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vice president of neuroscience at Voyager Therapeutics

The vice president of neuroscience at Voyager Therapeutics shared preclinical data on the company’s AAV-delivered RNA interference therapy, VY-1706.

Rajeev Sivasankaran, PhD, on Voyager’s Single-Dose AAV Strategy for Silencing Tau for Alzheimer Disease

Patient use of the 2 FDA-approved gene therapy products for sickle cell disease (SCD), Vertex Pharmaceuticals' and CRISPR Therapeutics’ 

 (exa-cel; marketed as Casgevy) and bluebird bio’s 

 (lovo-cel; marketed as Lyfgenia), in the commercial setting has remained low.

Despite the approval of both gene therapy products by the FDA occurring simultaneously on December 8, 2023, only 4 patients have begun treatment with lovo-cel in the commercial setting, while 20 patients are reported to have begun treatment with exa-cel. Alongside the number for lovo-cel, bluebird also pointed out that this year 4 patients have started on elivaldogene autotemcel (Lenti-D, Skysona), its marketed gene therapy for cerebral adrenoleukodystrophy, and 19 patients have started on betibeglogene autotemcel (beti-cel, Zynteglo), its marketed gene therapy for transfusion dependent thalassemia (TDT). As such, bluebird bio has reported 23 total cell collections for SCD/TDT gene therapies lovo-cel and beti-cel. The company expects that patient uptake of the 3 aforementioned therapies will continue to increase, with 85 patient starts expected across the therapies by the end of the year.

“We are seeing clear evidence that our commercial launch is accelerating, with over 20 cell collections completed in SCD and TDT to date in 2024, and more than 40 additional patients already scheduled to initiate the treatment journey for a bluebird gene therapy by the end of this year,” Andrew Obenshain, MBA, the chief executive officer of bluebird bio, said in a statement.

 “We are further encouraged by the commitment to provide patient access across both commercial and government payers, most recently conveyed through multiple positive Medicaid decisions and the growing number of published coverage policies for Lyfgenia, and we expect approximately 85 patient starts across our portfolio this year.”

Notably, unlike lovo-cel, exa-cel is indicated for both SCD and TDT. Since Vertex has not clarified the breakdown of patients treated with exa-cel for each disease, direct comparison is difficult. The approval in TDT came slightly later, with 

“Vertex delivered another strong quarter of revenue growth coupled with outstanding execution across the business, and we are increasing our full year product revenue guidance,” Reshma Kewalramani, MD, the chief executive officer and president of Vertex, said in an August 1, 2024, statement.

 “Our focus for the second half of the year remains on commercial execution in cystic fibrosis and the global launch of Casgevy, readying for the upcoming potential launches of the vanzacaftor triple in cystic fibrosis and suzetrigine in acute pain, while rapidly advancing a robust pipeline that is poised to deliver value for patients and shareholders for the long term.”

Beyond gene therapy for SCD and TDT, uptake of 

hemophilia gene therapies has also been slow

. Although BioMarin’s valoctocogene roxaparvovec (val-rox, marketed as Roctavian) was approved in June 2023, the first patient outside of clinical trials 

 until December 2023, at the Center for Inherited Bleeding Disorders (CIBD) in California.

 Just this month, BioMarin announced that 

 with regard to val-rox, with the company now mainly focusing on distributing the gene therapy in the United States, Germany, and Italy, the 3 countries in which it has been approved for use by relevant regulatory authorities and is reimbursed.

 UniQure and CSL Behring’s hemophilia B gene therapy, etranacogene dezaparvovec (marketed as Hemgenix), which was 

, has also been slow to be embraced by patients.

“We haven't seen a lot of patients dosed yet,” Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital, told 

 regarding Hemegenix earlier in 2024. “And I don't think that’s related to either the enthusiasm of the clinicians or the patients, but really just the mechanics of how to deliver this in the clinical space, as opposed to the research trials... We've seen that a lot of the hemophilia treatment center sites have got their components together, their infrastructure and personnel so that they can actually start delivering this in the commercial setting. And so, I'm really looking forward to seeing this making a difference in patients' lives in a real world setting.”

