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Company Presenters

4DMT_70Adaptimmune_95
Adverum_80AGTC
Avery_85BioCardia
blue bird bioB-Mogen_80
BrainStorm-(small)Capricor_80

Celixir_80Cellerant_60
Cynata_70Fibrocell_small-square_102x73
Frequency_95Histogen_70
Histogenics_80inRegen_85
Iovance_70Kiadis
LogicBio_70Mesoblast_small-square_102x73
Miromatrix_95MolMed_70
Neuralstem_90Orchard-Therapeutics_70
Oxford-BioMedica_small-square_websitePrecision-Biosciences_80
Regenerex_70ReNeuron_80
Sernova_60Synpromics_80
Thrive-Bioscience_80Tmunity_90
Vericel_small-square_websiteViaCyte_small-square_website

2018 Company Presenters

4DMT_180

PRESENTER
David Kirn, M.D., Chairman, CEO and Co-Founder

GENERAL INFORMATION
(Private)
Headquarters: Emeryville, CA
Number of Full-Time Employees: 35
Company Website: www.4dmoleculartherapeutics.com

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ABOUT 4D MOLECULAR THERAPEUTICS
4D Molecular Therapeutics (4DMT) is focused on the discovery and development of targeted and proprietary AAV gene therapy vectors and therapeutic products. The company’s robust discovery platform, termed Therapeutic Vector Evolution, empowers 4DMT to create customized gene delivery vehicles to deliver genes to any tissue or organ in the body, by optimal clinical routes of administration and with resistance to pre-existing antibodies. These proprietary and targeted products allow the company to treat both rare genetic diseases and complex large market diseases. 4DMT is creating a diverse and deep product pipeline through partnerships, while progressing internal 4DMT products toward clinical trials in parallel. 4DMT’s partners include: Pfizer, Roche, uniQure, AGTC and Benitec. 4DMT’s lead product is for the treatment of choroideremia, an inherited retinal disease.

Adaptimmune_210

ABOUT ADAPTIMMUNE
Adaptimmune, a leader in T cell therapy, is a multinational, clinical-stage biopharmaceutical company focused on developing novel immunotherapies using its proprietary SPEAR T cell receptor platform to treat solid tumors, which has the potential to transform the treatment of cancer. The company’s proprietary SPEAR T cell platform has generated a strong pipeline of affinity-enhanced T cell therapies, with multiple INDs open. Adaptimmune uses these therapies to harness the body’s own immune system to find and destroy diseased cells. The company’s SPEAR TCR therapies offer promise to patients that often have no other options. Adaptimmune is working hard to make that promise a reality. The company is based in the UK, in Oxford, and in the US, in Philadelphia.

Adverum_180

PRESENTER
Amber Salzman, Ph.D., President and CEO

GENERAL INFORMATION
(NASDAQ: ADVM)
Headquarters: Menlo Park, CA
Number of Full-Time Employees: 75
Company Website: www.adverum.com

RECENT NEWS
January 25, 2018 | Adverum Biotechnologies and Editas Medicine Extend Research Collaboration

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ABOUT ADVERUM BIOTECHNOLOGIES
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) deficiency and hereditary angioedema (HAE) as well as wet age-related macular degeneration (wAMD). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop and commercialize gene therapy products for ophthalmic diseases, and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum’s core capabilities include clinical development and in-house manufacturing expertise, specifically in process development and assay development.

AGTC_130

PRESENTER
Sue Washer, President and CEO

GENERAL INFORMATION
(NASDAQ: AGTC)
Headquarters: Alachua, FL
Number of Full-Time Employees: 86
Company Website: www.agtc.com

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ABOUT AGTC
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), and achromatopsia (ACHM CNGB3 and ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS), and otology. The clinical-stage XLRS and XLRP programs, the discovery program in ALD and two additional ophthalmology programs are being developed in collaboration with Biogen. In addition to its product pipeline, AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

Avery_190

PRESENTER
Jen Koevary, Ph.D., Chief Operating and Financial Officer

GENERAL INFORMATION
(Private)
Headquarters: Tucson, AZ
Number of Full-Time Employees: 2
Company Website: www.averytherapeutics.com

ABOUT AVERY THERAPEUTICS
Avery Therapeutics’ mission is to advance tissue engineered therapeutics to improve patient quality of life worldwide. Avery’s lead product, MyCardia™, is an allogeneic, iPSC-derived, engineered tissue for treatment of chronic heart failure and other cardiac conditions. The major advantages of MyCardia are the robustness, enabling minimally invasive implantation and the cryopreserved format, enabling large scale manufacturing, ease of transport and long term stability. Based on studies of MyCardia in gold-standard preclinical models of heart failure, MyCardia has the potential to provide a level of functional improvement that would translate to an improvement in heart failure class. Avery is on track to reach clinical trials by Q1 2019.

