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Axovant Gene Therapies to Present at 2019 Cell & Gene Meeting on the Mesa – GlobeNewswire

Posted: September 26, 2019 at 8:41 am

BASEL, Switzerland, Sept. 25, 2019 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that Parag Meswani, Pharm.D., senior vice president, commercial strategy & operations, will present at the annual Cell & Gene Meeting on the Mesa at 4:30 PM PT on October 3, 2019 in Carlsbad, California.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers.

A live video webcast of all company presentations will be available at: http://www.meetingonthemesa.com/webcast and will also be published on the conference website shortly after the event. Please visit http://www.meetingonthemesa.com for full information including registration.

About AxovantAxovant, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visitwww.axovant.com.

About RoivantRoivant Sciences aims to improve health by rapidly delivering innovative medicines and technologies to patients. It does this by building Vants nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. For more information, please visitwww.roivant.com.

Contacts:Media and InvestorsParag V. Meswani, Pharm.D.(212) 547-2523media@axovant.cominvestors@axovant.com

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Bluebird reveals positive gene therapy data – PharmaTimes

Posted: September 26, 2019 at 8:41 am

Bluebirdbio has announced updated results from a clinical development programmeevaluatingits investigational Lenti-D gene therapy inpatients with cerebraladrenoleukodystrophy (CALD).

The primary efficacy endpoint in the study isthe proportion of patients who are alive and free of major functionaldisabilities at month 24.Of those patients who have or would have reached 24months of follow-up and completed the study, 88% continue to be alive and majorfunctional disabilities-free in a long-term follow-up study.

Further data show that three out of the 32treated patients did not or will not meet the primary efficacy endpoint; twopatients withdrewfrom the study at investigator discretion, and oneexperienced rapid disease progression early on-study resulting in MFDs anddeath.

The company also announced that the 14 patientscurrently on-study have less than 24 months of follow-up and have shown noevidence of MFDs.

With the longest follow-up from the Phase II/IIIStarbeam study now up to five years, the data show that all boys with CALD whoweretreated with Lenti-D and were free of major functional disabilities at 24months continued to be major functional disabilities -free,explained DavidDavidson, chief medical officer of the company.

He continued, Importantly, there were noreports of graft failure or treatment-related mortality, and adverse eventswere generallyconsistent with myeloablative conditioning. These resultssupport the potential of Lenti-D as a treatment for CALD, which we hope maybecome an option for the boys and their families affected by this devastatingdisease.

CALD is the most severe manifestation ofadrenoleukodystrophy (ALD), a rare metabolic disorder. Approximately 35-40% ofboysdiagnosed with ALD will progress to CALD, typically between the ages ofthree and 12 years. It is characterised by a rapidly progressiveneurologicdecline leading to severe loss of neurologic function and death, in mostuntreated patients.

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BioMarin, Pioneer in Phenylketonuria (PKU) Therapies, Submits Clinical Trial Application (CTA) in UK for Investigational Gene Therapy for PKU -…

Posted: September 26, 2019 at 8:41 am

SAN RAFAEL, Calif., Sept. 26, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN), a pioneer in treatments for rare disease Phenylketonuria (PKU) and in gene therapies, today announced that it has submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to reduce blood phenylalanine (Phe) concentrations levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore Phe metabolism in patients with PKU, normalize plasma Phe level, and enable a normal diet. The company expects to start enrolling patients in a Phase 1/2 trial early next year and is actively preparing regulatory submissions for other countries. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and its second gene therapy development program. BMN 307 follows BioMarin's first investigational gene therapy program, valoctocogene roxaparvovec for severe hemophilia A, currently in a Phase 3 study.

