ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
January 16, 2020
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New Report Demonstrates Potential for Cell and Gene Therapies to Provide Cost Savings
January 10, 2020 – (ARM) – The report looks at sickle cell disease, hemophilia A, and multiple myeloma as case studies, and found that the total potential savings of cell and gene therapies for these indications range from 18% to 30% in annual total disease costs and productivity. This represents an aggregate cost savings of more than $33B over ten years.
BioNTech to Acquire Neon to Strengthen Global Leadership Position in T Cell Therapies
January 16, 2020 – (BioNTech) – BioNTech and Neon Therapeutics announced that they have entered into a definitive merger agreement under which BioNTech will acquire Neon in an all-stock transaction valued at approximately $67M.
CellFE Raises $3.6M in Seed Round Co-led by Cota Capital and Dynamk Capital
January 16, 2020 – (CellFE) – CellFE, an innovative startup developing a microfluidics based device to deliver gene-editing molecules, announced it has raised $3.6M in an initial Series Seed. The investment will be used to fuel the progress of the CellFE team towards the company mission of enabling the engineering of the next generation cell therapies.
Artisan Bio Announces Global Research and Discovery Collaboration Agreement with Takeda to Develop Next-Generation Cell Therapy Products
January 15, 2020 – (Artisan Bio) – Under the terms of the agreement, Artisan Bio will deploy its STAR platform and synthetic biology expertise to construct customized and precisely engineered cell therapies. Artisan will lead discovery efforts, including gene editing, and Takeda will be responsible for the development, manufacturing, and commercialization of the resulting cell therapy products.
Astellas Completes Acquisition of Audentes Therapeutics
January 15, 2020 – (Astellas Pharma) – Astellas announced that it has successfully completed the previously announced acquisition of Audentes Therapeutics. The $3B acquisition was announced in December 2019. 
Emendo Biotherapeutics Raises $61M to Advance Next Generation Genome Editing Therapeutics
January 15, 2020 – (Emendo Biotherapeutics) – Emendo Biotherapeutics is pioneering OMNI, a next-generation allele-specific gene editing platform that utilizing synthetic biology. In 2019, Emendo granted an option to Takeda to use the OMNI nuclease gene editing program for two research and development targets.
ASC Therapeutics and Vigene Announce Long-Term Strategic Partnership for Gene Therapy Development and Manufacturing
January 14, 2020 – (ASC Therapeutics) – Vigene will provide ASC with access to GMP manufacturing including viral vectors and plasmid DNA for its hemophilia A gene therapy clinical program, as well as a manufacturing platform for future gene therapy programs.
Astellas and Adaptimmune Enter Into Agreement to Co-Develop and Commercialize Stem Cell Derived Allogeneic CAR-T and TCR T-Cell Therapies
January 14, 2020 – (Adaptimmune Therapeutics) – Under the terms of the agreement, Adaptimmune may receive up to $897.5M in payments, including a $50M upfront payment.

Horizon Discovery to Provide Access to Novel Base Editing Technology
January 14, 2020 – (Horizon Discovery) – Horizon Discovery Group announced that it will provide access to a novel base editing technology licensed from Rutgers University for exclusive use in therapeutic, diagnostic and services applications. This technology is incorporated into Horizon’s next-generation gene editing platform to enable the development of novel therapeutics.
Actinium Pharmaceuticals Announces Iomab-ACT Program Gene Therapy Collaboration with UC Davis in Ongoing Clinical Trial for Patients with HIV-Related Lymphoma
January 13, 2020 – (Actinium Pharmacueticals) – In the trial, patients with relapsed or refractory HIV-related lymphoma are being treated with autologous stem cell gene therapy. This is the first gene therapy clinical trial that will utilize ARC based conditioning.