REFERENCES
1. bluebird bio reports second quarter 2024 results and highlights operational progress and 2024 guidance. News release. bluebird bio, Inc. August 14, 2024. Accessed August 15, 2024. https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-reports-second-quarter-2024-results-and-highlights
2. Stock dive for bluebird as sickle cell gene therapy lags behind Vertex rival. News article. Anna Bratulic. FirstWord Pharma. August 14, 2024. Accessed August 15, 2024. https://firstwordpharma.com/story/5885598
3. Vertex announces US FDA approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia. News release. Vertex Pharmaceuticals Incorporated. January 16, 2024. Accessed August 15, 2024. https://news.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-casgevytm-exagamglogene
4. Vertex reports second quarter 2024 financial results. News release. Vertex Pharmaceuticals Incorporated. August 1, 2024. Accessed August 15, 2024. https://investors.vrtx.com/news-releases/news-release-details/vertex-reports-second-quarter-2024-financial-results
5. Center for Inherited Blood Disorders Administers Country's First Gene Therapy Infusion to Treat Hemophilia A. News release. Center for Inherited Blood Disorders. January 11, 2024. Accessed August 15, 2024. https://firstwordpharma.com/story/5817834?from=article
6. BioMarin Announces Updated Strategy for ROCTAVIAN® to Focus on U.S., Germany and Ital. News release. BioMarin Pharmaceutical Inc. August 5, 2024. Accessed August 15, 2024. https://investors.biomarin.com/news/news-details/2024/BioMarin-Announces-Updated-Strategy-for-ROCTAVIAN-to-Focus-on-U.S.-Germany-and-Italy/default.aspx
7. FDA approves first gene therapy to treat adults with hemophilia B. News release. FDA. November 22, 2022. Accessed August 15, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b

bluebird bio has reported 23 total cell collections for SCD and TDT gene therapies lovo-cel and beti-cel and Vertex Pharmaceuticals has reported approximately 20 cell collections for SCD/TDT gene therapy exa-cel.

Topic

Uptake of Nononcology Gene Therapy Remains Slow in Hematology

(arsa-cel, OTL-200; Orchard Therapeutics) is a gene-edited cell therapy designed to restore arylsulfatase A (ARSA) enzyme activity in children with presymptomatic (PS) late infantile (LI), PS early juvenile (EJ), or early symptomatic (ES) early juvenile (EJ) metachromatic leukodystrophy (MLD).

 arsa-cel under the name Lenmeldy to become the first gene therapy for children with MLD to be approved in the United States, following its approval in the European Union, UK, Iceland, Switzerland, Liechtenstein and Norway under the name Libmeldy.1 Orchard Therapeutics previously announced that the FDA had accepted the biologics license application with 

The FDA’s decision was based on data from patients treated with arsa-cel across 2 clinical trials (NCT01560182; NCT03392987) and additional patients treated in expanded access frameworks. These patients had follow-up times ranging from 0.64 years to 12.19 years (median, 6.76).

“Metachromatic leukodystrophy, is a really important unmet need in the pediatric community. So, this is a disease that's rare, it affects about one in 40,000 children. And up until recently, we have had no curative treatment option for this,” Madeleine Powys, MBBS, Staff Specialist, Blood Transplant and Cell Therapies, The Children's Hospital at Westmead, told 

. "Autologous hematopoietic stem cell gene therapy has offered really promising new hope for these patients.”

