 23 genes associated with BBS. When research first began, there was only one. Four of them account for roughly 80 percent of people with symptoms of BBS. Those four genes are now part of a standard genetic testing panel which allows for a much earlier diagnosis. The earlier the diagnosis, the earlier services can be secured, and a plan can be put in action," said Tim.

"All of the research that has been done is exciting. When Nathanael was born, it felt like there was nothing and now, what we've been able to do over 13 years ... It feels like there's a lot of hope for people now."

Preparing for the task of riding 30 plus miles is a challenge. Tim and Nathanael spent a good part of the summer in training. They use a tandem bike for each of their training rides, which last anywhere from 15 to 25 miles per trip. Physical activity is a crucial part of Nathanael's care. Another symptom of BBS is the body's inability to recognize when it is full from food, so his parents keep a close eye on his diet and physical activity. Nathanael takes medicine to control his appetite.

"We've ridden a total of 165 miles since August in preparation for the Rocky Ride," said Tim.

For Nathanael, he enjoys the time with his father and feels a strong sense of accomplishment. "I like being able to do the bike ride itself. It's nice to be able to do exercise such as this," said Nathanael.

One hundred sixty-five miles is a lot of ground to cover. How does he find the energy?

"I don't know," he laughed.

All of Nathanael's senses are degenerating to some degree, with vision loss being the most severe.

According to his father, in first grade, he could see. Nathanael is now legally blind. He does the majority of his schoolwork on a computer screen with assistive technology.

"All of it has to be magnified and high contrast. It gets harder and harder, and then the question is - at what point is it more work to have him be able to see the screen as opposed to transitioning him to braille?" said Tim.

The Ogdens are humbled by the community support they have received.

"The biggest thing for anyone in the community to do is just to be there. A big part of having a child with a rare disease is the isolation. The feeling that no one knows about this, that no one understands," said Tim.

Families from across the West Chester Area School District are joining them for the ride.

"We're going to be barreling down the Schuykill River Trail - all 30 of us in red T-shirts to show that Nathanael doesn't have to do this alone. It's a big deal."

One repeat rider is Joe DiAntonio, Nathanael's principal at Peirce Middle School. DiAntonio will be riding with his son, who is a seventh-grader at Stetson Middle School.

"Nathanael is so special to our school," said DiAntonio. "He is everything that is right about Peirce. We want to support him in any way that we can. His attitude is so positive and the way he interacts with people. You just want to be around him."

Despite Nathanael's positive attitude, do Tim and Catherine ever question why this happened to their child?

"Absolutely. You can't get away from that. He lost the lottery. It was this totally bizarre thing that my wife and I had these genes that we didn't know we had. It's hard as a parent. You always want the best for your child."

For Tim, it was important to find something that he could do with his son and for other families with BBS. He helped start and is currently the president of the Bardet Biedl Syndrome Foundation, which provides support for families affected by the syndrome in North America and around the world.

"We're not really raising money for Nathanael. We are raising money that's going to benefit people in the future who have this syndrome and so he gets the feeling that there is something he can do. It's not about what he can't do, but what he can do to make the world a better place and help others," said Tim.

Tim and Nathanael drew inspiration for the Rocky Ride from the Rocky movies. The main character's attitude of determination is ever-present in Nathanael.

"Rocky doesn't win that first fight, but he still does it," said Tim. "That's the message that I want for Nathanael."

Nathanael's moto? "Keep pushing through it and try to find ways to be happy."

For more information on Bardet Biedl Syndrome, or to donate to the Rocky Ride, visit http://www.bardetbiedl.org/rockyride.

See original here:
Eighth-grader fights for himself and others with rare genetic syndrome - Daily Local News

NetworkNewsWire Editorial Coverage

NEW YORK, Sept. 24, 2019 /PRNewswire/ -- Advances in biotechnology look to deliver promising new treatment options against breast cancer. Independent researchers have found that Genprex Inc.'s (NASDAQ: GNPX) (GNPX Profile) TUSC2 prevented tumor growth against triple-negative breast cancer. Eli Lilly and Company (NYSE: LLY) is focusing on those patients with the greatest need and, in the process, has developed a new breast-cancer treatment option. Roche Holding (OTCQX: RHHBY) is expanding the use of its biotech to both identify and treat patients with challenging breast cancers. AbbVie Inc. (NYSE: ABBV) has been rapidly increasing its research, targeting more than 15 different types of cancer. AstraZeneca (NYSE: AZN) has seen hopeful results against metastatic breast cancer as it works in collaboration with a Japanese firm.

To view an infographic of this editorial, click here.

Battling Breast Cancer

Breast cancer is one of the most widely prevalent cancers in the world. Accounting for a quarter of all cancers in women worldwide, as well as a number of cases in men, it kills over half a million people every year.

Treatment for breast cancer varies, based on both the patient's circumstances and type of cancer. The development of treatments to suppress or even eliminate cancer has led to a high survival rate in wealthy countries, with around 85% of patients in the United States and United Kingdom surviving for at least five years from diagnosis. But even in these countries, survival depends upon the exact form of the cancer, how far it has progressed and whether a treatment has been developed for that particular form. Cancer is a difficult disease to defeat, and survival can depend upon the ability of scientists to counter a specific genetic defect in a specific set of cells.

Good News for Triple-Negative Breast Cancer Patients

Because cancers are so varied and challenging to tackle, every individual win is worth celebrating. That's why recent news relating to triple-negative breast cancer (TNBC) has brought excitement both for independent researchers and for Genprex Inc. (NASDAQ:GNPX), a clinical-stage, gene-therapy company whose tumor suppressor candidate 2 (TUSC2) was found to prevent tumor growth in TNBC.

TNBC covers a variety of cancers that do not express three specific types of receptor proteins. Most hormone therapies for breast cancer target one of these receptors, so tackling a cancer that doesn't feature any of those receptors is more difficult. TNBC is an extremely aggressive subtype of breast cancer associated with poor prognosis and high mortality rates. The lack of targeted treatment for triple-negative breast cancer makes it a particularly feared diagnosis. Because up to 20% of breast-cancer patients are fighting TNBC, finding therapies that effectively fight these forms of cancer is essential.

Recently TNBC patients have heard good news, thanks to research published in Nature. This research shows that TUSC2 prevented the growth of tumors in TNBC. And with Genprex already working on cancer treatments using TUSC2, including its lead drug candidate, Oncoprex(TM) immunogene therapy, the new research appears to validate the company's focus and direction.

Currently Genprex is conducting clinical and preclinical research to evaluate the effectiveness of TUSC2 when combined with targeted therapies and immunotherapies for non-small cell lung cancer. Existing preclinical data also suggest that TUSC2 may be effective against glioblastoma, head and neck cancer, kidney cancer and soft-tissue sarcomas. This new independent study raises the possibility that TUSC2 expression may also be used to treat this most aggressive subset of breast cancer.

"The results of the study evaluating TUSC2 for the treatment of triple-negative breast cancer are encouraging," said Genprex chairman and CEO Rodney Varner. "We believe that the data reported in this Nature article by independent researchers supports our belief that TUSC2 may be effective to treat a variety of cancers, including some of the most-deadly types of cancer."

This new report is a particularly encouraging moment for Genprex, as the study doesn't come from the company's own research work but instead verifies its value through an independent source. With multiple teams and researchers in multiple locations all evaluating and studying TUSC2 as a potential source for cancer treatment, the evidence is mounting that this approach may provide something new and valuable for doctors and patients.

Gene Therapy to Tackle Breast Cancer

Like many of the most promising cancer treatments, Genprex's TUSC2 treatment is a form of gene therapy.

A technology less than 50 years old, gene therapy remains on the cutting edge of modern medicine. The approach involves delivering new genetic material into the patient's body, where it is absorbed by cells. This rewrites the code of those cells, changing how they grow, reproduce, spread and die.

The growth and death of cells is fundamental to the challenge of cancer. Damaged genetic material leads cancer cells to develop in harmful ways and spread through the body, sometimes at a rapid rate. Treatments such as Genprex's Oncoprex can be used to write over the harmful DNA code and introduce a new gene. The result is that cancer itself is rewritten to reduce its harm.

Genetic treatments vary in the way they affect the body's cells. Some directly destroy cancerous cells. Others slow their reproduction, thereby reducing the spread of unhealthy tissue throughout the body. Other treatments make cells more susceptible to forms of treatment that might otherwise be ineffective.

TUSC2 treatment falls into both categories, helping cancer dells to die and preventing the growth of cancerous tumors. If successfully used, this approach could halt the growth of existing cancers and their spread through the body. TUSC2 is a vital element of treatment if cancer is to be prevented from running rampant through patients.

