ARM Sector Newsletter
The latest news in gene therapy, cell therapy, and tissue engineering
January 2, 2020
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Intrexon Appoints Helen Sabzevari, PhD, As New President And CEO And Will Change Name To Precigen To Reflect Healthcare Focus
January 2, 2020 – (Intrexon) – The new Precigen will encompass Intrexon's wholly-owned healthcare subsidiaries Precigen, ActoBio Therapeutics, Exemplar Genetics, and its majority ownership interest in Triple-Gene. Additionally, Intrexon has entered into an agreement to sell $35M of its common stock.
Sumitomo Dainippon Pharma Completes the Formation of the Strategic Alliance with Roivant Sciences
December 28, 2019 – (Sumitomo Dainippon Pharma) – As consideration for the Strategic Alliance, Sumitomo Dainippon Pharma has paid total of approximately $3B (approximately ¥330B) to Roivant.
Astellas Strengthens Immuno-Oncology Pipeline With Acquisition of Xyphos Biosciences
December 26, 2019 – (Astellas Pharma) – Astellas paid $120M upon closing, with potential future development milestone payments to increase the value to $665M. Xyphos’ first CAR-T cell product candidate is in preclinical development and scheduled to be tested in a first-in-human clinical study in 2021.
Abeona Announces Closing of $103.5M Underwritten Public Offering and Full Exercise of Underwriters’ Option to Purchase Additional Shares
December 23, 2019 – (Abeona Therapeutics) – The offering provides cash runway into 2021 and resources to fund continued clinical development of pipeline products, including the initiation and enrollment of the EB-101 pivotal Phase 3 VIITAL study.
Capricor Therapeutics Announces Closing of $5.1M Offering Priced At-the-Market
December 19, 2019 – (Capricor Therapeutics) – Capricor currently intends to use the net proceeds from the offering to fund research and development of its exosome technologies and for general corporate purposes.
Gamida Cell Announces Completion of Patient Enrollment in Ongoing Phase 3 Clinical Study of Omidubicel
January 2, 2020 – (Gamida Cell) – Gamida Cell announced that it has completed patient enrollment in its Phase 3 study of the company’s lead clinical program, omidubicel. Topline data from the study are expected in the first half of 2020.
Kadimastem Announces Promising Results of Cohort A of its Phase 1/2a Clinical Trial in ALS
January 2, 2020 – (Kadimastem) – The findings indicate that for a period of 3-4 months following treatment the progression of the disease was reduced, providing a potentially meaningful clinical benefit to the patients. In the 5- and 6-months post-treatment periods, a statistically significant difference was not observed between the rate of disease deterioration during these periods and the rate during the pre-treatment period.
MSB Files GvHD Clinical Efficacy and Safety Data with US FDA
January 2, 2020 – (Mesoblast) – The final module will be filed during January, and Mesoblast will request an expedited FDA review of the BLA under the product candidate’s existing Fast Track designation. If approved, remestemcel-L is planned to be launched in the US in 2020.
AVITA Medical Receives U.S. FDA Investigational Device Exemption Approval of Clinical Feasibility Study to Evaluate RECELL System for Vitiligo
December 30, 2019 – (AVITA Medical) – AVITA Medical announced that the FDA has approved the company’s Investigational Device Exemption application to conduct a feasibility study evaluating the safety and effectiveness of the RECELL Autologous Cell Harvesting Device for repigmentation of depigmented lesions associated with stable vitiligo.
Lineage Provides Update on Patient Enrollment in Phase 1/2a Clinical Study of OpRegen for the Treatment of Dry Age-Related Macular Degeneration
December 30, 2019 – (Lineage Cell Therapeutics) – The first Cohort 4 patient treated using both a new subretinal delivery system and new thaw-and-inject formulation of OpRegen has continued to demonstrate notable improvements in vision.
Prevail Therapeutics Announces IND Active for PR001 for Treatment of Neuronopathic Gaucher Disease
December 26, 2019 – (Prevail Therapeutics) – As previously reported, Prevail’s IND for PR001 for the treatment of nGD had been put on clinical hold by the FDA, and this clinical hold has now been removed. The trial is expected to be initiated in 1H 2020.
BioMarin Submits Biologics License Application to FDA for Valoctocogene Roxaparvovec to Treat Hemophilia A
December 23, 2019 – (BioMarin) – Subject to completion of the FDA's filing review, BioMarin anticipates the BLA review to commence in February 2020. BioMarin will provide an update in February 2020. BioMarin also announced that the EMA has validated the MAA for valoctocogene roxaparvovec in Europe.
Blood Cells That Are Invisible to the Immune System
December 27, 2019 – (Center for iPS Cell Research and Application) – Researchers have recently reported a way to manufacture the billions of platelets needed for a transfusion from iPS cells, removing the need of donors and enabling the production of platelets on demand. In their latest study, they show how gene editing can modify their manufactured platelets to eliminate the need for immune matching.
China Convicts 3 Researchers Involved in Gene-Edited Babies
December 30, 2019 – (AP News) – He Jiankui, who announced he had made the world's first germline genome edits to the embryos of twins born in November 2018, was sentenced to three years in prison and fined fined 3 million yuan ($430,000).
ARM Announces 2020 Cell & Gene Therapies State of the Industry Briefing
ARM will be hosting the 10th Annual Cell & Gene Therapies State of the Industry Briefing on Monday, January 13, at 8:00am PT. This event – which is free and open to the public – will include ARM’s annual yearly update on the sector’s progress, as well as two panels featuring top CEOs and executives highlighting future inflection points for cell and gene therapies in 2020. For those who are unable to attend in-person, a livestream will be available here.

In the meantime, check out some other recaps and predictions for the regenerative medicine sector here:
Director, Clinical & Regulatory Affairs
DiscGenics – Salt Lake City, UT
Translational Research Director
Project 8 – Remote
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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