Category Archives: Regenerative Medicine

BEDFORD, Mass.--(BUSINESS WIRE)--Anika Therapeutics, Inc. (NASDAQ: ANIK), a global, integrated regenerative therapies company leveraging its proprietary hyaluronic acid ("HA") technology platform, today announced plans to showcase its osteoarthritis pain management and joint preservation and restoration therapies at the 15th World Congress of the International Cartilage Regeneration and Joint Preservation Society (ICRS). The ICRS World Congress, which is being held October 5-8, 2019 in Vancouver, Canada, is the worlds largest gathering of scientists, clinicians and industry participants focused on clinical cartilage repair and basic cartilage research.

Anika is committed to expanding its portfolio of regenerative therapies to become the global leader in joint preservation and restoration, said Joseph Darling, President and CEO, Anika Therapeutics. We look forward to engaging with orthopedic specialists at this years ICRS World Congress to better understand the needs of patients and physicians across the joint preservation and restoration continuum of care.

Guided, hands-on demonstrations of HYALOFAST procedures using the HYALOFAST Arthroscopic Virtual Surgery Simulator will be available at the Anika booth. Details on Anikas joint preservation and restoration presentations at the 2019 ICRS World Congress are below:

October 6, 2019

Anika-Sponsored Lunch Symposium

11:30 am 12:15 pm PDT

Hyatt Regency Vancouver, Plaza C&B

Title: Preservation & Regeneration: A Joint Approach

Moderator: Sanjay Anand, Consultant Orthopaedic Surgeon - Stepping Hill Hospital, Stockport, Alexandra BMI Hospital, Cheadle - UK

Speakers:

October 7, 2019

Company-Sponsored Physician Education

10:45 am 11:15 am PDT

Hyatt Regency Vancouver, Anika Booth #15

About ICRS

The ICRS (International Cartilage Regeneration and Joint Preservation Society) is the main forum for international collaboration in cartilaginous tissue research that brings together basic scientists, clinical researchers, physicians and members of industry, engaged or interested in the field of articular biology, its genetic basis and regenerative medicine. It provides continuing education and training to physicians and scientists with an active interest in the prevention and treatment of joint disease to improve patient care through regenerative medicine approaches.

About Anika Therapeutics, Inc.

Anika Therapeutics, Inc. (NASDAQ: ANIK) is a global, integrated regenerative therapies company based in Bedford, Massachusetts. Anika is committed to delivering therapies to improve the lives of patients across a continuum of care from osteoarthritis pain management to joint preservation and restoration. The Company has over two decades of global expertise commercializing more than 20 products based on its proprietary hyaluronic acid (HA) technology platform. For more information about Anika, please visit http://www.anikatherapeutics.com.

Follow this link:
Anika to Showcase Joint Preservation and Restoration Portfolio at the 2019 World Congress of the International Cartilage Regeneration and Joint...

DUBLIN, Oct. 7, 2019 /PRNewswire/ -- The "Novel Cell Sorting and Separation Market: Focus on Acoustophoresis, Buoyancy, Dielectrophoresis, Magnetophoretics, Microfluidics, Optoelectronics, Traceless Affinity and Other Technologies, 2019-2030" report has been added to ResearchAndMarkets.com's offering.

Research and Markets Logo

The Novel Cell Sorting and Separation Market: Focus on Acoustophoresis, Buoyancy, Dielectrophoresis, Magnetophoretics, Microfluidics, Optoelectronics, Traceless Affinity, and Other Technologies, 2019-2030' report features an extensive study of the current landscape and future outlook of the growing market for novel cell sorting and separation technologies (beyond conventional methods). The study presents detailed analyses of cell sorters, cell isolation kits, and affiliated consumables and reagents, that are based on the aforementioned technologies.

Advances in the fields of cell biology and regenerative medicine have led to the development of various cell-based therapies, which, developers claim, possess the potential to treat a variety of clinical conditions. In 2018, it was reported that there were more than 1,000 clinical trials of such therapies, being conducted across the globe by over 900 industry players.

Moreover, the total investment in the aforementioned clinical research efforts was estimated to be around USD 13 billion. Given the recent breakthroughs in clinical testing and the discovery of a variety of diagnostic biomarkers, the isolation of one or multiple cell types from a heterogenous population has not only become simpler but also an integral part of modern clinical R&D. The applications of cell separation technologies are vast, starting from basic research to biological therapy development and manufacturing.

However, conventional cell sorting techniques, including adherence-based sorting, membrane filtration-based sorting, and fluorescence- and magnetic-based sorting, are limited by exorbitant operational costs, time-consuming procedures, and the need for complex biochemical labels. As a result, the use of such techniques has, so far, been restricted in the more niche and emerging application areas.

Amongst other elements, the report features:

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/ss4o5a

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

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

View original content:http://www.prnewswire.com/news-releases/novel-cell-sorting-and-separation-markets-2030-300932857.html

See the article here:
Novel Cell Sorting and Separation Markets, 2030 - Yahoo Finance

CLEMSON A group of South Carolina researchers working on new treatments for a variety of illnesses ranging from diabetes to heart disease cheered the decision to grant five years of funding to a biomedical research center based at Clemson University.

