ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
May 7, 2020
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ARM Announces Next Two Webinars in Series, Focusing on Biotech Fundraising During the Pandemic and Cell Therapies for COVID-19
May 7, 2020 – (ARM) – Tomorrow at 11am ET, speakers from Chardan Capital Markets, Sofinnova Investments, and Castle Creek Biosciences will discuss "What Biotechs Need to Know About Fundraising in the Midst of COVID-19." Topics include the current funding landscape, what investors are looking for when evaluating new and follow-on opportunities, and how companies can best prepare to fundraise in the midst of the pandemic. In addition, speakers from Athersys and Pluristem will join ARM for a webinar on "Cell Therapies for COVID-19" on Thursday, May 21. For more information, click here.
First Patients Dosed in Phase 2/3 Randomized Controlled Trial of Mesoblast’s Remestemcel-L for COVID-19 ARDS
May 6, 2020 – (Mesoblast) – The trial will randomize up to 300 ventilator-dependent patients in intensive care units to either remestemcel-L or placebo on top of maximal care. The trial will include up to 30 sites across North America.
Athersys Announces Commencement of Patient Enrollment in the MACOVIA Study, a Pivotal Phase 2/3 Trial Evaluating MultiStem Cell Therapy for COVID-19 Induced ARDS
May 5, 2020 – (Athersys) – The company’s program evaluating administration of MultiStem for the treatment of ARDS was recently granted Fast Track designation by the FDA based on the promising Phase 1/2 data from its previously completed MUST-ARDS trial.
Lineage Cell Therapeutics to Apply Allogeneic Dendritic Cell Therapy Program to COVID-19 Vaccine Development
May 5, 2020 – (Lineage Cell Therapeutics) – Lineage Cell Therapeutics announced that it has applied for grant funding from the California Institute for Regenerative Medicine to support the use of VAC, Lineage’s allogeneic dendritic cell therapy, toward the development of a potential vaccine against SARS-CoV-2, the virus which causes COVID-19. 
OPINION: A Pandemic Moves Peer Review to Twitter
May 5, 2020 – (Bloomberg) – This is the future for scientific communication that has been predicted since the spread of the internet began to enable it in the early 1990s, yet proved slow and fitful in its arrival. It involves more or less open access to scientific research and data, and a more-open review process with a much wider range of potential peers than the peer review offered by journals.
OPINION: How Biopharma Companies Can Keep ‘Feeding the Beast’ in the Time of Coronavirus
May 5, 2020 – (STAT News) – The capital needs of biopharma companies are unrelenting, even in the best of times. How much is needed? How should it be efficiently managed? How and when should we raise more?
A Coronavirus Vaccine Project Takes a Page From Gene Therapy
May 4, 2020 – (New York Times) – The vaccine employs a method already used in gene therapy: it uses a harmless virus as a vector, or carrier, to bring DNA into the patient’s cells. In this case, the DNA should instruct the cells to make a coronavirus protein that would stimulate the immune system to fight off future infections.
Bone Therapeutics Raises Additional €4M, With Total of €15M Committed
May 7, 2020 – (Bone Therapeutics) – Bone Therapeutics announced that it has received €4M as a result of issuing, to existing investors, subordinated bonds with the option to convert. 
Caribou Biosciences and MaxCyte Enter Into Clinical and Commercial License Agreement
May 7, 2020 – (MaxCyte) – Caribou will obtain non-exclusive clinical and commercial rights to use MaxCyte’s platform to develop CRISPR gene-edited allogeneic T cell therapies. In return, MaxCyte will receive undisclosed development and approval milestones and sales-based payments in addition to other licensing fees.
Lineage Cell Therapeutics Announces Early Exercise of Option With Cancer Research UK for Immuno-Oncology Cell Therapy Program
May 7, 2020 – (Lineage Cell Therapeutics) – Lineage Cell Therapeutics announced that Cancer Research UK’s Commercial Partnerships has permitted Lineage to conduct an early exercise of its option to acquire data from Cancer Research UK’s ongoing Phase 1 clinical trial of VAC2 in non-small cell lung cancer.