"This clinical trial application marks the latest milestone in BioMarin's 15-plus year commitment to the PKU community. BioMarin has brought the only two approved therapies for PKU to patients around the world," said Hank Fuchs, President, Worldwide Research and Development at BioMarin. "Leveraging our expertise in gene therapy, we are pleased to be adding a new gene therapy product, which is potentially a transformative solution, to build on our current achievements for the PKU community."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is found in most forms of protein. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychiatric-related issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

Phase 1/2 Study

The Phase 1/2 study will evaluate the safety efficacy and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The Phase 1/2 study consists of a dose-escalation phase, followed by a dose expansion phase. In addition, there is also an observational study in PKU patients to measure markers of disease and clinical outcomes over time inpatients.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has treated approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, in addition to the investigational gene therapy BMN 307 in development. BioMarin has conducted 40 clinical studies in PKU and has sponsored 38 external clinical studies. BioMarin researchers have authored 54 publications in medical and scientific journals on PKU, and BioMarin has supported another 52 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a corrected copy of the defective gene. The functional gene is inserted into a vector, containing a small DNA sequence, that acts as a delivery mechanism, providing the ability to deliver the functional gene to cells. The cells can then use the information to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has constructed, commissioned, and validated one of the first gene therapy manufacturing facilities of its kind in the world, which is located inNovato, California. This facility can support up to 4,000 doses per year, and the production process was developed in accordance with International Conference on Harmonisation guidance to facilitate eventual worldwide registration with health authorities. In 2018, the International Society for Pharmaceutical Engineering (ISPE) selected the Company's gene therapy manufacturing facility as the Facility of the Year Category Winner for Project Execution. The BMN 307 PKU gene therapy clinical production occurred at commercial scale in this facility. Production of material at commercial scale in the state of the art facility significantly reduces risk associated with making process changes later in development and can speed overall development timelines.

About Phenylketonuria

PKU, or PAH deficiency, is a genetic disorder affecting approximately 50,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most adult patients to adhere to the strict diet to the extent needed for achieving adequate control of blood Phe levels.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the development of BioMarin's BMN 307 program generally, BioMarin's planned submissions to regulatory authorities for BMN 307, BioMarin's gene therapy manufacturing capabilities, and the timing and results of BioMarin's planned Phase 1/2 trial of BMN 307. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:results and timing of current and planned preclinical studies and clinical trials of BMN 307; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; the risks related to commercialization of BMN 307 and our ability to manufacture sufficient quantities of BMN 307; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2019 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

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BioMarin, Pioneer in Phenylketonuria (PKU) Therapies, Submits Clinical Trial Application (CTA) in UK for Investigational Gene Therapy for PKU -...

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

Posted: September 26, 2019 at 8:41 am

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

REGULATORY LANDSCAPE: Cell & Gene Therapy

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

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

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

FULL-SERVICE SUPPORT

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

Product Development & Review

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

Quality, Manufacturing & Compliance

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

UNMATCHED EXPERTISE

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

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

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

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

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

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

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

ABOUT GREENLEAF

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

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

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

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

Posted: September 26, 2019 at 8:41 am

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

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

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

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

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

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

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

In the current study:

About Hackensack Meridian Health Hackensack University Medical Center

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

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

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BlueRock Therapeutics to Present at 2019 Cell & Gene Meeting on the Mesa – P&T Community

Posted: September 26, 2019 at 8:41 am

CAMBRIDGE, Mass., Sept. 25, 2019 /PRNewswire/ --BlueRock Therapeutics, today announced that Emile Nuwaysir, Ph.D., Chief Executive Officer of BlueRock and Vice Chairman of the Alliance for Regenerative Medicine (ARM), will present at the Cell & Gene Meeting on the Mesa, to be held Oct. 2-4 in Carlsbad, Calif.

The following are specific details regarding BlueRock's participation at the conference: Event: BlueRock Therapeutics Company PresentationDate: Wednesday, Oct. 2Time: 11:45 a.m. - 12:00 p.m. PDTLocation: Cognate Bioservices Ballroom,Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

Event: Panel: Cell and Gene Therapy for Neurological Product IndicationsDate: Wednesday, Oct. 2Time: 4:00 - 5:00 p.m. PDTLocation: BlueRock Therapeutics Ballroom, Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

A live video webcast of all company presentations and workshops will be available at: http://www.meetingonthemesa.com/webcast and will also be published on the conference website shortly after the event.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers.

