StemCell Therapy

Dublin, Dec. 04, 2019 (GLOBE NEWSWIRE) -- The "Biomarkers: Discovery Techniques and Applications - A Global Market Overview 2019" report has been added to ResearchAndMarkets.com's offering.

The report reviews analyzes and projects the global Biomarkers market for the period 2016-2025 in terms of market value in US$ and the compound annual growth rates (CAGRs) projected from 2018 through 2025.

The global market for Biomarkers is dominated by Oncology, in terms of application, estimated at a market share of 41.2% in 2018 and forecast to touch US$15.6 billion in 2019. The overall market for Biomarkers is projected to reach approximately US$50 billion by 2021.

The factors driving the Biomarkers market growth includes elevated demand for personalized medicine, technological advancement, immense utilization of biomarkers in drug discovery and development procedures, massive research on biomarkers, increasing awareness about the role of biomarkers in early disease diagnosis, increase research funding, and many more

Research Findings & Coverage

Key Market Trends

For more information about this report visit https://www.researchandmarkets.com/r/9jqzri

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

Excerpt from:
Analysis on the World Market for Biomarkers 2016-2019 & 2025: Key Trends, Discovery Techniques & Applications - Yahoo Finance

December 2019

Cambridge, UK: Global courier for cell and gene therapy, Biocair, will exhibit at Cell Therapy Manufacturing and Gene Therapy Congress between 3-6 December 2019. Taking place in Amsterdam, Cell Therapy Manufacturing and Gene Therapy Congress brings together key players from the industry to discuss the critical issues facing the development, scale-up and manufacture of cell-based and gene therapies.

Speaking about the logistics surrounding cell and gene therapy, Gert de Gier, Cell and Gene Business Development Director at Biocair, comments that with cell and gene therapies moving into the clinical trial phase for a wide variety of diseases, the results of these studies and their potential impact to patients are invaluable. Therefore the transportation of the materials and need for a secure supply chain is absolutely critical.

Patient samples can be bio-hazardous, time critical and have an extremely short shelf-life. Biocair has developed a highly customised project-based approach to the challenging requirements of the clinical and commercial cell and gene markets, providing chain of custody provision across every touchpoint, validated and pre-conditioned temperature-controlled packaging for all temperature ranges, total transparency in supply chain design and unrivalled short transit times (12 36hrs).

Biocairs specialist services are precisely documented in standard operating procedures and are regularly audited by its 12, 500 life science customers across the world. For more information, contact your local Biocair office or contact enquiries@biocair.com.

Notes to editors

Biocair is a specialist courier with over 30 years of dedicated experience in the pharmaceutical, biotechnology and life science sectors. The company has built up a unique, client-centric approach by employing scientists in front-line logistics positions and assembling a team of best-in-class industry experts in quality, cold chain and regulatory compliance amongst others. Biocair focuses on providing the most comprehensive service options available whilst delivering flexible, tailored, cost effective logistics solutions to all clients.

Biocair operates across Europe, Africa, Asia and the Americas.

Continued here:
Biocair Exhibits at Cell Therapy Manufacturing and Gene Therapy Congress 2019 - BioSpace

Dive Brief:

Astellas is paying up to join the gene therapy race.

At $60 per share, the Japanese pharma's takeover offer is more than double the $28.61 at which shares in Audentes traded at Monday's market close a multiple on par with Roche's bid for Spark but higher than the premium Novartis offered for AveXis, and Biogen for Nightstar Therapeutics.

Impressive clinical results, paired with the prospect of speedy development, have made gene therapies an attractive investment for larger drugmakers. And approvals for AveXis' Zolgensma (onasemonogene abeparvovec) and Spark's Luxturna (voretigene neparvovec) on data from only a few dozen patients indicates a FDA willing to clear new therapies on less information than usual, providing the benefit is clear enough.

*Over closing price the day preceding deal announcement SOURCE: Companies

For Astellas, its interest in Audentes appears linked as much to the options buying the biotech opens up as it is to the company's lead drug candidate.

Importantly, Audentes owns a sizable manufacturing plant capable of supporting clinical and commercial gene therapy production. Manufacturing the inactivated viruses used to deliver the one-time treatments has proved a bottleneck for the field, so acquiring capabilities could give Astellas a leg up in the future.

And Audentes this year secured a collaboration with Nationwide Children's Hospital in Ohio a hotbed for gene therapy science that gave the biotech several programs aimed at Duchenne muscular dystrophy and myotonic dystrophy type 1.

That work remains preclinical, however. Nearer term, Audentes offers Astellas a gene therapy candidate that could be submitted to regulators for approval as early as the middle of next year. Called AT132, the therapy is designed to treat x-linked myotubular myopathy, a rare and usually fatal neuromuscular disorder affecting infants and young boys.

Mutations in the MTM1 gene result in missing or dysfunctional myotubularin, a protein essential to skeletal muscle cell development. Without it, affected infants are extremely weak and often require ventilator assistance to breathe. Half of those diagnosed die in the first 18 months of life, according to estimates cited by Audentes.

AT132 proposes a genetic fix for the disease, delivering a functional copy of the MTM1 gene via a type of viral vector known as AAV8. Results from a Phase 1/2 study showed infants treated with the therapy were able to sit, stand and even walk. Importantly, the ten treated patients were able to significantly reduce their dependence on ventilatory support compared to two study participants who were used as a control.

Neuromuscular conditions like XLMTM and DMD require much higher doses of gene therapy than diseases of the eye, brain or liver, making Audentes manufacturing capacity a vital asset.

Audentes is also developing a therapy for Pompe disease, which, like DMD, is targeted by several other companies. A clinical-stage program in the ultra rare condition called Crigler-Najjar syndrome, however, appears to have been put on hold this year by Audentes.

Astellas said it will house Audentes in an independent subsidiary, and plans for the acquisition to form a foundation for a new research focus centered on genetic regulation. A statement on the deal seemed to hint more gene therapy partnerships could be forthcoming, or at least made possible by buying Audentes.

At $3 billion, Astellas' acquisition is the largest buyout of a gene therapy developer since Roche-Spark, as well as the largest in a run of deals brokered by the pharma, which is perhaps best known for its development of the prostate cancer drug Xtandi (enzalutamide).

Astellas' interest in Audenetes could ease some worries that would-be acquirers might put off dealmaking in gene therapy until the Federal Trade Commission reaches a decision in its review of Roche-Spark. Repeated delays have raised eyebrows among investors and analysts, who largely expected the deal to close rapidly.

"In light of the pending [Spark-Roche] transaction, some investors may find Astellas-[Audentes] as another potentially drawn-out and uncertain tie-up,' wrote Joseph Schwartz, an analyst at SVB Leerink, in a note to clients.

"However, our review of Astellas' product portfolio and R&D pipeline suggests minimal overlap with [Audentes]'s focus on neuromuscular diseases."

Schwartz and colleagues were able to identify only two drug candidates that could share a target with Audentes', potentially making a takeover from the much larger Astellas easier to clear.

Excerpt from:
Astellas joins gene therapy race with $3B Audentes buy - BioPharma Dive

LONDON, Dec. 3, 2019 /PRNewswire/ -- Alacrita, a leading life science consulting firm, with offices in the United States and the United Kingdom, announces the departure of Rob Johnson who leaves the company to take on a new role at a gene therapy startup. Anthony Walker, co-founder of Alacrita and managing partner, will take over leadership activities of the U.S. office.