Madeleine Powys, MBBS, on Lessons Learned With Libmeldy

Arsa-cel is currently being evaluated in 6 participants enrolled in a phase 3 clinical trial (NCT04283227). The trial enrolled participants with documented biochemical and molecular diagnosis of MLD, including low ARSA activity and identification of 2 disease-causing ARSA alleles, with novel mutations analyzed and excluded as common polymorphisms, alongside specific genotype criteria. Symptomatic individuals must have onset between 7 and 17 years of age, or pre-symptomatic participants must be less than 17 years at treatment initiation and have a sibling with late-juvenile MLD variant, along with normal cognitive and motor function, with seizures or disease signs revealed by instrumental evaluations not being exclusionary if present alone, and compliance with contraceptive use requirements if applicable, with signed informed consent or assent provided by the participant or guardian.

The exclusion criteria for the study include documented HIV infection, malignant neoplasia except localized skin cancer or hereditary cancer syndrome unless successfully treated with no recurrence, myelodysplasia, acute myeloid leukemia, or serious hematological disorders, current enrollment in other interventional trials, prior allogeneic HSPC gene therapy or gene therapy, symptomatic herpes zoster unresponsive to treatment, active tuberculosis unless on antibiotic prophylaxis, acute or chronic stable Hepatitis B or positive Hepatitis C RNA test result unless specific conditions are met, end-organ dysfunction, severe infection not responsive to treatment, or other severe diseases deemed inappropriate for the study by the investigator. Additionally, testing for other transmissible infectious agents may be considered, and participants with elevated liver enzymes or bilirubin may be included after discussion and agreement with the medical monitor.

The study’s primary measures involve assessing the OTpbmsL-200 Arylsulfatase A (ARSA) activity levels in cerebrospinal fluid (CSF) and the neuronal metabolite ratio of N-acetyl-aspartate (NAA) to creatine (Cr) in white matter brain regions, both evaluated at 24 months post-treatment, with changes from baseline being monitored.

Secondary measures involve evaluating changes in ARSA activity levels in CSF and neuronal metabolite ratio in white matter brain regions, assessing ARSA levels in peripheral blood mononuclear cells (PBMCs) and specific subtypes, comparing neuronal metabolite ratios to baseline, siblings, or untreated controls, determining engraftment through lentiviral positivity in bone marrow progenitors and vector copy number in BM cells and PBMCs, assessing severity scale for brain MRI, neurocognitive function, full neurological clinical examination, gross motor function classification, nerve conduction velocity, Vineland Adaptive Behavior Scale, and monitoring conditioning regimen and non-conditioning-related adverse events up to 8 years post gene therapy, along with hematological reconstitution, infusion-related reactions, immune response, abnormal clonal proliferation, replication-competent lentivirus, and integration site analysis findings over time.

, held February 4-9, in San Diego, California, by Vera Gallo, MD, staff pediatrician, San Raffaele Telethon Institute for Gene Therapy. The data were from 5 patients with LJ MLD that had follow-up ranging from 3.2 to 21.3 months (median, 17.3). At the time of treatment, 3 patients had PS MLD and 2 had ES MLD. The group included a 2.7-year-old girl (PS), a 9.9-year-old boy (ES), a 15.2-year-old boy (PS), a 6.6-year-old girl (PS), and 10.9-year-old boy (ES) as of the December 2023 cutoff date.

"This represents of course, the proof of principle that gene therapy with ex- vivo lentiviral gene therapy into stem cells can correct a lysosomal storage disorder (LSD)... and could be applicable to other LSDS. So we're working on new diseases to bring this approach of enzyme correction also to other LSDs," investigator Alessandro Aiuti, MD, PhD, deputy director, clinical research, and head, Clinical Research Unit, San Raffaele Telethon Institute for gene therapy, and Group leader, Pathogenesis and therapy, primary immunodeficiencies unit, and full professor, Università Vita Salute San Raffaele, and chief of clinic, Pediatric Immunohematology Unit, San Raffaele, told 

Data from these participants showed that they all had normal hematological reconstitution and engraftment that was consistent with arsa-cel outcomes seen in patients with early-onset MLD who have been treated in other settings. Time to neutrophil engraftment ranged from 26 to 42 days (median, 33) posttreatment and time to platelet engraftment ranged from 25 to 46 days (median, 26) posttreatment. ARSA activity reached supranormal levels in the PBMCs and in CD15+ cells and increased to normal levels in the CSF in 3 patients for whom central nervous system pharmacodynamic efficacy measures were available.At 90 days after administration of arsa-cel transduced bone marrow progenitors made up between 23.81% to 81.25% of bone marrow progenitors. At this time point, the PBMCs showed a median vector copy number (VCN) of 0.49 copies/cell and the CD15+ cells showed a median VCN of 0.82 copies/cell.