Allogene Therapeutics and SpringWorks Therapeutics Announce Clinical Collaboration to Evaluate ALLO-715 in Combination with Nirogacestat in Multiple Myeloma
January 13, 2020 – (SpringWorks Therapeutics) – Under the terms of the agreement, Allogene will sponsor and conduct the Phase 1 study to evaluate the safety, tolerability and preliminary efficacy of the combination, and will assume all development costs associated with the study. Allogene and SpringWorks will form a joint development committee to oversee the clinical study, which is expected to commence in the second half of 2020 pending discussions with regulators.
DiNAQOR AG Announces Research Collaboration and License Agreement with UCL to Investigate Gene Therapies for Heart Failure
January 13, 2020 – (University College London) – Under the terms of the agreement, DiNAQOR and UCL will collaborate to advance DiNAQOR’s two discovery cardiac gene therapy programs into clinical development. DiNAQOR will obtain exclusive worldwide rights to any therapies developed as part of the collaboration. If a product is successfully commercialized from the collaboration, UCL will be entitled to royalties on sales.
Editas Medicine And Sandhill Therapeutics, Inc. Announce Collaboration To Develop Engineered Cell Medicines To Treat Cancer
January 13, 2020 – (Editas Medicine) – Under the terms of the agreement, Editas Medicine obtains an exclusive license to Sandhill’s technology to research, develop and commercialize immuno-oncology engineered cell medicines for solid tumors originating within a given area of the body and an option to expand such license to two additional areas. In return, Sandhill will receive an upfront payment, development and sales-based milestone payments, and royalties on sales of resulting Editas products.
Helix Biopharma Corp. Closes $3M Private Placement
January 13, 2020 – (Helix BioPharma Corp) – The company intends to use the net proceeds of the private placement for working capital and research and development activities
Kyverna Therapeutics Secures $25M Series A Funding From Key Investors and Enters into Strategic Collaboration with Gilead Sciences
January 13, 2020 – (Kyverna Therapeutics) – Proceeds from the financing will be used to advance Kyverna's therapeutic strategy, which combines advanced T cell engineering and synthetic biology technologies to suppress and eliminate autoreactive immune cells at the root cause of inflammatory disease.
Muscular Dystrophy Collaboration Aims to Correct Muscle Stem Cells’ DNA
January 13, 2020 – (Harvard University) – Supported by research funding from Sarepta Therapeutics, under a multi-year collaboration agreement coordinated by Harvard’s Office of Technology Development, the project aims to use in-vivo genome editing, in mouse models of DMD, to fully and precisely restore the function of the dystrophin protein.
Nationwide Children’s Hospital Launching a Gene Therapy Manufacturing Company
January 13, 2020 – (Columbus Dispatch) – Nationwide Children’s Hospital in Columbus is creating Andelyn Biosciences, a for-profit subsidiary company, which will create gene therapy products. Andelyn will launch this summer and operate out of the Abigail Wexner Research Institute, which is a leader in developing gene therapies.
Iovance Biotherapeutics and Cellectis Enter Into a Research Collaboration and Exclusive Worldwide License Agreement
January 12, 2020 – (Iovance Biotherapeutics) – Iovance Biotherapeutics and Cellectis announced that the companies have entered into a research collaboration and exclusive worldwide license agreement whereby Iovance will license certain TALEN technology from Cellectis in order to develop tumor infiltrating lymphocytes that have been genetically edited to create more potent cancer therapeutics.
Generation Bio Announces $110M Series C Financing to Advance Lead Programs for Hemophilia A and PKU
January 10, 2020 – (Generation Bio) – Proceeds will be used to advance the company’s two lead liver-targeted programs for hemophilia A and phenylketonuria into IND-enabling studies and clinical development.
Indapta Therapeutics and Lonza Announce Strategic Partnership to Advance a Next-generation, Off-the-shelf, Allogeneic Immuno-oncology Therapy
January 10, 2020 – (Lonza) – Indapta Therapeutics is currently completing IND-enabling studies and plans to submit an IND application in late 2020 and initiate a first-in-human Phase 1/2 study in early 2021.