About BlueRock Therapeutics BlueRock Therapeutics, a wholly-owned and independently operated subsidiary of Bayer AG, is a leading engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock Therapeutics'CELL+GENE platform harnesses the power of cells for new medicines across neurology, cardiology and immunology indications. BlueRock Therapeutics' cell differentiation technology recapitulates the cell's developmental biology to produce authentic cell therapies, which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock's culture is defined by scientific innovation, highest ethical standards and an urgency to bring transformative treatments to all who would benefit. For more information, visit http://www.bluerocktx.com.

Company Contact:Candice Ellis, 978-879-7273Associate Director, Investor Relations and Corporate Communicationscellis@bluerocktx.com

Media Contacts: David Schull or Maggie BellerRusso Partners, LLC(646) 942-5631Maggie.beller@russopartnersllc.com

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Precision BioSciences to Present at Upcoming Industry and Investor Conferences – Yahoo Finance

Posted: September 26, 2019 at 8:41 am

Company to Participate in the Alliance for Regenerative Medicine Cell & Gene Meeting on the Mesa, Chardan Genetic Medicines Conference and Jefferies Gene Therapy Summit

DURHAM, N.C., Sept. 26, 2019 (GLOBE NEWSWIRE) -- Precision BioSciences, Inc. (DTIL), a genome editing company dedicated to improving life through the application of its pioneering, proprietary ARCUS platform, today announced that members of its senior leadership team will participate in three industry and investor conferences focused on gene editing and gene therapy in October. Presentations will consist of corporate overviews and a panel discussion. Participation at the Jefferies Gene Therapy Summit will be limited to private meetings.

Alliance for Regenerative Medicine Cell & Gene Meeting on the MesaDate: October 2-4, 2019Presentation Time: October 3, 4:15 p.m. PTPanel Time: October 4, 9:45 a.m. PTLocation: Carlsbad, CASpeaker: Michael Dombeck, Senior Vice President, Corporate Development

Chardan Genetic Medicines ConferenceDate: October 7-8, 2019Presentation Time: October 8, 8:30 a.m. ETLocation: New York, NYSpeaker: Matt Kane, Chief Executive Officer

Jefferies Gene Therapy SummitDate: October 8, 2019Location: New York, NYMatt Kane, Chief Executive Officer (investor meetings)

A live audio webcast of the Chardan presentation will be available here (webcast link) and up-to-date details for each event can be found on the "Events & Presentations" page in the Investors section of the Precision BioSciences website (www.precisionbiosciences.com). A replay of the Chardan presentation will also be archived for 30 days on the Events & Presentations page.

About Precision BioSciences, Inc.Precision BioSciences is dedicated to improving life (DTIL) through its proprietary genome editing platform, ARCUS. Precision leverages ARCUS in the development of its product candidates, which are designed to treat human diseases and create healthy and sustainable food and agriculture solutions. Precision is actively developing product candidates in three innovative areas: allogeneic CAR T immunotherapy,in vivogene correction and food. For more information regarding Precision, please visitwww.precisionbiosciences.com.

Investor Contact:Jason WongBlueprint Life Science GroupTel. (415) 375-3340 Ext. 4jwong@bplifescience.com

Media Contact:Cory TrombleeScient Public RelationsTel. (617) 571-7220cory@scientpr.com

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Rambo: Last Blood Ending & Real Meaning Explained | Screen Rant – Screen Rant

Posted: September 25, 2019 at 12:46 am

Warning: SPOILERS ahead for Rambo: Last Blood.