The National Institute of General Medical Sciences is providing $5.7 million to the South Carolina Bioengineering Center for Regeneration and Formation of Tissues. Its the institutes third round of competitive funding for the center, also called SCBioCRAFT.

Naren Vyavahare, the Hunter Endowed Chair of Bioengineering at Clemson University, is the principal investigator of the grant that created the the South Carolina Bioengineering Center for Regeneration and Formation of Tissues.

In its first decade, the center matched seasoned mentors with 23 early-career researchers. They went on to generate $35 million for research into spinal cord injuries, new ways of growing vascular tissue for grafts and a wide range of other biomedical challenges.

Researchers involved in the center have been awarded 24 patents, spun off four start-up companies and generated 304 articles in peer-reviewed publications.

SC BioCRAFT began operating in 2009 under the direction of Naren Vyavahare, the Hunter Endowed Chair of Bioengineering at Clemson.

It feels good to know that we have junior faculty who have been so successful and have their own independent labs because of this center, Vyavahare said. SC BioCRAFT is playing a key role in building the biomedical research infrastructure in South Carolina.

The centers primary mission is to increase the number of South Carolina biomedical researchers who receive funding for their work from the National Institutes of Health. The research theme revolves around regenerative medicine, a fast-growing field that offers the promise of repairing and regenerating diseased tissues.

With the latest round of funding, SC BioCRAFT researchers will be able to continue advancing tissue-regeneration research, recruit new faculty and build on collaborations that were established in the first decade.

Roger Markwald, an MUSC professor, plays a leading role in SC BioCRAFT.

Researchers with the center are also planning statewide educational programs and a voucher program that will help provide seed funding for new research projects. Further, the grant will give SC BioCRAFTs core facilities a chance to transition from federal funding to a fee-based system.

Martine LaBerge, chair of Clemsons bioengineering department, congratulated Vyavahare and the team for the funding.

SC BioCRAFT has given researchers early in their careers a chance to pursue innovative ideas, while learning from experienced mentors, she said. They have produced an exemplary body of work that is helping create a healthier society and nurturing South Carolinas burgeoning biomedical industry. Their funding is well deserved and being put to good use.

The center is a major force for bringing together researchers, clinicians and other health care professionals from across the state to advance biomedical research. Clemson researchers collaborate closely with colleagues at the Medical University of South Carolina and Prisma Health.

Roger Markwald, an MUSC professor, said the funding reaffirms that SC BioCRAFT is succeeding in its mission.

This collaborative center is helping improve patient care while enhancing biomedical research in South Carolina and encouraging investment in the biomedical industry, said Markwald, who is co-principal investigator on the grant that funds SC BioCRAFT. At the end of the day, patients and the state as a whole benefit most.

SC BioCRAFT is funded through a National Institute of General Medical Sciences program aimed at establishing Centers of Biomedical Research Excellence in 23 states and Puerto Rico. Funding for the centers comes in three phases.

The latest funding for SC BioCRAFT allows it to move into its third phase. SC BioCRAFT received $9.3 million in 2009 for its first phase and $11 million in 2014 for its second phase.

Clemson is now home to three Centers of Biomedical Research Excellence. SC BioCRAFT was the universitys first.

Last year, an $11 million grant funded the South Carolina Center for Translational Research Improving Musculoskeletal Health, or SC-TRIMH, a new research center that will bring together scientists from across South Carolina to change the way musculoskeletal disorders are diagnosed, treated and studied.

In 2016, a $10.5 million COBRE grant funded the Eukaryotic Pathogens Innovation Center, or EPIC. Since the award, EPIC investigators have generated more than $4.5 million in external funding and produced 35 publications.

Tanju Karanfil, vice president for research at Clemson, said that the success of SC BioCRAFT is helping fuel a trend toward collaboration among institutions.

Each institution brings its own strengths and ways of looking at the various healthcare challenges we face, he said. Bringing them together leads to innovative solutions that might have eluded us if we were to work on our own. SC BioCRAFT and our other Centers of Biomedical Research Excellence are great examples of that concept in action.

Anand Gramopadhye, dean of the College of Engineering, Computing and Applied Sciences, said SC BioCRAFTs funding will provide key support for health innovation in South Carolina.

SC BioCRAFT is enabling a collaborative, multidisciplinary research ecosystem that is helping create a healthier, more prosperous South Carolina, he said. I congratulate the team on its well-deserved success.

Read more:
Statewide team lands five years of funding for biomedical research - Clemson Newsstand

Over 20 cell and gene-based therapies are expected to be filed or to receive approval decisions over the next 18 months, according to the Alliance for Regenerative Medicine (ARM).

Earlier this year, the US Food and Drug Administration (FDA) laid out its action plan to deal with the large upswing in the number of investigational new drug (IND) applications for cell and gene therapies it is receiving.

The FDA commissioner has anticipated that he thinks there will be between 10-20 cell and gene therapies by 2025 every year, so this big pipeline that we are generating is definitely starting to produce results, Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM) said at Biotech Week Boston.

