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The chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.

Maria Escolar, MD, on the Importance of Newborn Screening for Treating Krabbe Disease

The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.

Maria Escolar, MD, on Continued Positive Results With Gene Therapy and UCBT in Krabbe Disease

The chief medical officer of Forge Biologics discussed updated data from the phase 1/2 RESKUE clinical trial that she presented at ASGCT’s 2023 conference.

Maria Escolar, MD, on Treating Krabbe Disease With Investigational Gene Therapy FBX-101

The chief medical officer of Forge Biologics discussed the potential advantages of the investigational combination therapy approach. 

Maria Escolar, MD, on Combining HSCT With Gene Therapy in Krabbe Disease 

The chief medical officer of Forge Biologics discussed clinical data presented at ESGCT. 

Maria Escolar, MD, on Addressing Unmet Needs in Krabbe Disease With Gene Therapy 

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 

Mission Bio, a company seeking to overcome limitations of bulk sequencing and standard single cell sequencing methods through the use of a unique platform, gave several presentation related to its technology 

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

, held May 13 to 17, 2024, in New Orleans, LA. 

 sat down with Brian Kim, MBA, the chief executive officer of Mission Bio, at the meeting to get his insight on the key takeaways of what the company brought to the conference this year.

Kim highlighted several of Mission Bio's presentations and discussed what the company is working on more generally, noting several collaborations. He also touched on the state of the field in general and expressed optimism for its trajectory in the future.

 Can you give some background about Mission Bio?

 Mission Bio is a single cell multiomic platform that is used for mostly clinical applications. It differentiates itself from other single cell companies in that it works with DNA and looks at proteins. Our platform is really used for customers who aren't necessarily trying to discover new things, which is a lot of what current single cell platforms are being used for, but [instead for] once you've discovered it and you know what you're looking for, to really validate it for either therapeutic purposes or for diagnostic purposes.

Can you give an overview of what the company is presenting at ASGCT this year?

We have a couple presentations here. One is with the work that we have done with Genentech, and another is a really important study that Ayal Hendel, PhD, has worked on with us. They're going to be, I think, really very exciting developments for our customers. One of the other big developments that we will have announced here at ASGCT is a partnership with Integrated DNA Technologies (IDT). This actually was a partnership that actually originated at ASGCT in 2024 in Baltimore. Essentially what we are doing is we are incorporating IDT's world-class rhAmpSeq technology into our assay. What this will effectively do is it will allow us to create a very sensitive assay that will look for on-target efficacy and off-target detection to really help characterize the safety of CRISPR-based medicines for our customers.

How would you summarize the big picture implications you would want the broader healthcare community to take away from Mission Bio's presentations this year?

I think this is an interesting time we're living in right now and cell and gene therapy is going through a transition period right now, but I think it's really important to note that it's a transition period. I don't think people should really lose too much sleep that this is going to go away. A lot of drug modalities and technologies have these ebbs and flows, but we just saw this morning there was an announcement by the 

 that a little baby was saved with a CRISPR-based therapeutic for a very rare genetic disorder that this child had. When you see things like that happening with patients with the therapies that we have right now, you know that it's going to go someplace in the future.

So as it relates to ASGCT and with Mission Bio we are absolutely committed to this space. We are continuing to develop our assays and our products really make an impact to our customers. To kind of give you a sense of it, our company is or has been involved in over 17 clinical trials for biopharma customers and our products right now are being adopted by 3 commercial and academic labs to be used as a laboratory-developed test. And so for us, we're committed to this space and we are in here for the long run because we know this momentary issue that we're facing will eventually turn the other way.

Are there any future plans for Mission Bio that you can discuss a bit?

I've been in post as the CEO of this company for about almost 18 months, and since I've been in place I've really focused on 4 key things: Revenue growth, continuing to innovate great products, clinical utility for our products, and really just continuing to optimize and really run a very optimized business performance and operations. I'm happy to note that revenue-wise, we are going to have a really strong year this year because of a lot of the work that our teams have been taking on. I'd say, from an innovation perspective, we are really being focused on the things that really matter to our customers. One of the big innovations that we will be announcing very soon is the incorporation of targeted gene expression into our assay. Right now, we work with DNA to get genotyping, we can detect proteins for immunophenotyping, and we'll now be able to incorporate into our assay RNA for targeted gene expression. We think that ability to analyze all these analytes for our customers is going to be very important. That's what our customers have been asking for from us for some time so we're really thrilled about being able to deliver this into the marketplace. 