BioCardia_190

PRESENTER
Peter Altman, Ph.D., CEO

GENERAL INFORMATION
(OTC: BCDA)
Headquarters: San Carlos, CA
Number of Full-Time Employees: 20
Company Website: www.biocardia.com

RECENT NEWS
January 29, 2018 | CardiAMP Cell Therapy Receives FDA Approval for Pivotal Trial in Chronic Myocardial Ischemia
January 5, 2018 | BioCardia Submits Clinical Trial to FDA for New Indication of Chronic Myocardial Ischemia for CardiAMP Cell Therapy

ABOUT BIOCARDIA
BioCardia is a clinical-stage regenerative medicine company developing novel therapeutics for cardiovascular diseases with large unmet medical needs. BioCardia is developing proprietary comprehensive biotherapeutic solutions for cardiovascular disease in its CardiAMP™ and CardiALLO™ therapies.

bluebird_190

PRESENTER
Jeff Walsh, Chief Financial and Strategy Officer

GENERAL INFORMATION
(NASDAQ: BLUE)
Headquarters: Cambridge, MA
Number of Full-Time Employees: 524
Company Website: www.bluebirdbio.com

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ABOUT BLUEBIRD BIO
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate for the treatment of cerebral adrenoleukodystrophy and its LentiGlobin™ BB305 product candidate for the treatment of transfusion-dependent β-thalassemia and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline.

B-Mogen_160

PRESENTER
Jeff Liter, President and CEO

GENERAL INFORMATION
(Private)
Headquarters: Minneapolis, MN
Number of Full-Time Employees: 8
Company Website: www.bmogen.com

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ABOUT B-MOGEN BIOTECHNOLOGIES
B-MoGen Biotechnologies is a genome engineering company that was established to address complex cell engineering challenges in order to accelerate the study of human diseases and the development of novel therapies. The B-MoGen team is equipped with proprietary tools and knowledge that have been developed into finely honed methods for efficient cellular engineering. The company utilizes this unique expertise to provide three distinct services that share complexity in gene delivery and gene editing. B-MoGen offers a non-viral transposon-based gene delivery platform to support the T cell immunotherapy industry, it is the first company in the world to demonstrate genome engineering of the mitochondria genome and the company offers fee-for-service custom cell engineering of both cell lines and, uniquely, primary human lymphohematopoietic cells for cancer research, drug screening and antibody validation.

BrainStorm_210_update

PRESENTER
Chaim Lebovits, President and CEO

GENERAL INFORMATION
(NASDAQ: BCLI)
Headquarters: Hackensack, NJ
Number of Full-Time Employees: 19
Company Website: www.brainstorm-cell.com

RECENT NEWS
January 3, 2018 | BrainStorm Granted GMP Certification for a Marketing-Level Manufacturing of NurOwn®

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ABOUT BRAINSTORM CELL THERAPEUTICS
BrainStorm Cell Therapeutics is a biotechnology company developing innovative, autologous stem cell therapies for highly debilitating neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig’s disease), Multiple Sclerosis (MS) and Parkinson’s Disease (PD).

Capricor_190

PRESENTER
Linda Marban, Ph.D., CEO

GENERAL INFORMATION
(NASDAQ: CAPR)
Headquarters: Beverly Hills, CA
Number of Full-Time Employees: 35
Company Website: www.capricor.com

RECENT NEWS
January 16, 2018 | Capricor Announces Licensing of Additional Patent Applications from Cedars-Sinai Medical Center for Cellular and Exosome-based Technologies

ABOUT CAPRICOR THERAPEUTICS
Capricor Therapeutics is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate to treat a variety of disorders.