In terms of safety, there were no serious adverse events (SAEs) deemed potentially related to arsa-cel. One nonserious AE deemed potentially related to the therapy, a case of erythematous rash, occurred in 1 patient. No SAEs deemed potentially related to the Busulfan conditioning regimen occurred; although cases of febrile neutropenia, cytopenia, and stomatitis were deemed to have potential relation to the regimen. An SAE of upper respiratory tract infection occurred in 1 patient. One patient with prolonged thrombocytopenia who had previously experienced treatment-failure with allogeneic transplant in the form of autologous reconstitution was administered thrombopoietin receptor agonist therapy for 5 months. No multilineage engraftment of transduced cells or malignancy occurred and there was no indication of clonal expansion or replication competent lentivirus.

1. FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy. News release. Orchard Therapeutics. March 18, 2024. Accessed March 18, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-children-metachromatic-leukodystroph
2. Orchard Therapeutics announces acceptance of biologics license application for OTL-200 in MLD and receives priority review. News release. Orchard Therapeutics. September 18, 2023. Accessed September 18, 2023. https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-acceptance-biologics-license
3. Fumagalli F, Calbi V, De Mattia F, et al. Long-term clinical outcomes of atidarsageneautotemcel (autologous hematopoietic stem cell gene therapy) preserves cognitive and motor development in early-onset metachromatic leukodystrophy with up to 12 years follow-up. Presented at: WORLDSymposium, held February 4-9, in San Diego, California. Poster #92
4. Gallo V, Calbi V, De Mattia F, et al. Lentiviral hematopoietic stem cell gene therapy (atidarsageneautotemcel) for late juvenile metachromatic leukodystrophy (MLD). Presented at: WORLDSymposium, held February 4-9, in San Diego, California. Poster #96

The gene-edited cell therapy has been approved as Lenmeldy by the FDA. 

Phase 3 Trial Seeks to Continue Supporting Arsa-Cel Gene Therapy for MLD 

A patient treated in Allogene Therapeutics’ phase 2 ALPHA3 clinical trial (NCT06500273), which is evaluating investigational CD19-directed allogeneic chimeric antigen receptor (CAR) T-cell therapy cemacabtagene ansegedleucel (cema-cel) as a first-line consolidation treatment for large B-cell lymphoma (LBCL), has died.

Notably, the patient was treated in an arm of the trial evaluating a lymphodepletion regimen consisting of fludarabine and cyclophosphamide and anti-CD52 monoclonal antibody ALLO-647 (FCA), and the patient’s death was attributed to this lymphodepletion regimen rather than to cema-cel itself. Specifically, the patient’s death was attributed to hepatic failure that was thought to have arisen from “disseminated adenovirus infection in the setting of immune suppression”. The patient’s death occurred 54 days after infusion and was attributed to ALLO-647 specifically.

ALPHA3 also includes a separate arm using only a standard fludarabine and cyclophosphamide (FC) lymphodepletion regimen, and Allogene noted that no adenoviral infections or hepatic failures have been reported in patients treated in the FC arm in ALPHA3, nor in any of the company’s other clinical trials utilizing an FC regimen. As such, Allogene stated that it has selected FC as the lymphodepletion regimen to be used going forward in ALPHA3, a decision it made following discussions with the FDA and with the study’s data and safety monitoring board and steering committee.

“The loss of a patient is always deeply saddening, and we extend our heartfelt condolences to the patient’s family,” David Chang, MD, PhD, the president, CEO, and cofounder of Allogene, said in a statement.