By the time the credits roll on Rambo: Last Blood, many people are dead, and a significant chapter of John Rambo's journey has come to an end. Last Blood is one of the most violent movies of the decade, laying bare the potential barbarism inherent in humanity. As stated in an earlier entry in the series, "When you're pushed, killing's as easy as breathing."

It came as a surprise when the film was announced, since Rambo 2008 served as something of a definitive ending for the long-running war-themed franchise. That film ended with Rambo returning home to Arizona after decades away from his country. However, even after the journey home, the battle still continues.

Related: John Rambo's Tragic Backstory (& How The Character Changed Over Time)

In addition to all the righteous blood-letting and testosterone-fueled declarations of murderous intent, Rambo: Last Blood is filled with important developments for the character of John Rambo, as well as the politically-charged themes that have defined the series since its inception. Despite the Mexico/Arizona setting, the film doesn't make any polarizing statements regarding the real-life political showdown at the US/Mexico border, but that doesn't mean it has nothing to say.

Many Neo-Westerns see their heroes head off to their final battle with no expectations of getting out alive. Last Blood feels different because, while Rambo is prepared to die, he is confident enough in his skills to tell his adopted family that he's going to return to his old life as a drifter after he leaves his old family farm. During the final battle itself, he brutally eviscerates the gang attacking his homestead, and he's only injured as a result of his own pride; he denies himself the quick and efficient kill on the gang leader, Hugo, opting instead to "make him feel his rage." While this leads to one of the most shockingly violent kills ever captured on film, with Hugo's heart being ripped out of his chest, it also leads to Rambo being grievously injured by his final opponent.

As the movie ends, Rambo struggles to maintain his composure as he makes his way to the front porch of his modest house, before collapsing into the embrace of a rustic rocking chair. He looks as though he might die, but his inner monologue muses about how he has to live because it's the only way to keep the memories of everyone he lost alive. From his Vietnam buddies to his adopted daughter, Rambo holds them all inside his heart, and he has to keep going, for their sake. It's not enough to kill for revenge, but to live on their behalf.

The closing credits of Last Blood feature a dramatic montage of Sylvester Stallone's iconic character across all five films in the series, and the final images show Rambo getting on a horse and riding away, not towards a setting sun, but towards a nearby mountain range. He's still wounded and bleeding, so it's a bit ambiguous whether or not Rambo could still die. However, if the movie performs well enough at the box office it's a pretty safe bet that he'll live on to appear in another sequel.

Related: Read Screen Rant's Review of Rambo: Last Blood

John Rambo's story beganseven years afterthe army veteran returned to America after fighting in Vietnam, one of the most infamous armed conflicts in the history of the world. It's arguably impossible to fully understand the damage done to America as a result of the Vietnam War, and Rambo represents all the guilt, pain, suffering, and trauma of that dark chapter in American history.

At the end of Rambo 2008, the eponymous war hero finally returned home. The closing shots of that film showed Rambo walking down the long road back to his family's farm. In Last Blood, Rambo seems to be in a positive place, but as he describes it, he hasn't conquered his demons - he just "keeps a lid on it, every day." It's implied that his self-prescribed therapy includes building a network of tunnels under his farm. Early on in the film, he gets a brief Vietnam flashback, and the imagery of his tunnels is indicative of the tunnels used by the Vietnamese to elude detection by American forces and launch sneak attacks.

Ironically, Rambo concludes his Vietnam journey by employing the same guerilla tactics that were used against him all those decades ago. As the Mexican gang attacks his farm, he pops out of spider holes, blasts a few foes, and immediately returns underground, completely disappearing before his enemies even have a chance to react. Inside the tunnels, he destroys his opponents with hand-made traps, including a spike pit which evokes the imagery of punji sticks, which were used to devastating effect against Americans in Vietnam. Like the Vietnamese during the war, Rambo is outnumbered and outgunned, but he's defending his home, and he knows his land better than the opponent does, which is why they never stood a chance. From this perspective, it's a provocative way for Rambo to end his journey, by using his own nightmares to overcome his latest foes. In a way, it could be seen as his way of making peace with his past.