Rob is a co-founder of Alacrita and was instrumental in establishing and growing the firm's U.S. presence. He guided it over the past seven years, leading the expansion of its consulting team there as well as of its portfolio of U.S. clients in the biotech and pharmaceutical sectors.

This continues a long history of Alacrita involvement in start-up companies. Alacrita consultants have played leading roles in a number of startup enterprises including Onyvax, Biotica Technology, Leucid Bio and other companies that have been spun out of university clients. Alacrita has worked with over 35 academic institutions on commercialization of early stage technology, including writing multiple business plans for successful spin-outs.

"Rob has been a driving force at Alacrita, leading our U.S. team and playing a major role in the progression of our firm in the United States. As we have grown there, his leadership has been effective and steady. Beyond his deep life-science expertise and leadership skill, he is a wonderful colleague who I have worked with for over 20 years. We could not be more excited for him in his new role," said Anthony Walker, Alacrita managing partner and co-founder.

"We are grateful for Rob's leadership of our U.S. operations. Rob worked tirelessly to help us build Alacrita into the transatlantic firm that it is today. We truly wish him well in his new endeavor," said Simon Turner, Alacrita managing partner.

"My time at Alacrita will always be meaningful. I am grateful for the incredible team that I have worked with all these years, for all that we have achieved together, as well as for the chance to help so many of our clients succeed with their projects. As I move on to a new challenge and chapter, I know I leave my post in capable hands that will continue the firm's strong trajectory upward," said Rob Johnson.

Anthony Walker, who takes over direction of the firm's U.S. office, leverages more than 30 years of experience, including over a decade spent buildingand managing a biotechnology company and nearly 20 years as a management consultant to the pharmaceutical and biotech industries.

Alacrita conducts more than 250 successful client assignments every year.The firm's team combines extensive industry experience (strategic, technical and commercial), advanced functional capabilities and a track record of success across the domain. Learn more by visiting http://www.alacrita.com.

For further information, please contact:

Anthony Walker, Managing Partner, U.S. Email: usa@alacrita.com Telephone: +1-617-714-9696 Address: 303 Wyman Street, Waltham, MA 02451

Simon Turner, Managing Partner, Europe Email: europe@alacrita.com Telephone: +44-(0)207-691-4915 Address: London BioScience Innovation Centre, 2 Royal College St, London, United Kingdom

Original post:
Alacrita's Rob Johnson Departs for Gene Therapy Space - P&T Community

The optimistic outlook is seen in an adorable 10-year-old girl named Jojo, who became the first patient to receive a gene therapy treatment, called AXO-AAV-GM1, during a human clinical trial this summer at the National Institutes of Health in Maryland. Auburn's College of Veterinary Medicine and the University of Massachusetts Medical School developed the treatment that has moved from helping cats with GM1 to hopefully helping children.

"Jojo is doing well and has experienced no major complications," said Dr. Doug Martin, professor in the Department of Anatomy, Physiology and Pharmacology in Auburn's veterinary college and the Scott-Ritchey Research Center. "Seeing all of the effort come together to help patientswho haveno treatment options today gives us great hope."

Auburn scientists for several decades have researched treatments to improve and extend the lives of cats affected by GM1. Martin is leading Auburn's effort, which was started by his mentor, Professor Emeritus Henry Baker.

To move the treatment toward human medicine, Martin developed a partnership with UMass Medical School researchers Drs. Miguel Sena-Esteves and Heather Gray-Edwards, an Auburn alumnaand they have worked collaboratively for 15 years, combining animal and human medicine studies to cure rare diseases that affect both animals and humans. In December 2018, the gene therapy was licensed to Axovant Gene Therapies Ltd., a clinical-stage company developing innovative gene therapies.

"This treatment is extremely promising because it has worked well in GM1mice and cats, and it is delivered by a single IV injection that takes less than an hour," Martin said. "We're hopeful that the treatment makes a real difference for patients and their families.

"The NIH is hoping to begin treating three or four more children in the next few months. As the trial progresses and more patients are treated, we'll have a good idea of whether the gene therapy helps children as much as it has helped the animals."

The NIH clinical trial is led by Dr. Cynthia Tifft, deputy clinical director at the National Human Genome Research Institute. "GM1 gangliosidosis is a devastating disease in young children, for which there are no currently approved treatment options. The development of a safe and effective gene therapy for these patients would be a welcome advancement in the field of pediatric lysosomal storage disorders affecting the brain," Tifft said.

For Auburn graduates Sara and Michael Heatherly of Opelika, whose son Porter was the first known case of GM1 in Alabama and died in 2016, the knowledge of a treatment is one of mixed emotions.

"We are excited to know there is hope for the future of children diagnosed with GM1," Michael Heatherly said. "We are thankful for everyone who has dedicated their time, resources and careers to move this treatment forward and to Axovant for bringing all of their work to life and making it a reality for GM1 patients.

"We understood early on the research would not help Porter, but we wanted to help spread the word of the research and the progress that was being made."

The Heatherlys gave Auburn researchers a reason to hope, and work harder for a cure. To honor the family, which held fundraisers for several years to support the research, the College of Veterinary Medicine's Scott-Ritchey Research Center incorporated Porter's likeness in a creative identity for the center.

SOURCE Auburn University

http://www.auburn.edu

See more here:
Auburn University research leads to gene therapy that provides hope for children with deadly disease - PRNewswire

DAYTON, Ohio, Dec. 3, 2019 /PRNewswire/ -- CSafe Global understands temperature control, gained from 40 years of experience in developing innovative temperature-controlled packaging - from solutions designed to transport vital pediatric vaccines to crucial life-saving cancer drugs. CSafe is known for protecting what matters most to pharmaceutical companies, so that patients receive what matters most to them.

CSafe recognizes the importance of thermally protecting shipments of life-enhancing medications as they are shipped to patients in need around the world. The criticality of temperature protection within the emerging Cell and Gene Therapy (CGT) space is further amplified due to the value and uniqueness of CGT products and the complicated supply chain. The new AcuTemp Plus Series has been developed with this in mind, and will confidently meet the evolving and increasingly demanding customer expectations for temperature performance, solution quality, and system robustness required of packaging solutions to protect shipments of novel cell and gene therapies.

"We are proud to expand our cold chain packaging offerings for the Cell and Gene Therapy market, and have focused and invested significantly in R&D to develop best-in-class solutions to meet the demanding product requirements for this emerging class of medication," stated Patrick Schafer, CEO of CSafe Global. "Test results of the new AcuTemp Plus Series have been very impressive with temperature performances outlasting other market available solutions by a large margin."

The AcuTemp Plus Series of packaging solutions has been designed with proprietary, high-performance ThermoCorVacuum Insulated Panels (VIP) to guarantee precise end-to-end control of internal payload temperatures. In addition to industry-leading performance, the innovative range has been developed with simplicity in mind, benefiting from optimized system components and solution design. The new series is available in multiple size configurations and temperature profiles, along with different integrated track and trace options to meet customer needs. Furthermore, the AcuTemp Plus Series is supported with a fully managed, end-to-end service infrastructure leveraging CSafe's industry-unique retest and reuse program - REPAQ.

With a market-leading portfolio of both active and passive temperature-management solutions, it is CSafe's mission to bring peace of mind to every life-science customer.

CONTACT: Josh Angliss, jangliss@csafeglobal.com

View original content:http://www.prnewswire.com/news-releases/csafe-expands-cold-chain-offerings-for-the-cell-and-gene-therapy-market-with-launch-of-high-performing-acutemp-plus-series-of-temperature-controlled-packaging-300968270.html

SOURCE CSafe Global

Original post:
CSafe expands cold chain offerings for the Cell and Gene Therapy market with launch of high-performing AcuTemp Plus Series of temperature-controlled...