Genprex's Oncoprex therapy delivers cancer-fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles. The nanovesicles are then administered intravenously. They flow through the bloodstream until they locate and are absorbed by the cancer cells. The TUSC2 gene then expresses proteins that are missing or in short supply, bringing the damaged cells closer to normal behavior. By rewriting the very fundamentals of the body, such treatments may save lives.

Disruptive Technology for the Medical Sector

"Disruptive" isn't a term that's often applied to medicine, where the aim is to avoid disrupting the health of the human body. But technology such Genprex's Oncoprex immunogene therapy is disruptive precisely because it could improve doctors' ability to preserve health. It may also change the course of cancer, as new technology often opens up new possibilities.

Since its first successful use in the 1980s, gene therapy has rightly been presented as a disruptive technology. It deals with health on a basic level, altering the building blocks of life. This is why Genprex was among the companies presenting at the 4th Annual Disruptive Growth Conference in New York this September. Oncoprex's multimodal mechanism allows it to tackle cancer in a number of different ways, reducing the cancer's spread, encouraging the death of cancer cells and modulating the response of the immune system to fight cancer. It can block mechanisms that cause resistance to other anti-cancer drugs and so increase the effectiveness of a broader course of treatment.

As both treatments and the business models of companies behind them develop, the battle against cancer is being transformed.

Founded more than 140 years ago, Eli Lilly and Company (NYSE:LLY) is one of the oldest players in the field of cancer treatment, a company that has spent more than a century making medicines to help people around the world. Lilly uses predictive and prognostic biomarkers to work out how a tumor is likely to behave and how it will respond to potential therapies. This approach allows more targeted treatment designed to tackle the patient's specific cancer, thereby increasing the likelihood of success in any given case. LLY's Verzenio treatment has been shown to be effective in cancer treatment, encouraging cell death and reducing the spread of tumors. The company's strategy is a targeted one, concentrating on those cancer patients with the greatest need, and so providing the greatest possible impact from its medicines.

Another long-established company, Roche Holding (OTC:RHHBY) was one of the first to provide targeted treatments such as those offered by gene therapy. The world's largest biotech company, Roche has poured a huge amount of resources into biopharmaceuticals and is a global leader in cancer treatments. The company recently announced the expanded use of its technology to identify TNBC patients and so to provide them with the targeted treatment they need to fight their cancer. The company's support for cancer patients extends beyond better medicine for those in wealthy countries. Roche is a supporter of a health-care approach in South Africa that takes treatment to the rails, providing mobile health-care facilities and services that include breast cancer screening for people who might otherwise lack access.

A research-driven biopharmaceutical company, AbbVie Inc. (NYSE:ABBV) is working on new treatments for more than 15 different cancers, including forms of breast cancer. The company's wide-ranging work means that insights from one area can affect work in another, allowing greater progress in the overall understanding of cancer and the development of specific, targeted treatments. AbbVie's oncology research has increased dramatically over the past six years, often through partnerships with other companies, allowing a rich cross-fertilization of ideas.

Based in the United Kingdom, AstraZeneca (NYSE:AZN) benefits from its proximity to some of the finest minds in the world and an elite recruitment pool. Like AbbVie, AstraZeneca has been making breakthroughs in a wide range of cancer types. Research in collaboration with a Japanese company has recently led to promising results for the treatment of metastatic breast cancer, as the company works to improve survival rates and quality of life for breast cancer patients.

With the attention of so many big players in the biopharmaceutical industry, promising treatments for cancer are developing fast.

For more information about Genprex, please visit Genprex Inc. (NASDAQ:GNPX).

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Gene Therapies with Potential to Conquer Tough-to-Tackle Breast Cancers content= - PR Newswire UK

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Synteract, an innovative CRO partnering with biotech and pharma companies to bring new medicines to market, has hired two experienced leaders to further strengthen its Centers of Development in Oncology and Rare/Orphan diseases. Given the enhanced strategic solutions that often result from the intersecting expertise of Synteracts centers of development, clients will derive further benefit from the therapeutic knowledge and clinical development acumen gained from these key additions.

Hassan Aly, M.D., Ph.D., has joined Synteract as senior medical director supporting Synteracts Oncology Center of Development and Derek Ansel has joined as director of Rare and Orphan Disease Development. Both are clinical strategists with multidisciplinary experience in developing and operationalizing global therapeutic strategies.

Rare disease, oncology, and hematology clinical trials pose unique challenges that require the ability to think broadly while applying very specific trial strategies, said Steve Powell, Synteract CEO. Both Hassan and Derek bring a wealth of knowledge, expertise, and creativity, coupled with a passion to help patients and families, that will help us to further advance trials in the most complex and acute areas of need.

Dr. Aly is a certified hematologist with over 20 years experience in oncology/hematology medical research and patient treatment, including nine plus years in the clinical research and pharmaceutical industry. He brings deep therapeutic expertise across all phases of oncology/hematology drug development, having consulted on complex study design, conduct, monitoring, and submission. Prior to joining Synteract, he worked at AstraZeneca, Boehringer-Ingelheim, and leading CROs.

In addition, Dr. Aly has authored or co-authored 105 papers on the treatment and biology of malignant hematological diseases in peer-reviewed medical periodicals and made more than 70 presentations at British and European conferences.

Prior to joining Synteract, Derek Ansel was a clinical strategy lead at the Center for Rare Diseases and a co-founder of the Gene Therapy Working Group at a leading CRO. Ansel has a breadth of clinical research experience spanning from the laboratory through clinical monitoring and project management, across Phases I-IV. He will work in a multidisciplinary, cross therapeutic team, where he will apply extensive gene and cell therapy expertise developed from his work in several liver-targeted gene therapy programs.

Ansel frequently speaks at industry events. He is a member of the ACRP and holds a Certified Clinical Research Associate (CCRA) credential. He is an IRB member at the Thomas Jefferson University in Philadelphia and is also a contributing member of Cochrane, a non-profit organization that promotes evidence-based decision making in healthcare.

Dr. Frank Santoro, chief medical officer for Synteract, commented, Hassan and Derek bring unique skillsets to our centers of development with a combination of medical, therapeutic, and operational know-how related to running complex trials. This expertise, along with their crossover experience in multiple therapeutic areas, offers exceptional insight to our clients looking to partner with Synteract for their clinical development needs.

About Synteract

Bringing Clinical Trials to Life represents Synteracts commitment to engage with drug developers, patients, investigators, and regulatory experts to bring insights to action and make better therapies a reality. Synteract supports biotech and pharma companies across all phases of drug development to help bring new medicines to market. Synteract has conducted nearly 4,000 studies on six continents and in more than 60 countries. It has contributed to more than 240 product approvals. Synteract offers notable depth of therapeutic expertise in oncology, dermatology, general medicine, neuroscience, pediatrics, and rare and orphan diseases. Connect on LinkedIn and Twitter.

Original post:
Synteract Augments Leadership Team for Oncology and Rare Disease Centers of Development - Business Wire

-- Webcast conference call to be held on Friday, Oct. 4, 2019 at 8:00 a.m. Eastern Time --

CAMBRIDGE, Mass., Sept. 25, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (SRPT), the leader in precision genetic medicine for rare diseases, today announced that on Friday, Oct. 4, 2019 at 8:00 a.m. Eastern Time (ET), it will host a webcast and conference call to present the 9-month functional results from the SRP-9003 gene therapy trial to treat Limb-girdle muscular dystrophy Type 2E, or beta-sarcoglyanopathy.

The presentation will include slides and will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-and-presentations and will be archived there following the call for one year. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 4986359. Please specify to the operator that you would like to join the "Sarepta hosted LGMD results call."

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics to Announce 9-Month Functional Results from the SRP-9003 Gene Therapy Trial to Treat Limb-girdle Muscular Dystrophy Type 2E, or...

For the latest events and activities happening in Greenwich, turn to For the record. To have your event included, submit a description, date, time, price and contact information. Photos are welcome. Drop us an email about your latest goings-on at gtcitydesk@scni.com.

Try Tai Chi

Tai Chi is a relaxing exercise that can loosen joints, improve balance and teach graceful movements to music. Fun and no pressure classes are held at 9 a.m. Thursdays in the auditorium at the First Congregational Church Auditorium on Sound Beach Avenue in Old Greenwich. The cost is $10 per one-hour session. Newcomers welcome. For info, call Joe at 203-504-4678.

Special Education parent forum

The Stamford JCC will present a Special Education Parent Forum: Pursuing a Specialized School Placement at 7 p.m. Thursday at 1035 Newfield Ave., Stamford. Free and open to the public. Registration is recommended with Liza Fahey at lfahey@stamfordjcc.org or 203-487-0946. Workshop speakers will include Lawrence Berliner, special education law attorney in Westport; and Clay Kaufman, head of school, and Sue Rappaport, assistant director of admissions and placement, at Eagle Hill School in Greenwich.