Cell and gene therapies moving through the pipeline

The industry advocacy group keeps track of cell and gene therapy pipelines and said that next year could preempt the FDAs prediction and see a gush of regulatory submissions and several approvals.

In gene therapies, there are four filings and two decisions expected in just 2019 covering the US, Europe and Japan, CEO Janet Lambert told conference delegates earlier this month.

Novartis/AveXis Zolgensma (onasemnogene abeparvovec) is awaiting EU and Japanese approval, having already won US approval in May). Meanwhile, PTC Therapeutics and Bluebird Bio are expected to file their gene therapies GT-AADC and Zynteglo (autologous CD34+ cells encoding A-T87Q-globin gene), respectively in the US, and Biomarin is likely to file its hemophilia product Valrox (Valoctocogene roxaparvovec) in the US and Europe before the end of the year.

In 2020, she said it is likely there will be another six gene therapy filings across major markets from Gensight Biologics, Audentes Therapeutics and Orchard Therapeutics.

These things have a way of moving around but still thats 12 gene therapies filings or decisions were expecting in the next 18 months.

There are also at least seven companies (and eight products) vying to file cell-based immune-oncology therapies in Europe or the US between now and the end of 2020, she said, based on company announcements. The firms, in the expected order that they will file, are: Bellicum Pharmaceuticals, Atara Biotherapies, Celgene, Bluebird Bio/Celgene, Kiadis Pharma, Poseida Therapeutics, and Iovance.

The range of indications targeted range from blood cancers, myeloma, melanoma, cervical cancer, she said, and many of these therapies are showing the same kind of outstanding clinical trial results that we showed in [recently] approved therapies.

A further three cell therapies San Bios SB263, Mesoblasts Remestemcel-L, Mesoblast/JCRs Temcell and three tissue-based therapies could also either be filed or receive a regulatory decision in the next 18 months.

These are just the near-term anticipated filings and approvals, as the ARM has tracked a total of 1,069 regenerative medicine clinical trials currently ongoing.

Ongoing clinical trials. Slide c/o Alliance for Regenerative Medicine

According to Lambert, this breaks down in the following way across all tech types and indications: 358 in Phase I, 617 in Phase II, and 94 in Phase III. Nearly 40% of these are gene-modified cell therapies, and close to 30% are gene-delivered or gene-edited products.

When broken down into therapeutic area, it is no surprise that around half of all current clinical trials are in oncology, though the rest of the trials are addressing a broad range of areas from Central Nervous System disorders to hematology to respiratory diseases.

A recent survey taken by Informas KNect365 supports the ARMs findings.

Out of 187 respondents, 60% said their company is focusing on oncology. And just over 40% said they are developing CAR-T products.

For access to the full report, click here.

The rest is here:
In the pipeline: Surge of cell and gene therapies likely - Bioprocess Insider - BioProcess Insider

Our Start-up of the Week is FeelTect, a Galway medtech start-up trying to improve treatment for patients with venous leg ulcers.

AlthoughFeelTect was founded in 2019, the start-ups journey really began in 2016, when Dr Andrew Cameron, one of the companys founders, took part in the BioInnovate Ireland programme.

While participating in the programme, Cameron spent time in University Hospital Galway and the Mayo Clinic in Minnesota, seeking to identify unmet clinical needs in the cardiovascular space.

With the guidance of Dr Georgina Gethin, head of the School of Nursing at NUI Galway, Cameron discovered the need for changes to the application and maintenance of evidence-based pressure during compression therapy of venous leg ulcers (VLUs), to improve healing outcomes and quality of life.

From there, an Enterprise Ireland Commercialisation Fund began in January 2018 in the school of Medicine in NUI Galway, where the BioInnovate programme was held. Cameron and his team went on to develop their product, Tight Alright, which is a pressure-sensing, connected health device for use during VLU compression therapy.

Data-driven therapy optimisation could ultimately redefine the standard of care for VLUs by providing tailored treatment plans for patients DR ANDREW CAMERON

In July 2019, the Health Innovation Hub Ireland supported FeelTect in an end-user study in the wound clinic of University Hospital Galway, which demonstrated that Tight Alright could achieve a six-fold improvement in targeted pressure application by experienced nurses.

That same month, FeelTect received an EIT Health Headstart award to contribute towards the further development and clinical validation of the technology. The company began an accelerator programme in Dublins NDRC in September to support the path to commercialisation.

With an undergraduate degree in chemical and biological engineering and a PhD in tissue engineering, FeelTect CEO Cameron came to Ireland from Australia in 2012 to work as a research fellow in the Tissue Engineering Research Group in the Royal College of Surgeons Ireland (RCSI).

The companys CTO, Dr Darren Burke, has a PhD in regenerative medicine, as well as extensive experience in quality and R&D engineering roles at well-established medical device companies such as Boston Scientific and Merit Medical.

Meanwhile, the CSO of FeelTect is Prof Garry Duffy, a professor of anatomy at NUI Galway. Duffy has coordinated two collaborative EU projects involving multiple academic and industrial partners across different countries. He is affiliated with both the Cram and AMBER research institutions in Ireland.