Celixir_190

PRESENTER
Ajan Reginald, CEO

GENERAL INFORMATION
(Private)
Headquarters: Stratford Upon Avon, UK
Number of Full-Time Employees: 44
Company Website: www.celixir.com

RECENT NEWS
January 8, 2018 | CTA Approval for Potentially Pivotal HeartcelTM Phase IIb Trial

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ABOUT CELIXIR
Celixir is a private British regenerative medicine company that discovers and develops life-saving and life-altering regenerative medicines for patients with the greatest medical need. Celixir, founded in 2009, is made up of a world-class team of scientists and biopharmaceutical executives, led by Nobel Laureate Professor Sir Martin Evans and former Roche Global Head of Emerging Technologies, Ajan Reginald. Celixir’s unique platform technology allows them to adopt an “off-the-shelf” approach to deliver regenerative medicines to patients. Celixir has developed world-leading expertise in the discovery and development of cellular therapies, and has built a management team and a series of collaborations to ensure that its medicines are successfully developed in the most efficient and effective manner.

Cellerant_160

PRESENTER
Ram Mandalam, Ph.D., President and CEO

GENERAL INFORMATION
(Private)
Headquarters: San Carlos, CA
Number of Full-Time Employees: 33
Company Website: www.cellerant.com

ABOUT CELLERANT THERAPEUTICS
Cellerant Therapeutics is a clinical-stage company developing innovative cell- and antibody-based immunotherapies for hematologic malignancies and other blood-related disorders. The company’s expertise in hematopoietic stem cell biology has resulted in multiple novel cell-based and antibody-drug conjugate (ADC) product candidates. Cellerant’s lead product, CLT-008 (human Myeloid Progenitor Cells), is a universal cell therapy designed to reduce infection during neutropenia. Neutropenia is a severe side effect of many chemotherapy regimens, particularly for hematologic cancers such as acute myeloid leukemia (AML), and can also result from exposure to acute radiation. The company’s antibody-based immunotherapies are based on novel cancer stem cell targets and Cellerant’s antibody-drug conjugate platform. Cellerant has identified multiple novel targets on leukemic stem cells, and are developing ADC products to enable efficient and selective targeting of leukemic stem cells, which will lead to more effective and durable treatments. The company currently has two ADC products, CLT030 and CSC012-ADC, in development.

Cynata-Therapeutics_160

PRESENTER
Ross Macdonald, Ph.D., CEO

GENERAL INFORMATION
(ASX: CYP)
Headquarters: Melbourne, Australia
Number of Full-Time Employees: 2
Company Website: www.cynata.com

RECENT NEWS
January 30, 2018 | December 2017 Quarterly Report
January 24, 2018 | Treatment Commences in Second Patient Cohort in Cynata’s World First Clinical Trial
January 23, 2018 | Cynata Executes MoU for Commercial Evaluation with Celularity, Inc.

ABOUT CYNATA THERAPEUTICS
Cynata Therapeutics is an Australian clinical-stage stem cell and regenerative medicine company that is developing a therapeutic allogenic stem cell platform technology, Cymerus™, originating from the University of Wisconsin-Madison. The proprietary Cymerus technology addresses a critical shortcoming in existing methods of production of multi-donor derived mesenchymal stem cells (MSCs) for therapeutic use, which is the ability to achieve consistent, economic manufacture at commercial scale. Cymerus utilizes iPSCs to produce MSCs through a proprietary process that is independent of donor limitations. It provides an “off-the-shelf” stem cell platform for therapeutic product use, with a pharmaceutical product business model and economies of scale. This has the potential to create a new standard in the emergent arena of stem cell therapeutics. A Phase I clinical trial of the company’s first MSC product is underway in graft-versus-host disease. Cynata has a strategic partnership with Fujifilm, the company’s largest shareholder.

Fibrocell_200

PRESENTER
John Maslowski, President and CEO

GENERAL INFORMATION
(NASDAQ: FCSC)
Headquarters: Exton, PA
Number of Full-Time Employees: 25
Company Website: www.fibrocell.com

RECENT NEWS
January 30, 2018 | Fibrocell Announces FDA Allowance to Initiate Pediatric Enrollment in Phase 1/2 Clinical Trial of FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

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ABOUT FIBROCELL
Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue. Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase I/II clinical trial for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in preclinical development of FCX-013, its product candidate for the treatment of moderate to severe localized scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with Intrexon Corporation, a leader in synthetic biology.