Farm to Table Dinner

Chef Geoff Lazlo brings his Farm to Table Dinner series to his hometown of Greenwich at the Greenwich Botanical Center, 130 Bible St., Cos Cob. The elegant four-course dinner kicks off with a signature cocktail and passed hors doeuvres as guests are treated to highlights of the propertys magnificent history, followed by a seated dinner featuring key ingredients of herbs and vegetables locally grown. Dinners will be from 6 to 9 p.m. on Sept. 26 and Oct. 3. For more information and tickets, visit greenwichbotanicalcenter.org.

Qigong at the GBC

Qigong expert Donna Bunte teaches classes at 10 a.m. Fridays at the Greenwich Botanical Center, 130 Bible St., Cos Cob. With roots in Chinese medicine, philosophy and martial arts, qigong is viewed as a practice to cultivate and balance qi (chi), translated as life energy. Qigong practice involves moving meditation, coordinating slow flowing movement, deep rhythmic breathing, and a calm meditative state of mind. For information, visit greenwichbotanicalcenter.org.

Abilis Has Talent Show

The third annual Abilis Has Talent Show is a star-studded evening fun for all ages. The event is held at the First Presbyterian Church at 1 W. Putnam Ave., Greenwich, on Friday. Tickets are $25 with advance reservation or $30 at the door. Register at abilis.us/calendar. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth through adulthood.

Enjoy Oktoberfest

Enjoy beer and brats for Oktoberfest on Friday at the St. Lawrence Club. Its one of the favorite Family Night Dinners as Chef Tony prepares an authentic German-style feast with beer & cheese soup, schnitzel, sauerkraut, potato salad and strudel. Cost is $25 for adults, $10 for kids. No tax, no tip. Society members get a free drink. The St. Lawrence Club is at 86 Valley Road, Cos Cob. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Bruce closes temporarily

The Bruce Museum will then be closed through Friday to facilitate work in the main galleries. When it reopens Saturday, admission will be free to all visitors through Jan. 31 while the main gallery spaces are renovated. During the renovation phase, the Permanent Science Gallery will remain open, as will the Bantle Lecture Gallery, Education Workshop, and Museum Store. The galleries will reopen Feb. 1, with the installation of major new art and science exhibitions. Visit BruceMuseum.org for more information.

Farmers Market

The Greenwich Farmers Market runs every Saturday from 9:30 a.m. to 1 p.m. into the fall in the Arch Street commuter lot. Enjoy fresh Connecticut-grown produce all season. Over a dozen farm vendors will be in attendance. The parking lot at Arch Street and Horseneck Lane is off Exit 3 of I-95. For more information, visit http://www.greenwichfarmersmarketct.com/.

Cocktails & Comedy

There will be a cocktails and comedy fundraiser to venefit The Undies Project from 7 to 10 p.m. Saturday at St. Catherine of Siena in Riverside. To purchase tickets and for more info, visit e.givesmart.com/events/dWy/.

Greenwich Symphony Orchestra opens season

The Greenwich Symphony Orchestra will feature five concerts during its 62nd season, beginning Saturday and Sunday. The opening concerts will feature Bartok, Divertimento for Strings; Dittersdorf, Concerto for Harp, with Barbara Allen on harp; Wieniawski, Violin Concerto No. 2, with Edita Orlinyte on violin; and Debussy, Iberia. Concerts are at the Greenwich High School Performing Arts Center, 10 Hillside Road, at 8 p.m. on Saturdays and 4 p.m. on Sundays. Tickets are $40 per person, $10 students. For more information, call 203-869-2664 or visit http://www.greenwichsymphony.org.

Puttin on the Dog

Puttin On The Dog, the popular annual fundraising event for Armonk-based Adopt-A-Dog, will be held from 10 a.m. to 4:30 p.m. Sunday in Roger Sherman Baldwin Park in Greenwich. Dogs and cats will be available adoption that day. The event will also feature a silent auction, childrens activities, pet demonstrations, vendors and food trucks as well as canine competitions. Dogs are welcome, as well as people of all ages. For more information about the organization, visit adopt-a-dog.org, call 914-273-1674 or email adoptions@adoptadog.org.

Springsteen at Seventy

The C. Parker Gallery presents Growin Up Bruce Springsteen at 70, a 70th birthday celebration featuring rare photographs, artwork, gold records and signed memorabilia showcasing The Boss. The exhibit will run through Sept. 29 at the gallery at 409 Greenwich Ave. Highlights include rare unpublished 1972 to 1975 photographs from Bruces earliest days performing in the Philadelphia area from the late Phil Ceccola. Debra Rothenberg, author of Bruce Springsteen In Focus 1980 2012, will showcase her photographs. Free. All works are available for purchase. To learn more, visit http://www.cparkergallery.com.

Archery open shoot

The Cos Cob Archers will hold an Open Shoot from 8 a.m. to 1 p..m. Sept. 29 as they open their club to the public and invite all to attend. Open Shoots are held once a month, generally on the last Sunday of each month; bad weather cancels. The club provides loaner compound bows, arrows and instruction for beginners. The cost $20 for shooting adults, $5 for kids under 16, and $10 for non-shooting adults includes lunch of grilled hamburgers, hot dogs and soft drinks. The Clubs trails cover 23 wooded acres and there are more than 40 shooting stations, with both paper and 3D targets. Cos Cob Archers is located at 205 Bible St. For more information, visit http://www.CosCobArchers.com.

Meters for a Cure ERG Challenge

Swim Across America Fairfield County and Greenwich Crew are teaming for the second annual charity rowing event Meters for a Cure ERG Challenge with proceeds supporting Swim Across America Fairfield County and its local beneficiary, the Alliance for Cancer Gene Therapy, the nations only foundation dedicated exclusively to funding cancer gene therapy research. On Sept. 29, hundreds of crew members, their families and friends, will come together to help raise money for cancer research at the Greenwich Water Club at 49 River Road, Cos Cob, and relay against each other on ERG machines (a rowing machine also known as an ergometer). Teams are divided by age group and each participant is asked to raise at least $100. Open to all. Register at swimacrossamerica.org/gwc2019 or on-site that day starting at 7:30 a.m.

Choral Evensong

Join Christ Church Greenwich in celebrating Saint Michael and All Angels with music by Rose, Sumsion, and Bainton sung by the Choir of Men & Boys with members of the St. Cecilia Choir of Girls. The concert will be at 5 p.m. Sept. 29 at the church at 254 E. Putnam Ave. Admission is free. Visit christchurchgreenwich.org/music/ for more information.

Duplicate Bridge Games

Weekly open duplicate Bridge games are held at 12:15 p.m. Mondays at the Greenwich YWCA. The games are sanctioned by the American Contract Bridge League, with masterpoint awards to top finishers. The card fee to play one session is $12. For more information, contact Steve Becker at 203-637-8927.

Exhibit of Bird Mobiles

The Greenwich Art Society Gallery will present a show called Bird Mobiles: Suspension Of Time through Sept. 30 at the Arts Council Building at 299 Greenwich Ave. The mobiles were created by local artist Susan McHale, who creates her art with discarded branches found on the beach. The process of making my Bird Mobiles is like catching an ephemeral moment suspended in time, the hanging flock fly by and seem to continue their journey, she says. Call 203-629-1533 for more information.

Error art installation

The Brant Foundation Art Study Center is presenting Urs Fischer: Error, a solo exhibition of works by Fischer from the last two decades, through Oct. 1 at 941 North St. Featuring some of the artists most notable large-scale sculptures and paintings from the Brant Collections, Error celebrates The Brant Foundations 10th anniversary at its Greenwich space with Urs Fischer, the first artist to present a solo exhibition at there in 2010. To book a tour, visit brantfoundation.org/exhibitions/error/.

ASOG Fall Art Show

As summer fades and fall beckons, the Art Society of Old Greenwich is preparing for its 2019 ASOG Fall Art Show. The exhibit takes place at the Greenwich Botanical Center in Cos Cob from Oct. 1 to Oct. 28, with an artists reception on Oct. 6 from 3 to 5 p.m. Visitors can view the artwork, mingle with the artists, listen to live music and sample refreshments. The Greenwich Botanical Center is open 9 a.m. to 4 p.m. weekdays.Free parking on premises. For more information, contact artsocietyoldgreenwich@gmail.com or visit http://www.asogct.com/fall-show.

