The Uncertainty of Regulating 3D Organ Printing – The Regulatory Review

Policymakers around the world are determining how to apply existing regulations to 3D organ printing.

When Selena Gomez suffered from Lupus, her best friend Francia Raisa donated her kidney to her, saving Gomezs life. For decades, people just like Gomez have escaped death due to heroic organ donations, either from living or deceased donors.

Today, 3D printingalso referred to as 3DPmay revolutionize the practice of organ donation. Although 3D printing is currently used to make jewelry, food, and art, it may soon be used for medical solutions such as organ donations and bionic limbs.

As a result, policymakers around the world seek increased regulation of 3DP organs. Yet 3D bioprinting does not clearly fit into existing regulatory frameworks.

Since bioprinting generally falls within the regulatory domain of regenerative medicine, medical devices, and biologic drugs, regulators face the challenge of applying existing rules to this uncertain field. As of now, it is unclear whether policymakers can effectively regulate bioprinting under existing regulations, or if a new, specific regulatory process will be necessary.

In determining how to regulate 3D organ printing, policymakers must juggle many possible concerns. Since the technology is still developing, a lot of uncertainty remains about what the actual risks and ethical concerns are. For example, one ethical concern is that 3DP organs may be available to wealthy people only, while less affluent individuals will be blocked out from using these organs.

Another concern is safety. Since 3DP may require stem-cell technology, and the patients own cells may be used for replication, it is difficult to assess the safety risks. Stem-cell therapy cannot be tested on a large sample of healthy people, which limits effective clinical analysis. Also, 3DP biotechnology may open up new uses beyond 3DP organs, such as enhancement of human capacities for military use. Developers could use the technology to make military officers or even terrorists less vulnerable to injury in battle, but this would open up a whole new challenge for law enforcement and national security.

The U.S. Food and Drug Administration (FDA) focuses on the regulation of 3D printed organs. FDA so far has only released guidance on 3DP, and the recommendations do not cover bioprinting.

A significant concern in the United States is that 3D printed organs do not fit into any clear category of law. First, they are not organs because they are not born alive at any stage of development. Second, they are not drugs because drugs are used orally rather than through an invasive surgery, and drugs are primarily meant to relieve illness while donated organs may completely cure an illness.

Some policymakers in the United States propose regulating 3DP organs as a biological product, defined as a virus, therapeutic serum, toxinor analogous productapplicable to the prevention, treatment, or cure of a disease or condition of human beings.

Research company Biogelx suggests that biological products may be a promising category for printed organs. Within biological products, a 3DP organ is comparable to proteins because to print the organ, clinicians replicate healthy human cells, which include such proteins, says Biogelx. Although existing regulatory frameworks often compare 3DP organs to medical devices, Biogelx asserts that these organs should not be regulated as medical devices. Medical devices are not made of biological material and are often metal or plastic devices that help an individuals standard of life, but 3DP organs are different since they cause a chemical reaction in the body and have the purpose of wholly replacing an existing organ, says Biogelx.

International policymakers are also struggling to find a sufficient regulatory framework. In Canada, Health Canada released draft guidance last year to develop regulations for medical device manufacturers working towards bioprinting. Health Canada has several concerns about bioprinting, and it suggests that manufacturers looking for bioprinting licenses should be required to submit information regarding the use of additives in materials, the verification of the software for the bioprinting design, the method of sterilizing the machines, and the process of safe removal and reuse of bioprinting materials and residues.

Finally, Europes 3DP health technology is regulated by the European Medical Devices Directive, the Active Implantable Medical Devices Directive, and the Invitro Diagnostic Medical Devices Directive. The Medical Devices Directive categorizes bioprinting devices into several risk classes. Across the different classes, devices ranked as higher risk are subjected to third party assessment and more stringent requirements for clinical data. The highest risk class, implantable devices such as 3D organs, requires an independent design dossier review. A design dossier assesses risk, evaluates clinical data, and demonstrates the technologys compliance with regulations and requirements.

Although 3D printing of organs is right around the corner, policymakers around the world lack the information necessary to make regulatory decisions in this space. Different countries have different approaches, but many of the leading nations in 3DP share similar concerns. With more information, regulators will have to decide if existing regulatory frameworks can adequately address the safety concerns of 3D printed organs.

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The Uncertainty of Regulating 3D Organ Printing - The Regulatory Review

Mustang Bio Announces Updated Clinical Data on MB-107 Lentiviral Gene Therapy for Patients with X-Linked Severe Combined Immunodeficiency -…

MB-107 preceded by low-dose busulfan conditioning continues to be well tolerated and results in development of functional immune system in newly diagnosed infants with XSCID

Enhanced transduction procedure is demonstrating improvements in older patients with XSCID who received prior hematopoietic stem cell transplantation

Data presented by St. Jude Childrens Research Hospital and National Institutes of Health at 61st American Society of Hematology Annual Meeting

NEW YORK, Dec. 09, 2019 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, announced today that updated Phase 1/2 clinical data for MB-107 lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID) were presented on Saturday by St. Jude Childrens Research Hospital (St. Jude) and today by the National Institutes of Health at the 61st American Society of Hematology (ASH) Annual Meeting.

MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude, and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Under a licensing partnership with St. Jude, Mustang intends to develop the lentiviral gene therapy for commercial use as MB-107. The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, The updated clinical data presented at the 2019 ASH Annual Meeting underscore the curative potential of MB-107 for newly diagnosed infants with XSCID, as well as its meaningful impact on older XSCID patients who received prior hematopoietic stem cell transplantation. St. Jude recently received the 2019 Smithsonian Magazine American Ingenuity Award for development of the lentiviral gene therapy, highlighting its potential to have an impact on this devastating disease. We are excited to be working with St. Jude and NIH to advance MB-107 and look forward to transferring the IND from St. Jude to Mustang in the first quarter of 2020.

Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical Study (Abstract Number: 2058)Poster presentation: Ewelina Mamcarz, M.D., Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Childrens Research Hospital, Memphis, TN, USA

Interim data from the multicenter Phase 1/2 clinical trial for infants under the age of two treated with the lentiviral gene therapy preceded by low exposure-targeted busulfan conditioning were published in the New England Journal of Medicine. Updated data presented at the 2019 ASH Annual Meeting include three more patients (n=11), 8 months additional median follow up (23.6 months; range: 1.5 to 33.9 months), more extensive analysis of T and B cell functional recovery, and detailed vector integration site studies.

Data Highlights:

The results from treatment with low-dose busulfan conditioning and the novel lentiviral gene therapy in newly diagnosed infants with XSCID continue to be very promising, said Dr. Mamcarz. We are pleased that the therapy has been well tolerated and all patients with a follow up of more than 3 months recovered from pre-existing infections, are off protective isolation and prophylactic antimicrobials, and have normal growth in respect to height and weight. This reinforces our belief that the lentiviral gene therapy has the potential to be an attractive alternative to current XSCID therapies.

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency (Abstract Number: 608)Oral presentation: Harry Malech, M.D., Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), Bethesda, MD, USA

Early outcome data for five older children and young adults with XSCID who received the lentivector (also known as lentiviral) gene therapy as salvage therapy after having previously received haplo-identical hematopoietic stem cell transplantation (HSCT) as infants without chemotherapy-based conditioning were previously reported and published in Science Translational Medicine. By 2016, three additional patients were treated, and the cohort of eight patients (referred to as Cohort A) has now been followed for 3 to 7 years. Among Cohort A, gradual clinical benefit in the clearance of chronic norovirus and associated improved abdominal complaints, malabsorption, growth and IgG production were observed, and four patients were able to cease immunoglobulin replacement therapy.

While the results were positive, the relatively inefficient transduction of hematopoietic stem/progenitor cells (HSPCs) required large quantities of vector. This resulted in relatively low VCN in myeloid cells in some patients, with delayed immune cell recovery and persistent clinical disease, especially in the last patient treated (patient 8). To address this, NIH developed a refined enhanced transduction (ET) procedure consisting of a single overnight transduction after 48 hours pre-stimulation in cytokines (Stem cell factor, Thrombopoietin, Flt3-ligand; 100ng/mL) and incorporated transduction enhancers LentiBoost 1:100 and dimethyl prostaglandin 2 (dmPGE2; 1uM).

The presentation at the 2019 ASH Annual Meeting included data from six patients (referred to as Cohort B) treated by NIH, including re-treatment of patient 8. The patients, who were aged 12 to 36, had significant problems with donor T cell infiltration of liver, bone marrow and kidneys, and were nearly absent of B and NK cells. The enhanced transduction procedure achieved much greater transduction efficiencies than were observed in Cohort A, with greater than 10-fold less vector, and resulted in faster immune reconstitution and more significant clinical benefit by 3 months.

We are encouraged by the significantly improved measures of early clinical outcomes from lentivector gene therapy in older children and young adults with XSCID using an enhanced transduction procedure with the addition of LentiBoost and dmPGE2, said Dr. Malech. Notably, we have seen an early appearance of B and NK cells at much higher levels in Cohort B than we previously observed in Cohort A, even at years after treatment. We look forward to continuing to closely monitor patients and report outcomes.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T and CRISPR/Cas9-enhanced CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking Statements This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

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Mustang Bio Announces Updated Clinical Data on MB-107 Lentiviral Gene Therapy for Patients with X-Linked Severe Combined Immunodeficiency -...

Top Florida Medical Spa, Amnion of Florida, Partners With Merakris Therapeutics to Advance Their Non-Surgical Treatment Options – Business Wire

ORLANDO, Fla.--(BUSINESS WIRE)--Amnion of Florida, a leading provider of alternative medicine utilizing cryopreserved placental cell allograft and advanced bioactive facial rejuvenation, is pleased to announce their vendor choice to round out regenerative anti-aging therapies.

Amnion announces a partnership with Merakris Therapeutics, LLC to advance the development of Merakris topical bioactive anti-aging hydrogel technology. Christopher Broderick, President, and Founder of Merakris Therapeutics stated, dedication to science-based outcomes is our primary focus, thus were delighted to be selected based upon our scientific approach to youth maintenance and rejuvenation technologies.

Amnion is focused on attracting women and men seeking affordable non-surgical options for youth maintenance via cell activated procedures, hair restoration, joint repair, dermal rejuvenation, and anti-aging treatments.

Our team of experienced medical professionals and aestheticians at Amnion are excited to utilize the Merakris Therapeutics product suite, including medical-grade, sterile filtered amniotic fluid serums and hydrogels at our newest Spa in Sanford, FL, said Eusebio Coterillo, President of Amnion.

In a constantly changing field, Amnion of Florida, under the guidance of the on-site medical staff, provides the highest level of quality products and procedures in cosmetic medicine. They offer cutting edge treatments that are proven by research, the use FDA cleared or registered products, and are widely published and peer endorsed.

More about Amnion of Florida

Amnion of Florida, based in Central Florida, is a leading provider of alternative medicine using cryopreserved placental cell transplants or allografts, processed from donated cellular birth tissue, which are natural alternatives to autologous regenerative medicine products. The primary function of our allogeneic regenerative treatments is to promote soft tissue joint/skin repair and regeneration mediated by growth factors and cells naturally found in placental tissue. These treatments have shown safety and efficacy in treating a variety of ailments including osteoarthritis, chronic ulcerative wounds, joint pain, skin rejuvenation, hair restoration, urinary incontinence, and ED. Learn more http://www.amnion.us.