As it relates to clinical utility, we're also really thinking and taking a hard look at where within diagnostics our platform will have the most relevance. We announced at the beginning of late 2024 a significant collaboration that we're doing with the University of Miami. 

University of Miami and C. Ola Landgren, MD, PhD, are world renowned for their work in treating multiple myeloma. The work we're doing with them is focused on how we can use our assay to actually help guide therapeutic selection after a patient relapses and have exhausted their frontline treatments. This is something that we're working with them on right now to actually collect the data, and we'll be able to say something more about this towards the end of the year. But if we can collect the data that actually shows that with our platform we can guide the therapy that our patients who have exhausted frontline therapies should go on, we think that this is going to be really meaningful to our customers. So that's another example of collaboration and working with the external world to really prove the clinical utility of our platform.

This transcript has been edited for clarity.

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

Brian Kim, MBA, the chief executive officer of Mission Bio, discussed the company’s single cell assay platform in the context of the broader developing field cell and gene therapy field.





Multiomic Single-Cell Sequencing and the Ebbs and Flows of the Gene Therapy Field

This is the second part of an interview with Brian Kim, MBA. For the first part, 

Mission Bio is seeking to overcome limitations of bulk sequencing and standard single cell sequencing methods with Tapestri, its platform that is intended to offer a multiomic single cell sequencing solution. The company recently presented data related to this platform 

interviewed Brian Kim, MBA, the chief executive officer of Mission Bio, to learn more about the company's activities and his thoughts on the field of cell and gene therapy in general. Kim discussed his thoughts on the trends currently being seen in the industry and also spoke about Mission Bio's goals for collaboration and partnerships.

 What are your thoughts on ASGCT this year and trends in the industry in general?

 We're really excited about CRISPR-based medicines. We are seeing more and more of it, especially with some of our early-stage customers. There's a lot of demand for it because of things like we just read in the 

 today. As such, we believe this is going to be a modality of the future and we're just at the cusp of some of the things that are being worked on. We've been involved with some of the earlier CRISPR-based medicines that have been launched and so this is a place we're very familiar with and this is something we're going to continue with. The second thing that we've heard is really this resurgence with AAV and viral-vector based therapeutics, and that was a little bit surprising to us. Maybe earlier on this year, there were maybe some premature announcements by the press about the decline of adeno-associated virus (AAV), but we're seeing quite the opposite here. As such, I think the only way that you're going to be able to see these things is actually to be able to attend these conferences like ASGCT and to actually hear from what our customers are saying, as opposed to just believing what you read in popular press.

Is there anything else you want to share?

We're really excited about where Mission Bio is going. One of the things that we committed to last year was really opening up partnerships and opportunities to collaborate with external parties. The significant work that we're doing with University of Miami to collect the clinical utility data is one example of it. The second one is with Integrated DNA Technologies (IDT). And we have a couple more of these announcements that we're going to be making in the back half the year. But we would just open the door and open the invitation to other companies out there that would like to work with us to give us a ring. We're open for business.

Brian Kim, MBA, the chief executive officer of Mission Bio, also discussed the company’s plans for collaboration.

The Emergence of CRISPR-Based Medicines and the Resurgence of AAV

Vertex Pharmaceuticals’ zimislecel (also known as VX-880), an investigational allogeneic insulin-producing islet cell therapy, has restored endogenous insulin secretion in patients with type 1 diabetes (T1D) treated in the phase 1/2 FORWARD clinical trial (NCT04786262).

 The data were announced in an oral presentation at the 85th Scientific Sessions of the American Diabetes Association® (ADA) , held June 20 to 23, in Chicago, IL, and simultaneously reported in the 

Among 12 patients with T1D with impaired hypoglycemic awareness treated in FORWARD, all 12 showed restored endogenous insulin secretion, as assessed via C-peptide. Furthermore, the patients showed cessation of severe hypoglycemia events and hit recommended glycemic control targets, which constituted A1C at less than 7% and time in range of greater than 70%. Use of exogenous insulin was cut down by a mean of 92% across all patients, with 10 of the 12 patients no longer using exogenous insulin at all.