Abilis Gardens & Gifts

Starting in the fall, Abilis Gardens & Gifts will be selling holiday gifts and its greenhouse will be full of holiday wreaths and greenery. It is must-shop store offering handcrafted candles, bath products, childrens gifts, jewelry, housewares and gifts, with fresh flowers, plants and micro greens in the greenhouse. Prices range from $5 to $40 and proceeds support Abilis programs. Abilis Gardens & Gifts provides job training for adults with special needs. Hours are 10 a.m. to 5 p.m. Mondays, Wednesdays and Fridays; 10 a.m. to 3p.m. Tuesdays; 10 a.m. to 7 p.m. Thursdays; and 10 a.m. to 2 p.m. Saturdays for October, November and December. Abilis Gardens & Gifts is at 50 Glenville St. and can be reached at 203-531-GIFT (4438). For more info, visit http://www.abilis.us.

An American Story

The Greenwich Historical Society will present a new exhibition on the immigrant experience called An American Story: Finding Home in Fairfield County from Oct. 2 to Jan. 6. The exhibit will include poignant stories of the grit and resilience of immigrants and refugees, including 12 who found home in Greenwich. An American Story is a celebration of the determination, resilience and courage of the human spirit. The stories illuminated in this timely exhibition reach across the world from five continents, shining a light on the ways that refugees and asylum seekers find hope and persevere in the face of daunting challenges for creating new lives in Fairfield County. The landmark exhibition is presented in partnership with the Connecticut Institute for Refugees and Immigrants. The Greenwich Historical Society is located at 47 Strickland Road, Cos Cob. For more information, visit greenwichhistory.org.

Talk on The Bottom of the World

The Bruce Museum Presents, a new series of monthly public programs featuring thought leaders in the fields of art and science, showcases experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The series continues with an event presented in cooperation with Columbia Universitys Lamont-Doherty Earth Observatory called From the Bottom of the World: The Art and Science of Antarctica from 6 to 8:30 p.m. Oct. 3. It will convene experts in the art and science of this unique and fragile continent. Two research scientists from Lamont-Doherty, Jonny Kingslake and Kirsty Tinto, will reveal their new findings, while photographer Rick Sammon, who has twice visited and documented the Antarctic landscape, will remind us of its beauty. Leonard Jacobs moderates. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-869-0376.

Breast Cancer Awareness

To kick off Breast Cancer Awareness Month, Greenwich Town Hall will raise the Breast Cancer Alliances flag at 9 a.m. Oct. 3 with First Selectman Peter Tesei; Dr. Barbara Ward, director of breast care services at Greenwich Hospital, and Mary Jeffery, president of the BCA. The event will continue as Richards of Greenwich hosts the BCAs GoForPink breakfast called Love, Loss and Cancer: An Intimate Conversation with authors Lee Woodruff and Allison Gilbert at 10 a.m. Oct 3. The organizations annual luncheon, which takes place at 11 a.m. Oct. 21 at the Hyatt Regency Greenwich, will feature celebrity chef and restaurateur Todd English. For more info, go to http://www.BCAgoforpink.org, contact Info@breastcanceralliance.org or call 203-861-0014.

Dinner With a Cause

The Social Justice Committee of the Parish of St. Catherine of Siena and St. Agnes is sponsoring its Dinner with a Cause from 6:30 to 8:30 p.m. Oct. 3 at the Church Hall. The featured guest is Cathleen Caron, founder of Justice in Motion, a nonprofit dedicated to service the needs of migrant workers. Come for a light dinner at 6:30, presentation at 7:15 followed by Q & A. Free will offering to benefit Justice in Motion. For information on the nonprofit, visit https://justiceinmotion.org/about-us. RSVP to parkerelizabeth@sbcglobal.net or 203-637-8092.

Outdoor Arts Festival

More than 80 new and returning artists will showcase their work on the grounds of the Bruce Museum on Oct. 5 and Oct. 6, as the Greenwich museum celebrates the 38th edition of its annual Outdoor Arts Festival. The event will feature juried works, including paintings, mixed media in 2D and 3D, drawings and graphics such as digital media, sculpture and photography. There will be food-truck offerings and free art activities for children. Free parking offered in all nearby municipal lots. Festival admission is free to Bruce Museum members and children less than 5 years old; entry is $10 for non-members. Festival-goers can also tour the museum. Fore more info, visit BruceMuseum.org.

YWCAs walk against domestic violence

On Oct. 6, YWCA Greenwich is hosting a communitywide walk to raise awareness and funds for YWCA Greenwich Domestic Abuse Services and education. The family-friendly event takes place at Bruce Park. Registration will open at 8:30 a.m. Members of the community are encouraged to form teams, come with friends and family, and even dogs are welcome. To preregister, go to ywcagrn.org/walkthewalk. Proceeds will support the work of YWCA Greenwich Domestic Abuse Services. October is Domestic Violence Awareness and Prevention Month. YWCA is the state-designated provider of domestic abuse services for victims of domestic violence in Greenwich. If you or someone you know is a victim of domestic abuse, call the YWCA 24/7 hotline at 203-622-0003. For more information about the walk, contact Jackie Stam at 203-869-6501 x102 or j.stam@ywcagreenwich.org.

Teddy Bear Clinic

The Teddy Bear Clinic Greenwich Hospitals largest community event will celebrate its 21st anniversary from noon to 3 p.m. Oct. 6 at 49 Lake Ave. in the Greenwich Medical Building parking lot behind the hospital. Geared for children ages 3 through 12, this popular free event will take place rain or shine. Children and their families are invited to experience the friendly face of medicine at this event, staged as a mini-hospital under the tents. Visit with Greenwich Hospital doctors, nurses, technicians and other staff during this fun, interactive day of learning. Peer through a microscope, climb aboard an ambulance, check out the X-ray machine and test your balance by negotiating an obstacle course. Families can learn about nutritious food, strong bodies, sun protection, sports safety and other healthy habits. Children are encouraged to bring a favorite stuffed animal or doll for an exam. Depending on the diagnosis, dolls, bears and other fuzzy patients may receive splints, stitches, surgery or X-rays. Free parking in the hospitals staff lot on Lake Avenue. For information, call 203-863-3627.

First Sunday bird walks

A First Sunday Bird Walk will be held at Greenwich Point Park from 9 to 11:30 a.m. on Oct. 6, Oct. 13 and Oct. 20 to see the migrating hawks and raptors. Bring binoculars and meet near the main concession stand at the south end of the beach. The Bird Walks are a series of free, year-round, monthly bird walks providing friendly and informative birding. Guides are knowledgeable local naturalists and birders. Everyone from beginner to expert, of all ages is welcome. No fees or registration are required. For more information, visit birdwalk.home.blog/. Co-sponsored by Wild Wings and Friends of Greenwich Point.

Lecture on Classical Americana

The Greenwich Decorative Arts Society will present Classical Americana: The Life and Legacy of Richard Hampton Jenrette, a lecture by Peter M. Kenny, co-president of the Classical American Homes Preservation Trust. The event will be from 1:15 to 3 p.m., with refreshments to follow, on Oct. 7 at the Bruce Museum. Insights will be offered into the numerous houses he restored, his approach to their interior decoration and his role as a collector of classical American furniture from the workshops of New Yorks premier cabinetmakers of the early 19th century. Kenny was the Ruth Bigelow Wriston Curator of American Decorative Arts and Administrator of the American Wing at the Metropolitan Museum. Admission for nonmembers is $25. Space is limited. Reservations required for members and guests by Sept. 30. For reservations and information: email greenwichdecorativearts@gmail.com or visit http://www.greenwichdecorativearts.com.

Blood drives

The American Red Cross urges people of all races and ethnicities to give blood or platelets to help increase the diversity of the blood supply. The vast majority of blood types fall into one of the major blood groups. However, for patients with rare blood types or those who receive regular blood transfusions, blood must be matched closely - beyond the primary A, B, O and AB blood types - to reduce the risk of developing complications from transfusion therapy. The best match may be someone of the same racial or ethnic group. That includes patients who need blood transfusions to treat complications from sickle cell disease, a genetic disease that is most common among people of African descent or Latino descent. Local blood drives will be held from 1:30 to 6:30 p.m. Oct. 7 at Christ Church Greenwich, 254 E. Putnam Ave., and from 9 a.m. to 2 p.m. Oct. 8 at the Boys & Girls Club of Greenwich, 4 Horseneck Lane. Appointments can be made by downloading the free Red Cross Blood Donor App, visiting RedCrossBlood.org, or calling 1-800-RED CROSS (1-800-733-2767). As a thank-you, donors who give blood or platelets during October will be entered for a chance to win one of five $500 gift cards.

Outdoor movie night

As part of the Greenwich Reads Together program, there will be an outdoor screening of the movie Fahrenheit 451 at 7:30 p.m. Oct. 12 on the front lawn of the Greenwich Arts Council at 299 Greenwich Ave. Legendary French director Franois Truffaut brings to life a terrifying care-free future in which Guy Montag (Oskar Werner) questions his actions after meeting Clarisse (Julie Christie) and begins to rebel against society. Free and open to all, but registration is encouraged at http://www.greenwichlibrary.org. For more information, call 203-862-6750 or visit greenwichartscouncil.org. Co-sponsored by Alliance Francaise of Greenwich, The Avon Theater, The Commission on Aging, Greenwich Arts Council, Greenwich Library, and Sebass Events & Entertainment.