Frequency_200

PRESENTER
David Lucchino, Co-Founder, President and CEO

GENERAL INFORMATION
(Private)
Headquarters: Woburn, MA
Number of Full-Time Employees: 23
Company Website: www.frequencytx.com

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ABOUT FREQUENCY THERAPEUTICS
Frequency Therapeutics is a clinical-stage company harnessing the body’s innate regenerative biology with its Progenitor Cell Activation (PCA) regenerative platform. Frequency has successfully completed a first-in-human study of FX-322, the company’s lead PCA candidate for hearing restoration. The Phase I study provided crucial data on safety and pharmacokinetics associated with intratympanic delivery of FX-322 as the company moves forward toward additional clinical trials in the US to monitor clinical response. Frequency expects to initiate Phase II clinical work for its hearing regeneration program in 2H 2018. Frequency announced a three-way partnership with the US Army and the Harvard Stem Cell Institute to investigate small molecule regeneration for several disease indications involving muscle. The company also announced a Juvenile Diabetes Research Foundation award for the development of small molecule approach to create insulin and GLP-1 secreting cells. The company has secured $45M in financing.

Histogen_160

PRESENTER
Stephen Chang, Ph.D., Chairman

GENERAL INFORMATION
(Private)
Headquarters: San Diego, CA
Number of Full-Time Employees: 17
Company Website: www.histogeninc.com

ABOUT HISTOGEN
Histogen is a regenerative medicine company developing innovative products from cells grown under simulated embryonic conditions, including low oxygen and suspension. Through this patented technology process, newborn cells are directed to naturally produce vital proteins and growth factors from which the company has developed its rich product portfolio. Histogen’s focus is on stimulating a patient’s own stem cells to regenerate tissues. The company’s lead therapeutic product stimulates hair growth, addressing a multi-billion dollar global market. Applications of Histogen’s technology span multiple therapeutic and aesthetic areas include skin care, orthopedics indications such as cartilage formation and spinal disk repair, wound healing and dermal fillers.

Histogenics_200

ABOUT HISTOGENICS
Histogenics is a leader in the development of restorative cell therapies that may offer rapid-onset pain relief and restored function. Histogenics’ lead investigational product, NeoCart, is designed to rebuild a patient’s own knee cartilage to treat pain at the source and potentially prevent a patient’s progression to osteoarthritis. NeoCart is one of the most rigorously studied restorative cell therapies for orthopedic use. Histogenics recently completed enrollment of its NeoCart Phase III clinical trial and expects to report top-line, one-year superiority data in the third quarter of 2018. NeoCart is designed to perform like articular hyaline cartilage at the time of treatment, and as a result, may provide patients with more rapid pain relief and accelerated recovery as compared to the current standard of care. Histogenics’ technology platform has the potential to be used for a broad range of additional restorative cell therapy indications.

inRegen_200

PRESENTER
Tim Bertram, Ph.D., CEO

GENERAL INFORMATION
(Private)
Headquarters: Winston-Salem, NC
Number of Full-Time Employees: 3
Company Website: www.inregen.com

ABOUT INREGEN
inRegen is a clinical-stage biotechnology company developing autologous cellular therapies (ACT) to treat chronic kidney disease (CKD). The company’s technology is based on proprietary breakthroughs in cell biology and manufacturing. Phase II clinical trials have demonstrated the value of inRegen’s investigational product in diabetic patients with late-stage progressive diabetic kidney disease. Patients stabilized renal function and remained dialysis-free for over four years post-injection. inRegen holds the promise of transforming medical care for the treatment of renal failure, potentially delaying or eliminating the need for dialysis or kidney transplant. InRegen produces renal ACT (REACT) by using proprietary methods of manufacturing and delivery. This therapy triggers regeneration of new functional kidney tissue that eliminates or reduces many of the risks of today’s current standards-of-care for kidney failure. Because the new functioning nephrons are made from the patient’s own cells, there is no risk of rejection and lifelong immunosuppressive therapy.

Iovance_170

PRESENTER
Maria Fardis, President and CEO

GENERAL INFORMATION
(NASDAQ: IOVA)
Headquarters: San Carlos, CA
Number of Full-Time Employees: 65
Company Website: www.iovance.com

RECENT NEWS
January 29, 2018 | Iovance Biotherapeutics, Inc. Announces Closing of $172.5 Million Common Stock Public Offering
January 24, 2018 | Iovance Biotherapeutics Announces Preliminary Phase 2 Data for TIL Treatment in Head and Neck and Cervical Cancers

ABOUT IOVANCE BIOTHERAPEUTICS
Iovance Biotherapeutics is a clinical-stage biotechnology company focused on the development of cancer immunotherapy products for the treatment of various cancers. The company’s lead product candidate is an adoptive cell therapy using tumor-infiltrating lymphocyte (TIL) technology being investigated for the treatment of patients with metastatic melanoma, recurrent and/or metastatic squamous cell carcinoma of the head and neck and recurrent and metastatic or persistent cervical cancer.