With regard to safety, adverse events (AEs) were noted to be in line with expectations for islet infusion procedures and the immunosuppressive drug regimens. There were no other AEs seen in the treated patients.

“Hypoglycemia remains a dangerous risk for individuals with T1D who are dependent on exogenous insulin administration,” investigator Michael Rickels, MD, MS, the Willard and Rhoda Ware professor in diabetes and metabolic diseases in the Division of Endocrinology, Diabetes & Metabolism, at University of Pennsylvania Perelman School of Medicine, who presented the data, said in a statement.

 “These findings indicate the potential for a novel cellular therapy that restores endogenous insulin secretion to improve outcomes for T1D patients who have been struggling to achieve glycemic control.”

Before the end of the year, it is expected that FORWARD, which is currently in phase 3, will have finished enrollment and dosed around 50 patients. Vertex also plans to initiate a separate study that will evaluate VX-880 in 10 adult patients who have previously received a kidney transplant and are thus already receiving an immunosuppressive therapy regimen.

 by Vertex following 2 patient deaths that were deemed unrelated to VX-880.

 The VX-880 trial had previously been placed on hold by the FDA in May 2022, after the FDA determined there was insufficient information to support dose escalation, despite an Independent Data Monitoring Committee’s recommendation to advance patients to part B of the study and full dosing.

 At the time, Carmen Bozic, MD, the executive vice president of Global Medicines Development and Medical Affairs and the chief medical officer of Vertex, expressed surprise at the FDA’s decision. The FDA later 

REFERENCES
1. Stem cell-derived islet therapies shown to reduce the need for injectable insulin. News release. American Diabetes Association. June 20, 2025. Accessed June 20, 2025. https://diabetes.org/newsroom/press-releases/stem-cell-derived-islet-therapies-shown-reduce-need-injectable-insulin
2. Vertex provides pipeline and business updates in advance of upcoming investor meetings. News release. January 7, 2024. Accessed June 20, 2025. https://www.businesswire.com/news/home/20240107188174/en/Vertex-Provides-Pipeline-and-Business-Updates-in-Advance-of-Upcoming-Investor-Meetings
3. Vertex provides updates on phase 1/2 clinical trial of VX-880 for the treatment of type 1 diabetes. News release. Vertex Pharmaceuticals. May 2, 2022. Accessed June 20, 2025. https://news.vrtx.com/press-release/vertex-provides-updates-phase-12-clinical-trial-vx-880-treatment-type-1-diabetes
4. Vertex announces FDA has lifted the clinical hold on VX-880 phase 1/2 clinical trial for the treatment of type 1 diabetes. News release. Vertex Pharmaceuticals. July 5, 2022. Accessed June 20, 2025. https://www.businesswire.com/news/home/20220705005299/en/Vertex-Announces-FDA-Has-Lifted-the-Clinical-Hold-on-VX-880-Phase-12-Clinical-Trial-for-the-Treatment-of-Type-1-Diabetes

The patients also showed cessation of severe hypoglycemia events and hit recommended glycemic control targets.

Vertex’s Islet Cell Therapy Zimislecel (VX-880) Restores Endogenous Insulin Secretion in Type 1 Diabetes

the European Hematology Association (EHA) 2025 Congress

, held June 12 to 15, both virtually and in Milan, Italy, Galapagos presented new data regarding GLPG5101, an investigational. CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy product. During the conference, 

 interviewed Omotayo Fasan, MRCP, DTM&H, MBBS, the head of the clinical development program at Galapagos, about the findings.

Fasan discussed the design of GLPG5101 and went over the key data points and their implications. He also touched on future plans for the therapy and other cell therapy products in the company's pipeline.

 Can you give some background context about Galapagos’ EHA presentation?

 At EHA 2025, Galapagos is presenting new data from its ATALANTA-1 study, an ongoing phase 1/2 clinical trial of GLPG5101, a CD19-directed CAR-T cell therapy candidate for relapsed/refractory nonHodgkin lymphoma (r/r NHL). This therapy is manufactured using Galapagos’ innovative decentralized platform, which enables delivery of fresh, stem-like early memory T-cells in a median vein-to-vein time of just 7 days.