Honoring Columbus

The St. Lawrence Society will celebrate Columbus Day on Oct. 14 with an event honoring First Selectman Peter Tesei. Festivities begin with a 9 a.m. flag raising at Town Hall, followed by a breakfast at The Club and ending with cocktails, presentation of awards and an elegant Italian dinner back at the Club at 6:30 p.m. The cost for dinner is $100 per person. The St Lawrence Club is 86 Valley Road, Cos Cob. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Lush Landscapes benefit

Landscape designer James Doyle will present a program on Dream Locations and Special Places at 11:30 a.m. Oct. 17 at the Belle Haven Club as a benefit for Community Centers Inc., a Greenwich-based social service agency. Doyle, founder and principal of Greenwich-based Doyle Herman Design Associates, has developed an award-winning design business with projects in the United States, Europe and the Middle East. His work combines his horticultural expertise with strong design philosophy to bring to life unique and innovative landscapes. Projects range from small courtyards to large country estates. CCI, which has been serving the community for 65 years, is dedicated to building skills that empower clients to overcome educational, social and economic barriers. Tickets may be purchased on the CCI website at ccigreenwich.org.

Walk/Run for Abilis

Abilis will host its 15th annual Walk/Run for Abilis on Oct. 20 at Greenwich Point Park. This fun family-friendly event includes childrens activities, a Bubble Bus, music, arts & crafts, a 1-mile wheelchair and stroller-accessible walk and a 5k run. This event is one of Abilis largest fundraiser events of the year and attracts hundreds of participants for a morning of fun. Registration is $40 for adult runners; $20 for child runners (ages 11 to 17) and free for children under 11. There is no cost for the walk portion of the event. To register in advance, donate and or volunteer, visit abilis.us/walkrun. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth throughout adulthood in the area.

Model sailboat regatta

The Old Greenwich-Riverside Community Center will hold its popular annual Model Sailboat Regatta from 1 to 4 p.m. Oct. 20 at Binney Park. Model sailboat enthusiasts of all ages are welcome, with categories for homemade boats, motorized and remote-controlled boats spread out throughout the afternoon. Hundreds of families are expected to attend. Many families set up a picnic for the day as kids run around and enjoy Binney. The OGRCC provides arts and crafts activities and music. The regatta raises money for the Scholarship Program that supports over 70 families in the community each year for everything from soccer to childcare to summer camps. To buy a ticket, visit myogrcc.org. Walk-ins are welcome. For more info, call 203-637-3659.

TAGs grand prix fundraiser

The Transportation Association of Greenwich will hold its fall fundraiser, the Greenwich Grand Prix, from 3 to 5 p.m. Oct. 27 at the RPM Raceway in Stamford. The proceeds of the go-kart racing event will be used to purchase a new 20-passenger vehicle with wheelchair lift. The go-kart racing teams are from Greenwichs top car dealers, who will be competing for the Greenwich Grand Prix Championship. The community is invited to cheer on the teams and support TAG to raise these much-needed funds. Tickets are on sale now. For more information on tickets, teams, donations and sponsorship, visit ridetag.org/greenwich-grand-prix.

Nostalgia dining

A Remember Maneros Steak Dinner will be held at 6:30 p.m. Nov. 1 at The St. Lawrence Club, 86 Valley Road, Cos Cob. Join the club and reminisce over the beloved and sorely missed Greenwich landmark restaurant. Diners can linger with family and friends over a mouth-watering meal of Steak Tid-bits, garlic bread, fried onions, gorgonzola salad and more. Theres no tax or tip, and members of the St. Lawrence Society get a free drink. Cost is $40 for members, $45 for guests and $25 for kids. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Celebrating documentaries on artists

The Bruce Museum Presents, a new series of monthly public programs featuring thought leaders in the fields of art and science, showcases experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The series continues with Rewind/Fast-Forward: Celebrating the Artist Documentaries of Olympia Stone from 6 to 8:30 p.m. Nov. 7. Stone is an independent producer of documentary films about art and artists. Her first documentary, The Collector, explored the five-decade career of her father, famed NYC gallery owner and art collector Allan Stone. For the event, join Stone and two of her artist subjects James Grashow (The Cardboard Bernini) and Elizabeth King (Double Take: The Art of Elizabeth King) for a retrospective of these films, and their maker. Moderated by Leonard Jacobs. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-8690376.

Bacon Brothers perform for charity

The Bacon Brothers, a band started by the award-winning actor Kevin Bacon and his brother Michael, an Emmy-winning composer, will perform a rare intimate concert in Greenwich on Nov. 9 at a fundraiser for Fairfield Countys Inner-City Foundation for Charity & Education. The event, the 28th annual fall gala to benefit The Inner-City Foundation, will be held at the Hyatt Regency in Old Greenwich. It is the largest fundraiser of the year for the Inner-City Foundation, which provides support to local Fairfield County programs and organizations that provide education, food, shelter, help for addiction, domestic violence and much more to the neediest in Fairfield County. The band also includes Paul Guzzle (bass, backing vocals), Joe Mennonna (keyboards, accordion), Tim Quick (lead guitar, mandolin and backing vocals) and Frank Vilardi (drums). For more information and tickets, tables or sponsorships, visit innercityfoundation.org or call 203-416-1363.

Sip & Shop Art Show

Stop by the Holiday Sip & Shop Art Show at Abilis Gardens & Gifts from 5 to 8 p.m. Dec. 4. Enjoy wine, cheese and other refreshments while shopping with a 20 percent discount in the entire store. The Art Show will feature paintings and digital art created by adults who are supported by Abilis. To learn more, visit abilis.us/calendar. Abilis is a nonprofit organization that supports more than 700 individuals with special needs and their families annually from birth throughout adulthood in the area.

Talk on art and climate change

The Bruce Museum Presents is a new series of monthly public programs featuring thought leaders in the fields of art and science. It will showcase experts on compelling subjects of relevance and interest to members and visitors to the Bruce Museum. The program continues with Can Art Drive Change on Climate Change? An Evening with Alexis Rockman from 6 to 8:30 p.m. Dec. 5. Among current American artists profoundly motivated by nature and its future from the specter of climate change to the implications of genetic engineering Rockman holds a high place of honor. Join the Bruce for a night of discussion and debate featuring Rockman and a panel of thought leaders in contemporary art and science. Doors open at 6 p.m. for a reception with light bites and beverages, followed by the panel discussion and Q&A from 7 to 8:30 p.m. Seats are $30 for Museum members, $45 for nonmembers. To reserve a seat, visit brucemuseum.org or call 203-869-0376.

SLS golf tourney

The St. Lawrence Society will hold its 29th annual Charity Golf Tournament on May 11. Enjoy a perfect day of golf, food and fun. Get your foursomes together and join the fun. Cost is $175 for everything: the luncheon, cart, golf, cocktail hour and dinner. For cocktails and dinner only, its $100. Starts at 11:30 a.m. at E. Gaynor Brennan Golf Course, with a 12:45 p.m. shotgun start, scramble format. Golf is followed by cocktail hour, antipasti and a prime rib dinner back at the Club. To RSVP, visit http://www.stlawrencesociety.com/events or call 203-618-9036.

Originally posted here:
Greenwich Symphony Orchestra concerts and other things to do - CT Insider

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Newswise The Childrens Cancer Institute at the Joseph M. Sanzari Childrens Hospital at Hackensack Meridian Health Hackensack University Medical Center and the John Theurer Cancer Center have announced they are participating in a multicenter Phase I/II clinical trial of an investigational gene therapy from bluebird bio, Inc. This trial is specifically for adolescents and adults with severe sickle cell disease (SCD) who cannot be effectively treated using standard therapies such as antibiotics, vitamins, blood transfusions or any pain relieving medications. The study is evaluating the safety and effectiveness of LentiGlobin for sickle cell disease, a gene therapy produced using the patients own modified stem cells to treat their sickle cell disease.

By using the patients own cells to produce functional hemoglobin that can prevent sickling of their red blood cells, LentiGlobin for SCD offers patients the opportunity to treat their disease without the need to have a matched bone marrow donor. The John Theurer Cancer Center is one of a limited number of centers internationally, and the Joseph M. Sanzari Childrens Hospital is the only pediatric site in New Jersey, where the study, which is enrolling patients age 12-50, is taking place.