Shares of Regenxbio (RGNX) have risen by 190% since I initially uncovered and recommended a position in this gene therapy pioneer in 2016. The stock has appreciated by just 52% since my February 2018 update piece.

With the recently announced acquisition of collaboration partner Audentes Therapeutics (BOLD) by Astellas Pharma for $2.7 billion (also a prior recommendation), I thought it's time to revisit this gene therapy "picks and shovels" play.

Chart

Figure 1: RGNX daily advanced chart (Source: Finviz)

When looking at charts, clarity often comes from taking a look at distinct time frames in order to determine important technical levels to get a feel for what's going on. In the above chart (daily advanced), we can see a downtrend taking place from April into Q3, with share price bottoming in the low 30s in September. Recently, the stock received a boost after news of Audentes Therapeutics being acquired (had licensed NAV technology for various indications including XLMTM, Crigler-Najjar and CPVT). Readers might recall that when collaboration partner AveXis was acquired for $8.7 billion in 2018, its suitor Novartis (NVS) was required to pay them $100 million upfront as an accelerated licensee payment (while remaining on the hook for $80 million in commercial milestones plus royalties on Zolgensma). A move to highs last witnessed in 2018 would equate to a bit less than a double from today's levels.

In my last update piece, I touched on the following keys to our bullish thesis:

Figure 3: Internally developed pipeline (Source: corporate presentation)

Figure 4: Collaboration partner pipeline (Source: corporate presentation)

Let's take a look at recent events and how they've affected our thesis here.

In late July Regenxbio and Swiss biotech firm Neurimmune announced an exclusive license to develop novel AAV gene therapies using NAV vectors to deliver human antibodies against targets implicated in chronic neurodegenerative diseases, including tauopathies. Both companies will be jointly responsible and share development costs equally, with each having co-development and co-commercialization options (or can elect to receive a phase-based worldwide royalty instead of continued development investment).

On July 31st Regenxbio announced a license agreement with Pfizer (PFE), specifically non-exclusive global license to NAV AAV9 vector for commercialization of gene therapies for the treatment of Friedreich's ataxia. In return for these rights, Regenxbio received an upfront payment, and has the potential to receive ongoing fees, development and commercial milestone payments, and royalties on net sales of products incorporating their IP.

On October 30th, it was disclosed that the company exercised its option under the previously announced option and license agreement to Clearside Biomedical's (CLSD) in-office SCS Microinjector for the delivery of adeno-associated virus-based therapeutics, including RGX-314 delivery to the suprachoroidal space to potentially treat wet age-related macular degeneration, diabetic retinopathy, and related conditions where chronic anti-vascular endothelial growth factor (VEGF) treatment is currently the standard of care. Deal terms were modest, involving a small upfront payment, up to $34 million in development milestones, up to $102 million in sales milestones and mid-single digit royalties on net sales of products incorporating SCS Microinjector.

Figure 5: Widespread retinal transduction observed in both subretinal and suprachoroidal delivery of AAV8 in non-human primates (Source: Corporate Presentation)

Keep in mind that Regenxbio is currently suing the FDA, as they disclosed a clinical hold for RGX-314 suggesting the agency opted for this action "without notice or explanation". The clinical hold is related to issues associated with delivery systems currently being used (seems agency is not a fan of subretinal injections, which allows for a more direct approach but can have a complicated adverse event profile (involves potential events such as intraocular inammation, retinal detachment, ocular hemorrhage, etc). A more convenient intravitreal approach is being utilized by Adverum Biotechnologies (ADVM). While such differences may not be that big of a deal in clinical trials, you can bet that when and if such gene therapies make it to the market, those that offer the ideal mix of high convenience and best safety profile will be the most likely to win this high stakes race.

Figure 6: Subretinal injection versus intravitreal injection (Source: Adverum corporate presentation)

For the third quarter of 2019, the company reported cash and equivalents of $417.1 million as compared to net loss of $34.6 million. Research and development costs nearly doubled to $35.7 million, while SG&A increased to $12.4 million

Regarding RGX-314 in treating wet AMD and diabetic retinopathy, we are informed of the clinical hold related to use of third-party commercially available devices used to deliver the gene therapy candidate. Specifically, the company states that all 42 patients have been dosed in the phase 1/2a study and that the partial clinical hold on IND is not related to the gene therapy candidate itself. Management guides for phase 2b study in wet AMD to get underway in Q1 2020, around which time IND will be filed for the study in diabetic retinopathy. We are reminded that data here continues to be encouraging, with no drug-related serious adverse events and subjects in Cohort 5 showing reduction of over 80% from the mean annualized injection rate during the 12 months prior to receiving RGX-314. Additionally, durable effects on vision and retinal thickness had been demonstrated over 1.5 years in the third cohort, with 50% of subjects remaining free of anti-VEGF injections more than 1.5 years after RGX-314 administration.

Figure 7: Cohort 5 injections pretreatment and after being treated with RGX-314 (Source: Corporate Presentation)

Management continues to state that they are evaluating in-office delivery of RGX-314 to the suprachoroidal space and will unveil plans for this route of administration next year.

As for RGX-501 for the treatment of Homozygous Familial Hypercholesterolemia (HoFH), interim data from Cohort 2 is expected at the end of the year (keep in mind this time they are using corticosteroid prophylaxis). Also at the end of the year, we'll get an interim data update on the phase 1/2 study of RGX-121 for the treatment of MPS II.

As these gene therapy companies are increasingly valued on the basis of their manufacturing capabilities as reflected in recent buyouts (including Audentes Therapeutics), it's worth noting that Regenxbio's new cGMP production facility (allows for production of NAV Technology-based vectors at scales up to 2,000 Liters) is expected to be operational in 2021.

As for partnered efforts, Novartis revealed Q3 Zolgensma sales of $160 million resulting in royalty revenue to Regenxbio of $9.2 million. Audentes Therapeutics' AT132 in XLMTM is well on its way to potential regulatory approval with BLA to be filed mid-2020.

To conclude, even after the recent bounce this gene therapy "picks and shovels" has continued upside ahead. With 20+ partnered product candidates, one can almost think of it as a mutual fund of sorts as investors gain exposure to various rare disease programs as well as programs targeting more prevalent conditions. On the other hand, the company has much more to prove with wholly-owned programs, as the anti-VEGF market is known for being intensely competitive with multiple "next-generation" approaches being tried out by larger companies aiming to garner a slice of this lucrative pie. Consider Baker Brothers' recent royalty deal, purchasing a 4.5% rate for $225 million (assumes KSI-301 worth over $5 billion using back of the envelope math). Another overlooked catalyst is phase 1 data for Danon Disease program with collaboration partner Rocket Pharmaceuticals (RCKT) in 2020, a lucrative indication of high unmet medical need considering estimated US+ EU prevalence of 15,000 to 30,000 patients.

For readers who are interested in the story and have done their due diligence, Regenxbio remains a Buy and I suggest patiently accumulating dips over the next couple of quarters.

Additional dilution in the near term does not look likely considering the current cash position and operational runway. Disappointing data for wet AMD and other programs not to mention setbacks in the clinic (especially delays or safety/tolerability concerns) would weigh on the stock. Failure to get the recent clinical hold lifted would weigh on the stock and the company's competitive position in the wet AMD market (and related indications). Competition for certain indications should not be ignored, especially by peers with significantly greater resources.