Venous leg ulcers are painful, odorous and unsightly wounds that affect 1pc of the western population, rising to 3pc to 4pc of the population of people aged 65 years and older, Cameron told Siliconrepublic.com.

He added that there are 2.2m new cases of VLUs in the US and Europe every single year, with a serviceable addressable market of 923m.

The average VLU healing time is four months. 20pc dont heal within a year. These wounds, which are associated with age and obesity, result from a failure of the valves in the veins of the leg (known as venous insufficiency) that causes an accumulation of blood in the lower limbs and a breakdown of subcutaneous tissues.

Its estimated that treatment costs for VLUs in the UK are between 597m and 922m per year, while in the US treatment costs can be up to $14.9bn annually.

According to Cameron, the average cost of treating a single VLU is approximately 8,400 under current treatment times. The proven and widely accepted treatment for the condition is compression therapy, where bandages are used to apply pressure to the leg.

This overcomes the venous insufficiency, restoring venous return and promoting healing. However, if insufficient pressure is applied, compression therapy is ineffective. Excessive application of pressure can impede arterial circulation and cause danger to the patient, he said.

Therefore, it is extremely important that pressure is applied accurately. Yet, studies have shown that targeted pressure is only achieved about 10pc of the time during compression bandage application. Tight Alright was developed to remove the uncertainty from the treatment of VLUs.

FeelTects device uses wearable, connected health technology to measure and monitor sub-bandage pressure during compression therapy, both inside and outside of the clinical setting.

This is quite significant, according to Cameron. The digital capabilities of Tight Alright potentially enable data-driven therapy optimisation, which could ultimately redefine the standard of care for VLUs by providing tailored treatment plans for patients.

Unlike existing products, Tight Alrights accompanying mobile app can incorporate training, educational and motivational material to enhance user engagement with their therapy. The start-ups team predicts that the product can reduce healing time by five weeks, from an average of 17 weeks when patients are treated by unspecialised nurses.

While FeelTects initial business strategy is to improve the treatment of VLUs with a connected health device, the companys long-term plan is to form a digital wound care platform that encompasses information on the treatment received by a patient, as well as the state of their wound.

This will allow for data correlation that can direct optimised, individualised treatment regiments, Cameron said.

In this way, Tight Alright also aligns with FeelTects ambition to develop strategic partnerships with other wound care companies that could benefit from Tight Alrights digital capabilities, whilst in turn providing access to their distribution channels. Product development expertise and clinical validation resources.

Cameron added that he has receiveda lot of positive traction and accolades for the technology, includingMost Cost Effective Product at the IMSTA Medtech Awards last week, but plans for market entry arent on the cards until the first quarter of 2022.

Dr Andrew Cameron at the IMSTA Medtech Awards in October 2019. Image: FeelTect

We are currently commencing fundraising for a seed investment round, Cameron said.

It will cover the costs of design freeze, scalable manufacturing, clinical validation, regulatory approval, IP, operations and personnel for 24 months. Were also in the process of submitting funding applications to various funding agencies to support our funding requirements.

Want stories like this and more direct to your inbox? Sign up forTech Trends, Silicon Republics weekly digest of need-to-know tech news.

Original post:
Galway medtech FeelTect is going out on a limb to treat venous leg ulcers - Siliconrepublic.com

LabCentral 238 will straddle both buildings and have a connecting bridge. The facility will include dozens of modest-sized bioreactors that are used to make biologic drugs produced from living organisms.

Its not just more of the same, Johannes Fruehauf, cofounder and president of LabCentral, said of the project scheduled to be announced Tuesday.

His nonprofit has rapidly expanded as average monthly rents for traditional lab space in white-hot Kendall Square have approached $100 a square foot, raising demand for a less costly alternative.

LabCentral 238 is scheduled to open in autumn 2021, Fruehauf said. It will lease shared space to about 20 venture-backed startups that employ a total of about 300 people, he said. The project is getting a boost from a Tokyo-based drug maker and the state.

Astellas Pharma Inc., the Japanese firm, will invest $12.5 million in the biomanufacturing incubator, plus $450,000 in LabCentrals existing 103,000-square-foot complex a short walk away at 700 Main St.

Astellas has a regenerative medicine subsidiary in Marlborough and will be a founding sponsor of LabCentral 238. Its executives hope to get an early look at promising startups at the new outpost and maybe even make deals with them.

We need more access to innovative startup companies, especially in the cell therapy and gene therapy space, said Yoshitsugu Shitaka, president of the Astellas Institute for Regenerative Medicine, whose company will have an office in LabCentral 238.

The Massachusetts Life Sciences Center, a quasi-public agency that promotes the states biotechnology industry, plans to contribute $5 million to LabCentral 238, said Fruehauf. All told, the new incubator will cost $30 million to $40 million to build.

One of the goals of state officials is to encourage more biomanufacturing jobs in Massachusetts. Such jobs typically pay less than what scientists earn but often only require associate degrees or one-year certificates instead of advanced degrees.

Massachusetts consistently ranks at or near the top of states with high-paying biopharma research jobs. But it lagged behind nine other states in its total biomanufacturing positions, according to the most recent report by the Massachusetts Biotechnology Council trade group.