Sickle cell affects 100,000 Americans. It affects one in every 365 African American births and one in every 16,000 Hispanic American births, said Alfred P. Gillio, M.D., director, Childrens Cancer Institute and section chief, Pediatric Stem Cell Transplantation and Cellular Therapy Program, Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center. This trial is for patients who have severe sickle cell disease and seek advanced treatment options but do not have a well-matched stem cell donor. Only 15% of sickle cell patients have a matched sibling donor and only 25 percent of patients have a matched unrelated volunteer donor.

Sickle cell affects every organ in a patients body, said Stacey Rifkin-Zenenberg, D.O., FAAP, pediatric hematologist/oncologist, Childrens Cancer Institute, and section chief, Pain and Palliative Care, Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center. This disease really has a tremendous effect not only on the patient, but also the family.

Sickle cell disease is an inherited disease caused by a mutation in the beta-globin gene, resulting in abnormal hemoglobin and sickle-shaped red blood cells. Symptoms and complications of the disease include anemia, infections, stroke, poor quality of life and early death. To date, the only cure for sickle cell disease is receiving a stem cell transplant from a matched donor, but this is not a therapeutic option for many patients. Supportive care including hydroxyurea and blood transfusions can ameliorate symptoms of the disease. To date, without a marrow donor, there has been no alternate curative therapy. Life expectancy of a person with sickle cell disease is 20 to 40 years of age. In some cases, patients using disease modifying medications can live to 50 or 60.

This therapy may be a major advance for sickle cell patients and so far, the results look very promising, said Scott D. Rowley, M.D., FACP, hematologist, medical director, Stem Cell Transplantation and Cellular Therapy and medical director, BMT Cell Lab, John Theurer Cancer Center, Hackensack Meridian Health Hackensack University Medical Center, who is enrolling adult patients. This investigational treatment, which is a one-time therapy, may be an option for our patients who have no other treatment options.

The results from early clinical studies are encouraging, said Dr. Gillio. With this treatment, the patient is their own donor and we are modifying their own cells to add copies of a functional beta globin gene.

In the current study:

About Hackensack Meridian Health Hackensack University Medical Center

Hackensack Meridian Health Hackensack University Medical Center, a 781-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the countys first hospital, it is now part of the largest, most comprehensive and truly integrated health care network in New Jersey, offering a complete range of medical services, innovative research and life-enhancing care, which is comprised of 34,100 team members and more than 6,500 physicians. Hackensack University Medical Center is ranked #2 in New Jersey and #59 in the country in U.S. News & World Reports 2019-20 Best Hospital rankings and is ranked high-performing in the U.S. in colon cancer surgery,lung cancersurgery,COPD, heart failure, heart bypass surgery, aortic valve surgery,abdominal aortic aneurysm repair, knee replacement and hip replacement. Out of 4,500 hospitals evaluated, Hackensack is one of only 57 that received a top rating in all nine procedures and conditions. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades Americas 50 Best Hospitals Award for five or more years in a row. Beckers Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2018. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 26 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its sixth consecutive designation in 2019. Hackensack University Medical Center has created an entire campus of award-winning care, including: John Theurer Cancer Center, a consortium member of the NCI-designated Georgetown Lombardi Comprehensive Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Womens and Childrens Pavilion, which houses the Joseph M. Sanzari Childrens Hospital and Donna A. Sanzari Womens Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guides list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to THE NORTHERN TRUST PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Childrens Cancer Institute housed at the Joseph M. Sanzari Childrens Hospital. To learn more, visit http://www.HackensackUMC.org.

Original post:
Joseph M. Sanzari Childrens Hospital and John Theurer Cancer Center Launch Clinical Trial Evaluating Gene Therapy for Severe Sickle Cell Disease in...

A study in Molecular Therapy describes how neuroD1-based gene therapy turns glial cells abundant support cells in the brain into neurons, repairing damage that results from stroke and significantly improving motor function in mice.

Once further developed, this NeuroD1-based gene therapy could potentially be used to treat stroke, the researchers suggest.

The current treatment for stroke has a narrow time window, typically within a few hours after the occurrence of stroke, says lead author Yuchen Chen, a postdoctoral fellow at Penn State, in a media release.

Many patients cannot receive the treatment in time and as a result, often suffer from permanent disability caused by irreversible neuronal loss. There is an urgent need to develop a new therapy to regenerate new neurons and restore lost brain functions among stroke patients.

The human brain has approximately 86 billion neurons. While mini-strokes can be tolerated, moderate stroke involving the loss of billions of neurons leaves detrimental effects that do not spontaneously recover.

So, the critical question that is still unanswered in the neuroregeneration field is how can we regenerate billions of new neurons in a patients brain after stroke? said Gong Chen, professor of biology and Verne M. Willaman Chair in Life Sciences at Penn State and leader of the research team. The biggest obstacle for brain repair is that neurons cannot regenerate themselves. Many clinical trials for stroke have failed over the past several decades, largely because none of them can regenerate enough new neurons to replenish the lost neurons.

Gong Chen and his team pioneered a new approach to regenerate functional neurons using glial cells, a group of cells surrounding every single neuron in the brain that provide essential support to neurons. Unlike neurons, glial cells can divide and regenerate themselves, especially after brain injury.

I believe that turning glial cells that are already present in the brain into new neurons is the best way to replenish the lost neurons, Gong Chen states. These glial cells are the neighbors of the dead neurons in the brain and are likely to share the same ancestral cellular lineage.

Gong Chens team previously reported that a single genetic neural factor, NeuroD1, could directly convert glial cells into functional neurons inside mouse brains with Alzheimers disease, but the total number of neurons generated was limited. The research team believed that this limited regeneration was due to the retroviral system used to deliver NeuroD1 to the brain. In the current study, the research team used the AAV viral system, which is now the first choice for gene therapy in the nervous system, to deliver NeuroD1 into mouse motor cortex that had suffered from stroke.

Many neurons die after stroke but surviving glial cells can proliferate and form a glial scar in the stroke areas. The AAV system was designed to express NeuroD1 preferentially in the glial cells that form these scars, turning them directly into neuronal cells. Such direct glia-to-neuron conversion technology not only increased neuronal density in the stroke areas, but also significantly reduced brain tissue loss caused by the stroke.

Interestingly, the newly converted neurons showed similar neuronal properties to the neurons that were lost after stroke. This suggests a potential impact of the local glial lineage on the converted neuronal identity, the release explains.

The most exciting finding of this study is to see the newly converted neurons being fully functional in firing repetitive action potentials and forming synaptic networks with other preexisting neurons, Gong Chen adds. They also send out long-range axonal projections to the right targets and facilitate motor functional recovery.

A separate collaborative work led by Gregory Quirk, professor at the University of Puerto Rico, further tested the NeuroD1-based gene therapy in a rat stroke model. Quirk and colleagues also found that this direct glia-to-neuron conversion technology can rescue cognitive functional deficits induced by stroke.

Because glial cells are everywhere in the brain and can divide to regenerate themselves, our study provides the proof-of-concept that glial cells in the brain can be tapped as a fountain of youth to regenerate functional new neurons for brain repair not only for stroke but also for many other neurological disorders that result in neuronal loss, Yuchen Chen comments.

Our next step is to further test this technology and ultimately to translate it into clinically effective therapies to benefit millions of patients worldwide.

[Source(s): Penn State, Science Daily]

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Gene Therapy Could Help Improve Functional Recovery After Stroke - Physical Therapy Products

WASHINGTON, D.C., Sept. 24, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM) and the National Association of Managed Care Physicians (NAMCP) Medical Directors Institute announced today the joint release of their recent study of medical director and manufacturer perspectives on value demonstration and reimbursement for cell- and gene-based regenerative and advanced therapies.

The study, entitled Roadmap for Navigating Cell and Gene Therapy Value Demonstration and Reimbursement in U.S. Managed Care, characterizes step-by-step considerations for achieving appropriate patient access to transformative and potentially curative therapies in the U.S. managed care setting. The findings identify key issues relevant to value demonstration and access to potentially curative therapies at a pivotal time for the industry, as several products have reached the market, with dozens more in late-stage clinical trials.

The initial wave of cell and gene therapies has launched into an environment that was not built with transformative or curative therapies in mind, said Eric Faulkner, Vice President, Precision and Transformative Medicine at Evidera and lead author for the publication. Its crucial for payers, providers, patients, and other stakeholders to align on expectations on value demonstration to ensure sustainable access.

In the next two to three years, the sector expects the number of marketed cell and gene therapies to more than triple, said Janet Lambert, CEO for the Alliance for Regenerative Medicine. We will improve patient lives if we ensure patients have timely access to these treatments and value assessment and reimbursement processes have been structured with these unique therapies in mind.

Appropriate reimbursement and access to transformative therapies is critical to constantly improve managed care practice, appropriate patient access and outcomes. This effort is one step to achieving that goal by identifying and acknowledging the key acceptance requirements and stakeholder perspectives as these therapies enter the marketplace, said Bill Williams, MD, Executive Vice President of the NAMCP Medical Directors Institute.