Author's Note: I greatly appreciate you taking the time to read my work and hope you found it useful. Consider clicking "Follow" next to my name to receive future updates and look forward to your thoughts in the Comments section below.

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Disclosure: I am/we are long CLSD, RCKT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: Disclaimer: Commentary presented is NOT individualized investment advice. Opinions offered here are NOT personalized recommendations. Readers are expected to do their own due diligence or consult an investment professional if needed prior to making trades. Strategies discussed should not be mistaken for recommendations, and past performance may not be indicative of future results. Although I do my best to present factual research, I do not in any way guarantee the accuracy of the information I post. I reserve the right to make investment decisions on behalf of myself and affiliates regarding any security without notification except where it is required by law. Keep in mind that any opinion or position disclosed on this platform is subject to change at any moment as the thesis evolves. Investing in common stock can result in partial or total loss of capital. In other words, readers are expected to form their own trading plan, do their own research and take responsibility for their own actions. If they are not able or willing to do so, better to buy index funds or find a thoroughly vetted fee-only financial advisor to handle your account.

Read the original post:
Regenxbio: Revisiting This Gene Therapy 'Picks And Shovels' Play - Seeking Alpha

New GMP conditioning of PEIpro-GMP designed to support clinical phase and commercialization stages of production

Strasbourg, France December 3, 2019 Polyplus-transfection(R) SA, the leading biotechnology company that supports gene and cell therapy by supplying innovative transfection solutions, today announces the availability of the first GMP-grade transfection reagent PEIpro(R)-GMP in additional conditioning that will ensure additional flexibility and cost-efficiency.

Polyplus-transfection commercialized PEIpro-GMP at the end of 2018. This was the first transfection solution for the gene and cell therapy industry that is compliant with global cGMP viral vector manufacturing requirements. Polyplus-transfection is now expanding the PEIpro-GMP packaging options from a 1 liter single-use bag to several conditioning options from 10 milliliters up to 1 liter in both single-use bags and bottles.

The use of closed-systems and single-use technologies for clinical trials and commercial manufacturing of viral vectors continues to increase. Polyplus-transfection is now providing two sizes of PEIpro-GMP in bags to ensure aseptic connections to successful perform large-scale transfection in closed sterile systems. Polyplus-transfection now additionally supplies PEIpro-GMP in bottle conditioning that will allow the adaption to different manufacturing scale-up and scale-out strategies. These increasing scale-out strategies require the performance of multiple smaller-scale transfections in multiple cell culture units at the same time. Demand for transfection reagents has thus increased for the smaller as well as for the larger conditioning options. The increases in options will therefore result in additional flexibility for therapeutic developers in the setup of cost-efficient viral vector manufacturing processes.

Polyplus-transfection has been developing strong supplier-manufacturer relationships over eighteen years, and certainly since the conception of the modern gene and cell therapy industries. As a result, we have unrivalled first hand visibility on the ever-changing needs of the gene and cell therapy markets, said Claire Wartel, PhD, director of quality and compliance, Polyplus-transfection. The focus of the market has evolved to commercialization and late-stage clinical stages. This means the therapeutic production scale and strategy needs to be adaptable to the production of each viral vector, whilst complying with cGMP manufacturing requirements. To provide this critical function in making available therapies to patients, Polyplus-technology is now able to offer a sustainable solution for viral vector platforms. This will benefit gene and cell therapy developers, the wider industry, and most importantly, the patient community reliant on the ongoing development of these therapies.

Claire Wartel recently gave more information on Identifying and mitigating risks in the viral vector supply chain in a podcast interview that can be accessed here.

About Polyplus-transfection SA

Polyplus-transfection SA is a biotechnology company specializing in nucleic acid delivery solutions located close to the University of Strasbourg in Eastern France and a market leader for transfection reagents for cell & gene therapy. Polyplus-transfections vast portfolio of delivery solutions can be used for all types of applications, from research and process development, all the way to clinical trials. For more information, please visit the Polyplus-transfection web site at: http://www.polyplus-transfection.com.

Read the original post:
Polyplus-transfection expands GMP portfolio to deliver flexibility for gene and cell therapy manufacturing - BioSpace

By Murielle Gonzalez 2-Dec-2019

Pharmaceuticals

New building in Stein also hosts the production of difficult-to-manufacture solid dosage forms

A cell processing specialist at work in new cell and gene facility in Stein. Photo as seen on Novartis website

Novartis has announced the opening of a new manufacturing facility for cell and gene therapies in Stein, Switzerland.

Our site in Stein is vital for new launches of solid and liquid drugs, explained Steffen Lang, Global Head of Novartis Technical Operations and member of the Novartis Executive Committee.

Lang continued: The construction of the new manufacturing facility is another investment in the production of breakthrough cell-based therapies that can potentially change the lives of patients.

In addition to manufacturing areas for novel CAR-T cell therapies, the new building also hosts the production of innovative, difficult-to-manufacture solid dosage forms, such as tablets and capsules.

Last September, the first clinical production of a cell and gene therapy badge was successfully completed, Novartis said.

Unlike conventional drug production, cell and gene therapy asks for the manufacture of a personal dose for each patient.

For this purpose, patients who have already undergone various therapies have a small amount of their own blood cells taken, which are then sent to Stein.

Here we enrich part of the white blood cells, the T cells, and genetically modify them so that they can recognize and fight the cancer cells in the patients blood, said Dorothea Ledergerber, project manager of the Stein plant for cell and gene therapies.

The altered cells are then sent back to a hospital and administered to the patient by infusion.

For Ledergerber, Novartis is doing pioneering work here. She explained: We have the unique opportunity to offer patients for whom there have been no other therapeutic options a totally new perspective by using these novel CAR-T cell therapies.

Novartis celebrated the opening in the presence of Federal Councillor Alain Berset and other guests on 28th November 2019.

Read more:
Novartis opens facility for innovative cell and gene therapies in Switzerland - Cleanroom Technology

Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the world's priciest medicines.

The full scope of Novartis' $500 million plan, revealed to Reuters in an interview with the company's gene therapy chief, has not been previously disclosed. It is second only to Pfizer, which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patient's cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

"There's so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies," said David Lennon, president of AveXis, Novartis's gene therapy division. "We need internal manufacturing capabilities in the long term."

The approach is not without risks.

Bob Smith, senior vice president of Pfizer's global gene therapy business, acknowledged drugmakers take a "leap of faith" when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

PUSHING THE LIMITS

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartis's Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia.

The proliferation of these treatments is pushing the limits of the industry's existing manufacturing capacity.

Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio, PTC Therapeutics and Krystal Biotech are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc, developer of a gene therapy for hemophilia, which constructed one of the industry's largest manufacturing facilities in 2017.

REGULATORY SCRUTINY

The FDA is keeping a close eye on standards.

This comes amid the agency's disclosure in August that it is investigating alleged data manipulation by former executives at Novartis' AveXis unit.

AveXis had switched its method for measuring Zolgensma's potency in animal studies. When results using the new method didn't meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

But the scandal has highlighted the importance of having a consistent manufacturing process for gene therapies, industry executives say.

According to four of them, the FDA has stressed in recent meetings the need for continuity in production processes all the way from the development of a drug to its commercialization.

By bringing production in-house, drugmakers may avoid pitfalls such as the need to switch to a larger facility if contract manufacturers' capacity proves limited, executives say.

The FDA is finalizing new guidelines for gene therapy manufacturing, expected at the end of the year.

"Manufacturing consistency is always a major concern for the agency," FDA spokeswoman Stephanie Caccomo told Reuters.