We need to present Massachusetts to the rest of the world as a turnkey state where you can discover it, develop it, commercialize it, and manufacture it all here, said Travis McCready, president and CEO of the Massachusetts Life Sciences Center.

Researchers who lease space at LabCentral say that once they identify new potential medicines at the incubator, they often have to wait months or years to pay contract manufacturing organizations, or CMOs, to actually make the substances for clinical trials, according to McCready and Fruehauf. Thats because CMOs are often too busy working for large drug companies.

LabCentral 238 will make early versions of the drugs more quickly and at a lower cost, although the substances wont meet the rigorous standards that CMOs must follow to allow the compounds to be tested in patients. Then, if the substances still seem promising, the startups at LabCentral will pay CMOs to produce them at the higher standards.

Some of the startups in the new lab may have already been LabCentral tenants, but they dont have to be, Fruehauf said.

The other tenants at 238 Main St. are expected to include one of the worlds biggest drug makers. Bayer AG, the German pharmaceutical giant, said in April it has signed a 12-year lease for 62,100 square feet on the top two floors and a common area, for a total of $100 million. Bayer is expanding its presence in Kendall Square.

LabCentral has grown rapidly since it was founded six years ago at 700 Main St., where technology pioneer Thomas A. Watson received the first long-distance phone call in the 1800s, and Polaroid founder Edwin Land kept an office a century later. It originally opened with 28,000 square feet on the ground floor of 700 Main St. but has expanded repeatedly because of demand.

About 320 scientists and entrepreneurs from 70 startups currently rent lab benches, offices, or suites, and the space doesnt come cheap. A biotech entrepreneur told the Globe this year that he was paying $4,600 a month for a lab bench and a couple of shelves.

But he and other renters say the fees are worth it. They get access to millions of dollars in laboratory equipment, tips from other young scientists in the incubator, amenities like unlimited espresso and two nap rooms, and face time with local drug developers and venture capitalists.

The state has more than two dozen incubators, according to the Massachusetts Life Sciences Center.

Jonathan Saltzman can be reached at jsaltzman@globe.com

More here:
LabCentral to double its Kendall Square space - The Boston Globe

Oct. 5, 2019 11:01 UTC

SAN FRANCISCO--(BUSINESS WIRE)-- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, today announced new positive data from ASPIRO, the clinical trial evaluating AT132 in patients with X-Linked Myotubular Myopathy (XLMTM). The data are being presented today at the 24th International Annual Congress of the World Muscle Society by Dr. James J. Dowling, Hospital for Sick Children, Toronto, Canada, during the Clinical Trial Highlights 7 session scheduled to begin at 1:00pm Central European Summer Time (CEST)/7:00am Eastern Time (ET).

"The new ASPIRO data shared today builds upon the encouraging efficacy and safety profile seen to date with AT132, stated Dr. Dowling. Treated patients across both dose cohorts show significant reductions in ventilator dependence and the progressive attainment of developmental motor milestones, suggesting that AT132 has the potential to deliver transformative benefit to patients and families living with XLMTM.

We remain focused on our goal of rapidly progressing AT132 toward global regulatory approvals, stated Natalie Holles, President and Chief Operating Officer of Audentes. Importantly, we have fully enrolled 14 patients into the ASPIRO dose escalation cohorts, and plan to complete enrollment of the ASPIRO pivotal expansion cohort imminently. We remain on track to submit a BLA in the United States in mid-2020 and a MAA in Europe in the second half of 2020.

Data Summary

The newly reported data include safety and efficacy assessments as of the August 7, 2019 data cut-off date for 12 patients enrolled in the ASPIRO dose escalation cohorts. The data includes 48 weeks or more of follow-up for seven patients enrolled in Cohort 1 (1x1014 vector genomes per kilogram (vg/kg); six treated and one untreated control) and 24-48 weeks of follow-up for five patients in Cohort 2 (3x1014 vg/kg; four treated and one untreated control). Key assessments include neuromuscular function as assessed by the achievement of motor milestones and improvement in CHOP INTEND score, and respiratory function as assessed by reduction in ventilator dependence and improvement in maximal inspiratory pressure (MIP). Todays presentation does not include new muscle biopsy data.

Efficacy

Patients receiving AT132 have achieved significant and durable reductions in ventilator dependence, an endpoint considered to be closely correlated with morbidity and mortality in XLMTM patients. To date, the first seven patients treated (all six treated patients in Cohort 1 and the first patient treated in Cohort 2) have achieved ventilator independence. All treated patients continue to show gains in neuromuscular function, with the first seven patients treated achieving the ability to rise to a standing position, or walk.

Safety

AT132 has been generally well-tolerated and has shown a manageable safety profile across both dose groups. Since the last data update in May 2019, there has been one new serious adverse event (SAE) in Cohort 2, an episode of joint swelling that resolved without treatment. Results to date indicate no clinically meaningful differences in the safety and tolerability profile of AT132 between the 1x1014 vg/kg and 3x1014 vg/kg dose cohorts.