The study concludes with several key learnings for therapeutic developers and payers:

The study results are available to download here.

About The Alliance for Regenerative MedicineThe Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

About the NAMCP Medical Directors InstituteThe NAMCP Medical Directors Institute is a non-profit membership association, which was established to provide tools, education, and resources to medical directors, practicing physicians, and other healthcare professionals. NAMCP Medical Directors Institutes mission is to help Medical Directors from purchasers, health plans, and provider systems make effective and informed decisions, respond to opportunities and challenges in managed care, while helping improve healthcare outcomes, and ultimately the lives of members and patients. To learn more about the NAMCP Medical Directors Institute or to become a member, visit http://www.namcp.org

Lyndsey Scull202-213-7086lscull@alliancerm.org

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ARM and NAMCP Publish Recommendations to Increase Patient Access in Joint Study, "Roadmap for Navigating Cell and Gene Therapy Value...

PARIS--(BUSINESS WIRE)--

Regulatory News:

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190922005072/en/

GenSight Biologics (SIGHT.PA) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today reported the first set of results from Week 96 of the RESCUE Phase III clinical trial. The trial evaluated the efficacy and safety of a single intravitreal injection of GS010 (rAAV2/2-ND4) in 39 subjects whose visual loss due to 11778-ND4 Leber Hereditary Optic Neuropathy (LHON) commenced up to 6 months prior to study treatment. Week 96, which marks the time when individual patient profiles can be analyzed, is the last of the scheduled readouts for the RESCUE trial and completes the data collection from GS010s pivotal trials in Europe.

The results point to continued efficacy of GS010 two years past injection, with best-corrected visual acuity (BCVA) sustaining a clinically meaningful improvement over nadir. Having been treated early in the course of the disease, RESCUE patients vision initially deteriorated to a worst point, or nadir, before beginning to recover.

Note: A mixed model of analysis of covariance (ANCOVA) was used with change from baseline as the response, and subject, eyes of the subject as random factor, treatment and the baseline LogMAR value as covariates in the model.

When visual acuity is measured from the post-baseline nadir, the visual acuity of GS010-treated eyes in fact recovered significantly from the worst BCVA reading post-baseline.

Table 1: BCVA, Change from Nadir*, RESCUE

Week 48

Week 72

Week 96

N

Mean (SD)

N

Mean (SD)

N

Mean (SD)

All-GS010 Eyes

36

+12.8 (17.9)

34

+20.6 (26.3)

34

+24.9 (3.8)

All-Sham Eyes

36

+11.8 (15.6)

33

+21.7 (25.1)

34

+22.3 (3.8)

Note: *Nadir defined as worst BCVA measured in LogMAR after baseline, up to the week of interest; baseline reading was excluded from consideration. Mean change was calculated using observed values (no data imputation).

GS010-treated eyes regained more than two-thirds of the initial loss occurring in the most acute phase of the disease. This improvement from nadir (-0.498 LogMAR mean improvement, or +24.9 ETDRS letters equivalent) corresponds to 5 lines of Snellen acuity and is far above the 3-line threshold commonly accepted as a clinically meaningful level of visual improvement. Such recovery of vision is unprecedented in any gene therapy trial. Moreover, these results demonstrate the durability of improvement seen in earlier readouts of this trial.

Vision in sham-treated eyes evolved in parallel fashion, continuing the bilateral improvement already observed in earlier Phase III readouts. The picture that has emerged is one of durable, bilateral recovery from the effects of the acute phase in LHON.

The BCVA results from RESCUE show a remarkable correspondence with those from the REVERSE trial, which studied the treatment of subjects at 6 to 12 months after onset of vision loss.

The visual evolution in RESCUE appears to be a phase shift of the REVERSE curves, with an additional impact from the acute phase. This coherence will be more rigorously explored in a meta-analysis of the pooled data from the two trials, which is planned for this year.

In previous analyses of these trials, LHON clinical experts confirmed that the visual recovery seen in these trials are at odds with and much superior to their observations from clinical practice. That natural history of vision in untreated LHON patients stands in strong contrast to acuities seen in both RESCUE and REVERSE.

A natural history study conducted by Santhera1 provides another way of assessing the results in RESCUE. In that study, 28% of subjects who had the 11778A mutation achieved the following definition of spontaneous clinically relevant recovery (CRR) from nadir in at least one eye:

By comparison, 58% of RESCUE subjects achieved this definition of CRR in at least one eye at Week 96, with GS010-treated eyes as likely to achieve this as sham-treated eyes (58% vs. 45%, p = 0.0956).

The results from the RESCUE study are encouraging and convincing, particularly because we are seeing a similar pattern to the REVERSE study results, said Dr. Mark L. Moster, Neuro-Ophthalmology, Wills Eye Hospital, Professor of Neurology and Ophthalmology at Thomas Jefferson University, Philadelphia, PA, and Principal Investigator in the RESCUE and REVERSE trials. Patients in RESCUE were treated before the nadir so, as expected, they continued to worsen early on. But then from week 48 until week 96 they experienced a recovery from the nadir. That is much better than the natural history in any prior studies.

Story continues

Examination of other visual functions and biomarkers, including Contrast Sensitivity, show that these measures stabilized at Week 96. Based on preliminary analysis of the safety data, GS010 was well-tolerated after 96 weeks. There were no ocular serious adverse events or discontinuations that were due to ocular events. The ocular adverse events most frequently reported in the therapy group were mainly related to the injection procedure, except for the occurrence of intraocular inflammation (accompanied by elevation of intraocular pressure in some patients) that is likely related to GS010, and which was responsive to conventional treatment and without sequelae. There were no systemic serious adverse events or discontinuations that were related to study treatment or study procedure.

These results are remarkable, showing the durable difference that GS010 can make for patients who would otherwise go blind due to the onset of LHON, commented Bernard Gilly, Co-founder and Chief Executive Officer of GenSight. These findings, which we will be discussing at the meetings we have planned with regulatory authorities, form a compelling core for the clinical and non-clinical data that support our marketing authorization application in Europe. GenSight is excited to have reached this milestone in GS010s clinical development and energized by the prospect of pulling it all together for our European dossier.

GenSight is planning to schedule a pre-submission meeting with the EMA in early 2020 and expects to submit application for marketing approval in Europe in the third quarter of 2020.

An End of Phase II meeting with the U.S. Food and Drug Administration (FDA) has been requested and is expected for November 2019.

GenSight will host a conference call today, September 23, 2019, at 10:30am CEST in French, and at 2.30pm CEST (8.30am EST) in English, to discuss in greater detail these results and the roadmap to submission.

Webcast & Conference call in French

Dial-in numbers:

United States: +1 212 999 6659 France: +33 (0) 1 7037 7166 United Kingdom: +44 (0) 20 3003 2666 Password: GenSight

Webcast link: https://channel.royalcast.com/webcast/gensightbiologicsfr/20190923_1/

Webcast & Conference call in English

Dial-in numbers:

United States: +1 212 999 6659 France: +33 (0) 1 7037 7166 United Kingdom: +44 (0) 20 3003 2666 Password: GenSight

Webcast link: https://channel.royalcast.com/webcast/gensightbiologicsen/20190923_1/

A replay of the calls and webcasts will be available by using the above links.

Reference:

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

About GS010

GS010 targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function.

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of patients have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. The estimated incidence of LHON is approximately 1,400 to 1,500 new patients who lose their sight every year in the United States and Europe.

About RESCUE and REVERSE

RESCUE and REVERSE are two separate randomized, double-masked, sham-controlled Phase III trials designed to evaluate the efficacy of a single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects affected by LHON due to the G11778A mutation in the mitochondrial ND4 gene.

The primary endpoint will measure the difference in efficacy of GS010 in treated eyes compared to sham-treated eyes based on BestCorrected Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks post-injection. The patients LogMAR (Logarithm of the Minimal Angle of Resolution) scores, which are derived from the number of letters patients read on the ETDRS chart, will be used for statistical purposes. Both trials have been adequately powered to evaluate a clinically relevant difference of at least 15 ETDRS letters between treated and untreated eyes adjusted to baseline.

The secondary endpoints will involve the application of the primary analysis to bestseeing eyes that received GS010 compared to those receiving sham, and to worseseeing eyes that received GS010 compared to those that received sham. Additionally, a categorical evaluation with a responder analysis will be evaluated, including the proportion of patients who maintain vision (< ETDRS 15L loss), the proportion of patients who gain 15 ETDRS letters from baseline and the proportion of patients with Snellen acuity of >20/200. Complementary vision metrics will include automated visual fields, optical coherence tomography, and color and contrast sensitivity, in addition to quality of life scales, biodissemination and the time course of immune response. Readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials are conducted in parallel, in 37 subjects for REVERSE and 39 subjects for RESCUE, in 7 centers across the United States, the UK, France, Germany and Italy. Week 96 results were reported in 2019 for both trials, after which patients were transferred to a long-term follow-up study that will last for three years.