More about Merakris Therapeutics, LLC

Merakris Therapeutics, based in Research Triangle Park, North Carolina, is focused on researching, developing, and marketing regenerative healthcare products. Merakris is pioneering commercially scalable biotherapeutic technologies derived from stem cells that have various clinical applications. Our vision is to improve global patient care and outcomes through the pioneering and innovation of acellular regenerative biotechnologies. Learn more at http://www.merakris.com.

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Top Florida Medical Spa, Amnion of Florida, Partners With Merakris Therapeutics to Advance Their Non-Surgical Treatment Options - Business Wire

MacroGenics Presents Flotetuzumab Data in Patients with Primary Induction Failure and Early Relapsed Acute Myeloid Leukemia at the 2019 ASH Annual…

Rockville, MD, Dec. 09, 2019 (GLOBE NEWSWIRE) --

MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, today announced updated results from a Phase 1/2 dose expansion study of flotetuzumab, an investigational, bispecific CD123 x CD3 DART molecule in patients with primary induction failure and early relapsed acute myeloid leukemia (AML). The data were presented in an oral session at the 61st Annual Meeting of the American Society of Hematology (ASH) in Orlando, FL, taking place December 7-10, 2019.

Patients with AML who have failed primary induction therapy or relapsed early after an initial response represent a significant unmet medical need. A remission rate of 32% observed in the ongoing study of flotetuzumab in this extremely challenging patient population is noteworthy, said Geoffrey Uy, M.D., Associate Professor, Department of Medicine, Division of Oncology at the Washington University School of Medicine in St. Louis. Importantly, by implementing a lead-in dosing schedule for flotetuzumab, as well as early intervention with tocilizumab in this study, we were able to mitigate cytokine release syndrome, known to be associated with T-cell engagers.

In the Phase 1/2 (NCT02152956) open-label, dose expansion study, 30 patients classified as primary induction failure or early relapsed AML who had received a median of four prior therapies were treated with flotetuzumab at the recommended phase 2 dose (RP2D) of 500 ng/kg/day by continuous infusion. Data were reported as of the cut-off date of November 1, 2019. The study is currently ongoing, with additional patients being enrolled.

Responses, including complete remission (CR), CRh (CR with partial hematological recovery) and CRi (CR with incomplete hematological improvement) per a modified International Working Group (IWG) Response Criteria for AML, are summarized in the table below. Four responders received allogeneic hematopoietic stem cell transplantation as consolidation therapy and remain in remission after 6 to 21 months.

The most common treatment-related adverse event (TRAE) was infusion-related reaction/cytokine release syndrome (IRR/CRS) that occurred in all (30/30) patients. However, most CRS events observed were of short duration and mild to moderate (grade 1 or 2) in severity, with only one grade 3 event reported in one patient.

Based on the encouraging data from this study, and pending anticipated discussions with the FDA in the first half of 2020, we are planning for a potential registration-enabling study of flotetuzumab in this high unmet need population of patients with refractory AML, who have limited treatment options, said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics.

A separate oral presentation described translational research that showed an inflammatory (IFN--related) gene expression signature in a subset of patients with AML that correlated with a lack of response to induction chemotherapy. Furthermore, the same gene signature was associated with patients more likely to respond to flotetuzumab, supporting the mechanism being exploited by this molecule. In addition, AML patients with an immune-infiltrated tumor micro-environment show high expression of immune checkpoint molecules, including PD-L1, which provides a scientific rationale for combining flotetuzumab with checkpoint blockade as a potential mechanism for enhanced anti-leukemic activity. MacroGenics has initiated a study combining flotetuzumab with MGA012, an anti-PD-1 antibody, given the strong preclinical and translational data that indicate the combination may enhance CD123-directed T cell killing.

Flotetuzumab Presentations at ASH

Oral Presentations

Poster Presentations

These slide and poster presentations are available on the Events & Presentations page on MacroGenics' website at http://ir.macrogenics.com/events.cfm.

Conference Call & Webcast

MacroGenics management and external guest speakers will host a conference call and audio webcast today at 8:00 p.m. ET to review the flotetuzumab data presented at the ASH Annual Meeting and discuss ongoing clinical development plans.

To participate in the MacroGenics ASH 2019 Conference Call, please dial (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and provide the Conference ID: 3625435. A listen-only slide and audio webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company's website at http://ir.macrogenics.com/events.cfm. A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company's website for 30 days.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a hematopoietic stem cell malignancy characterized by differentiation arrest and uncontrolled clonal proliferation of neoplastic precursors that prevent normal bone marrow hematopoiesis. Nearly 20,000 new cases of AML are diagnosed in the U.S. each year, with a median age of 69 years at diagnosis. Approximately 40-50% of newly diagnosed patients fail to achieve a complete remission with intensive induction therapy (primary induction failure) or experience disease recurrence after a short remission duration (<6 months; early relapsed). A very small number of these patients are expected to respond to salvage therapy. In addition, although new targeted agents have been approved for the treatment of frontline or relapsed/refractory AML in recent years, approximately 50% of patients have no known targetable mutations.

About Flotetuzumab

Flotetuzumab (also known as MGD006) is a clinical-stage molecule that recognizes both CD123 and CD3. CD123, the interleukin-3 receptor alpha chain, has been reported to be over-expressed on cancer cells in AML and other hematologic malignancies. The primary mechanism of action of flotetuzumab is believed to be its ability to redirect T lymphocytes to kill CD123-expressing cells. To achieve this, the DART molecule combines a portion of an antibody recognizing CD3, an activating molecule expressed by T cells, with an arm that recognizes CD123 on the target cancer cells.

Flotetuzumab is currently being evaluated in the U.S. and Europe in a Phase 1/2 dose expansion study designed to assess the safety, tolerability, and initial anti-leukemic activity of the molecule in patients with relapsed/refractory AML. The U.S. Food and Drug Administration has granted orphan drug designation to flotetuzumab for the treatment of AML. A Phase 1/2 study in combination with MGA012, a proprietary anti-PD-1 monoclonal antibody, in patients with relapsed/refractory AML is being conducted ex-U.S. MGA012 (also known as INCMGA00012) was exclusively licensed to Incyte Corporation in 2017 under a global collaboration and license agreement; MacroGenics retains the right to develop its pipeline molecules with MGA012. MacroGenics retains global development and commercialization rights to flotetuzumab.