Next steps in the AT132 development program include the completion of enrollment and follow-up of patients in the ASPIRO pivotal expansion cohort, designed to confirm the safety and efficacy profile of AT132 at a dose of 3x1014 vg/kg, and preparations for filing of a Biologics License Application (BLA) for AT132 in the United States planned in mid-2020 and filing of a Marketing Authorization Application (MAA) in Europe planned for the second half of 2020.

About X-linked Myotubular Myopathy

XLMTM is a serious, life-threatening, rare neuromuscular disease that is characterized by extreme muscle weakness, respiratory failure, and early death. Mortality rates are estimated to be 50 percent in the first 18 months of life, and for those patients who survive past infancy, there is an estimated additional 25% mortality by the age of 10. XLMTM is caused by mutations in the MTM1 gene that lead to a lack or dysfunction of myotubularin, a protein that is needed for normal development, maturation, and function of skeletal muscle cells. The disease affects approximately 1 in 40,000 to 50,0000 newborn males.

XLMTM places a substantial burden of care on patients, families and the healthcare system, including high rates of healthcare utilization, hospitalization and surgical intervention. More than 80 percent of XLMTM patients require ventilator support, and the majority of patients require a gastrostomy tube for nutritional support. In most patients, normal developmental motor milestones are delayed or never achieved. Currently, only supportive treatment options, such as ventilator use or a feeding tube, are available.

About AT132 for the treatment of X-linked Myotubular Myopathy

Audentes is developing AT132, an AAV8 vector containing a functional copy of the MTM1 gene, for the treatment of XLMTM. AT132 may provide patients with significantly improved outcomes based on the ability of AAV8 to target skeletal muscle and increase myotubularin expression in targeted tissues following a single intravenous administration. The preclinical development of AT132 was conducted in collaboration with Genethon (www.genethon.fr).

AT132 has been granted Regenerative Medicine and Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track, and Orphan Drug designations by the U.S. Food and Drug Administration (FDA), and Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA).

About Audentes Therapeutics, Inc.

Audentes Therapeutics (Nasdaq: BOLD) is a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases. We are leveraging our AAV gene therapy technology platform and proprietary manufacturing expertise to develop programs across three modalities: gene replacement, vectorized exon skipping, and vectorized RNA knockdown. Our product candidates are showing promising therapeutic profiles in clinical and preclinical studies across a range of neuromuscular diseases. Audentes is a focused, experienced and passionate team driven by the goal of improving the lives of patients.

For more information regarding Audentes, please visit http://www.audentestx.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the timing of regulatory filings for AT132 and planned activities for the ASPIRO pivotal expansion. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates and obtain regulatory approval of and ultimately commercialize its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to protect intellectual property and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191005005003/en/

Continued here:
Audentes Therapeutics Presents New Positive Data from ASPIRO, the Clinical Trial Evaluating AT132 in Patients with X-Linked Myotubular Myopathy...

Liver disease patients could one day benefit from a new cell therapy that has just completed its first clinical trial.

Researchers who tested the potential treatment in patients with liver cirrhosis where long term damage produces scarring found the therapy had no significant adverse effects.

Now the team, based at the Universitys MRC Centre for Regenerative Medicine, is to gauge the effectiveness of the treatment which is based on white blood cells called macrophages, that are key to normal liver repair.

The next stage of the trial will measure whether the therapy helps the liver to reduce scarring and stimulate regeneration. The results should be known within the next two years.

At present the only successful treatment for end-stage liver cirrhosis which claims around 14,000 lives in the UK each year (British Liver Trust) is an organ transplant. The safety trial is a vital step forward in finding an alternative therapy.

During the trial scientists took cells from the blood of nine patients with the disease and turned them into macrophages, in the Scottish National Blood Transfusion Services (SNBTS) cell therapy facility.

The new cells were then re-injected into the patient with the hope of repairing the damaged organ from within.

Causes of liver cirrhosis include infections such as hepatitis C, obesity, alcohol excess and some genetic and immune conditions.

Liver cirrhosis is a major healthcare issue in the UK and is one of the top five killers. The results from this first safety trial are encouraging and we can now progress to testing how effective it is in a larger group of people. If this was found to be effective it would offer a new way to tackle this important condition.

The research which was published in the journal Nature Medicine, received funding from the Medical Research Council and was conducted in partnership with the SNBTS and the Cell and Gene Therapy Catapult.

Read the original here:
Cell therapy safe for liver patients, trial shows - Mirage News

One of the most promising and rapidly developing areas in the treatment of pain is regenerative medicine. These treatments helpthe body heal or rebuild itself. While, the body can naturally heal many types of injuries, newer techniques, such as stem cell therapy, may enhance the bodys ability to heal. But, what is regenerative medicine and how does it work?

Regenerative medicine uses cutting-edge therapies to help the body replace, re-engineer, or regenerate human cells, tissues, or organs that were damaged or injured. The most common applications of regenerative medicine are in new organ growth orrepair and cellular therapies. These both use a patients own cells to stimulate repair tissue and improve function.

The AABB reports that up to one in three people in the U.S. could benefit from regenerative medicine.