 “This event, which prompted an early review of the trial data, compelled us to make a decisive choice—one that may ultimately help bring this potentially lifesaving therapy to patients more quickly. The ability to administer cema-cel following standard FC lymphodepletion in an outpatient setting will simplify study treatment and has the potential to accelerate trial enrollment and streamline regulatory review, ultimately transforming care for patients.”

As a result of the change, ALPHA3 is now a 2-arm trial, in which patients are randomly assigned to receive either cema-cel with FC lymphodepletion or to undergo standard of care observation. The company stated that a futility analysis comparing minimal residual disease conversion for ALPHA3 is planned for the first half of 2026, and that the trial currently has 50 active clinical sites in the United States and Canada. Allogene also pointed out that currently, none of its actively enrolling clinical trials for its therapeutic candidates include the use of ALLO-647.

Among Allogene’s other investigational CAR T therapy products is ALLO-329, an investigational allogeneic CAR T therapy that targets both CD19+ B cells and CD70+ activated T cells, with the intention of eliminating dysfunctional cells of both types in rheumatology indications including active refractory moderate-to-severe systemic lupus erythematous; active severe/refractory idiopathic inflammatory myopathy, specifically including dermatomyositis, immune mediated necrotizing myopathy, and antisynthetase syndrome; and active refractory diffuse systemic sclerosis.

received fast track designation from the FDA

 for the aforementioned indications in April 2025 Furthermore, the CAR T product utilizes the company’s Dagger technology, which is intended to reduce or eliminate the need for lymphodepletion by making the CAR T more resistant to rejection by the patient’s immune system.

"Receiving these designations for ALLO-329 underscores the versatility and transformative promise of this next-generation allogeneic CAR-T investigational product in redefining the autoimmune treatment landscape," Zachary Roberts, MD, PhD, the executive vice president of research and development and the chief medical officer at Allogene, said in an April 2025 statement.

 "Leveraging our extensive expertise, we've developed this off-the-shelf CAR T specifically for autoimmune diseases, prioritizing both scalability and the reduction or elimination of lymphodepletion—a key barrier in this patient population."

REFERENCES
1. Allogene Therapeutics moves forward with the standard fludarabine and cyclophosphamide (FC) lymphodepletion regimen in the ALPHA3 trial for cemacabtagene autoleucel (cema-cel) in first-line consolidation for large B-cell lymphoma. News release. Allogene Therapeutics Inc. August 1, 2025. Accessed August 4, 2025. https://ir.allogene.com/news-releases/news-release-details/allogene-therapeutics-moves-forward-standard-fludarabine-and
2. Allogene granted three U.S. FDA fast track designations (FTD) for ALLO-329, a next-generation dual-targeted CD19/CD70 allogeneic CAR T, for the treatment of lupus, myositis and scleroderma. News release. Allogene Therapeutics Inc. April 7, 2025. Accessed August 4, 2025. https://ir.allogene.com/news-releases/news-release-details/allogene-granted-three-us-fda-fast-track-designations-ftd-allo
3. Allogene Therapeutics secures U.S. FDA IND clearance for ALLO-329, advancing its next-generation allogeneic CAR T into autoimmune diseases. News release. Allogene Therapeutics Inc. January 28, 2025. Accessed August 4, 2025. https://ir.allogene.com/news-releases/news-release-details/allogene-therapeutics-secures-us-fda-ind-clearance-allo-329

The patient’s death was attributed to anti-CD52 monoclonal antibody ALLO-647 rather than to cema-cel itself.

Patient Dies After Receiving Monoclonal Antibody Lymphodepletion Regimen in LBCL Trial for Allogene’s Cema-Cel

“I think we're very excited about the progress the preclinical data that we've generated so far, and we're on track for continuing to our GLP tox studies, and we look forward to an IND filing in 2026.”

 spoke with Rajeev Sivasankaran, PhD, vice president of neuroscience at Voyager Therapeutics, who presented new preclinical data at 

the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting

, held May 13 to 17, 2025, in New Orleans, Louisiana. His presentation focused on VY-1706, an investigational gene therapy intended to treat Alzheimer disease by delivering tau-silencing siRNA through a single intravenous (IV) dose of adeno-associated virus (AAV).