LogicBio Therapeutics Announces Collaboration With Takeda to Develop New Genome Editing Candidate LB-301 for the Treatment of Crigler-Najjar Syndrome
January 10, 2020 – (LogicBio) – Under the agreement, LogicBio and Takeda will collaborate to further research and develop LB-301. Takeda will provide funding for the research program under the collaboration agreement and will have an exclusive option to negotiate an exclusive, worldwide license to LogicBio’s LB-301 program. 
Novartis Opens Its Largest Gene Therapy Plant to Support Zolgensma
January 10, 2020 – (BioProcess International) – The facility currently employs more than 300 talented scientists, engineers, analysts and manufacturing and operations personnel. While the plant is open, it will not become operational until early next year.
PACT Pharma Raises $75M in Oversubscribed Series C Financing to Develop Fully Personalized NeoTCR-T Cell Therapies for Patients with Cancer
January 10, 2020 – (PACT Pharma) – PACT will use the Series C proceeds to expand the scope of its clinical plan to investigate NeoTCR-T cell products targeting multiple neoantigens for a spectrum of solid tumor types. In addition to clinical expansion, PACT will open in 2020 a next-gen GMP manufacturing facility in South San Francisco.
Jasper Therapeutics Announces Expansion of Series A Financing, Bringing Total Corporate Fundraising to $50M+
January 9, 2020 – (Jasper Therapeutics) – Jasper plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191, designed to replace toxic chemotherapy and radiation therapy as conditioning regimens to prepare patients for curative stem cell and gene therapy.
First Patient Dosed With Cellectis’ New Allogeneic UCART123 Product Candidate for Relapsed/Refractory Acute Myeloid Leukemia
January 15, 2020 – (Cellectis) – Cellectis announced the first patient dosing in AMELI-01, the Phase 1 dose escalation clinical trial evaluating a new UCART123 product candidate in relapsed/refractory acute myeloid leukemia.
GeneTx and Ultragenyx Announce IND Application Active for GTX-102 in Patients With Angelman Syndrome
January 15, 2020 – (Ultragenyx) – GeneTx and Ultragenyx announced that GeneTx’s IND application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome, filed with the FDA is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020.
Iovance Biotherapeutics Completes Patient Dosing in Registration-Enabling Cohort 4 of the C-144-01 Melanoma Study with Lifileucel
January 15, 2020 – (Iovance Biotherapeutics) – Iovance remains on track to submit a BLA subsequent to discussions with FDA, for regulatory approval of lifileucel in late 2020.
Tmunity Announces First Patient Dosed in Phase 1 Clinical Trial with CART-TnMUC1
January 15, 2020 – (Tmunity Therapeutics) – Tmunity announced that it has dosed the first patient in its Phase 1 CART-TnMUC1-01 clinical trial with the Tn/STn glycoform of mucin 1 CAR-T therapy in patients with TnMUC1-positive advanced cancers. This is a first-in-human trial with a CAR-T therapy targeting this antigen.
AVROBIO Announces Clinical and Regulatory Progress for Gaucher Disease Program
January 14, 2020 – (AVROBIO) – AVROBIO announced that it has received IND clearance from the FDA for AVR-RD-02, its investigational gene therapy for the treatment of Gaucher disease.
NantKwest and ImmunityBio Announce Complete Response in Metastatic Pancreatic Cancer in the First Patient to Receive PD-L1 Tumor-Targeted NK Cells with N-803 IL-15 Fusion Protein
January 14, 2020 – (NantKwest) – After five infusions of PD-L1.t-haNK and N-803, the patient’s tumor metastasis resolved completely per CT/Pet scan and the complete response has been confirmed by a repeat CT/Pet scan. The patient continues to receive ongoing treatment.