One of the biggest concerns regarding Rambo: Last Blood was how it would handle its setting, the border between Mexico and The United States. Rambo lives in Arizona, near the border, and his adopted family are Mexican immigrants. The story of Last Blood doesn't make any grand declarations about the political situation between America and Mexico; it just uses the circumstances as a backdrop for a personal story of revenge and man's proclivity towards brutality.

Related:Rambo: Last Blood Cast & Character Guide

The story is kicked off when John's adopted daughter, Gabrielle, crosses over into Mexico in an effort to find her birth father, who abandoned her years before. Most of the characters in Mexico are portrayed as villains, but that's because Rambo is explicitly looking in all the seediest places. There are several Mexicans who are shown in a positive light, from Rambo's new family to Paz Vega's character, a journalist seeking justice for her sister's murder at the hands of the gang.

As for the border itself, there is no mention of the current President's obsession with building a wall to keep people out of the country, and the film even takes steps to show how it would be ineffective. There are several scenes involving border crossing: the first shows Gabrielle crossing through a legal checkpoint, but one shows Rambo simply driving through a thin fence, and another shows the Mexican gang using an underground tunnel to go from one country to the next. Ironically, for those who decried the movie as being wall-building propaganda, Rambo would have been hindered by a wall, while the villains would have been unaffected. Make of that what you will.

Could there be a Rambo VI? Or, to stick with the series' unorthodox naming conventions, Rambo 6: Last Blood Part II? To quote the tagline from Rambo 2008, "Legends never die; they reload." Rambo is a distinctly American character, and there will always be more stories to tell with him. In his world, warriors are needed to protect the innocent, punish the wicked, and aid those in need. As long as there is life in his bones, John Rambo will meet that need.

Assuming he survives his wounds, Rambo ends Last Blood the way he began back in 1982's First Blood. He's alone, drifting across the country, looking for meaning in a country that would rather pretend he never existed than admit it was a mistake to send him and his generation off to die in the jungles of Vietnam. The national tragedy of Vietnam is still embedded deep in the American collective unconscious. It's part of this country's DNA. As long as this remains the case, Rambo will always be relevant, and more stories can continue to be told using the character.

More: First Bloods Original Ending Killed Rambo: Heres Why It Was Changed

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‘Prassthanam’ Review: Sanjay Dutt Came Close To Taking Saaho’s Spot Of Worst Movie Of The Year – Mashable India

Posted: September 25, 2019 at 12:46 am

This article is part of our Mash'O'Meter review series, where Mashable India delves deep and obsessively into movies and shows. Sit back, grab a cuppa. This is going to be a long ride.

I think I have to make this a habit now because the CBFC clearly doesn't know how to rate movies. Prassthanam is rated U/A i.e. this movie can be watched by anyone if they're under adult supervision. But in case you're planning to take your children, just be warned because this movie has a lot of violence, a really uncomfortable rape scene, and storytelling that can turn their mind to jelly. On a side-note, if you're planning to watch this movie alone, please bring meds because you're going to have a headache before the credits roll.

Prassthanam is written and directed by Deva Katta, who also made the 2010 original Telugu film of the same name. It stars Sanjay Dutt, Jackie Shroff, Manisha Koirala, Ali Fazal, Satyajeet Dube, and lest anyone forgets, Amyra Dastur. The story revolves around the family of politician Baldev Pratap Singh (Dutt) and how their hunger for power destroys them completely. Pretty simple, innit? Well, then watch how Katta muddles the hell out of it for over 2 hours!

How's the writing by Deva Katta?

Prassthanam features a lot of crime. But the greatest crime is committed by Katta on the female characters in the movie. I can't find a reason why he even thought of making women a part of his script. Because all they did was look pretty, whine, be someone's love interest or... umm... die. They are used as objects in order to have some form of bearing on the plot. None of them were made to take active or organic decisions. They were just there like an afterthought to make the men look more 'manly'.

SEE ALSO: Prassthanam - Cover Your Ears! There's A Lot Of Screaming In This Sanjay Dutt-Led Trailer

Coming to the men, there are just too many of them. There can only be a certain amount of testosterone in a movie until it becomes too much. There are so many men that there's no room of nuance or understandable character arcs. And that's why after every other moment, they're reduced to slapping each other, shouting into the open sky or smoking so much that it will make your lungs burn. But the worst side-effect of adding too much manliness into the screenplay is the metaphors spewed by the characters.

To make matters worse, the structure is very rickety. The movie starts in the present day. Then it goes to 6 months prior to that moment. Then it jumps to what all happened 25 years ago, all within a span of 20 minutes (approximately). And you might be thinking that that must have a purpose, right? Well, it doesn't because there's just so much plot and plot-twists that it dampens the impact of the non-linear storytelling.

How's the direction by Deva Katta?

To be honest, it was difficult to determine what Katta was going for in the first 20-minutes of Prassthanam. Because all of it was very basic stuff. There was no flavour in the scenes. Then came the moment that started Baldev's political career. In that scene, Dutt took on an entire army of goons with just two sickles and a ball of steel. People went flying into random stacks of cans. Other people were running around randomly. And I realised Katta's style of direction: swaying between over-the-top and Kyuki Saas Bhi Kabhi Thi levels of bland.

SEE ALSO: Sanjay Dutt Is Ready To Start Shooting For Munnabhai 3 But There's One Small Issue

Then there are the Dutch angles/tilts i.e. shots where the camera is kept at an angle for dramatic purposes. Most filmmakers use the Dutch tilt when they have to show something is askew or something surprising is going to happen. But Katta uses it anywhere and everywhere. Where someone like a Christopher McQuarrie, Anurag Kashyap or Quentin Tarantino can impress you with their Dutch tilts, Katta just drops them on you and leaves you with a feeling of disgust. And the reason why that feels noticeable is because the conversational scenes are shot in grey environments and cut boringly.

Finally, there are the action sequences and the item song. Some moments of Prassthanam's action sequences are good. I mean, there's potential. If Katta chooses to focus only on that and not on the plot (because clearly that's not his strong suit), I guess he can do wonders. But since he hasn't mastered it yet, they're jarring and dull all at the same time. And then there's that item song which is completely unnecessary (all item songs are unnecessary but this was particularly unnecessary). Because it felt like Katta didn't have any confidence in Satyajeet to sell the fact that his character is debauch AF.

How's the acting by the cast of Prassthanam?

If somebody makes a mash-up of every scene that Satyajeet Dubey is in and leaves the rest on the cutting room floor, I think that would make for a much, much better movie. That's because Dubey's on fire in Prassthanam. His character arc isn't something original. But the odd mix of repulsion and empathy that he brings to the screen was quite fascinating to watch. This is the second time he's starring alongside Ali Fazal, and those were clearly the highlight of the movie.

SEE ALSO: Getting Geeky with Ali Fazal

The only person who wasn't overshadowed by Dubey's powerhouse performance was Ali Fazal. The international star (FYI, he has done Furious 7, Victoria & Abdul, and is about to star alongside Gal Gadot) brought a lot of Guddu-wala charm from Mirzapur. There are two moments where he breaks down emotionally and it was too compelling to tell if he was acting or actually having a breakdown on-screen. However, the sad part is that his character is written in a way that it supplements Dutt's Baldev, thereby robbing him of a lot of solo moments to act the sh*t out of.

The rest of the cast is just horrendous. Dutt is just bad. Manisha Koirala is wasted. Jackie Shroff is wasted more than Koirala (The dude literally has a handful of lines in the movie for one of the stupidest reasons ever!). Chunky Pandey botches the hell out of the horribly written dialogues given to him. Amyra Dastur just hangs around for a bit, dances while looking beautiful and then returns at the tail end of the movie only to be forgotten all over again. In summary, it's painful.

