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Lysosomal Disorders

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The program director of the center for psychiatric oncology & behavioral sciences at Mass General Cancer Center

Jamie Jacobs, PhD, the program director of the center for psychiatric oncology & behavioral sciences at Mass General Cancer Center, discussed her presentation at ASCO this year.

Using a Digital App to Aid Caregivers for Patients Undergoing Hematopoietic Stem Cell Transplant

The program director of the center for psychiatric oncology & behavioral sciences at Mass General Cancer Center discussed her presentation at ASCO this year.

Jamie Jacobs, PhD, on Using a Digital App to Aid Caregivers for Patients Undergoing Hematopoietic Stem Cell Transplant

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 

March Biosciences is currently developing MB-105, an investigational CD5-directed chimeric antigen receptor T-cell (CAR-T) therapy, with the intent of addressing unmet needs in the treatment of relapsed/refractory (r/r) CD5-positive T-cell lymphoma. Notably, the design of a recently launched phase 2 clinical trial (NCT06534060) for the product is being presented by the company at 

the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting

, held May 30 to June 3, in Chicago, Illinois.

 got in touch with Sarah Hein, PhD, the chief executive officer and cofounder of March Biosciences, to learn more about MB-105 and the key takeaways from the presentation. Hein explained the therapy's intended role, discussed the trial, and also touched on future plans for the program, highlighting the company's openness to new collaboration opportunities.

: Can you give some background context about MB-105 and how it is meant to address unmet needs?

MB-105 is an autologous cell therapy for CD5-positive hematological cancers. It's currently in a phase 2 trial for T-cell lymphoma, which is a severely under-addressed hematological malignancy, and is also applicable to other CD5-positive cancers, including T-cell leukemia, mantle cell lymphoma, chronic lymphocytic leukemia, and potentially other T-cell driven diseases.

Can you give an overview of the trial design that March Bio will be presenting at ASCO this year?

The phase 2 trial that we're presenting at ASCO is targeted towards T-cell lymphoma. If you're familiar with this indication, unfortunately once patients enter the r/r setting, there really is not a standard of care. These patients have about a less-than-20% survival over about 3 years, and that's because there's a handful of salvage therapies available to these patients, but not a lot else

 As a consequence of that what's really interesting about CD5 is it's widely expressed across both peripheral T-cell lymphoma (PTCL) and cutaneous T-cell lymphoma (CTCL). It covers about 70% to 80% of the patients, and we have a trial design that is therefore available to patients with PTCL at second line or above and CTCL at third line or above. It's a Simon 2-stage design, and multicenter across the entire United States. We have targeted 12 sites that we're activating currently, and we began enrolling earlier this year and announced our first patient dosed about a month ago.

What are the big picture implications that you would want doctors and the broader healthcare community to take away from the ASCO presentation?

Please, if you have patients that are in this indication, there really aren't a lot of trials available to these patients at all, much less these more powerful immunotherapies or cell therapies that have these larger response rate opportunities. We've seen really strong responses from the clinicians due to that. If you have patients that meet the criteria and are eligible for this trial, you'll be able to see through the presentation all of our network sites. We are always open to talking to additional sites as well. We have, as I said, 12 sites currently. It is a Simon 2-stage design, so we'll get our first initial readout later this year, and then we'll be rolling into that 2b portion of the trial, as well. We are always are trying to reach as many patients as we can through that enrollment process.

Are there any challenges with the MB-105 program that still need to be addressed, or additional future plans that you can discuss?

The challenges are similar to what you see in in any advanced therapy. Really, our main major focus has been on manufacturing as quickly as possible and reaching as many patients as possible, as quickly as possible. We have developed a pipeline and a production capacity that we feel is more than adequate to meet the needs of this trial and beyond, but as you know, with moving these products into later-stage development and continuing to expand the trial, there's always going to be new opportunities for improvement as we continue to expand this product. Really, our goal is to get this to an approval standpoint as quickly as we can, as quickly as is reasonable and safe for the patients, because the need is just incredibly high.

Is there anything else you want to share with our audience?

I always like to say, if you have interacted with T-cell lymphoma in particular, but really with any of these CD5-positive indications, and understand the need the way that we do—we're always looking for opportunities to collaborate. That could look like additional clinical sites, it could look like partnerships down the road. Our goal here is to rapidly accelerate this as quickly as possible, and that's a team effort. So my invitation is always for people who are enthusiastic about the space and who see the patient need to reach out to us. We're open to those conversations.

This transcript has been edited for clarity.

Click here to view more coverage of ASCO's 2025 Meeting.

Sarah Hein, PhD, the chief executive officer and cofounder of March Biosciences, discussed the company’s trial design presentation at ASCO’s 2025 meeting.

The Phase 2 Trial for CD5-Directed CAR-T MB-105 in R/R T-Cell Lymphoma

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

The CEO of Mission Bio also discussed the company’s plans for collaboration.

Brian Kim, MBA, on the Emergence of CRISPR-Based Medicines and the Resurgence of AAV 

“...[E]arlier on this year, there were maybe some premature announcements by the press about the decline of 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.”