Kiadis_160

PRESENTER
Karl Hard, Head of IR and Communications

GENERAL INFORMATION
(KDS: NA)
Headquarters: Amsterdam, The Netherlands
Number of Full-Time Employees: 60
Company Website: www.kiadis.com

RECENT NEWS
January 16, 2018 | Kiadis Pharma Proposes Former Cipla CEO Subhanu Saxena as New Supervisory Board Member

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ABOUT KIADIS PHARMA
Kiadis Pharma’s allodepleted T cell immunotherapy product can make haploidentical hematopoietic stem cell transplantations (HSCT) safer and more effective. Phase II data with lead product ATIR101™ given after a HSCT in patients with blood cancer shows a strong and clinically relevant improvement over literature for the Baltimore protocol, without severe chronic graft-versus-host-disease. The company submitted an MAA to the EMA in April 2017, for approval of ATIR101™ as an adjunctive treatment in HSCT for malignant disease. Kiadis received Day 120 questions in September 2017 and expects a potential (conditional) approval in 4Q18 and launch in 2H19. Kiadis is conducting a Phase III trial across Europe and North America (head-to-head against the Baltimore protocol). The first patient was enrolled in December 2017. In September 2017 the FDA granted ATIR101™ the Regenerative Medicine Advanced Therapy (RMAT) designation. ATIR101™ has been granted Orphan Drug Designations both in the US and Europe.

LogicBio_170

PRESENTER
Fred Chereau, CEO

GENERAL INFORMATION
(Private)
Headquarters: Cambridge, MA
Number of Full-Time Employees: 16
Company Website: www.logicbio.com

RECENT NEWS
January 2, 2018 | LogicBio Therapeutics Announces Expansion of Leadership Team through Multiple Key Hires

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ABOUT LOGICBIO THERAPEUTICS
Launched in 2016, LogicBio is a leading genome-editing company founded by pioneers in gene therapy from Stanford University, with a mission to develop transformative medicines for life-threatening diseases. LogicBio’s technology includes GeneRide™, a promoterless, nuclease-free genome editing technology, which drives high expression of therapeutic proteins by harnessing homologous recombination – a naturally occurring process – to safely integrate transgenes site-specifically into the genome to provide a durable cure from a one-time treatment; and a proprietary library of adeno-associated viral vectors which have proven highly effective and safe in clinical trials. This combination of next generation genome editing and proven delivery technology allow LogicBio to target a wide range of diseases not addressable today, including early-onset conditions in young patients. With a world-class global team, headquartered in Cambridge, MA, LogicBio is working to deliver the next wave of innovation in genetic medicine.

Mesoblast_200

PRESENTER
Donna Skerrett, M.D., Chief Medical Officer

GENERAL INFORMATION
(NASDAQ: MESO | ASX: MSB)
Headquarters: Melbourne, Australia
Number of Full-Time Employees: 75
Company Website: www.mesoblast.com

ABOUT MESOBLAST
Mesoblast is a global leader in developing innovative cell-based medicines. The company has leveraged its proprietary technology platform, which is based on specialized cells known as mesenchymal lineage adult stem cells, to establish a broad portfolio of late-stage product candidates. Mesoblast’s allogeneic, ‘off-the-shelf’ cell product candidates target advanced stages of diseases with high, unmet medical needs including cardiovascular conditions, orthopedic disorders, immunologic and inflammatory disorders and oncologic/hematologic conditions.

Miromatrix_200

PRESENTER
Jeff Ross, Ph.D., CEO

GENERAL INFORMATION
(Private)
Headquarters: Eden Prairie, MN
Number of Full-Time Employees: 28
Company Website: www.miromatrix.com

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ABOUT MIROMATRIX
Miromatrix is a leading regenerative medicine company bioengineering transplantable organs including livers, kidneys and hearts based on its patented perfusion decellularization and recellularization technology, with collaborators including the Mayo Clinic, Mount Sinai Hospital and Texas Heart Institute. In addition to whole organ development, Miromatrix has successfully developed, manufactured and commercialized a series of porcine organ-derived biological products. MIROMESH®, is the only liver-derived biologic mesh for soft tissue reinforcement. Miromatrix released one-year prospective multicenter clinical trial data for the treatment of hiatal hernias demonstrating no reintervention. MIRODERM®, is the only liver-derived wound care product and a recent case-series published in WOUNDS® demonstrated its ability to successfully treat diabetic foot ulcers that had previous failed other advanced biologics. A multicenter clinical study is currently enrolling to further demonstrate its ability to treat this challenging patient population. Miromatrix is leveraging these successes with the company’s recellularization technology to fully address the transplantable organ shortage.