UK Regulatory Authority Approves Cynata’s Phase 2 Clinical Trial in Critical Limb Ischaemia
January 14, 2020 – (Cynata Therapeutics) – Cynata Therapeutics announced that it has received approval from the UK Medicines and Healthcare products Regulatory Agency to proceed with its Phase 2 clinical trial of the Cymerus mesenchymal stem cell product CYP-002 in patients with critical limb ischemia.
Abeona Therapeutics Initiates Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa
January 13, 2020 – (Abeona Therapeutics) – The majority of patients targeted for enrollment have completed the pre-screening process at Stanford, and the company expects the first patient in the VIITAL study to be treated in the first quarter of 2020.
Adaptimmune Announces that SPEAR T-cell Platform Delivers Initial Responses in Four Solid Tumor Indications
January 13, 2020 – (Adaptimmune Therapeutics) –Adaptimmune reported two confirmed Partial Responses (PRs) – one in a patient with liver cancer and one in a patient with melanoma. There are also two unconfirmed PRs – one in a patient with gastro‑esophageal junction cancer and one in a patient with head and neck cancer.
Axovant Announces Positive 12-Month Data on AXO-Lenti-PD and Provides Updates Across Gene Therapy Pipeline Programs
January 13, 2020 – (Axovant) – At 12 months post-dosing, patients in the first dose cohort demonstrated an average 22-point change from baseline in motor function as assessed by the UPDRS Part III “OFF” score, which represents a 37% improvement.
BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy
January 13, 2020 – (BioMarin) – BioMarin announced today that the US FDA and the MHRA in the UK have granted the company IND status and approved its CTA respectively for its investigational gene therapy candidate BMN 307. The company expects to start dosing patients in a Phase 1/2 study in the first quarter of 2020.
bluebird bio Announces Launch in Germany of ZYNTEGLO Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β⁰/β0 Genotype
January 13, 2020 – (bluebird bio) – bluebird bio has established a collaboration with University Hospital of Heidelberg as the first qualified treatment center in Germany. In addition, bluebird has entered into value-based payment agreements with multiple statutory health insurances in Germany. This is the first time ZYNTEGLO is commercially available.
Precision BioSciences Announces FDA Acceptance of IND for PBCAR269A, a BCMA Targeted Genome Edited Allogeneic CAR T Therapy Candidate for Multiple Myeloma
January 13, 2020 – (Precision BioSciences) – The FDA has also granted Orphan Drug Designation to PBCAR269A for the treatment of multiple myeloma. Precision plans to initiate dosing in this Phase 1 clinical trial in 2020.
Celyad Successfully Doses First Patient with CYAD-02 in CYCLE-1 Trial for r/r AML and MDS
January 12, 2020 – (Celyad) – Preliminary data from the Phase 1 CYCLE-1 trial are expected during second half 2020.
PTC Therapeutics Submits Marketing Authorization Application to EMA for PTC-AADC Gene Therapy to Treat AADC Deficiency
January 13, 2020 – (PTC Therapeutics) – PTC expects the Committee for Medicinal Products for Human Use (CHMP) opinion in 2H 2020. The company plans to submit a BLA in the US in Q2 2020.
Adverum Biotechnologies Reports Additional Clinical Data from First Cohort of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD
January 11, 2020 – (Adverum Biotechnologies) – Additional data for the first cohort included zero rescue injections for any patient. Vision was maintained as demonstrated by stable mean Best Corrected Visual Acuity compared to baseline.
LogicBio Therapeutics Announces Submission of IND for LB-001 for Methylmalonic Acidemia (MMA) and Highlights Key Corporate Milestones
January 10, 2020 – (LogicBio) – LogicBio plans to initiate a Phase 1/2 trial in pediatric MMA patients in the first half of 2020, with preliminary data expected in the second half of 2020.
UC Davis Health to Utilize Orgenesis Point of Care Platform for the Development, Commercialization and Supply of Cell and Gene Therapy Products
January 10, 2020 – (Orgenesis) – Under the collaboration agreement, UC Davis Health will utilize Orgenesis’ POCare platform to develop, commercialize and supply cell and gene products and therapies.
