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.

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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.

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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.

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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.

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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.

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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.

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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

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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/

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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.

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Cell therapy safe for liver patients, trial shows - Mirage News