MolMed_170

PRESENTER
Luca Alberici, Ph.D., Chief Business Officer

GENERAL INFORMATION
(MLMD.MI)
Headquarters: Milan, Italy
Number of Full-Time Employees: 186
Company Website: www.molmed.com

ABOUT MOLMED
MolMed S.p.A. is an Italian biotechnology cell and gene company focused on research, development, manufacturing and clinical validation of innovative anti-cancer therapies. MolMed built an original dual business model, including research and development on proprietary onco and onco-hematology products, and third party GMP development and manufacturing services. In both areas MolMed delivered outstanding results, owning one of the first authorized cellular therapies (Zalmoxis®, first patient-specific cell therapy, devoted to the treatment of adult patients affected by leukemia or other high-risk hematological malignancies) authorized by EMA for all EU countries, as well as the only facility authorized in Europe for commercial ex-vivo cell and gene products (Strimvelis and Zalmoxis) manufacturing, serving relevant international partners, such as GSK, Cellectis, Rocket Pharma and others. Founded in 1996 as an academic spin-off of the San Raffaele Scientific Institute, one of the leading private scientific research institutes in Italy, MolMed is listed on the (MTA) Milan stock exchange.

Neuralstem_200

PRESENTER
Karl Johe, Ph.D., Chief Scientific Officer

GENERAL INFORMATION
(NASDAQ: CUR)
Headquarters: Germantown, MD
Number of Full-Time Employees: 6
Company Website: www.neuralstem.com

ABOUT NEURALSTEM
Neuralstem is a clinical-stage biopharmaceutical company developing novel treatments for nervous system diseases of high unmet medical need. The company’s lead asset and stem cell therapy candidate, NSI-566, is a spinal cord-derived neural stem cell line being tested in Amyotrophic Lateral Sclerosis (ALS), chronic spinal cord injury (cSCI) and ischemic stroke. Additionally, NSI-189, is a chemical entity in development for major depressive disorder (MDD) and for Angelman syndrome, irradiation-induced cognitive impairment, neuropathy associated with Type 1 and Type 2 diabetes and stroke. These product candidates are based on the company’s proprietary neural stem cell technology.

Orchard_160

PRESENTER
Mark Rothera, President and CEO

GENERAL INFORMATION
(Private)
Headquarters: London, UK
Number of Full-Time Employees: 71
Company Website: www.orchard-tx.com

RECENT NEWS
January 17, 2018 | Orchard Therapeutics Appoints Frank Thomas as Chief Financial Officer and Chief Business Officer

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ABOUT ORCHARD THERAPEUTICS
Orchard Therapeutics is a biotechnology company incorporated in September 2015 and dedicated to transforming the lives of patients with rare disorders through innovative gene therapies. The company’s programs will use the potential of ex-vivo autologous hematopoietic stem cell gene therapy to restore normal gene function in severe and life-threatening inherited disorders. Orchard works in partnership with the world’s leading research centers to harness the life-giving potential of gene therapy. The company’s mission is to transform the lives of patients with rare disorders through innovative gene therapies. Orchard’s vision is to become the leader in gene therapy for primary immune deficiencies and inherited metabolic disorders.

Oxford-BioMedica_200

PRESENTER
Jason Slingsby, Head of Business Development

GENERAL INFORMATION
(LON: OXB)
Headquarters: Oxford, UK
Number of Full-Time Employees: 320
Company Website: www.oxfordbiomedica.co.uk

RECENT NEWS
January 17, 2018 | Oxford BioMedica Announces £3 Million Grant Awarded by Innovate UK
January 23, 2018 | Oxford BioMedica Notes the US FDA Priority Review for Kymriah™ for Adults with r/r DLBCL and EMA Accelerated Assessment for Children, Young Adults with r/r B-cell ALL and Adult Patients with r/r DLBCL

ABOUT OXFORD BIOMEDICA
Oxford BioMedica is a leading gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford BioMedica has built a sector leading lentiviral vector delivery platform (LentiVector®), which the company leverages to develop in vivo and ex vivo products themselves and with partners. The company has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of CNS disorders (Parkinson’s), oncology (CAR-T), and ophthalmology. Oxford BioMedica has also entered into a number of partnerships including with Novartis, Sanofi, GSK, Orchard Therapeutics and Immune Design, through which it has long-term economic interests in other potential gene and cell therapy products. Oxford BioMedica is a fast growing company in Oxfordshire, UK and employs more than 320 people.