Passage Bio Announces Expansion of Gene Therapy Collaboration With University of Pennsylvania
May 7, 2020 – (Passage Bio) – Under the expanded agreement, Passage will pay $5M annually to Penn to fund research across numerous technology applications for gene therapy. In addition to five additional program options and an extension of the relationship through 2025, Passage will receive exclusive rights to IP arising from this research and related indications.
Avectas and ONK Therapeutics to Develop Off-the-Shelf Cancer Therapy
May 6, 2020 – (Avectas) – Under the collaboration, ONK will be responsible for rapidly progressing the potential therapy to a Phase 1 clinical trial. ONK will receive upfront fees, development and commercial milestones, and royalties. Avectas has also, through a holding company, acquired a minority shareholding in ONK.
Magenta Therapeutics and AVROBIO Announce Collaboration to Evaluate Targeted Antibody-Drug Conjugate as a Potential Conditioning Regimen for Lentiviral Gene Therapies
May 6, 2020 – (Magenta Therapeutics) – Under the collaboration, Magenta and AVROBIO will jointly evaluate MGTA-117 in conjunction with one or more of AVROBIO’s investigational gene therapies. Magenta will retain all commercial rights to MGTA-117. AVROBIO will retain all commercial rights to its gene therapies and will be responsible for the clinical trial costs related to the evaluation of MGTA-117 with AVROBIO’s gene therapies.
Santhera Signs Agreements in Gene Therapy Research for Congenital Muscular Dystrophy With Rutgers University
May 6, 2020 – (Santhera) – Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy. Under the agreements, Santhera gains rights to intellectual property developed at Rutgers on certain gene constructs that will be further studied under a collaboration agreement.
Genprex Enters Into Exclusive Worldwide Patent and Technology License Agreement for Combination of Its TUSC2 Gene Therapy With Immunotherapies
May 5, 2020 – (Genprex) – The license agreement provides for payment to MD Anderson of an up-front license fee and annual maintenance fees, with the potential for milestone payments, sublicensing fees, and product royalties.
Pluristem Announces Pricing of Its Registered Direct Offering for Aggregate Proceeds of $15M
May 5, 2020 – (Pluristem) – Pluristem intends to use the net proceeds from the offering for working capital, including funding towards its Phase 2 study of PLX cell therapy in the treatment of complications arising from COVID-19 and other clinical trial activities, investment in capital equipment and other general corporate purposes.
Harvard’s Wyss Institute Joins Forces With Cytosurge to Improve CRISPR-Based Multiplexed Gene Editing
May 4, 2020 – (Wyss Institute) – The Wyss Institute for Biologically Inspired Engineering at Harvard University and Cytosurge AG announced that they will collaboratively investigate CRISPR-based approaches to more effectively introduce multiple edits into the genome of single cells, while minimizing CRISPR-related toxicity.
Legend Biotech and Noile-Immune Biotech Announce Collaborative Research and Licensing Agreement
May 4, 2020 – (Legend Biotech) – Under the multi-year collaboration agreement, Legend and Noile-Immune will work together on up to two select cancer targets. Legend will gain the right to incorporate the PRIME technology into its CAR-T and TCR-T cell programs and will provide to Noile-Immune a total of up to $70M per selected target for certain development, regulatory, and commercial milestone payments.
LifeArc and MRC Partnership Creates New £16M Fund to Establish Gene Therapy Innovation Hubs
May 4, 2020 – (LifeArc) – The Medical Research Council and independent medical research charity LifeArc are making £16M available to establish a network of centers that will offer clinical grade viral vectors, and translational and regulatory guidance to support academic-led patient trials of new gene therapies.
BioMarin Enters Into in a Preclinical Collaboration and License Agreement With DiNAQOR to Develop Gene Therapies for Rare Genetic Cardiomyopathies
May 3, 2020 – (BioMarin) – BioMarin announced that the company has entered into a preclinical collaboration and license agreement with DiNAQOR to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR will receive an undisclosed upfront payment and is eligible to receive development, regulatory, and commercial milestones on product sales in addition to tiered royalties on worldwide sales.