About MacroGenics, Inc.

MacroGenics is a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer. The Company generates its pipeline of product candidates primarily from its proprietary suite of next-generation antibody-based technology platforms, which have applicability across broad therapeutic domains. The combination of MacroGenics' technology platforms and protein engineering expertise has allowed the Company to generate promising product candidates and enter into several strategic collaborations with global pharmaceutical and biotechnology companies. For more information, please see the Company's website at http://www.macrogenics.com. MacroGenics, the MacroGenics logo and DART are trademarks or registered trademarks of MacroGenics, Inc.

Cautionary Note on Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the Company's strategy, future operations, clinical development of the Company's therapeutic candidates, milestone or opt-in payments from the Company's collaborators, the Company's anticipated milestones and future expectations and plans and prospects for the Company and other statements containing the words "subject to", "believe", "anticipate", "plan", "expect", "intend", "estimate", "project", "may", "will", "should", "would", "could", "can", the negatives thereof, variations thereon and similar expressions, or by discussions of strategy constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and enrollment of future clinical trials, expectations of expanding ongoing clinical trials, availability and timing of data from ongoing clinical trials, expectations for regulatory approvals, other matters that could affect the availability or commercial potential of the Company's product candidates and other risks described in the Company's filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views only as of the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so, except as may be required by law. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date hereof.

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MacroGenics Presents Flotetuzumab Data in Patients with Primary Induction Failure and Early Relapsed Acute Myeloid Leukemia at the 2019 ASH Annual...

Orchard Therapeutics Showcases Clinical Data at the 61st American Society of Hematology Annual Meeting – BioSpace

BOSTON and LONDON, Dec. 08, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, will be presenting new registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, FL.

On Sunday, December 8, 2019, investigators will describe ongoing clinical progress for two lead development programs in the companys primary immune deficiencies portfolio: OTL-103, an investigational gene therapy in development for the treatment of Wiskott-Aldrich syndrome (WAS) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy; and OTL-101, an investigational gene therapy in development for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

In addition, on Monday, December 9, 2019, investigators will deliver an oral presentation featuring updated data from the ongoing clinical proof-of-concept study of OTL-203, an investigational gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at SR-Tiget.

To learn more about Orchards approach to ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapy, conference attendees can visit booth #2228 in the Exhibition Hall.

Full presentation details are below:

Poster Presentation Details

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010Publication Number: 3346Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded CohortPublication Number: 3345Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

Oral Presentation Details

Extensive Metabolic Correction of Hurler Disease by Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II TrialPublication Number: 607Session: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersDate and time: Monday, December 9, 7:00am ET

About ADA-SCID and OTL-101Severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the ADA gene resulting in a lack of, or minimal, immune system development.1-4 The first symptoms of ADA-SCID typically manifest during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive, and without treatment the condition can be fatal within the first two years of life. The incidence of ADA-SCID is currently estimated to be one in 500,000 live births in the United States and between one in 200,000 and one in 1 million in Europe.3 OTL-101 is an autologous, ex vivo, hematopoietic stem cell-based gene therapy for the treatment of patients diagnosed with ADA-SCID being investigated in multiple clinical trials in the United States and Europe, including a registrational trial at the University of California, Los Angeles (UCLA). OTL-101 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of ADA-SCID, and Breakthrough Therapy Designation from the FDA.

About WAS and OTL-103Wiskott-Aldrich Syndrome (WAS) is a life-threatening inherited immune disorder characterized by autoimmunity and abnormal platelet function and manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. Without treatment, the median survival for WAS patients is 14 years of age. Treatment with stem cell transplant carries significant risk of mortality and morbidities. OTL-103 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in April 2018 and has been developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The global incidence of WAS is estimated to be about 100-260 births per year, with a global prevalence of 2,900-4,700 patients.

About MPS-I and OTL-203Mucopolysaccharidosis type I (MPS-I) is a rare inherited neurometabolic disease caused by a deficiency of the IDUA (alpha-L-iduronidase) lysosomal enzyme required to break down glycosaminoglycans (also known as GAGs or mucopolysaccharides). The accumulation of GAGs across multiple organ systems results in the symptoms of MPS-I including neurocognitive impairment, skeletal deformity, loss of vision and hearing, hydrocephalus, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births.5 There are three subtypes of MPS-I; approximately 60 percent of MPS-I patients have the severe Hurler subtype and, when untreated, these patients rarely live past the age of 10.Id Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy.

About Orchard Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the UK and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, including any cryopreserved formulations of such product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Orphanet. SCID due to ADA deficiency. 2Whitmore KV, Gaspar HB. Front Immunol. 2016;7:314. 3Kwan A, et al. JAMA. 2014;312:729-738. 4Sauer AV, et al. Front Immunol. 2012;3:265. 5Beck et al. The Natural History of MPS I: Global Perspectives from the MPS I Registry. Genetics in Medicine 2014, 16(10), 759.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics Showcases Clinical Data at the 61st American Society of Hematology Annual Meeting - BioSpace

Olympic Hall of Fame Inductee Gary Hall Jr. Joins C3 International Board of Advisors – BioSpace

GARDEN GROVE, Calif., Dec. 9, 2019 /PRNewswire/ --C3International, a biopharmaceutical company that has played a leadership role in the emerging cannabinoid therapeutics health sector, today announced that Gary Hall, Jr. has joined the Board of Advisors for C3 International.