ClinicalTrials.gov Identifiers: REVERSE: NCT02652780 RESCUE: NCT02652767

About REFLECT

REFLECT is a multi-center, randomized, double-masked, placebo-controlled study to evaluate the safety and efficacy of bilateral injections of GS010 in subjects with LHON due to the NADH dehydrogenase 4 (ND4) mutation.

The trial planned to enroll 90 patients with vision loss up to 1 year in duration and will be conducted in multiple centers in Europe and in the US.

In the active arm, GS010 will be administered as a single intravitreal injection to both eyes of each subject. In the placebo arm, GS010 will be administered as a single intravitreal injection to the first affected eye, while the fellow eye will receive a placebo injection.

The primary endpoint for the REFLECT trial is the BCVA reported in LogMAR at 1-Year post-treatment in the secondaffected/notyetaffected eye. The change from baseline in secondaffected/notyetaffected eyes receiving GS010 and placebo will be the primary response of interest. The secondary efficacy endpoints include: BCVA reported in LogMAR at 2-Years post-treatment in the secondaffected/notyetaffected eye compared to both placebo and the firstaffected eye receiving GS010, OCT and contrast sensitivity and quality of life scales. The first subject was treated in March 2018, and enrolment was completed in July 2019, ahead of schedule.

ClinicalTrials.gov Identifiers: REFLECT: NCT03293524

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GenSight Biologics Reports Sustained Efficacy and Safety at 96 Weeks in RESCUE Phase III Clinical Trial of GS010 for the Treatment of Leber Hereditary...

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The global Gene Therapy market report focuses on the principal advertise players:

Novartis, Kite Pharma, Inc., GlaxoSmithKline PLC, Spark Therapeutics Inc., Bluebird bio Inc.

which contributes to the two points, estimation and level of administrations with moderate development. These sections are moreover gathered into the sub-segments for an in-depth examination and recognition of the explicit Gene Therapy market.

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Alongside the report includes the most slanting substances of the complete Gene Therapy market. The report provides a demand for the individual segment region wise. It demonstrates distinct segments of type, end-user, and region of the global Gene Therapy market. This can help to grasp the position of the market in the globe. Further, the report includes the limitations that constraints the Gene Therapy market advancement.

by Vector Type (Viral Vector and Non-viral Vector), Gene Type (Antigen, Cytokine, Tumor Suppressor, Suicide, Deficiency, Growth Factors, Receptors, and Others), and Applications (Oncological Disorders, Rare Diseases, Cardiovascular Diseases, Neurological Disorders, Infectious Disease, and Other Diseases)

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The Gene Therapy market report states the organizations, vendors, and manufacturers. The complete Gene Therapy industry showcase gives end-customer demands, different market changes, constraining segments, and managerial adaptability. Moreover, Gene Therapy report serves in-detail forecast evaluations based on the current business designs and analytical systems. The Gene Therapy market evaluation is complete relying upon the quantitative and intellectual procedure to provide a precise idea of the present and future figure incline. The Gene Therapy market report all around includes the diagrams, charts, graphs, etc. which exemplify the status of the specific business on the global and regional stage.

1. Understand the ongoing and future Gene Therapy Market in both developed and emerging markets.

2. The report assists in planning the business strategies by highlighting the Gene Therapy business precedence.

3. The report sheds light on the segment expected to dominate the Gene Therapy market.

4. Forecasts the regions expected to reckon the fastest growth.

5. The recent developments in the industry and description of the Gene Therapy industry leaders along with their market share and strategies.

6. Rescue time on the entry-level analysis because the report serves very crucial info regarding growth, size, key players and segments of the business.

7. Save and lessen time carrying out entry-level research by recognizing the growth, Gene Therapy market size, leading players and segments in the global market.

While gathering the information we perform an interview with suppliers, raw material suppliers, and others to get supply-side Gene Therapy sector stats. To grab sales retailers, traders and market dealers gather Gene Therapy sector data. Likewise, in order to study demand-side statistics, we interview end-users and conduct custom surveys using secondary research techniques, Gene Therapy company reports, government information, demographics collect Gene Therapy production sales and consumption statistics.

How can our report help you?

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Tags:Gene Therapy market development, Gene Therapy market in key countries, Gene Therapy trends, Gene Therapy market forecast, Gene Therapy industry overview

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Global Gene Therapy Market with Indepth Analysis, Competitive Players, Future Trends and Forecast to 2025 : Novartis, Kite Pharma, Inc.,...

YORBA LINDA, Calif. (PRWEB) September 26, 2019

LabRoots, the leading scientific social networking website offering premier, interactive virtual events and webinars, will host its 2nd Annual CRISPR Virtual Event, on October 3, 2019. Since the first conference was held in 2018, research has continued to advance rapidly, however many questions remain about the science, applications, and the use of biotechnological techniques for editing genomes.

CRISPR 2019 resumes the global dialogue on these issues by bringing together a broad range of participants including biomedical researchers, academia and industry, and renowned scientists to explore topics such as CRISPR Biology, CRISPR in Gene Editing Applications, and Ethical and Regulatory dimensions and concerns of Gene Editing.

CRISPR has evolved into various technologies for basic research with new applications. Now, in its 2nd year, this premier event focused on the discovery and classification of CRISPR systems, gene therapy, animal model development for medical research, agriculture and food security will feature eleven prominent speakers, and a keynote delivery by Dr. Marie-Christine Birling, Head Associate of the Genetic Engineering and Model Validation Department at the Institut Clinique de la Souris on generating rodent model by CRISPR/Cas9 genome editing.

CRISPR generates a high level of interest, said Dimitri Perrin, Senior Lecturer in Data Science, Queensland University of Technology and member of the organizing committee. Research in this area intersects with a number of disciplines, from the basic biology of CRISPR systems to applications in gene editing (for instance in model organisms, health or agriculture) and reflections on how to ethically use and adequately regulate these technologies. It has been a pleasure to work with LabRoots to bring you an exciting program of excellent researchers across these domains, added Perrin.

Our inaugural conference was a huge success, said Greg Cruikshank, Chief Executive Officer of LabRoots. CRISPR 2019 returns highlighting momentum and recent progress made in gene editing and biotechnology and its potential moving forward. LabRoots continues to be dedicated to developments in life sciences using its unique stage for sharing data and research critical to the scientific community.

LabRoots interactive platform allows attendees to watch, learn and connect seamlessly across all desktop and mobile devices. By joining this online event, viewers can earn one Continuing Education credit per presentation for a maximum of 14 credits.

To register for the event and for more information, click here. Participants can follow the conversation online by using #LRcrispr.

About LabRoots LabRoots is the leading scientific social networking website, and primary source for scientific trending news and premier educational virtual events and webinars and more. Contributing to the advancement of science through content sharing capabilities, LabRoots is a powerful advocate in amplifying global networks and communities. Founded in 2008, LabRoots emphasizes digital innovation in scientific collaboration and learning. Offering more than articles and webcasts that go beyond the mundane and explore the latest discoveries in the world of science, LabRoots users can stay atop their field by gaining continuing education credits from a wide range of topics through their participation in the webinars and virtual events.

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LabRoots Announces Second Annual Conference on CRISPR Technologies - PR Web

Kari Branham, M.S., a genetic counselor at Kellogg, worked with Zions family to help them understand the genetic basis for Zions condition.

SEE ALSO: Retinitis Pigmentosa in Children: 5 Facts Families Should Know

We have seen such amazing progress with these conditions over the last 15-20 years,says Branham. We used to tell patients and their families that we would have to wait and see what happens, but now we can actually do something to help.

By going through the gene therapy process, Branham says the team is hopeful that this has changed Zions prognosis.

The treatment is designed to stop or slow the death of specialized cells in the retina, called photoreceptors, that send visual information to the brain.

Patients who have Leber congenital amaurosis have night blindness, says Besirli. One of the first treatment effects after receiving Luxturna is that (patients) are telling us that they function much better in dark.

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They can play outside much longer, they can navigate around the house and dont need nightlights anymore and can participate in indoor sports. Thats been a huge change in their lives.

Seven months after treatment, Zion, now age 7, and his family are back to their normal routine in Montrose, Mich., and monitor his progress during follow-up appointments at Kellogg.

Zion says hes looking forward to playing football and, with improved vision -- playing outside at night with his brothers.

We hope that with Zion we have changed the trajectory for him to the point that in his 20s he wont have significant vision loss we see with him now, says Branham.

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7-Year-Old Receives New FDA-Approved Retina Gene Therapy - University of Michigan Health System News