Emerging Therapy Solutions

Cell Therapy News! Mesoblast Limited has announced that the US Food and Drug Administration (FDA) has approved Ryoncil (remestemcel-L-rknd), an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) for pediatric patients with steroid-refractory acute graft-versus-host-disease (SR-aGvHD). Emerging Therapy Solutions (ETS) helps payers and other risk-bearing entities prepare for and manage risk related to the pipeline of upcoming cell and gene therapies. Reach out to ETS to learn more at 877.445.4822. https://bit.ly/49JOz81

National Marrow Awareness Month is observed every November to honor those who donate marrow for stem cell / bone marrow transplantation (BMT) and to thank the physicians who perform these lifesaving treatments. Join us in raising awareness about the importance of bone marrow transplants and consider becoming a donor. Emerging Therapy Solutions (ETS) has negotiated rates with top hospitals around the country that offer BMT / stem cell transplants. View our Adult and Pediatric Transplant Program Guides at https://bit.ly/4g9f3SB for a list of these facilities. https://bit.ly/4g1SaAn

Gene Therapy News! PTC Therapeutics has announced that the US Food and Drug Administration (FDA) has approved Kebilidi™ (eladocagene exuparvovec-tneq; PTC-AADC), a gene therapy for the treatment of children and adult patients with aromatic L–amino acid decarboxylase (AADC) deficiency, including the full spectrum of disease severity. According to the announcement, this is the first-ever gene therapy approved in the United States that is directly administered to the brain. The therapy received accelerated approval based on safety and clinical efficacy findings in the ongoing PTC-AADC-GT-002 global clinical trial, with confirmatory data coming from long-term follow-up of patients already previously treated. Emerging Therapy Solutions (ETS) helps payers and other risk-bearing entities prepare for and manage risk related to the pipeline of upcoming cell and gene therapies. Reach out to ETS to learn more at 877.445.4822. https://bit.ly/3AGSCF5

ETS is excited to attend the 18th Annual Personalized Medicine Conference from November 12-14 in Boston! According to the event website, this conference will look at the importance of ongoing investment in research and development programs to advance personalized medicine, and will explore strategies for widening access to the related tests and treatments. We look forward to engaging in this interesting forum and connecting with individuals from multiple sectors of the health care system including academic researchers, investors, business leaders, and health system administrators. Dr. Julie England, ETS Chief Medical Officer will be at this year’s conference. If you're attending the conference and would like to schedule a meeting with Dr. England, please email [email protected]. Hope to see you there! 👋 #PMC2024 #PersonalizedMedicine https://bit.ly/3YOxHYC

This week, Abeona Therapeutics announced that the US Food and Drug Administration (FDA) has accepted its Biologics License Application (#BLA) resubmission for Pz-cel (prademagene zamikeracel), its investigational #genetherapy for recessive dystrophic epidermolysis bullosa (RDEB). The BLA resubmission is supported by clinical efficacy and safety data after a one-time administration of pz-cel. The FDA has set a Prescription Drug User Fee Act (#PDUFA) target action date of April 29, 2025. Pz-cel was previously accepted for Priority Review by the FDA and may be eligible for a Priority Review Voucher should the therapy be approved. Emerging Therapy Solutions (ETS) helps payers and other risk-bearing entities prepare for and manage risk related to the pipeline of upcoming cell and gene therapies. Reach out to ETS to learn more at 877.445.4822 or visit us at https://bit.ly/48MXuFm. https://bit.ly/3Z1BUcX

Cell Therapy News! Autolus Therapeutics has announced that the US Food and Drug Administration (FDA) has approved Aucatzyl® (obecabtagene autoleucel; obe-cel), a chimeric antigen receptor (CAR) T-cell therapy for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). Approval was based on results from the FELIX clinical trial of obe-cel in adults with r/r B-ALL. Emerging Therapy Solutions (ETS) helps payers and other risk-bearing entities prepare for and manage risk related to the pipeline of upcoming cell and gene therapies. Reach out to ETS to learn more at 877.445.4822 or visit us at https://bit.ly/3CjksYq. https://bit.ly/48LMU1o

ETS is looking forward to the Precision Health Information Network Summit next week in Nashville! This event brings together committed leaders across the healthcare industry to discuss the data, digital infrastructure, and economic challenges facing precision medicine. Hope to see you there! 👋 https://bit.ly/48f5s9P

Discover the many benefits of being an Emerging Therapy Solutions (ETS) client—like access to our expert-led educational webinars! These ETS-hosted webinars feature leading physicians discussing a range of topics related to cell and gene therapy, transplants, rare conditions, and more. As a valued ETS client, you can gain 24/7 access to all past webinars through our Knowledge Center. Interested in accessing this valuable resource? Contact us at [email protected] or visit https://bit.ly/488m3MP to learn more.

Staff spotlight! 📣 Kelsey Brandner, RN, BSN, OCN, is a Senior Clinical Specialist at Emerging Therapy Solutions (ETS). "What I like most about my job is helping our clients and facility partners with their variety of needs so that their patients can access great care. The nerd in me also loves learning about all of the advances in medicine that allow for treatment of rare conditions and cancers." We are so fortunate to have Kelsey on the ETS team!

September is #BloodCancerAwarenessMonth, a month dedicated to raising awareness about the types of blood cancer and the signs and symptoms. Types of blood cancer include leukemias, lymphomas, myelomas, myelodysplastic syndromes, and myeloproliferative neoplasms. The US Food and Drug Administration (FDA) has approved a number of #celltherapies for treating certain blood cancers, and there are numerous clinical trials evaluating investigational therapies as well. For a list of FDA-approved cell therapies for blood cancers, visit the ETS Approved Therapies resource at https://bit.ly/47QIsxR