NJII Launches Biocentriq Cell & Gene Therapy Manufacturing Center
May 3, 2020 – (NJII) – BioCentriq is the only process development and clinical manufacturing facility for cell and gene therapies located in Newark, NJ, and the first of its kind to be located on a university campus in the United States.
Rubius Therapeutics Announces Dosing of First Patient in Phase 1/2 Trial of RTX-240 for the Treatment of Solid Tumors
May 7, 2020 – (Rubius Therapeutics) – RTX-240 is an allogeneic, off-the-shelf red cell therapy that is engineered to mimic the human immune system by stimulating adaptive and innate immunity to generate an anti-tumor immune response.
Cabaletta Bio Receives FDA Fast Track Designation for DSG3-CAART for the Treatment of Mucosal Pemphigus Vulgaris
May 6, 2020 – (Cabaletta Bio) – The company plans to initiate its Phase 1 DesCAARTes trial to evaluate the safety and tolerability of DSG3-CAART in relapsed and/or refractory patients in 2020. DSG3-CAART is based on technology licensed from and has been developed in collaboration with the University of Pennsylvania.
Immunicum Receives RMAT Designation from FDA for Ilixadencel in Kidney Cancer
May 6, 2020 – (Immunicum) – The latest results of the Phase 2 MERECA trial showed an ORR for the ilixadencel treatment group of 42.2% (19/45), versus 24.0% (6/25) for the sunitinib control group.
Kiadis Pharma Announces FDA Approval to Start NK-Realm Phase 2 Clinical Trial for Natural Killer Cell Therapy Produced With PM21
May 6, 2020 – (Kiadis Pharma) – The first 6 patients in the NK-REALM study will be evaluated as a safety lead-in. The study is designed to confirm earlier proof-of-concept data in 24 patients, which showed that adjunctive treatment with K-NK002 has the potential to substantially improve outcomes for patients in need of a HSCT.
Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy
May 6, 2020 – (Lineage Cell Therapeutics) – Some OpRegen-treated patients are able to see better, have less growth in their area of GA, and are able to read faster, all of which represent significant enhancements to vision and quality of life metrics. In addition to these individual results, the pooled data continues to suggest a treatment effect in both visual acuity and GA progression.
Adverum Biotechnologies Reports Positive Interim Data from Cohorts 1-3 of OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD
May 4, 2020 – (Adverum Biotechnologies) – ADVM-022 continues to be well tolerated with a favorable safety profile in all three cohorts. The therapy continues to show robust efficacy, with 6/6 patients in the high dose cohort rescue injection free, and 8/11 patients in the lower dose cohorts rescue injection free.
Freeline Receives Orphan Drug Designation From the FDA for FLT190 for the Treatment of Fabry Disease
May 4, 2020 – (Freeline Therapeutics) – Freeline recently announced it had received Orphan Drug Designation for FLT190 from the European Commission, based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency.
New Gene Therapy for Color Blindness Tested in Patients
April 30, 2020 – (Ludwig Maximilian University of Munich) – Researchers report that the therapy was safe, and that there was also a clear positive effect in terms of efficacy. The patients' visual function improved somewhat, both in terms of focus and in relation to contrast and color vision.
Focused Ultrasound Opening Brain to Previously Impossible Treatments
May 6, 2020 – (University of Virginia) – University of Virginia researchers are pioneering the use of focused ultrasound to defy the brain's protective barrier so that doctors could deliver many treatments, including gene therapies, directly into the brain to battle neurological diseases.
Novel Technology Aims to Improve Treatment of Neurological Diseases
April 16, 2020 – (Princeton University) – The research team's engineered promoters occupy far less space than existing promoters used in gene therapy, allowing the transport of larger genes or multiple genes.
Executive Director
ARM Foundation for Cell and Gene Medicine – Remote
Business Manager (US)
Ovizio Imaging Systems – US East Coast
Senior Application Scientist – Cell Therapy
Treefrog – Pessac, France
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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