Highlighting the pressures on the industry, Sarepta Therapeutics, which largely outsources manufacturing, delayed a clinical trial of its Duchenne treatment in August, telling investors it wanted to avoid any questions from regulators about consistency in producing its therapy at commercial scale.

ENOUGH GROWTH FOR ALL?

"Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense, if you can do it, to build out your own facilities and more and more gene therapy companies have started to do that," said Krish Krishnan, chief executive of Krystal Biotech Inc.

Krystal, which is developing therapies for rare skin diseases, has built one manufacturing facility and plans to invest more than $50 million in a new one it will start constructing in December.

MeiraGTx, which focuses on gene therapies for eye conditions, estimates it is currently spending roughly $25 million a year on manufacturing, including process development.

Despite such moves, however, contract manufacturers like Lonza and Thermo Fisher are confident their businesses will continue to grow due to the strength of demand.

Thermo Fisher has told investors its Brammer gene therapy manufacturing division, acquired in May, could soon earn $500 million in revenue a year, double its projected 2019 earnings.

Lonza CEO Marc Funk is also optimistic.

"Demand in gene therapy has increased," he said in an interview. "We believe this is going to continue in the coming years."

Continued here:
Pfizer, Novartis lead pharma spending spree on gene therapy production - Japan Today

MUNICH, Germany, Nov. 28, 2019 (GLOBE NEWSWIRE) -- ViGeneron GmbH, a gene therapy company, announced the closing of its series A financing round led by WuXi AppTec and Sequoia Capital China. The proceeds will enable ViGeneron to accelerate its proprietary viral vector-based gene therapy platforms and drive product development in its two lead ophthalmic gene therapy programs.

ViGenerons pipeline in gene therapy addresses ophthalmic diseases with high unmet medical need, including two programs in development for undisclosed indications where no approved treatment options are currently available.

The companys two novel next-generation gene therapy platforms are geared towards addressing the limitations of existing adeno-associated virus (AAV)-based gene therapies. The vgAAV vector platform, based on novel engineered AAV capsids, enables a superior transduction of target cells and is designed to efficiently cross biological barriers. These attributes allow vgAAV vectors to target a broad spectrum of cell types in the retina and potentially other tissues, such as central nervous system tissue; enabling intravitreal, less invasive treatment administration. For larger genes (>5Kb) the company has developed the innovative REVeRT vector platform. This platform uses an innovative vector approach to pack split genes into individual vgAAV vectors and generate a full-length protein via mRNA trans-splicing.

ViGeneron is a spin-off of the Ludwig-Maximilians-University (LMU) in Munich. The companys founding team includes highly experienced executives and internationally renowned experts with track records in developing retinal gene therapy programs from discovery to clinical stage: Dr. Caroline Man Xu (Co-founder and CEO), Prof. Dr. Martin Biel (Scientific Co-founder), and Prof. Dr. Stylianos Michalakis (Scientific Co-founder).

Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron said: The evolution of medicines from small molecules to proteins has driven increased therapeutic benefits in the past; the next generation of gene therapies holds tremendous promise for patients. We are passionate about bringing innovations to patients. This financing is an important validation of our next-generation gene therapy technology platforms and ophthalmic development programs. With these top-tier investors and a strong ophthalmologic network supporting us, we are now in an excellent position to accelerate our development programs.

Edward Hu, Co-CEO of WuXi AppTec, commented: We are impressed by ViGenerons vgAAV vector platform and the innovative REVeRT vector platform. These gene therapy platform technologies will potentially generate superior gene therapy products to treat a wide range of diseases that are traditionally difficult to treat. We look forward to supporting the company to deliver on its great promises for the patients in need.

About ViGeneronViGeneron is dedicated to developing innovative gene therapies to treat ophthalmic diseases with high unmet medical need, as well as partnering with leading biopharmaceutical players in other disease areas. The companys pipeline is built on two proprietary adeno-associated virus (AAV) technology platforms. The first, vgAAV gene therapy vector platform, allows superior transduction efficiency and intravitreal, less invasive treatment administration. The second, REVeRT vector platform, targets diseases caused by mutations in large genes. Privately-owned ViGeneron was founded in 2017 by a seasoned team with in-depth experience in AAV vector technology and clinical ophthalmic gene therapy programs and is located in Munich, Germany. For further information, please visit http://www.vigeneron.com.

About WuXi AppTecWuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable companies in the pharmaceutical, biotech and medical device industries worldwide to advance discoveries and deliver groundbreaking treatments to patients. As an innovation-driven and customer-focused company, WuXi AppTec helps our partners improve the productivity of advancing healthcare products through cost-effective and efficient solutions. With industry-leading capabilities such as R&D and manufacturing for small molecule drugs, cell and gene therapies, and testing for medical devices, WuXi AppTecs open-access platform is enabling more than 3,700 collaborators from over 30 countries to improve the health of those in need and to realize our vision that "every drug can be made and every disease can be treated".

About Sequoia Capital ChinaThe Sequoia team helps daring founders build legendary companies. In partnering with Sequoia, companies benefit from our unmatched network and the lessons we've learned over 47 years. As The Entrepreneurs Behind The Entrepreneurs, leading venture capital firm Sequoia Capital China is renowed for investing early in many successful companies and focuses on four sectors: TMT, healthcare, consumer/serive, and industrial technology.

Contact:ViGeneron GmbH info@vigeneron.com

Media and Investor Relations:MC Services AGShaun Brown/ Julia von Hummelphone: +49 (0)89 21022880 vigeneron@mc-services.eu

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ViGeneron announces closing of series A financing to drive development of next generation gene therapy pipeline - GlobeNewswire

Certain levels of plasma protein adenosine deaminase 2 (ADA2) are sensitive and specific, and can be used as a novel biomarker of macrophage activation syndrome in patients with systemic juvenile idiopathic arthritis (JIA), according to research results published in the Annals of the Rheumatic Diseases.

Researchers sought to evaluate the role and function of ADA2 as a novel biomarker of macrophage activation syndrome because of the overlapping clinical features between macrophage activation syndrome and active systemic JIA.

A reference range for plasma ADA2 activity was first established using samples from 324 healthy individuals (174 children and 150 adults). Spectrophotometric assays showed that patients with biallelic ADA2 mutations had a near absence of ADA2 activity compared with carriers who had approximately half-normal plasma ADA2 activity.

After establishing the reference range, the researchers compared ADA2 levels in children with Kawasaki disease, pediatric systemic lupus erythematosus, and juvenile dermatomyositis with age-matched healthy controls. Investigators established that ADA2 levels did not correlate with markers of disease activity and were not a general marker of systemic inflammation in any of the 3 disorders.

Among the JIA population, most patients showed plasma ADA2 activity levels that were comparable with age-matched healthy controls; however, a small subset had levels well above the upper limit of normal. After stratification by JIA category, investigators found that ADA2 activity was present in children with oligoarticular and polyarticular JIA, enthesitis-related arthritis, and psoriatic arthritis.

Using multiple macrophage activation syndrome biomarkers, researchers compared ADA2 activity in patients with JIA and created a correlation matrix based on Spearman rank correlation r values. The comparisons demonstrated that ADA2 levels correlated with ferritin, interleukin-18, and C-X-C Motif Chemokine Ligand 9. ADA2 activity also correlated well with increased aspartate aminotransferase levels in macrophage activation syndrome, but not with conventional markers of inflammation.

Although the patterns displayed by these markers were different, all the markers were sensitive and specific in discriminating macrophage activation syndrome from systemic JIA using the upper limit of normal as a cutoff.