Mission Bio's Tapestri platform is intended to provide a multiomic single cell sequencing solution for companies and institutions developing cell and gene therapy products. The company is currently working to build upon the features of its platform and foster collaborations to expand its reach, and gave presentations related to these topics at 

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

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

 spoke with Brian Kim, MBA, the chief executive officer of Mission Bio, about what he is paying attention to at the meeting this year and about the broader trends in the field. Kim drew attention to the growing interest in CRISPR-based medicines, specifically alluding to a story covered by major news outlets, including 

. This story covered the case of KJ, an infant with severe carbamoyl phosphate synthetase 1 deficiency who 

was treated with a personalized CRISPR-based gene editing therapy

 by investigators at Children’s Hospital of Philadelphia in the context of an n-of-1 trial. Kim indicated that this story demonstrates the growing prominence of CRISPR gene editing in the field of cell and gene therapy. Although, he also pointed out that adeno-associated virus (AAV) vector-based gene therapy approaches seem to be having a resurgence of interest, despite some news outlets predicting that this modality is on the decline. Kim emphasized that the on-the-ground experience at ASGCT this year shows a different story about where the field is compared to the mainstream press. Afterwards, he also touched on Mission Bio’s collaboration efforts and stated that the company continues to be open to new partnership opportunities.

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

REFERENCES
1. World's first patient treated with personalized CRISPR gene editing therapy at Children’s Hospital of Philadelphia. News release. Children’s Hospital of Philadelphia. May 15, 2025. Accessed May 15, 2025.https://www.chop.edu/news/worlds-first-patient-treated-personalized-crispr-gene-editing-therapy-childrens-hospital

Videos

The CEO of Mission Bio discussed the company’s single cell assay platform in the context of the broader developing field cell and gene therapy field. 

Brian Kim, MBA, on Multiomic Single Cell Sequencing and the Ebbs and Flows of the Gene Therapy Field

“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 New York Times 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.”

Mission Bio is seeking to overcome limitations of bulk sequencing and standard single cell sequencing methods through the use of its Tapestri platform for single cell sequencing. At 

, held May 13 to 17, 2024, in New Orleans, LA, the company gave several presentations related to its platform and the ways in which it is contributing to the development of the field of cell and gene therapy.

 spoke with Brian Kim, MBA, the chief executive officer of Mission Bio, to learn more. Kim explained that the company offers a single-cell multiomic platform focused on validation for therapeutic and diagnostic purposes and highlighted the presentations the company gave at ASGCT this year. Kim also took a broader view and emphasized that despite current challenges in cell and gene therapy, the field is progressing, with recent news of patient successes reinforcing its potential. He pointed out that Mission Bio remains committed for the long haul, having supported over 17 clinical trials and working to expand adoption of its assays. Future plans for the company include adding targeted gene expression to their platform and seeking to foster more collaborations.

The CEO of Mission Bio discussed the company’s single cell assay platform in the context of the broader developing field cell and gene therapy field.

Brian Kim, MBA, on Multiomic Single-Cell Sequencing and the Ebbs and Flows of the Gene Therapy Field

The chief executive officer and cofounder of March Biosciences discussed the company’s trial design presentation at ASCO’s 2025 meeting.

Sarah Hein, PhD, on the Phase 2 Trial for CD5-Directed CAR-T MB-105 in R/R T-Cell Lymphoma

“If you're familiar with this indication, unfortunately once patients enter the r/r setting, there really is not a standard of care. These patients have about a less-than-20% survival over about 3 years, and that's because there's a handful of salvage therapies available to these patients, but not a lot else.”

Patients with relapsed/refractory (r/r) T-cell lymphoma currently have few options. As such, the unmet need for new therapies for this indication is quite substantial. One potential candidate to fulfill this role is MB-105, an investigational CD5-directed chimeric antigen receptor T-cell (CAR-T) therapy currently being evaluated for the treatment of r/r CD5-positive T-cell lymphoma. The therapy, which was invented at Baylor College of Medicine and is being developed by March Biosciences, is being evaluated in an ongoing phase 1 clinical trial (NCT03081910) that was launched in 2017 and a phase 2 clinical trial (NCT06534060) that recently launched on February 15, 2025.

2025 American Society of Clinical Oncology (ASCO) Annual Meeting

, held May 30 to June 3, in Chicago, Illinois, March Biosciences is giving a presentation on the design of the new phase 2 clinical trial. Ahead of the meeting, 

 sat down with Sarah Hein, PhD, the chief executive officer and cofounder of March Biosciences, to learn more.

Hein pointed out that MB-105 targets CD5, a protein expressed in 70% to 80% of peripheral and cutaneous T-cell lymphoma cases, making it broadly applicable. She also noted that the trial, which began enrolling patients earlier this year, uses a Simon 2-stage design and is being conducted at 12 sites across the United States. March Biosciences announced its first patient dosed about a month ago.

Hein highlighted the urgent need for innovative therapies in this space and encouraged physicians to refer eligible patients. She stressed the importance of reaching patients quickly. She also spoke about how while manufacturing and scale-up remain challenges, the company has built infrastructure to support current and future development. Hein additionally emphasized openness to new clinical sites and partnerships.

Jamie Jacobs, PhD

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