VY-1706 is still in preclinical development and utilizes the AAV platform to deliver a potent, selective siRNA payload directly to affected brain regions. The therapy demonstrated strong pharmacologic activity in the P301S mouse model of tauopathy, which shows early and progressive tau accumulation. A single IV administration of VY-1706 resulted in broad central nervous system biodistribution and significantly reduced tau mRNA and protein expression, including reductions in pathologic tau.

In nonhuman primate studies, VY-1706 achieved dose-dependent delivery and greater than 50% knockdown of tau protein in the cortex, hippocampus, and other brain regions relevant to AD. Notably, the treatment was well-tolerated, with no clinical signs or changes in cardiac or liver enzymes and no elevation of neurofilament light chain, a surrogate marker for AAV-associated neurotoxicity. “We did not see any clinical signs or clinical chemistry changes, including cardiac enzymes and liver enzymes,” Sivasankaran told 

The company expects to initiate GLP toxicology studies later this year and aims to submit an investigational new drug application in 2026. VY-1706 is 1 of 2 tau-targeting programs at Voyager, alongside a monoclonal antibody currently in human trials.

Click here to view more coverage of the 2025 ASGCT Annual Meeting.

Videos

BioCardia has stated that it plans to request a meeting with the FDA to discuss the pathway to approval for its CardiAMP cell therapy system, an investigational autologous cell therapy for the treatment of ischemic heart failure (HF).

The company noted that it plans to submit the meeting request by the fourth quarter of this year and that the meeting will focus on the approvability of the product based on available clinical data. CardiAMP is currently being evaluated in the pivotal phase 3 CardiAMP HF clinical trial (NCT02438306). Although the study did not meet its primary end points, BioCardia believes a number of factors may allow a pathway to approval for CardiAMP. The company cited as examples of these factors the breakthrough therapy designation previously granted to CardiAMP by the FDA, longer-term follow-up data from CardiAMP HF, the FDA’s prior 510(k) approval of Marrowstim, a primary component of CardiAMP cell therapy, and the confirmatory phase 3bCardiAMP HF II clinical trial (NCT06258447), which is currently enrolling patients at several sites in the United States. BioCardia also noted that is plans to submit a DeNovo 510(k) application for the Helix Transendocardial Delivery System (Helix) to the FDA in the third quarter of 2025. Helix is also a component of CardiAMP.

In addition to the updates regarding US regulatory activities, BioCardia also announced it expects that a clinical consultation with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), which the company requested in July of this year, will take place in the fourth quarter of 2025. BioCardia pointed out that if it and the agency are aligned during the meeting, it may be able to submit for market approval of CardiAMP in Japan.

“This discussion may be influenced by other sponsor’s applications to PMDA for allogeneic cell therapies to treat the same clinical indication expected to require chronic immunosuppression and open chest surgical delivery of cells,” BioCardia stated in a press release.

 “Such laboratory manufactured therapies may receive early conditional approval under Japan’s adaptive framework for the approval of regenerative medical products with a post marketing study. The CardiAMP Cell Therapy is regulated as a medical device as the autologous cells are processed at point of care.... The point of care CardiAMP cell processing platform is already approved and in clinical use in Japan for orthopedic applications by Zimmer Biomet Japan under the trade name BioCUE.”

Data from CardiAMP HF were presented earlier this year at the American College of Cardiology 74th Annual Scientific Session & Expo, held March 29 to 31, 2025, in Chicago, IL.

 A study-wide increase in survival and decrease in major adverse cardiac and cerebrovascular events (MACCE) was reported, although BioCardia noted that the primary composite efficacy end point did not reach a statistically significant result. Furthermore, the company stated that compared to patients receiving an optimized heart failure medication regimen alone, patients treated with CardiAMP experienced 3.6% fewer heart death equivalents, showed a relative risk reduction in heart death equivalents of 20.9%, had 8.7% fewer nonfatal MACCE, and displayed a relative risk reduction in nonfatal MACCE of 44.6%. A 5.5-point improvement on The Minnesota LIVING WITH HEART FAILURE Questionnaire (MLHFQ), considered clinically meaningful, was also observed for patients treated with CardiAMP.

“This clinical trial was conducted because there remains a large group of heart failure patients today who are insufficiently responsive to optimized heart failure medication,” coprincipal investigator Amish N. Raval, MD, FACC, a professor of medicine at the University of Wisconsin School of Medicine and Public Health, who presented the data, said in a March 2025 statement.

 “In this rigorous CardiAMP-HF Trial, patients who received the novel cell therapy adjunctive to medication experienced decreased mortality and MACCE, with improved quality of life, when compared to those on medication alone. These benefits appeared to be greater in patients with elevated NTproBNP - comprising fully half of treated patients - reaching statistical significance in the composite measure of these outcomes. The trial shows us that CardiAMP therapy has the potential to safely and significantly improve survival and quality of life for heart failure patients in distress, encompassing a large group of patients we see in daily practice.”

REFERENCES
1. BioCardia provides update on timing of regulatory activities seeking FDA approval and Japan PMDA approval for CardiAMP® cell therapy for heart failure and Helix™ Transendocardial Delivery Catheter. News release.BioCardia, Inc. August 4, 2025. Accessed August 5, 2025. https://www.biocardia.com/investors/press-releases/id/1037?pressReleaseId=233
2. BioCardia phase 3 CardiAMP-HF trial of novel cardiac cell therapy for ischemic heart failure shows increased survival, decreased cardiac events, and improved quality of life at two years. News release. BioCardia, Inc. March 31, 2025. Accessed August 5, 2025. https://www.biocardia.com/investors/press-releases/id/1037?pressReleaseId=217

The company noted that it plans to submit the meeting request by the fourth quarter of this year and that the meeting will focus on the approvability of the product based on available clinical data.

BioCardia Reveals Plans to Request Meeting With FDA Regarding CardiAMP Heart Failure Cell Therapy

In Episode 5 of ImmunoLogic, Michael T. Lotze, MD, discussed the evolution and future of tumor infiltrating lymphocyte (TIL) therapies.

ImmunoLogic, Episode 5: "Tumor Infiltrating Lymphocytes" With Michael T. Lotze, MD

We're excited to introduce another episode of the 

an assistant professor of microbiology and the director of the Fraietta Lab at the University of Pennsylvania. Fraietta is often joined by cohost 

 a clinical fellow in oncology (MSTR Program) at the University of Pennsylvania. Our hope is that this show will serve as a critical forum for clinicians, translational researchers, and biotech professionals to explore the evolving landscape of immunotherapy. We're seeking to bridge cutting-edge research with practical applications and delve into how immune-based therapies transition from innovative discoveries to transformative patient care solutions

a professor of surgery, immunology, and bioengineering at the University of Pittsburgh, about his work on tumor infiltrating lymphoctye (TIL) therapy. Fraietta and Lotze covered topics including the history of TIL research, the potential of TIL therapy in different tumor types, Lotze and his colleagues' new reciprocal learning model hypothesis, and concluded with some philosophical reflections on cancer research.

 is tailored for an audience fluent in the language of medicine and biotechnology, offering data-driven insights while maintaining accessibility. This podcast is an essential resource for those driving the future of immunotherapy.

Interested in cohosting an episode or being a guest? Have feedback to share? 

If you'd like to get in touch to talk about the show, contact our editorial director, Matt Hoffman: 

In episode 5 of ImmunoLogic, Michael T. Lotze, MD, discusses the evolution and future of tumor-infiltrating lymphocyte therapies.

Rajeev Sivasankaran, PhD

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