Precision_190

PRESENTER
Matt Kane, CEO

GENERAL INFORMATION
(Private)
Headquarters: Durham, NC
Number of Full-Time Employees: 94
Company Website: www.precisionbiosciences.com

ABOUT PRECISION BIOSCIENCES
Precision BioSciences is dedicated to improving life. The company develops products designed to cure genetic disease, overcome cancer and feed the planet. To make this vision a reality, Precision has brought together the world’s leading minds in genome editing and ARCUS, the first therapeutic grade editing platform. This simple and uniquely powerful genome editing method allows the company to create products that solve significant problems in oncology, genetic disease, agriculture and beyond.

Regenerex_170

PRESENTER
Suzanne Ildstad, M.D., CEO

GENERAL INFORMATION
(Private)
Headquarters: Louisville, KY
Number of Full-Time Employees: 10
Company Website: www.regenerex.com

ABOUT REGENEREX
Regenerex, an innovative, late clinical-stage cellular therapy company, is developing its FCRx platform technology with the goal of eliminating the burden of chronic immunosuppression in select organ transplant and severe autoimmune conditions. In an ongoing Phase II study, the company’s proprietary allogeneic cell therapy product, FCR001, has been shown to induce tolerance in recipients of solid organ transplants from unmatched and unrelated donors, allowing living donor kidney transplant recipients to wean completely off immunosuppression one year after transplant. FCR001 Phase II subjects achieved these results utilizing nonmyeloablative conditioning which minimizes toxicities typically associated with a standard Hematopoietic Stem Cells (HSC) transplant. FDA has granted Regenerex clearance to proceed with a pivotal Phase III study in Living Donor Kidney Transplant. FDA has also granted FCR001 Orphan Drug designation and Regenerative Medicine Advanced Therapy (RMAT) designation.

ReNeuron_180

PRESENTER
Richard Beckman, M.D., Chief Medical Officer

GENERAL INFORMATION
(LON: RENE)
Headquarters: Pencoed, Wales, UK
Number of Full-Time Employees: 66
Company Website: www.reneuron.com

RECENT NEWS
January 26, 2018 | Phase II Stroke Data Presented at AHA Conference

ABOUT RENEURON
ReNeuron is a leading, clinical-stage cell therapy development company. Based in the UK, ReNeuron has therapeutic candidates in clinical development for motor disability as a result of stroke, for critical limb ischemia and for the blindness-causing disease, retinitis pigmentosa. ReNeuron is also advancing its proprietary exosome technology platform as a potential new nanomedicine targeting cancer and as a potential delivery system for gene therapy treatments. ReNeuron’s shares are traded on the London AIM market under the symbol RENE.L.

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PRESENTER
Philip Toleikis, Ph.D., President and CEO

GENERAL INFORMATION
(TSX-V: SVA; OTCQB: SEOVF)
Headquarters: London, Ontario, Canada
Number of Full-Time Employees: 8
Company Website: www.sernova.com

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ABOUT SERNOVA CORP.
Sernova Corp. is a clinical-stage regenerative medicine company commercializing products which benefit patients with chronic metabolic, neurological and hematological diseases with first therapeutic applications in diabetes and hemophilia. Sernova’s platform technologies include the Cell Pouch System™, an implantable medical device for the long-term survival and function of therapeutic cells, therapeutic cells (porcine and human donor islets, stem cell processes) and micro-encapsulation-based technologies which provide local immune protection to therapeutic cells enabling potential elimination of antirejection drugs.