This study represents a breakthrough in operationalizing decentralized manufacturing, making fresh CAR-T therapy more accessible and feasible across multiple sites and lymphoma subtypes. The EHA presentation demonstrates the potential of this novel “fresh and fast” approach with a favorable safety profile. The low incidence of high-grade toxicities makes such a therapy ideal for development as an out-patient therapy, meaning less patients are hospitalized to treat side-effects and lower costs for the healthcare system.

What are some of the key findings Galapagos is presenting at EHA this year?

Key highlights from the EHA presentation include:

High Fresh Cell Utilization: 95% of patients received fresh CAR-T products, avoiding cryopreservation, which is associated with reduced toxicity, good expansion, and an early memory phenotype cell profile.

Short Vein-to-Vein Time: Approximately 9 out of 10 patients achieved infusion within 7 days of leukapheresis, thereby avoiding the need for cytotoxic bridging therapy —significantly faster than the 4–6 weeks typical of current CAR-T therapies.

Favorable Safety Profile: Only 1 grade 3 cytokine release syndrome (CRS) and 1 grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS) were reported, with most grade ≥3 adverse events being hematologic and expected.

Low Attrition: Just 5% of patients dropped out prior to infusion, outperforming industry benchmarks where dropout ranges from 10–30%.

Robust In Vivo Expansion: Consistent expansion of CAR-T cells and maintenance of a stem-like early memory phenotype across all tumor types and dose levels.

How would you summarize the big-picture implications that doctors and the broader healthcare community should take away from these results?

Galapagos’ EHA results suggest a meaningful advance in CAR-T therapy delivery and safety. The decentralized, 7-day manufacturing approach demonstrates that fresh, early memory CAR-T cells can be reliably delivered in a decentralized manner, enhancing physician confidence and expanding treatment access—especially for patients too fragile to wait weeks for traditional CAR-T production.

The potential benefits to patients include:

Rapid vein-to-vein time of 7 days, potentially affording more patients a CAR-T option.

Minimized attrition rates and postleukapheresis dropout rates.

Reduced toxicity and high-grade complications.

Elimination of the need for cytotoxic bridging therapy.

Enhanced outcomes and experiences for both patients and providers by offering a faster, scalable and more accessible therapeutic option.

What are the next steps for this research? Are there any challenges that still need to be addressed?

Galapagos plans to initiate pivotal development for GLPG5101 in 2026, starting with mantle cell lymphoma (MCL) as the lead indication, and subsequently following with multiple phase 3 indications as the phase 1/2 cohorts fully enroll. We are aiming for regulatory submissions starting in 2028. Preparations include expanding decentralized manufacturing units (DMUs) across Europe and the US and onboarding additional clinical sites.

Is there anything not covered by the previous questions that you want to share with our audience of doctors and healthcare professionals?

Beyond GLPG5101 in 8 relapsed/refractory B-cell malignancies, Galapagos is developing a broader pipeline, including GLPG5301, an antiBCMA CAR-T for r/r multiple myeloma (PAPILIO-1 phase 1/2 study) and a preclinical T-cell therapy program (uza-cel) in MAGE-A4 positive solid tumors such as Head & Neck cancer, in partnership with Adaptimmune. This platform is not just a delivery mechanism but a transformative ecosystem of 3 key components—integrating an end-to-end xCellit® workflow and monitoring software system, a decentralized, functionally closed, automated manufacturing platform for cell therapies (using Lonza’s Cocoon®), and proprietary quality control and product release processes.

The data from ATALANTA-1 of GLPG5101 demonstrate the potential for our lead CAR-T product candidate—and it also suggest the strong potential of decentralized, fast and fresh cell therapy approaches. By reducing barriers to access and expanding the eligible patient population, Galapagos is working to set a new standard in how advanced personalized cell therapies can be delivered reliably, rapidly, and globally.

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The head of the clinical development program at Galapagos discussed the company's data presented at EHA's 2025 Congress.

Maria Escolar, MD

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