Patients with osteoarthritis of the hips and knees can use PRP therapies to stimulate the bodys own healing processes and reduce inflammation. Stem cell therapy could be used to revitalize worn-out intervertebral discs.In the very near future, patients could benefit from lab-grown bladder or heart transplants. These transplants would be derived from the patients very own cells to reduce the risk of transplant rejection or infection.

Regenerative medicine began when the first bone marrow and solid organ transplants were done decades ago. The first successful kidney transplant was in 1954. Successful liver, pancreas, and heart transplants took place in the 60s. From this perspective, regenerative medicine is a fairly established medical practice.

From there, regenerative medicine arose as atruly a cross-disciplinary approach to healthcare. As UPMC notes:

Regenerative medicine is a new field that brings together experts in biology, chemistry, computer science, engineering, genetics, medicine, robotics, and other fields to find solutions to some of the most challenging medical problems faced by humankind.

In the following TED talk, Alan Russell talks a bit aboutthe history of regenerative medicine andwhy so many healthcare professionals are excited about its potential uses.

There are three main types of regenerative therapy, as ARMI explains:

While scientists are working on new types of regenerative medicine that can actually create new body parts from a patients own cells and tissues, this research is still in its infancy. Regenerative medicine for self-repair and cellular therapy, however, is performed on patients today.

Regenerative cellular and self-repair therapies use the following processes:

When an embryo is developing in the womb, it starts out as a mass of stem cells. As the embryo grows, the stem cells multiply and become the specialized cells that make up a body.

Stem cells are body cells that have the potential to become the cells for many types of bodily tissues. Human embryos are a widely recognized and controversial source of stem cells, but stem cells can also be found in the body tissues of adults, such as bone marrow and blood as well as placental tissue. Other adult cells (e.g., skin cells) can be modified through the injection of specific genes or molecules to become stem cells. The various sources of stem cells often have different properties. They are generally capable of becoming some, but not all, types of cells.

Various treatments that involve the extraction of stem cells and their injection into damaged tissues have been developed. Some of these treatments involve taking a persons own stem cells and injecting them into a site of injury. Other treatments rely on stem cells from adult donors. Bone marrow is perhaps the most commonly-accessed source of adult stem cells for regenerative therapies. However, harvesting stem cells from bone marrow is still a surgical procedure requiring anesthesia or sedation.

Though medical research on these treatments is progressing, stem cell treatments are generally considered experimental. They are only approved in the United States as a part of clinical trials. These treatments hold great promise because they may be able to treat certain conditions, such as discogenic back pain, that are difficult to effectively relieve with other methods.

This video from Mayo Clinic gives a quick primer on stem cells.

Another regenerative medicine treatmentis called platelet rich plasma (PRP) therapy. This technique was developed in the 1970s and has been adopted in a variety of medical practices, such as orthopedic surgery, plastic surgery and sports medicine. This treatment gained wider recognition after a number of professional athletes were reported to use it to aid in their recoveries.

PRP therapy involves drawing a small volume of blood from the patient and spinning this blood in a centrifuge. This separates the blood into different layers, each containing concentrated amounts of the substances naturally found in blood: serum in the top layer, white blood cells and platelets in the middle layer, and red blood cells in the bottom layer.

The middle layer, known as PRP concentrate, is then separated from the rest of the blood. This PRP concentrate contains three to five times the normal concentration of the platelets and growth factors that are used in the bodys own healing process. This PRP concentrate is then injected back into the patients body at the site of their injury, for example, into the site of an injured tendon or ligament. This process is then repeated over the course of weeks with additional PRP concentrate being drawn and injected each time.

The platelets injected release hundreds of different proteins involved in the regeneration of tissues. Though research on this treatment continues, the existing research has shown increased healing of several different types of body tissues. Because the patient is injected only with concentrate from their own blood, PRP therapy avoids safety issues of ensuring donor compatibility when using blood and tissue donation.

A pain doctor from Arizona explains how platelet-rich plasma therapy works. You can also find out more about PRP therapy in our posts on the subject.

The process for acquiring the cells for regenerative therapy is different for each type, but the application of the therapy is a simple, straight-forward process no matter the cell type.

The acquisition of adult stem cells typically requires surgery. Amniotic cells come from the amniotic membrane of the placenta, so a placenta is required. This can be a patients own placenta thats been saved, or it can be from a family member. Immediate family members are more likely to be a close-enough match to ensure the cells wont be rejected.

Platelet-rich plasma requires a blood draw. A patients blood is spun in a centrifuge to separate the platelets. The concentrated platelets are combined with residual blood, and the resultant compound is injected into the painful location on the body. From beginning to end, the process of drawing and preparing the blood and injecting the platelet-rich plasma takes between one and two hours.

After the regenerative cells have been injected, the patient is usually able to go home immediately. While physicians often suggest avoiding any strenuous activity for a few days, its possible to go back to regular day-to-day activities right away. Some irritation, soreness, bruising, or other minor discomforts might be present for a few days, but generally there are few side effects of regenerative therapy.