Synpromics_180

PRESENTER
David Venables, Ph.D., CEO

GENERAL INFORMATION
(Private)
Headquarters: Edinburgh, UK
Number of Full-Time Employees: 32
Company Website: www.synpromics.com

RECENT NEWS
January 25, 2018 | Synpromics Announces New Partnership with UCL to Develop Novel Gene Therapies for Blood-based Disorders
January 17, 2018 | Synpromics Appoints Sarah Haecker Meeks, PhD as Vice President of Business Development

ABOUT SYNPROMICS
Synpromics is the leader in gene control, improving human health by enabling safer, more effective cell and gene medicines through proprietary genomics, bioinformatics and intelligent data driven design. Synpromics enables unprecedented control of gene expression through custom designed synthetic cell-type, or condition, specific promoters. Custom control of gene expression is fundamentally important in the creation of gene and cell therapy products. The technology created by the Synpromics team allows tighter control of gene expression, facilitating the creation of novel promoters tailored to: Tissue-type, e.g., liver, muscle, CNS; Developmental stage, e.g., lineage specificity in stem cells; Cell affliction, e.g., pathogen-infected; Inducible by biological, chemical or environmental stimuli; Eliminate off-target gene expression. By enhancing the specific activity of the promoter it is possible to reduce vector dose, increasing the safety profile of the therapeutic, lowering immunogenicity, reducing off-target side effects.

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PRESENTER
Thomas Farb-Horch, President and CEO

GENERAL INFORMATION
(Private)
Headquarters: Boston, MA
Number of Full-Time Employees: 15
Company Website: www.thrivebio.com

ABOUT THRIVE BIOSCIENCE
Thrive Bioscience is developing a family of instruments that solve major roadblocks in research, drug discovery/development and regenerative medicine. These problems result from the current widespread practice of manually-performed cell and stem cell culture (including iPSCs). Cell culture is central to biomedical research and the delivery of cell therapeutics — an $18 billion market per year, performed in over 80,000 labs in over 200,000 non-smart incubators worldwide. Thrive will deliver its first revenue instruments to internationally recognized customers, including the Harvard Stem Cell Institute for automating the difficult task of growing iPSCs and the Broad Institute of MIT and Harvard for a sequencing/drug discovery project. Thrive has a significant IP portfolio of 23 patent applications, has raised $16 million of financing to-date and will be raising its Series B mid-year. Thrive’s instruments integrate a broad range of technologies — robotics, optics, microscopy, AI and image analysis.

Tmunity_200

PRESENTER
Usman Azam, M.D., President and CEO

GENERAL INFORMATION
(Private)
Headquarters: Philadelphia, PA
Number of Full-Time Employees: 2
Company Website: www.tmunity.com

RECENT NEWS
January 22, 2018 | Tmunity Therapeutics Raises $100 Million in Series A Financing to Advance Portfolio of Next-Generation T cell Immunotherapies to Transform the Treatment of Cancer

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ABOUT TMUNITY THERAPEUTICS
At the heart of Tmunity is a relentless focus on creating proprietary products by integrating T cell discovery, development and production capabilities. Tmunity has established a broad, exclusive research and development collaboration and license agreement with the University of Pennsylvania (UPenn) in the field of T cell therapies in oncology, infectious diseases and autoimmune diseases. Tmunity’s portfolio consists of key T cell therapy platforms designed to produce engineered T cells that activate or suppress immunity. These approaches include: T cell receptors, regulatory T cells, gene transfer, allogeneic T cells and bionic T cells, as well as certain CAR-T programs.

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PRESENTER
Nick Colangelo, President and CEO

GENERAL INFORMATION
(NASDAQ: VCEL)
Headquarters: Cambridge, MA
Number of Full-Time Employees: 250
Company Website: www.vcel.com

RECENT NEWS
January 2, 2018 | Vericel Initiates Collaboration with Innovative Cellular Therapeutics

ABOUT VERICEL
Vericel develops, manufactures and markets autologous cell-based therapies for patients with serious diseases and conditions. The company markets two cell therapy products in the US. MACI® (autologous cultured chondrocytes on porcine collagen membrane) is a third generation autologous cellularized scaffold product that is indicated for the repair of single or multiple symptomatic, full-thickness cartilage defects of the adult knee, with or without bone involvement. Epicel® (cultured epidermal autografts) is a permanent skin replacement for the treatment of patients with deep dermal or full thickness burns greater than or equal to 30% of total body surface area.

ViaCyte_200_website

PRESENTER
Paul Laikind, Ph.D., President and CEO

GENERAL INFORMATION
(Private)
Headquarters: San Diego, CA
Number of Full-Time Employees: 49
Company Website: www.viacyte.com

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ABOUT VIACYTE
ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte has two products in clinical development. The PEC-Direct™ product candidate delivers stem cell-derived PEC-01 pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap™ (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is headquartered in San Diego, California. The company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF.