View in your browser January 4, 2018

FDA Approves Spark Therapeutics’ LUXTURNA (voretigene neparvovec-rzyl), aOne-Time Gene Therapy for Patients with Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy December 19, 2017 – (Spark) – LUXTURNA is first gene therapy for a geneticdisease, first and only pharmacologic treatment for an inherited retinal disease andfirst adeno-associated virus vector gene therapy approved in U.S.

Atlas Venture Launches Generation Bio January 4, 2018 – (Atlas Venture) – Generation Bio is developing a breakthroughclass of genetic medicines based on its proprietary GeneWave technology, whichdelivers high levels of durable gene expression and can be re-dosed to titrate andsustain effect.

BioNTech Secures USD 270 Million in Series A Financing January 4, 2018 – (BioNTech) – BioNTech will use the capital to further advance itsclinical pipeline of individualized immunotherapies covering a number of newapproaches including mRNA and CAR-T / T-Cell receptors for the treatment ofcancer and other diseases with high medical need.

Sangamo and Pfizer Announce Collaboration for Development of Zinc Finger ProteinGene Therapy for ALS January 3, 2018 – (Sangamo) – Under the terms of the collaboration agreement,Sangamo will receive a $12 million upfront payment from Pfizer. Sangamo will beresponsible for the development of ZFP-TF candidates. Pfizer will be operationallyand financially responsible for subsequent research, development, manufacturingand commercialization for the C9ORF72 ZFP-TF program and any resultingproducts.

Spark Therapeutics Announces First-of-Their-Kind Programs to Improve PatientAccess to LUXTURNA January 3, 2018 – (Spark) – Spark Therapeutics announced three new payerprograms: an outcomes-based rebate arrangement with a long-term durabilitymeasure, an innovative contracting model and a proposal to CMS under whichpayments for LUXTURNA (voretigene neparvovec-rzyl) would be made over time.

NexImmune Raises $23 Million to Advance Endogenous Cellular ImmunoTherapyinto Clinical Development January 1, 2018 – (NexImmune) – Series A financing co-led by ArrowMark Partnersand Barer & Son Capital Financing supports initiation of Phase I/II trial of AIM ACT inrelapsed/refractory Hematologic Malignancies in 2018.

Muscular Dystrophy Biotech Solid Biosciences Files for a $100 Million IPO December 29, 2017 – (Nasdaq) – The Cambridge, MA-based company was foundedin 2013 plans to list on the Nasdaq under the symbol SLDB. Solid Biosciences filedconfidentially on August 4, 2017.

Histogenics Corporation and MEDINET Co., Ltd Enter Into Licensing Agreement forDevelopment and Commercialization of NeoCart for the Japanese Market December 21, 2017 – (Histogenics) – Transaction terms include a $10 million up-front payment with a potential of $87 million in total milestones, and tiered royaltieson sales.

Janssen Enters Worldwide Collaboration and License Agreement with LegendBiotech to Develop Investigational CAR-T Anti-Cancer Therapy December 21, 2017 – (Janssen) – Under terms of the agreement, Legend will grantJanssen a worldwide license to jointly develop and commercialize LCAR-B38M inmultiple myeloma with the Legend team of experts.

Salk Institute President Elizabeth Blackburn Announces Retirement December 21, 2017 – (Salk) – Salk Institute President Dr. Elizabeth Blackburn hasannounced her plans to retire and step down as the Institute’s head at the end of thissummer. The Board of Trustees, led by Dan Lewis, who was elected Chair inNovember, will commence a search for Blackburn’s successor beginning in January.

Juno Therapeutics and Thermo Fisher Scientific Announce CAR T ManufacturingPartnership December 20, 2017 – (Thermo Fisher Scientific) – Under the seven-yearnonexclusive licensing and supply agreement, Juno has obtained rights to use CTSDynabeads CD3/CD28 magnetic beads as part of the clinical and commercialmanufacturing processes for its current and future CAR T therapies.

Orchard Therapeutics Announces $110M Series B Financing to AdvanceTransformative Gene Therapy Pipeline December 20, 2017 – (Orchard) – Proceeds of the Series B round will be used torapidly progress Orchard's pipeline, including the global launch of OTL-101, and toexpand their manufacturing infrastructure and business development activity.

Tessa Therapeutics Closes USD 80 Million Financing Round Led by Temasek December 20, 2017 – (Tessa) – Tessa will use the proceeds from this funding roundto further advance its clinical pipeline and to bring new therapies, based on thecompany’s Virus Specific T cell platform, into clinical trials.

Institute for Clinical and Economic Review Releases Draft Evidence Report on CAR-T Therapy for B-cell Cancers December 19, 2017 – (ICER) – Both drugs are chimeric antigen receptor T-cell(CAR-T) therapies and were approved earlier this year as potentially curativetreatments for certain cancers.

Intellia Therapeutics Names John Leonard, M.D., President and Chief ExecutiveOfficer

December 18, 2017 – (Intellia) – Dr. Leonard succeeds Nessan Bermingham, Ph.D.,Intellia’s founding President and CEO, who is returning to the venture capitalindustry.

CIRM Grants ViaCyte $1.4M to Create Immune‐Evasive Pluripotent Stem Cell Lines December 15, 2017 – (ViaCyte) – The California Institute for Regenerative Medicine(CIRM) approved a grant of $1.4 million to support the initial development ofimmune-evasive pluripotent stem cell lines. The focus of the project will be togenetically engineer the Company’s CyT49 pluripotent stem cell line.

Audentes Announces Positive Interim Data from First Dose Cohort of ASPIRO, aPhase 1/2 Clinical Trial of AT132 in Patients With X-Linked Myotubular Myopathy January 4, 2018 – (Audentes) – The key assessment of respiratory function in thisfirst data set from Cohort 1 is a measurement of maximal inspiratory pressure(MIP), for which values ≥ 80 cmH20 are considered normal in healthy children less

than 5 years of age.

Phase III Clinical Trials for Stem Cell-Based Cartilage Regeneration Therapy HaveStarted January 4, 2018 – (Osaka University) – This is the first clinical trial of regenerativetherapy in Japan in terms of using allogeneic stem cells and the commercial use ofstem cell bank at the Medical Center for Translational Research (MTR)*1 of OsakaUniversity.

Adverum Biotechnologies Doses First Patient in the ADVANCE Phase 1/2 ClinicalTrial of ADVM-043 for A1AT Deficiency December 28, 2017 – (Adverum) – Adverum Biotechnologies announced the dosingof the first patient in the ADVANCE Phase 1/2 clinical trial of ADVM-043 for alpha-1antitrypsin deficiency. The ADVANCE clinical trial is designed to evaluate the safetyand protein expression following a single administration of ADVM-043, Adverum’snovel gene therapy candidate.

Organovo Receives Orphan Designation From U.S. FDA for 3D BioprintedTherapeutic Liver Tissue Treatment of Alpha-1 Antitrypsin Deficiency December 26, 2017 – (Organovo) – “This is a critical milestone that supports ourongoing development of 3D bioprinted tissues for therapeutic use. We remain ontrack for filing an Investigational New Drug application with the FDA in calendar-year2020, as we continue to conduct safety and dosing investigations in small animal

disease models and move to defining and scoping IND enabling studies,”said Taylor J. Crouch, CEO, Organovo.

Mesoblast Receives FDA Regenerative Medicine Advanced Therapy Designation forIts Cell Therapy in Heart Failure Patients With Left Ventricular Assist DevicesDecember 21, 2017 – (Mesoblast) – This RMAT designation allows for multi-disciplinary, comprehensive interactions with the FDA to support the efficientdevelopment of and potential accelerated approval pathway for Mesoblast’sallogeneic MPCs in the treatment of heart failure patients with LVADs.

Abeona Therapeutics Enrolls First Patient in ABO-101 Phase 1/2 Clinical Trial forMPS IIIB December 20, 2017 – (Abeona) – ABO-101 has been granted Orphan ProductDesignation by the U.S. Food and Drug Administration and received Rare PediatricDisease Designation as a pre-requisite component of the FDA’s Priority ReviewVoucher process.

REGENXBIO Announces IND Active for Phase I/II Trial of RGX-121 to TreatMucopolysaccharidosis Type II December 19, 2017 – (REGENXBIO) – “The goal of the RGX-121 program is todevelop a single-dose treatment for MPS II that can prevent the progression ofneurocognitive decline experienced by children with the disease, which addressesone of the shortcomings of the current standard of care, enzyme replacementtherapy,” said Stephen Yoo, M.D., Chief Medical Officer of REGENXBIO.

BioMarin Doses First Patient in Global GENEr8-1 Phase 3 Study of ValoctocogeneRoxaparvovec Gene Therapy for Severe Hemophilia A December 19, 2017 – (BioMarin) – BioMarin Pharmaceutical announced that it hasdosed the first patient in the global GENEr8-1 Phase 3 study with the 6e13 vg/kgdose for valoctocogene roxaparvovec, an investigational gene therapy for thetreatment of patients with severe hemophilia A. This is the first of two Phase 3studies in the global Phase 3 program to dose a first patient.

Gene Therapy Restores Normal Blood Glucose Levels in Mice with Type 1 Diabetes January 4, 2018 – (Cell Stem Cell) – A study published January 4th in Cell Stem Celldemonstrates that a gene therapy approach can lead to the long-term survival offunctional beta cells as well as normal blood glucose levels for an extended period oftime in mice with diabetes.

Cellular Barcoding Helps Scientists Understand the Behavior of Stem Cells January 3, 2018 – (NIH) – By tagging bone marrow cells of mice with a genetic label,or barcode, researchers were able to track and describe the family tree of individualblood cells as they form in their natural environment. The scientists discovered thatthese cells regenerate differently than their counterparts do after a blood celltransplant.

NIH Discovery Brings Stem Cell Therapy for Eye Disease Closer to the Clinic January 2, 2018 – (NIH) – Scientists at the National Eye Institute report that tinytube-like protrusions called primary cilia on cells of the retinal pigment epithelium areessential for the survival of the retina’s light-sensing photoreceptors. The discoveryhas advanced efforts to make stem cell-derived RPE for transplantation into patientswith geographic atrophy, otherwise known as dry age-related macular degeneration,a leading cause of blindness in the U.S.

Cellular Dynamics International Sends Human iPSC-Derived Cardiomyocytes ToInternational Space Station On NASA Resupply Mission December 27, 2017 – (CDI) – The purpose of the scientific research project utilizingCDI's iCell Cardiomyocytes in space is to validate the function of NASA's newBioculture System for automated cell culture on the International Space Station andto study human cardiac cell function in microgravity.

Hearing is Believing in Gene Therapy’s Promise December 20, 2017 – (Rice) – Gene editing could someday help people at risk ofhearing loss from genetic mutations, according to research by a new Rice Universityfaculty member.

Scientists Use Gene Editing To Prevent A Form Of Deafness In Mice December 20, 2017 – (Broad Institute) – Once inside the ear, the gene editor "homesin on the mutant gene" and cuts the DNA "so that mutant gene can no longer poisonthe hair cells and cause the hair cells to eventually die," said David Liu, a geneticengineer at Broad Institute, the Massachusetts Institute of Technology and HarvardUniversity.

(Re)-Acquiring the Potential to Become Everything December 19, 2017 – (Helmholtz Zentrum München) – A new study in ‘NatureGenetics’ identifies a specific population of pluripotent embryonic stem cells that canreprogram to totipotent-like cells in culture.

NICE Final Draft Guidance Recommends Gene Therapy for Rare ‘Bubble BabySyndrome’ January 3, 2018 – (NICE) – The draft guidance, which is the first time NICE hasapplied its new, higher cost effectiveness limits for treatments for very rareconditions, recommends Strimvelis when no suitable matched related stem celldonor is available.

Remarks by Dr. Scott Gottlieb on FDA’s Role in Advancing a Modern Framework forGene Therapy December 19, 2017 – (FDA) – We need to make sure that we’re taking policy stepsto enable these innovations to efficiently advance to benefit patients, while wemaintain our gold standard for ensuring the safety and efficacy of new products.

An $850,000 Price Tag on Gene Therapy Shouldn’t Freak You Out—Yet January 4, 2018 – (MIT Technology Review) – “The challenge is like going frombeing an apartment renter to a condo buyer and being shocked at [the] purchaseprice,” says Mark Trusheim, who directs MIT’s New Drug Development Paradigmsprogram.

In Vivo's Top 10 For 2017 January 3, 2018 – (In Vivo) – In Vivo's 10 most-read thought leadership pieces oflast year including ARM's white paper on new payment and financing models forcurative regenerative medicines.

In a Milestone Year, Gene Therapy Finds a Place in Medicine December 28, 2017 – (Washington Post) – Gene therapies to treat cancer and evenpull off the biblical-sounding feat of helping the blind to see were approved by U.S.regulators, establishing gene manipulation as a new mode of medicine.

Four Amazing Things Gene Editing Did in 2017 December 27, 2017 – (MIT Technology Review) – Gene editing hasn’t cured disease

in a human being yet—but it is getting closer. With the debut of CRISPR a few yearsago, advances in the technology have been happening at a breakneck pace.

CRISPR Isn't Enough Anymore. Get Ready for Gene Editing 2.0. December 26, 2017 – (Wired) – This year, newer, flashier gene editing tools beganrolling off the production line, promising to outshine their first-generation cousin. So ifyou were just getting your head around Crispr, buckle up. Because gene-editing 2.0is here.

Personalized Supply Chains for Cell Therapies December 15, 2017 – (Cell & Gene Therapy Insights) – This article aims to provideinsight into the function and implementation strategy for COPs, while alsoconsidering their strategic position and connectivity with other adjacent systemswithin the autologous cell therapy supply chain. Additionally, the article reviews theroles of key supply chain stake- holders and the systems they interact with toultimately provide a seamless experience for physicians and patients.

About ARM

The Alliance for Regenerative Medicine (ARM) is the preeminent global advocate for regenerative and advancedtherapies. ARM fosters research, development, investment and commercialization of transformational treatmentsand cures for patients worldwide. By leveraging the expertise of its membership, ARM empowers multiplestakeholders to promote legislative, regulatory and public understanding of, and support for, this expanding field.Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. tospecifically represent the interests of the companies, research institutions, investors and patient groups thatcomprise the entire regenerative medicine community. Today, ARM has more than 270 members and is the leadingglobal advocacy organization in this field. To learn more about ARM or to become a member, visitwww.alliancerm.org.

Copyright © 2018 Alliance for Regenerative Medicine, All rights reserved.

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