The injected regenerative cells should stimulate the healing and the growth of new tissue. For instance, in degenerative conditions like osteoarthritis, this may mean the growth of new cartilage around joints. With torn muscles or ligaments, regenerative therapy should support the growth of new tissues to heal the injury.

Chronic pain is often the after-effect of degeneration, injury, or illness. Part of the body is left damaged somehow, and that damage causes pain. Most pain therapies concentrate on controlling the pain with medications, injections, or devices that block pain signals in one way or another.

Regenerative therapies are well-suited to treating musculoskeletal pain, or pain thats caused by something in the muscles or bones. Conditions that can respond well to regenerative therapies include:

Compared to other chronic pain therapies, regenerativemedicine has an extremely low risk of side effects.

Regenerative therapies typically involve nothing more than an injection to the painful area of the body, making them very safe. Because the injected compound, whether its stem cells, amniotic cells, or platelet-rich plasma, is usually from the patients own body to begin with, theres almost no risk of infection.

Additionally, one of the biggest risks in the transplant of biological materials, such as organ transplants, is rejection. This is when the body realizes that the transplanted material is foreign and attacks it. However, since the cells used in regenerative therapy are the patients own natural cells, there is no chance at all of rejection.

Stem cell therapy utilizing adult stem cells from the bone marrow is the exception. The harvesting process requires a surgical procedure, which carries the same risks as any other surgery. If, however, the procured stem cells are used on the same person theyre from, this still has the benefit of being extremely low-risk for infection, with no risk of rejection.

It often takes at least two weeks to notice any difference in pain after regenerative therapy, although in some people it may take even longer. In some cases, there may be no discernable pain reduction after regenerative therapy. However, because of its low risk of side effects, it may still be worth discussing this treatment option with a physician.

Regenerative medicine is already available today, in many different applications. According to the NIH:

Naturealso keeps a running list of the scholarly articles about current regenerative medicine research and trials.

As to wide-spread availability of these therapies, that depends. Since stem cell and regenerative medicine is still in its infancy, many insurance providers dont cover the cost of these procedures. That means many procedures are paid out of pocket. You may find discounted optionsby reaching out to pain clinics and independent medical groups who are performing their own studies and patient trials onregenerative medicine.

Once there is more solid research backing these procedures, regenerative medicine should become more easily available and cost-effective for all patients.

From made-to-order organs to smart biomaterials that could help form functional tissues, the possibilities and future of regenerative medicine is endless. The NIH notes:

Imagine a world where there is no donor organ shortage, where victims of spinal cord injuries can walk, and where weakened hearts are replaced. This is the long-term promise of regenerative medicine, a rapidly developing field with the potential to transform the treatment of human disease through the development of innovative new therapies that offer a faster, more complete recovery with significantly fewer side effects or risk of complications.

Organizationslike the Stanford Medicine Institute for Stem Cell Biology and Regenerative Medicine and Mayo Clinics Center for Regenerative Medicine areat the forefront of researching new applications for regenerative medicine.

Many healthcare researchers today are grappling with the questions of what is regenerative medicine and what potential uses it can have for patients. To learn more about the future of regenerative medicine, check out the following articles:

Regenerative medicine clinics can be found in larger medical institutions like Mayo Clinic or Stanford Medicine, however there are also local doctors in your area who are running stem cell and regenerative medicine trials for their patients. Pain clinics, in particular, are offering regenerative therapies for conditions like arthritis and back pain.

You can find a pain clinic in your area by clicking the button below. The clinicsServices page will list which regenerative therapies they offer. Or, you can call their office and ask.

Read the original:
What Is Regenerative Medicine? - Pain Doctor

OPTIMAL Pain & Regenerative Medicine focuses on treating patients through restorative and pain management solutions. We offer state of the art biologic treatments that have changed the way medicine is practiced today. Our practice specializes in the treatment of neck and back pain, arthritis, neuropathy, diseases, headaches, joint and soft-tissue injuries and sports related injuries.

Dr. Scott Berlin, Dr. Andrew Cottingham,Dr. Michael Phillipsand Dr. Tibor Racz of OPTIMAL Pain & Regenerative Medicine strive to be at the forefront in providing cutting edge pain management and regenerative medicine options to the Dallas-Ft. Worth metroplex. As a group of board certified anesthesiologists, their collective expertise includes a full range of innovative treatment options. No matter what your stage of life, OPTIMAL delivers comprehensive, current and most importantly, compassionatemedical care.

To date, it is estimated that over 116 million American adults are affected by some degree of chronic pain. Pain management is important for patients who are suffering from acute, chronic and cancer pain. Pain management includes injection/interventional therapy, implantable therapies, physical therapy,medication managementand other suitable treatment options.

Regenerative medicine offers asolution to patients who have conditions that seem beyond repair. Regenerative medicine is a process of replacing the bodys owns cells, tissues or organs to restore normal function. OPTIMAL specializes in minimally invasive bone marrow aspirate concentrate (BMAC), a stem cell therapy for back pain, as well as platelet rich plasma therapy (PRP) for joints or soft tissue injuries.

To get to learn more about our practice please review our physician profiles and patient education.

See the original post:
Pain Management & Regenerative Medicine Specialists DFW