Final verdict

Prassthanam stands atop a simple plot about how power corrupts everyone, even familial relations. But then it crumbles under the weight of an over-complicated screenplay, a tedious run-time, and too many slaps! And with a slew of mediocre to bad movies (The Zoya Factor, Rambo: Last Blood, Pal Pal Dil Ke Paas) coming in this week, it's a relief that there's Srikant Tiwari/The Family Man to get us through the weekend.

Cover artwork by Dhawal Bhanushali/Mashable India

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Sperm-like biobots swim toward seeding stem cells – The Sociable

Posted: September 25, 2019 at 12:45 am

Biobots modeled after sperm cells can now swim, which means they could one day seed stem cells to deliver drugs, perform minimally invasive surgery, and target cancer.

Our first swimmer study successfully demonstrated that the bots, modeled after sperm cells, could in fact swim

Conceived at the University of Illinois, researchers announced on Monday that they have built microscopic biohybrid robots (biobots) that can swim.

The biobots are modeled after nature, in particular sperm cells, and are propelled by muscles and nerves derived from rats.

Read More:Nature is intelligent: Pentagon looks to insects for AI biomimicry design

Research teams led by Taher Saif and Rashid Bashir worked together to develop the first self-propelled biohybrid swimming and walking biobots powered by beating cardiac muscle cells derived from rats, according to the National Science Foundation.

Our first swimmer study successfully demonstrated that the bots, modeled after sperm cells, could in fact swim, Saif said.

The two-tailed, sperm-inspired biobots are powered by skeletal muscle tissue stimulated by on-board motor neurons. Upon exposure to light, the neurons fire to actuate the muscles.

A major obstacle to anyone trying to fuse machine with organic matter is compatibility, yet the team discovered that the neuromuscular tissue was compatible with their synthetic biobot skeletons.

Read More:Could quantum computing and exotic materials facilitate AI-human cyborgs?

According to Saif, this advance could lead to the development of multicellular engineered living systems with the ability to respond intelligently to environmental cues for applications in bioengineering, medicine and self-healing materials technologies.

In other words, a future version of these sperm-inspired biobots could one day be swimming through your body to seed stem cells, target illnesses, or administer drugs internally.

Potentially broad impact in robotics, bioengineering, and health

Saif laid out his vision back in 2014 when the project began, and five years later it is becoming a reality.

The long-term vision is simple. Could we make elementary structures and seed them with stem cells that would differentiate into smart structures to deliver drugs, perform minimally invasive surgery or target cancer? he said at the time.

According to the research paper published in the Proceedings of the National Academy of Sciences (PNAS), the significance for such a breakthrough is great.

This paves the way for the development of biohybrid embodied platforms as models to gain deeper understanding of motor control, with potentially broad impact in robotics, bioengineering, and health.

However no two living machines can be expected to develop the same, and although they have created biobots that can swim, it may be difficult to consistently replicate those abilities.

One may move faster or heal from damage differently from the other a unique attribute of living machines, said Saif.

On a related note, the Pentagon has been researching bioelectronics for military use.

In February, the Defense Advanced Research Projects Agency (DARPA) launched the Bioelectronics for Tissue Regeneration (BETR) program for the intelligent healing of complex wounds like those suffered on the battlefield.

Read More:Bioengineering soldiers for smart, adaptive wound recovery

Wounds are living environments and the conditions change quickly as cells and tissues communicate and attempt to repair. An ideal treatment would sense, process, and respond to these changes in the wound state and intervene to correct and speed recovery, said BETR program manager Paul Sheehan, in a statement.

It seems that DARPA could look to the University of Illinois for inspiration, if it hasnt already.

The swimming biobot project is part of a larger National Science Foundation-supported Science and Technology Center on Emergent Behaviors in Integrated Cellular Systems, which also produced walking biobots developed at Illinois in 2012.

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