Seven patients with macrophage activation syndrome had available serum samples. Consistent with previous analyses, investigators found that ADA2 levels were generally higher during a confirmed episode of macrophage activation syndrome compared with other time points. Researchers noted that patients who experienced recurrent episodes of macrophage activation syndrome exhibited ADA2 levels near the upper limit of normal even when macrophage activation syndrome was absent.

Whether ADA2 contributes to the pathophysiology of [macrophage activation syndrome] is not clear, the researchers concluded. The [physiologic] function of ADA2 remains to be determined.

Reference

Lee PY, Schulert GS, Canna SW, et al. Adenosine deaminase 2 as a biomarker of macrophage activation syndrome in systemic juvenile idiopathic arthritis [published online November 9, 2019]. Ann Rheum Dis. doi: 10.1136/annrheumdis-2019-216030

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Novel Biomarker of Macrophage Activation Syndrome Identified in Juvenile Idiopathic Arthritis - Rheumatology Advisor

CHICAGO - About 54 million adults have doctor-diagnosed arthritis, according to The Arthritis Foundation. Almost 300,000 babies and children have arthritis or a rheumatic condition

For Chelsea May, those numbers hit too close to home. Her 14-year-old daughter, Jenna, suffers from Juvenile Psoriatic Arthritis and Uveitis. She was diagnosed four years ago.

To help fight spread awareness of the debilitating disease and raise money for research, Jenna's family will host their third annual bake sale on Dec. 6. The bake sale will take place at the Mt. Greenwood Holiday Stroll from 4 p.m. to 7 p.m.

"We are also looking for donations of baked goods as well," May said.

The donations and money received from the bake sale will be donated to the Arthritis Foundation's Jingle Bell Run which will take place on Dec. 14.

The Arthritis Foundation's Jingle Bell Run is the original festive race for charity, bringing people from all walks of life together to champion arthritis research and resources.

Less than a week after the bake sale, Jenna's family will gear up for the Jingle Bell Run in Chicago. If you would like to donate or join Jenna's run team, click here.

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Jingle Bake Sale To Benefit The Arthritis Foundation - Beverly, IL Patch

ATLANTA There is no significant difference in the rate of serious infections in patients with rheumatoid arthritis (RA) receiving upadacitinib 15 mg or adalimumab, according to study results presented at the 2019 American College of Rheumatology/Association of Rheumatology Professionals (ACR/ARP) Annual Meeting, held November 8 to 13, 2019, in Atlanta, Georgia.

However, the results indicated that patients receiving upadacitinib 15 mg and 30 mg had higher rates of herpes zoster compared with patients receiving adalimumab or methotrexate.

The study included data from 5 randomized double-blind, placebo- or active-controlled phase 3 trials of upadacitinib 15 mg (included in all 5 trials) or 30 mg daily (included in 4 trials) in patients with RA.

Researchers calculated the exposure adjusted event rates (EAERs; events/100 patientyears [PY]) of treatment-emergent adverse events (AEs) for the integrated placebo (3 trials; 12/14 weeks), the integrated methotrexate (2 trials; mean exposure, 36 weeks), the originator adalimumab (mean exposure, 42 weeks), upadacitinib 15 mg (mean exposure, 53 weeks), and upadacitinib 30 mg (mean exposure, 59 weeks) groups.

In all 5 phase 3 trials, 3834 patients received 1 dose of upadacitinib 15 mg (n=2630) or 30 mg (n=1204) with no option to switch doses, for a total of 4020.1 PY of upadacitinib exposure.

Similar to patients who received adalimumab, patients who received upadacitinib 15 mg had EAERs of overall serious adverse events (SAEs) and AEs that led to discontinuation. Compared with methotrexate, upadacitinib 15 mg and 30 mg had higher EAERs of both SAEs and AEs.

The most commonly reported AEs were upper respiratory tract infection, nasopharyngitis, and urinary tract infections, all of which occurred more frequently among patients in the upadacitinib groups compared with the placebo group.

Patients in the upadacitinib 15 mg and adalimumab groups had comparable rates of serious infection events (SIEs); however, rates of SIEs were higher among patients receiving upadacitinib compared with methotrexate. Compared with patients receiving methotrexate and adalimumab, patients receiving either dose of upadacitinib had higher rates of herpes zoster.

Among patients receiving upadacitinib, those in the 15-mg group had lower rates of SIEs and herpes zoster compared with the 30-mg group.

All treatment groups had similar rates of malignancies, excluding non-melanoma skin cancer, and adjudicated major adverse cardiovascular events and venous thromboembolic events. In addition, all treatment groups had comparable rates of death.

Compared with the other treatment groups, upadacitinib 30 mg had a higher rate of non-melanoma skin cancer; however, the rates of non-melanoma skin cancer for both the upadacitinib groups fell within the range reported for patients with RA treated with disease-modifying antirheumatic drugs.

Visit Rheumatology Advisor for live coverage and more news from the 2019 ACR/ARP Annual Meeting.

Reference

Cohen S, van Vollenhoven R, Winthrop K, et al. Safety profile of upadacitinib in rheumatoid arthritis: integrated analysis from the SELECT phase 3 clinical program. Presented at: 2019 ACR/ARP Annual Meeting; November 8-13, 2019; Atlanta, GA. Abstract 509.

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Rates of Serious Infection Events Similar With Upadacitinib, Adalimumab in Rheumatoid Arthritis - Rheumatology Advisor

In September, the world of entertainment news buzzed with word that Kim Kardashian West tested positive for lupus and rheumatoid arthritis. The star underwent further tests, however, resulting in a diagnosis of psoriatic arthritis instead. While all three autoimmune disorders share some signs and symptoms, psoriatic arthritis is generally considered to have a better prognosis than lupus. That said, the conditions can co-exist and lupus has gotten a reputation for being difficult to diagnose, especially in the absence of the butterfly-shaped rash on ones cheeks and nose.

Im so relieved. The pain is going to come and go sometimes, but I can manage it and this is not going to stop me, Kardashian said in an article in response to receiving her psoriatic arthritis diagnosis. Her relief at not having lupus is understandable, given that lupus can affect a greater number of organs and systems in the body and is considered to be life-threatening.

Lupus, rheumatoid arthritis and psoriatic arthritis are examples of some conditions that are often considered when an individual is undergoing diagnosis for certain autoimmune diseases, because they share several symptoms and can trigger positive results in the same diagnostic tests. Kim Kardashian received the initial news that she had lupus or rheumatoid arthritis likely due to positive antinuclear antibody (ANA) test results.

An ANA is a blood test ordered when a doctor, usually a rheumatologist, suspects that a patient has a particular kind of autoimmune disorder. This test checks for the existence of autoantibodies, which are produced when a persons body is, in effect, attacking itself and several areas of the body are affected. A positive ANA test usually indicates that the doctors suspicions are confirmed, and then other factors (like medical and family history) need to be considered and more tests done to arrive at a diagnosis.

Psoriatic arthritis is usually diagnosed between the ages of 20 and 50, and occurs in women and men equally. While there is no cure, appropriate and early treatment can help prevent major damage to affected parts of the body.

Psoriatic arthritis appears in a minority of individuals who have already been diagnosed with psoriasis, an autoimmune skin condition with which Kim Kardashian and her mother, Kris Jenner, had already been diagnosed. Psoriatic arthritis affects around 520,000 individuals in the United States alone.

The autoimmune condition is believed to be caused by a combination of genetic factors and environmental triggers. So while some people inherit psoriatic arthritis-related genes, only a subset of those individuals will go on to develop the condition. In these cases, the disease could be triggered by other illnesses or infections, various forms of extreme stress, poor diet, smoking, and so on.