Gary Hall, Jr. is a healthcare-focused board member, patient advocate and key opinion leader with expertise in strategic alliance, marketing, clinical distribution, sports science, medical research and patient engagement. Hall has over 80 keynote presentations internationally, 10 Olympic medals in swimming and is an Olympic Hall of Fame Inductee. Using accomplishment in sports as a platform to advance diabetes advocacy for over 20 years, Gary amassed an influential network of policy makers, politicians, healthcare industry executives, diabetes care specialists, medical research, nutrition, philanthropic, sport, physical activity, obesity prevention, sports medicine and sports science organizations.

Gary has testified three times before Senate subcommittees on healthcare related issues and was a featured speaker at the 2016 Vatican hosted Cellular Horizons conference on stem cell therapy. He is the first recipient of USA Swimming's Humanitarian Award. One of his health community health initiatives was recognized at the Aspen Institute's Project Play Summit. He has presented at the American Heart Association, American Diabetes Association, American College of Sports Medicine, International Olympic Committee Medical Commission and National Youth Sports Health & Safety Institute conferences.

Idrasil is the first standardized form of medical cannabis. It offers all of the medicinal analgesic and therapeutic benefits of cannabis but is a superior alternative to opiates and life-threatening narcotics because physicians and caregivers can provide patients with safe, non-addictive, measurable dosages.

"The health benefits of CBD seem apparent and C3 International is the only company I've found that has Cannabinoid in a divisible, consistently dosed tablet," said Gary Hall Jr. "It's an honor to join the team. I look forward to providing perspective on potential athletic and healthcare applications. Idrasil aims to be the worldwide leader in medical cannabis."

"We are incredibly excited to welcome such an influential medical activist like Gary Hall Jr. onto our Board of Advisors," said Steele Clarke Smith III, Chairman and CEO of C3 International. "Hall will bring his vast expertise to our innovative biopharmaceutical company and we can't wait to work with him!"

Conditions treated with Idrasil include, but are not limited to, AIDS; anorexia; arthritis; autism; anxiety/depression; cancer; chronic pain; glaucoma; migraines; persistent muscle spasms; Parkinson's; seizures; severe nausea; Tourette's Syndrome, and any other chronic or a persistent medical symptoms that substantially limit major life activities as defined in the Americans with Disabilities Act of 1990.

Idrasil consists of a proprietary blend of concentrated cannabis extract that is 100% natural and organic. C3's proprietary process isolates all of the cannabinoids from the cloned cannabis plant, resulting in pure natural extraction in pill form to eliminate the unwanted euphoria and social risks associated with smoking cannabis products and unpredictable dosages of edible confections. Idrasil is a natural product that looks like any pill on the market.

Idrasil is aseptically processed and bacteria-free with a manufacturing process that is FDA-compliant. Idrasil is manufactured in a sterile ISO 9001 certified laboratory with a standardized and consistent 12.5mg, 25mg, or 100mg dose pill. Idrasil is categorized by the FDA as Generally Regarded As Safe (GRAS).

About Idrasil and C3 International

The mission of C3 International, Inc. is to manufacture the unique Intellectual Property for Idrasil, a major advancement in the standardization and administering of natural cannabinoids, in a tablet. Idrasil is a holistic alternative to addictive opiates and life-threatening narcotics, without euphoria. For more information on C3 International, Inc. visit http://www.c3internationalinc.com. To learn more about Idrasil, go to http://www.idrasil.com.

DISCLAIMER: THESE STATEMENTS HAVE NOT BEEN EVALUATED BY THE FOOD AND DRUG ADMINISTRATION. FURTHERMORE, NONE OF THE INFORMATION ON THIS PAGE SHOULD BE USED AS A SUBSTITUTE FOR THE ADVICE OF AN APPROPRIATELY QUALIFIED PHYSICIAN OR OTHER HEALTHCARE PROVIDER. THIS INFORMATION IS ONLY MEANT FOR INFORMATIONAL PURPOSES.

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Olympic Hall of Fame Inductee Gary Hall Jr. Joins C3 International Board of Advisors - BioSpace

U.S. Stem Cell Training | Regenerative Medicine Training …

In 2011 I attended training and education on adult stem cell harvesting, isolation, and separation techniques led by Kristin Comella. She laid a solid foundation for us by educating us on how stem cells function, practical applications, and current research and results. When we went into the lab portion, Kristin was very thorough and meticulous when walking us through the steps. We were given a detailed, printed protocol to follow that made things very clear and easy to replicate. Kristin was eager to help out, answer questions, and show us the most efficient ways to perform each step of the procedure. Kristin is very knowledgeable and passionate about her research and adult stem cell therapies and has continued to be a valuable resource to us at SouthPointe Family Physicians. She always replies promptly to any questions or concerns we may have and keeps us up-to-date on the latest protocols and findings! Overall, working with Kristin has been a fantastic experience, and I'm excited to continue learning more from her in this field!

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U.S. Stem Cell Training | Regenerative Medicine Training ...

Stem Cell Therapy Treatment – Melbourne Stem Cell Centre

Thank you for contacting Melbourne Stem Cell Centre. We appreciate you taking the time to enquire about our treatment options. On Monday 27th May MSCC notified the media with a press release detailing the great results of our randomised control trial treating knee Osteoarthritis. This release has created a high influx of calls and emails, we ask that you be patient with us getting back to you.

In the meantime, please feel free to browse over the below information to better acquaint yourself with our current therapies and general pricing.

Why Stem Cells?

Mesenchymal stromal Stem Cells are rudimentary cells with the potential to stimulate other cells within the body. Melbourne Stem Cell Centre doctors can harvest your adult stem cells in a small liposuction procedure. The stem cells are then isolated from the other cells extracted during the procedure, including fat cells. The stem cells are then specially prepared and expanded, in the laboratory of our partner Magellan stem cells, before being used for osteoarthritis treatment. Our recent clinical trial has showed both statistical and clinically significant pain and function improvement following stem cell therapy in 85 per cent of patients.

Why MSCC?

MSCCs treatment protocol has been ethically approved by Charles Sturt University. This uses a pure, high-dose stem cell population (as this has had success in recent scientific literature) and verification of the cell number, cell viability, and sterility is conducted off site, prior to injection. All patients are strictly followed up to assess outcome and to add to the growing scientific knowledge regarding stem cell therapies. Your response to treatment will be regularly followed up to maximise potential improvement.

What conditions to we treat?

Currently at Melbourne stem cell centre we treat osteoarthritis. There may be future treatments for other conditions and we regularly inform all our patients of any developments. To keep up to date with upcoming treatments and trials we recommend you visit the ANZCTR trial registry website http://www.anzctr.org.au .

Do I need a consultation first?

Prior to any treatment you will receive a thorough arthritis health care consultation with one of our musculoskeletal specialists. This includes a full biomechanical assessment, discussion of current pain management treatments and review of formal imaging. Your suitability for other biological therapeutic treatment options will be assessed. X-rays or MRI less than 6 months old is preferred for this appointment. The cost of the consultation is $240 on the day; if you bring a GP referral letter, you are eligible for a Medicare rebate of approximately. $70.

How much does it cost?

The cost for stem cell therapy depends on the individualised treatment plan formulated by your physician. Costs typically range from $7,100-$11,650AUD for the treatment of one joint. This includes the lipo-harvest procedure and two stem cell injections. Any additional treatment would be on top of this fee. Unfortunately, there are no Medicare or private health insurance rebates on our treatment.

I live interstate

MSCC offer the option of having the initial consultation and assessment conducted via phone. This would require you to send a CD of your X-rays and/or MRI in the post with a phone registration form. The cost of the phone consult is $150AUD (with no Medicare rebate). If you decide to have stem cell therapy, your treatment will involve at least 3-5 trips to Melbourne over the course of 12 months.

I live overseas

MSCC offer the option of having the initial consultation and assessment conducted via phone. This would require you to send a CD of your X-rays and/or MRI in the post with a phone registration form. The cost of the phone consult is $150AUD. Your stem cell therapy would require 3 separate trips to Melbourne for 2-3 weeks each. You may decide to combine the first two trips into an extended stay in Australia.

*The cost for stem cell therapy is 100% out of pocket. Medicare or private health funds do not subsidize any costs in the treatment of mesenchymal stem cell therapy.

If you would like a call to book an initial consultation with one of our specialists, please click the request a call button on our website and one of our team will give you a call.

With warmest regards,

Reception TeamMelbourne Stem Cell Centre

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Stem Cell Therapy Treatment - Melbourne Stem Cell Centre

ASH 2019 Roundup: The Latest on CAR T, New Treatments for CLL, and Using Genes to Predict a Common Side Effect – On Cancer – Memorial Sloan Kettering

The annual meeting of the American Society of Hematology (ASH) brings together leaders from around the world who treat people with blood cancers and other blood disorders. Doctors and researchers from Memorial Sloan Kettering presented their research at the meeting,held this year in Orlando. Below are some highlights from the past few days.

Chimeric antigen receptor (CAR) T cell therapies have been in the spotlight at the ASH meeting. Many scientists in the field are excited by the prospect of using the genome-editing tool CRISPR to engineer more-potent CAR T cells.

At MSK, researchers Michel Sadelain, Renier Brentjens, and Isabelle Rivire and their colleagues are at the forefront of CAR T science. Just last year, the team published results from the longest-running clinical trial of CAR therapy, showing which patients benefitted most. This year at ASH, another member of that team, MSK medical oncologist Jae Park, presented the results of a retrospective study that provides clues into who may benefit most from a stem cell transplant following CAR therapy.

A stem cell transplantreplaces a persons blood-forming stem cells with those from a donor. It can be a cure for some people with cancer, yet it comes with serious potential risks, such as life-threatening infections and graft-versus-host disease. Previous research from Dr. Park and his colleagues had suggested that there was no clear survival advantage to a stem cell transplant in adults with acute lymphoblastic leukemia (ALL) who were treated with CAR T cells. But the scientists were curious to find out if a group of patients would benefit from the procedure.

Of 53 adults with ALL who received CAR T cells at MSK, 16 of those who had a complete response to the CAR therapy went on to have a stem cell transplant. Dr. Park and his colleagues looked for correlations between how well these patients did and several variables: age, a prior stem cell transplant, the presence of the Philadelphia chromosome, the amount of disease measurable at the time of the CAR therapy, the severity of cytokine release syndrome, and neurotoxicity.

The only variables that seemed to make a difference were age and neurotoxicity. Specifically, being younger and having no severe neurotoxicity during CAR therapy were associated with improved overall survival following a stem cell transplant. Dr. Park cautioned that the small size of the study limits drawing firm conclusions. Nevertheless, the results point to a group of people for whom the potential benefits of a stem cell transplant may outweigh the risks. Further research is needed.

MSK hematologist-oncologist Anthony Mato presented the results of several studies aimed at improving care for people with chronic lymphocytic leukemia (CLL). One study dealt with identifying the best treatment options for people with CLL who have had to stop taking the targeted drug venetoclax (Venclexta). Dr. Mato and colleagues conducted a retrospective study of 326 people with CLL from around the world who received venetoclax and then switched to another drug.

They looked at how well these people did on various treatments specifically BTK inhibitors, PI3K inhibitors, CAR T therapy, and stem cell transplantation. Of these, they found that BTK inhibitors were an effective post-venetoclax treatment, provided the patients had not yet received BTK inhibitors or had not developed resistance to these drugs. They also found that a stem cell transplant was a good option that provided lasting benefits.

Together, these studies show the progress we at MSK are making in developing much-needed therapies for people with CLL.

In a second presentation, Dr. Mato discussed the results of a first-in-human trial of a BTK inhibitor called LOXO-305. This targeted drug works differently than existing BTK inhibitors. The trial, which enrolled 13 people, showed that LOXO-305 was safe. It also provided proof of concept that this approach may be effective at helping people with CLL and other B cell cancers, including those who have developed resistance to existing BTK inhibitors.

Finally, Dr. Mato discussed results from a study that tested a combination of targeted drugs given at lower dosages to people with CLL. Combination approaches have the potential to preempt the emergence of drug resistance but are often too toxic when each drug is given at full strength.

The phase I study tested a lower-dose combination of the BTK inhibitor everolimus (Afinitor, Zortress) and pomalidomide (Pomalyst). It included 33 people with CLL and lymphoma who lacked an approved treatment option. Dr. Mato and his colleagues found that this combination, named DTRM-555, was generally safe and showed signs of effectiveness in several people. A phase II study of the combination is underway.

Together, these studies show the progress we at MSK are making in developing much-needed therapies for people with CLL, Dr. Mato says.

Blood clots are a life-threatening side effect in people being treated for cancer. Known risk factors for blood clots include cancer type and stage, obesity, blood cell counts, and treatment with chemotherapy. However, it is still difficult for doctors to predict the risk of blood clots in individual people.

To find out if a tumors molecular profile could predict the risk of blood clots, MSK hematologist Simon Mantha and colleagues conducted a study in which they analyzed tumor DNA sequences for 341 genes in 11,695 people with cancer.

As Dr. Mantha presented at the ASH meeting, the scientists found that mutations in several genes STK11, MET, KEAP1, CTNNB1, and KRAS increase the risk of blood clots in people with cancer.

Dr. Mantha hopes these findings will ultimately translate into better methods to predict who is most at risk for this dangerous complication.

Read about other MSK science at ASH hereand here.

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ASH 2019 Roundup: The Latest on CAR T, New Treatments for CLL, and Using Genes to Predict a Common Side Effect - On Cancer - Memorial Sloan Kettering

Injectables Are the Future of K-Beauty Trends in America – Yahoo Lifestyle

Before meeting up with me for dinner to celebrate my first time in Korea, my Seoul-based friend Jessica got off the plane after a business trip in Los Angeles and drove straight to her dermatologist or "dermie," as she affectionately calls him for Botox on her nose and jawline. After our stomachs were filled with dak galbi and soju, I scooted closer to Jessica in our booth and asked her about her latest dermie appointment. She began to list the dozen-plus cosmetic procedures she's tried since moving to Seoul from New York City eight years ago. Botox was her gateway injectable, starting with her jawline for a narrower, V-shaped silhouette. Since then, Jessica's gotten fillers in her forehead, chin, nose, and lips.

She also mentioned the "Chanel" injection, a cocktail of vitamins, minerals, and antioxidants that is supposed to tighten and brighten skin (she didn't notice a difference, though). I listened to all of this slightly slack-jawed. On the other side of Jessica was our friend who grew up in South Korea he was utterly unfazed. There, the idea of a 33-year-old signing up for regular cosmetic injections is par for the course. An estimated one in three South Korean women between the ages of 19 and 29 has undergone a cosmetic procedure, according to a 2015 Gallup poll.

Earlier in the week, I skipped Seoul's stunning palaces and animal-cafe tourist traps to go straight to Gangnam. The neighborhood's streets are lined with high-rises filled with full-service, multilevel plastic surgery and dermatology clinics, many complete with in-house pharmacies, stem cell laboratories, and hotel rooms for out-of-towners. Hundreds of people cycle in and out daily for touch-ups and treatments, with the nonchalance of stopping by a salon for a blowout. Inside these buildings, the future of injectables is being determined by a discerning audience of South Koreans who prize flawless skin, small faces, and round, youthful features. To maximize efficacy and move patients along, the spaces are divided by treatment (like the "filler room" in one clinic I visited, where chairs are lined up for patients to receive their injections, one next to the other). And unlike in the U.S., where privacy surrounding cosmetic work is prized, waiting rooms are sprinkled with patients wearing a full face of numbing cream as they stand by for their cosmetic procedures.

I met with Korean doctors who spoke very casually of thread-lifting vaginas or injecting Botox into the calves, applications I had never heard of (or even imagined).

In the U.S., we have 32 FDA-approved dermal fillers. It can take many years of testing (and bureaucracy) for a new one to see the light of a doctor's office. In South Korea, however, regulation is less stringent, and a person looking for injectables in Seoul has many more options to choose from. Formulas that the FDA has not approved because of a lack of studies on efficacy and safety or potentially serious side effects, like injectable skin-brighteners spiked with glutathione (an antioxidant that can deactivate the body's melanin-producing enzymes), are used regularly in Korea. And while American doctors certainly use neurotoxins and hyaluronic acid fillers off-label (injecting Botox to lift lips, for example), I met with Korean doctors who spoke very casually of thread-lifting vaginas or injecting Botox into the calves, applications I had never heard of (or even imagined).

"It's common for Koreans to go to the dermatologist weekly, sometimes even daily, for maintenance treatments."

When it comes to the latest trends and technology in injectables, Seoul is ahead of the curve. "A lot of Korean [patients] are first-time adopters," says Yongjoon Noh, a plastic surgeon at Banobagi Plastic Surgery & Aesthetic Clinic in Seoul. "New fillers, new materials, new plastic surgery techniques they want to try them all." A big upside to all this: With so many options available, cosmetic injections are vastly cheaper in Korea than they are in the U.S. Alternatives to Botox, like Medytox and Botulax, can be priced as low as $30 for a treatment (compared to about $400 in the U.S.). "It's common for Koreans to go to the dermatologist weekly, sometimes even daily, for maintenance treatments," says Y. Claire Chang, a New York City-based dermatologist who frequently travels to Seoul to learn about the latest advancements in cosmetic dermatology.