Positive Final 2-Year Topline Data in Completed DC Vaccine Phase 1/2 Trial in AML Patients
January 10, 2020 – (Medigene) – The secondary outcome measures evaluating key clinical parameters show that after 24 months of treatment, overall survival rate was 80% and progression free survival rate was 55%.
Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase Deficiency
January 9, 2020 – (Ultragenyx) – In Cohort 3, there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status. Across all nine patients dosed in the study, up to six patients have demonstrated a response.
Locus Biosciences Initiates World’s First Controlled Clinical Trial for a CRISPR Enhanced Bacteriophage Therapy
January 8, 2020 – (Locus Biosciences) – Locus is enrolling patients for this trial across several clinical sites in the United States. The company received clearance from the FDA for its IND application in Q4 2019 and began activating its study sites shortly thereafter.
The Kill-Switch for CRISPR That Could Make Gene-Editing Safer
January 15, 2020 – (Nature) – More than 50 anti-CRISPR proteins have now been characterized, each with its own means of blocking the cut-and-paste action of CRISPR systems. Some are using these proteins as switches to more finely control the activity of CRISPR systems in gene-editing applications for biotechnology or medicine.
Rejuvenating the Brain: More Stem Cells Improve Learning and Memory of Old Mice
January 9, 2020 – (TU Dresden) – Researchers developed a method to stimulate the small pool of neural stem cells that reside in the brain in order to increase their number and, as a result, to also increase the number of neurons generated by those stem cells. Surprisingly, additional neurons could survive and form new contacts with neighbouring cells in the brain of old mice.
UK Accounts for Over 12% of Global Cell and Gene Therapy Clinical Trials
January 15, 2020 – (CGT Catapult) – The UK and the NHS are providing the right environment and infrastructure to allow innovative cell and gene therapies to reach patients. International companies recognise the appeal of the UK cell and gene therapy ecosystem and are sponsoring the majority of the UK commercial clinical trials, 77% of the total 127 ongoing trials.
FDA and NIH Let Clinical Trial Sponsors Keep Results Secret and Break the Law
January 13, 2020 – (Science) – In 2017, NIH and FDA enacted a long-awaited “final rule” to clarify expectations and penalties for failing to disclose trial results. The rule took full effect two years ago, giving trial sponsors ample time to comply. But a Science investigation shows that many still ignore the requirement, while federal officials do little or nothing to enforce the law.
Gene Therapy: Keeping Costs from Negating Its Unprecedented Potential
January 13, 2020 – (CVS Healthcare) – CVS Healthcare identifies four complementary approaches in the white paper that health plans and employers can utilize to reduce the cost impact of gene therapies: extending the National Medical Excellence Program model, evolving the role of specialty pharmacy, value-based contracting, and financial protection programs.
Gene Therapies Test Europe’s Willingness to Pay
January 15, 2020 – (Financial Times) – The drive to cure deadly diseases is about to enter a new and demanding phase as the pharmaceutical industry prepares for the first time to test the appetite for hyper-expensive gene therapies in Europe.
At 16, She’s a Pioneer in the Fight to Cure Sickle Cell Disease
January 11, 2020 – (New York Times) – Helen Obando is the youngest person ever to get a gene therapy that scientists hope will cure the disease, which afflicts 100,000 Americans.
Director / VP, Finance and Operations
Alliance for Regenerative Medicine – Washington, DC
Marketing and Communications Manager
International Society for Cell & Gene Therapy – Vancouver, Canada
About ARM

The Alliance for Regenerative Medicine (ARM) is the preeminent global advocate for regenerative and advanced therapies. ARM fosters research, development, investment and commercialization of transformational treatments and cures for patients worldwide. By leveraging the expertise of its membership, ARM empowers multiple stakeholders 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. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit
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