Around 40 percent of psoriatic arthritis patients have one or more close family members with psoriasis or psoriatic arthritis diagnosis, which strongly indicates that the disease is hereditary. Interestingly, recent research has suggested that psoriasis patients who go on to develop psoriatic arthritis have a different genetic profile than those who do not. And the most well-studied of the psoriatic arthritis genes belong to a family of genes called the human leukocyte antigen (HLA) complex, which help the body tell the difference between its own proteins and viral or bacterial proteins.

According to Genetics Home Reference by the U.S. National Library of Medicine, Variations of several HLA genes seem to affect the risk of developing psoriatic arthritis, as well as the type, severity, and progression of the condition.

Ive been feeling so tired, so nauseous, and my hands are really getting swollen. I feel like I literally am falling apart. My hands are numb, Kardashian said on a recent episode of Keeping Up with the Kardashians.

These kinds of descriptions are common in all three conditions lupus, rheumatoid arthritis, and psoriatic arthritis though each patient presents with a different array of symptoms, and all with varying degrees of severity. The main symptoms of psoriatic arthritis are pain, stiffness, and swelling in affected joints, along with chronic fatigue. Joints near the end of the fingertips and tips of the toes are often affected, as are bones in the spine.

The symptoms of psoriatic arthritis tend to worsen over time, though some patients experience periods of remission when symptoms temporarily improve. Compared to rheumatoid arthritis, psoriatic arthritis is more likely to cause swelling in the smallest joints of the fingers and toes, foot pain (in the heel and/or sole of the foot), and lower back pain caused by inflammation in vertebral joints. Patients with psoriatic arthritis are also more likely to experience symptoms on one side of the body or in different appendages on each side (in other words, it tends to be an asymmetric disease), whereas patients with rheumatoid arthritis are more likely to experience symptoms that affect both sides of the body equally (symmetric disease).

Most if not all patients with psoriatic arthritis also have psoriasis, an autoimmune condition that causes red, scaly patches of skin that can be itchy, painful and embarrassing. Psoriasis usually precedes the onset of psoriatic arthritis by several years. People with psoriatic arthritis commonly experience fingernail changes, too, such as the formation of a pitted or ridged nail surface, or the nails become separated from the nail beds.

There are several treatment options for psoriatic arthritis, which include nonsteroidal anti-inflammatory drugs (NSAIDs) to reduce inflammation and pain, immunosuppressants to suppress the immune system, disease-modifying antirheumatic drugs (DMARDs) to slow the progression of the disease, and newer medications that minimize the activity of certain enzymes involved in the inflammatory process. Treatment plans may also involve steroid injections administered directly into affected joints, or joint replacement surgery in cases where the disease has significantly progressed.

Kristen Hovet covers genetics, medical innovations and the intersection of sociology and culture. The North Dakota native is based in Vancouver, Canada, where she is working on a masters degree in health communication at Washington State University. Follow her on her website or Twitter @kristenhovet

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Kim Kardashian West's battle with psoriatic arthritis: Will understanding the genetics of the autoimmune disorder point to a cure? - Genetic Literacy...

AbbVie's (ABBV - Get Report) fledgling rheumatoid arthritis treatment Rinvoq is showing promise and appears to be tracking "meaningfully ahead of expectations," according to analysts at Piper Jaffray.

Analyst Christopher Raymond reiterated the firm's overweight rating on theNorth Chicago biopharma while increasing its price target to $92 from $90.

At last check Tuesday AbbVie was trading down 1.2% at $86.

Piper Jaffray's bullish outlook on Rinvoq is fueled by the results of a new survey of 100 rheumatologists, who provided mostly positive feedback about the drug. A rival treatment still enjoys success, but Rinvoq could soon be on its heels, the analyst wrote.

"While indications of Xeljanz (PFE - Get Report) entrenchment do exist, fully 33% of doctors indicate they would be early Rinvoq adopters, just because of AbbVie's involvement," Raymond wrote.

"And in terms of reported sales contact," AbbVie's rheumatology focus on Rinvoq appears to rival that "of its Humira effort, and then finally, when compared to the prior two rheumatoid arthritis launches (Olumiant and Kevzara), unaided awareness, brand familiarity and initial user base are all head-and-shoulders above."

"[Just] months post-launch, ... Rinvoq registers 1% patient share," Raymond wrote. But "asked to delineate most recent scripts, doctors give Rinvoq 8% share. When asked to projectshare in six months, rheumatologists indicate Rinvoq's share will grow to 6%."

"In that Rinvoq largely takes a back seat to [AbbVie treatment] Skyrizi as a key growth driver in the minds of most investors we speak with, we see this feedback as a welcome source of upside in coming quarters," Raymond wrote.

AbbVie is a holding in Jim Cramer's Action Alerts PLUS Charitable Trust Portfolio. Want to be alerted before Cramer buys or sells ABBV? Learn more now.

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AbbVie Affirmed Overweight at Piper Jaffray on Potential of Arthritis Drug - TheStreet.com

While theres no cure for knee osteoarthritis, a combination of strategies can help relieve your pain and keep you active.

Cleveland Clinic is a non-profit academic medical center. Advertising on our site helps support our mission. We do not endorse non-Cleveland Clinic products or services.Policy

Although the cornerstones of treatment are exercise and physical therapy and pain medications and steroid injections are also options you can also try knee braces, shoe inserts or simply wearing more supportive shoes.

Knee braces can be helpful for managing your pain, says physical therapist Dawn Lorring, PT, MPT. The location and severity of your symptoms will drive which brace works best for you.

Osteoarthritis is caused by the breakdown of cartilage (thats the cushioning material that covers the ends of bones in joints.) This causes pain and stiffness.

In the knee joint, arthritis can occur at any of three points where the bones come in contact:

Sleeve braces. People who have mild pain or stiffness that limits their activities can try a sleeve-type brace. These provide compression, which can reduce swelling and warm the joint. This might relieve the stiffness.

These braces also provide added support. If your knee feels unsteady or wobbly, a compression-type brace can be helpful, Lorring says. Some of them have plastic stays or a hinge on the side, which provides a little more support. She recommends getting one that has an opening at the knee cap.

Sleeve braces arent covered by insurance, but they are relatively inexpensive, ranging from $10 to 100.

Web brace. A more advanced brace is a sleeve with silicone webbing over the front. As you bend and straighten your knee, the webbing tightens in certain areas. This provides extra support to the knee.

A regular sleeve brace provides compression all over. The brace with the webbing also provides guidance for how the knee cap moves, Lorring says.

This type of brace might be the most helpful for someone with osteoarthritis beneath the knee cap. A web brace costs about $100.

Unloader brace. When arthritic changes are between the femur and tibia, a device called an unloader knee brace may help, especially if one side is more arthritic than the other. These have a metal band that goes around the thigh and another one around the calf, connected by a hinged bar. This creates a frame that can be adjusted to shift pressure (unload) from one side of the knee to the other.

If the inside of your knee hurts, the brace can be adjusted to put more force on the outside of your knee, unloading weight off the inside, Lorring explains.

These are less beneficial if your arthritis symptoms are similar on both the inside and outside of the joint.

Unloader knee braces are expensive ($500 to $1,000), but they can be covered by insurance. Youll need a doctors prescription and documentation that it is medically necessary.

Shoes and inserts. Various foot problems (like high arches or flat feet) or just the particular way you walk can affect the alignment of your body. That might be putting more pressure on your knee joints. You may get some relief by choosing better shoes or wearing shoe inserts (also called orthotics).

Because everyone is different, theres no universal advice for shoes or inserts. Lorring recommends consulting a physical therapist or an expert in foot mechanics who can observe how you walk and help you pick out shoes or shoe inserts that match your needs. I encourage people to look at running shoes because there are more support options, she says.

The goal with orthotics is to make sure your foot is moving in the best way it can so your knee isnt getting more force than it should, Lorring says. There are a wide variety of shoe inserts and heel wedges that you can buy in a drug store or online. You can also get them custom made or save some money and get semi-custom ones. Like with shoes, you need to get inserts and wedges that are specific to your needs.

You can have an insert that doesnt add much arch support but it adds cushion, which can be beneficial if you walk on the outside of your foot, Lorring says. However, if your foot rolls inward too much, you may need more arch support.

You can get heel wedges that are sloped in one direction or the other, which is similar to the action of an unloader brace. It shifts pressure from one side of the knee to the other.

Ultimately, you have to find what works for you, Lorring says.

This article originally appeared in Cleveland Clinic Arthritis Advisor.

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Could a Knee Brace Help Ease Your Osteoarthritis Pain? - Health Essentials from Cleveland Clinic

Study Assesses Risk of Infections, Cancer With Interleukin Inhibitors in Rheumatic Disease

According to the study, the risks may be comparable to those reported for other biologics approved for the treatment of rheumatic diseases.

A number of IL-1, IL-6, IL-12/23, and IL-17 inhibitors are used as therapy options in rheumatologic diseases. Although IL inhibitors have demonstrated efficacy, there are limited data regarding their safety profile and it is unknown to what extent IL inhibitors may increase the risk of serious infections and cancer.

To assess this risk, researchers conducted a systematic review and meta-analysis using data from clinical trials that evaluated IL inhibitor therapies and reported safety data. The meta-analysis included 74 studies including 29,214 patients. The studies included tocilizumab, secukinumab, anakinra, ixekizumab, rilonacept, sarilumab, sirukumab, ustekinumab, brodalumab, guselkumab, clazakizumab, canakizumab, and olokizumab.

Diseases in the study included rheumatoid arthritis (RA), psoriatic arthritis, ankylosing spondylitis, gout, juvenile idiopathic arthritis, giant call arteritis, systemic lupus erythematosus, primary Sjogren syndrome, systemic sclerosis, familial Mediterranean fever, and osteoarthritis.

Overall, 69 studies included data for serious infections across all rheumatic diseases, comprising a total of 24,236 patients. The pooled analysis showed an increased risk of serious infections with the use of IL inhibitors compared with placebo.

Fourteen of the studies reported the incidence of opportunistic infection, with 9998 patients in these trials. Oral candidiasis was the most commonly reported opportunistic infection, according to the analysis. Others included herpes zoster, esophageal candidiasis, Mycobacterium tuberculosis, atypical mycobacterial infections, and histoplasmosis. Overall, the results showed an increased risk of opportunistic infections with the use of IL inhibitors compared with placebo.

A total of 46 studies with approximately 21,000 patients had data on the incidence and type of cancers across all rheumatic diseases. A total of 141 cases of cancer reported in the treatment groups and 28 in the control groups. The analysis found that, overall, there was an increased risk for cancer with IL inhibitors compared with placebo.

The researchers noted that, although the review suggest that the risk of cancer may be increased with longer IL inhibitor therapy, the results are not conclusive and should be further investigated by long-term data.

This analysis provides estimates of toxic effects for infections and cancer associated with the use of IL inhibitors that can inform shared decision-making when patients and clinicians are contemplating the use of IL inhibitors for rheumatologic diseases, they concluded.

References

Bilal J, Berlinberg A, Riaz IB, et al. Risk of infections and cancer in patients with rheumatologic diseases receiving interleukin inhibitors: a systematic review and meta-analysis. JAMA Network Open. 2019. Doi: 10.1001/jamanetworkopen.2019.13102.

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Study Assesses Risk of Infections, Cancer With Interleukin Inhibitors in Rheumatic Disease - Pharmacy Times

BACKGROUNDS:

Rheumatoid arthritis (RA) is a systemic autoimmune disease that confers one of the strongest risks for cardiovascular disease (CVD) morbidity and mortality than in general population. Pulse wave velocity (PWV) and augmentation index (AIx) are composite measures of arterial stiffness (AS) and associated with CV risk.

The aim of this study was to systemically review the evidence regarding the relationship between PWV, AIx and RA, as well as underlying influential factors.

Eligible literatures were searched in PubMed, EMBASE and The Cochrane Library published up to February 28, 2019 in English. The pooled weight mean difference (WMD) with its 95% confidence interval (CI) was calculated using random-effect model analysis.

A total of 38 studies were finally incorporated in the meta-analysis. The results indicated that, compared to controls, RA patients had significantly increased levels of carotid-femoral (cf)-PWV (WMD=1.10m/s, 95% CI: 0.84-1.35), brachial-ankle (ba)-PWV (WMD=0.20m/s, 95% CI: 0.12-0.28), cartoid-radial (cr)-PWV (WMD=0.51m/s, 95% CI: 0.23-0.79), AIx (WMD=4.79%, 95% CI: 1.34-8.24) and AIx normalized to a 75 beats/minute heart rate ([emailprotected]) (WMD=5.78%, 95% CI: 3.82-7.74) (all p <0.001). Meta-regression and subgroup analysis demonstrated significant association of cf-PWV with age, disease duration and erythrocyte sedimentation rate (ESR) in RA.

Overall, there is increased PWV level in patients with RA, and this alteration is associated with age, disease duration and ESR.

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Assessment of Aortic Stiffness in Patients With Rheumatoid Arthritis Using Pulse Wave Velocity: An Update Meta-analysis - DocWire News

OBJECTIVE:

To determine the prevalence of comorbidities in rheumatoid arthritis (RA), discover which comorbidities might predispose to developing RA, and identify which comorbidities are more likely to develop after RA.

We performed a case-control study using a single-center biobank, identifying 821 cases of RA (143 incident RA) between January 1, 2009, and February 28, 2018, defined as 2 diagnosis codes plus a disease-modifying antirheumatic drug. We matched each case to 3 controls based on age and sex. Participants self-reported the presence and onset of 74 comorbidities. Logistic regression models adjusted for race, body mass index, education, smoking, and Charlson comorbidity index.

After adjustment for confounders and multiple comparisons, 11 comorbidities were associated with RA, including epilepsy (odds ratio [OR], 2.13; P=.009), obstructive sleep apnea (OR, 1.49; P=.001), and pulmonary fibrosis (OR, 4.63; P<.001), but cancer was not. Inflammatory bowel disease (OR, 3.82; P<.001), type 1 diabetes (OR, 3.07; P=.01), and venous thromboembolism (VTE; OR, 1.80; P<.001) occurred more often before RA diagnosis compared with controls. In contrast, myocardial infarction (OR, 3.09; P<.001) and VTE (OR, 1.84; P<.001) occurred more often after RA diagnosis compared with controls. Analyses restricted to incident RA cases and their matched controls mirrored these results.

Inflammatory bowel disease, type 1 diabetes, and VTE might predispose to RA development, whereas cardiovascular disease, VTE, and obstructive sleep apnea can result from RA. These findings have important implications for RA pathogenesis, early detection, and recommended screening.

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Comorbidities As Risk Factors for Rheumatoid Arthritis and Their Accrual After Diagnosis - DocWire News