Many of the most popular injectable techniques are specific to Korean beauty standards: plump apples of the cheeks and rounded foreheads, as well as the aforementioned V-shaped jawlines. But other techniques, like using Botox to create the impression of poreless skin, or a thin hyaluronic acid filler to softly upturn the corners of the mouth, are likely to start creeping into practices in the U.S. in fact, they've already arrived in some. Want to know what's on the horizon? During my week in Korea, these were the procedures I heard about over and over again.

"Glass skin" is the Korean ideal of a poreless, translucent complexion (like a pane of glass). And the key to getting it is meso-Botox (or colloquially, "skin Botox"). For years, Korean celebrities have sworn by these shallow injections, especially before major events, says Shin Hye Won, a dermatologist at Oracle Clinic in Seoul. Just a few miles east, at Thema Dermatologic Clinic, they have 30 to 40 patients a day who undergo the procedure, says dermatologist Lim Ee Seok. The cost? About $300. (In the U.S., Chang offers it for $950 to $1,200.) Rather than being injected into the muscles to prevent and smooth wrinkles, botulinum toxin is placed just below the skin's surface, at about 40 to 50 sites along the jawline, forehead, and undereye areas. As a result, pores tighten, which makes skin appear smoother and brighter, and excessive acne-causing sebum stops forming. The effects last between three and four months.

The fruity nickname is a reference to the lip shape this filler technique creates. In the past, Angelina Jolie's lips were the most requested look in Seoul (just as they were in the U.S.), says Kang Jong Bum, a dermatologist at JY Plastic Surgery & Dermatology in Seoul. But as of 2019, Koreans prefer the more targeted plumping that many K-pop stars are known for. Instead of giving lips an allover fullness, dermatologists administer a hyaluronic acid filler (like Juvederm or Restylane, or domestic options Yvoire or Neuramis) to the middle areas of the upper and lower lip, enhancing the Cupid's bow. Imagine two double-stemmed cherries on their sides, the stems forming the outline of the edges of your smile. The results last for at least six months and cost about $150 to $250 in Korea.

After the center of the lips are plumped, the same hyaluronic acid filler is often administered just above the outer corners of the mouth a technique called lift edge filler. As the name suggests, the treatment raises the edges of the lips into a soft smile. As we age, the area around our mouth loses volume, and the outer corners start falling into an unintentional frown. Lift edge filler counters the droop and balances out all that fruity volume in the center of the lips, explains Kang.

"It's really popular here for people with resting bitch face. It helps them look softer."

My best friend, CJ, who lives in Gangnam and works in the K-pop industry, knows people who get the procedure for other reasons. "It's really popular here for people with resting bitch face," she says. "It helps them look softer." Like other lip injections, lift edge filler lasts about six months to a year; swelling and small bumps can result, usually for two to three days after the injection, before dissolving. Banobagi offers a cherry lip filler and lifts edge filler combination. A permanent smile is also trending in South Korea. One of the coordinators at JY Plastic Surgery & Dermatology mentioned increased requests for surgical smile lifts, where surgeons create incisions in the same areas targeted by lift edge filler, so your face rests naturally with the corners turned up.

My Korean is limited, but I do know the word for thread lift (also, not to brag, egg and grandpa). And that was a good thing because the term came up often in my reporting. At one point, Kang asked if I wanted to try a thread lift myself. I'm 27, with skin that is more plump than saggy, so I was extremely confused by the offer. Thread lifts in Korea, Kang clarified, are not the "facelift lites" that we consider them here.

"We use thread lifting to make it look like a patient has had a nose job without actually doing a nose job,"

Sure, the mechanics of thread lifts in Korea are the same as they are in the U.S.: Dissolvable, fine-barbed threads are passed underneath the skin with a large needle. Then, as the needle is pulled out, the barbs grab onto skin and pull it upward, stimulating collagen and tightening and lifting the skin. Korean-style thread lifts, though, are less invasive because finer threads are used; they alter the shape of the face, slimming the jawline or changing the contours of the nose.

"We use thread lifting to make it look like a patient has had a nose job without actually doing a nose job," says Choi Jun Young, the lead plastic surgeon at JY Plastic Surgery & Dermatology, of his most requested thread lift procedure. The thread is injected between the nostrils, and in about 15 minutes patients can walk out with the bridge and tip of their nose angled higher. The results last around a year or two and run about $250 to $420.

"These days, it's not about fixing a problem but preventing it," says Lim. He believes the future of cosmetic dermatology in Korea is the "booster shot," an injection designed to rev up skin's natural powers of regeneration and moisturize and brighten skin not to change the contours of your face.

The most sought-after booster shot at JY is Jalupro, a solution of amino acids and sodium hyaluronate (a form of hyaluronic acid) manufactured in Switzerland. Kang says it builds up collagen (to best effect when done as a series of injections over several weeks), so the skin becomes plumper and acne scars start to fade away. Jalupro can be injected all over your face, but Kang likes to target wrinkle-prone areas like the forehead, around the mouth, and around the eyes. It can also supplement laser treatments for stretch marks. My friend Jessica loved the results so much that she got it once a week for three months.

Rejuran which contains PDRN, short for polydeoxyribonucleotide is the booster shot that many doctors in Korea believe holds the most promise. PDRN is extracted from a segment of salmon DNA that is a 95 percent match to that of humans. This small DNA chain is known for its anti-inflammatory and tissue-repairing properties. When injected into the skin, Rejuran is said to shrink pores, diminish the appearance of fine lines and wrinkles, even skin tone, and balance oil production. Unlike skin Botox, which focuses on instantly smoothing the surface, PDRN could heal sun damage or acne overtime at the cellular level, based on early in vitro research. Typically, patients get three monthly injections ($100 to $340 each), for results that last up to a year.

Read more about injectables:

Now, watch a dermatologists entire skin-care routine:

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Originally Appeared on Allure

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Injectables Are the Future of K-Beauty Trends in America - Yahoo Lifestyle