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Lysosomal Disorders

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The senior researcher at Seattle Children’s discussed the development and validation of A2-CAR-CISC EngTreg cells.

Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells

The senior researcher at Seattle Children’s discussed advantages of engineered Tregs compared with natural Tregs. 

Subhash Tripathi, PhD, on Developing Safe, Specific Engineered Treg Cell 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 

Chimeric antigen receptor T-cell (CAR-T) therapies have provided important new treatment options for patients with hematologic malignancies such as diffuse large B-cell lymphoma (DLBCL). Although, recent research has suggested that CAR-T therapies may carry a risk of causing second primary malignancies. 

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

, held May 30 to June 3, in Chicago, Illinois, Deevyashali Parekh, MBBS, an internal medicine resident at SUNY Upstate Medical University Hospital, presented new research on the prevalence of second primary malignancies following treatment with FDA-approved CAR-T therapies for DLBCL based on electronic medical record (EMR) data. In an interview with 

, Parekh discussed the key findings and their implications, emphasizing that more data is still needed on this topic.

 Can you give some background context about your presentation?

 Around 40% of patients with DLBCL relapse or don't respond to first line therapy. In this setting, 3 approved CAR T-cell treatments have shown promising results. Longitudinal studies are required for long-term outcomes and adverse effects with these treatments, however a body of data exists showing a notable increase in second primary malignancies after these treatments.

What are some of the key findings you presented?

In this study using a deidentified database of EMR data, our study demonstrated an increase in risk of developing myelodysplastic syndrome and acute myeloid leukemia compared to propensity matched patients with relapsed/refractory DLBCL not receiving CAR-T therapy.

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

The big-picture implication of this data is that while CAR-T therapy is exhibiting robust responses in patients who have progressed on 2 or more lines of prior treatment, longitudinal studies on long-term follow-up after this treatment are necessary to understand the risk of developing second primary malignancies and weigh the risk versus benefit of these novel therapies.

What are the next steps for this research?

CAR-T treatments are receiving approval and wider usage in recent times. It has been established in smaller cohort studies that hematological second primary malignancies develop in around 12 months after CAR-T therapy and solid tumor malignancies take greater than 24 months. Balanced, propensity matched studies of longer-term follow-up is needed in this cohort to establish whether the risk of second primary malignancies is statistically significantly increased and whether this is due to longer survival in this patient population versus a risk from CAR-T therapy.

This transcript has been edited for clarity.

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

REFERENCE
1. Parekh D. Secondary primary malignancies (SPMs) with CAR-T cell therapy in RR-DLBCL from real-world data. Presented at the 2025 ASCO Annual Meeting, held May 30 to June 3, in Chicago, Illinois. Abstract #7066

The internal medicine resident at SUNY Upstate Medical University Hospital discussed the implications of findings from a deidentified database of EMR data.

Deevyashali Parekh, MBBS, on Evaluating the Prevalence of Second Primary Malignancies After CAR T-Cell Therapy

This is the second part of an interview with Shahzad Raza, MD. For the first part, 

The hematologist/oncologist at the Cleveland Clinic discussed next steps after early promising results were presented at ASCO’s 2025 meeting.

Shahzad Raza, MD, on Future Plans for CAR-T NXC-201 in R/R Light Chain Amyloidosis

“This is very different than continuing to bring patients to the clinic every week do therapy. A treatment holiday—freedom from more treatments —at least for our timeline, I think would be an important point for these patients. I think the future studies should focus on treatment holidays...”

Immix Biopharma and its subsidiary Nexcella are currently developing NXC-201, an investigational autologous BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy, for the treatment of relapsed/refractory (r/r) light chain (AL) amyloidosis. Notably, early data from the phase 1b/2 NEXICART-2 clinical trial (NCT06097832) evaluating the therapy in the United States were recently presented at 

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

, spoke with Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic who coauthored the study presented at ASCO, about 

. Afterwards, Raza was asked about future plans for evaluating the CAR-T product in AL amyloidosis and unanswered questions and challenges that still need to be addressed. Raza emphasized that while patients with advanced cardiac amyloidosis (stage 3b) were excluded from the current trial, future studies should explore its use in this high-risk group if safety and efficacy continue to hold. Raza stressed the urgent need for effective second-line treatments and suggested offering CAR-T therapy earlier in the disease course. He highlighted the potential for “treatment holidays,” where patients could avoid continuous therapies after receiving CAR-T, which could improve quality of life.

Raza also spoke about challenges in patient eligibility for treatment, emphasizing the importance of being free from active infections and other disease. He then mentioned his excitement about another presentation at ASCO, which focused on the use of teclistamab, a Bispecific T-cell engager (BiTE), for AL amyloidosis. He spoke about how teclistamab and CAR-T may allow for patients to have multiple treatment options for r/r AL amyloidosis in the future.

REFERENCE
1. Landau H. Safety and efficacy data from Nexicart-2, the first US trial of CAR-T in R/R light chain (AL) amyloidosis, NXC-201. Presented at the 2025 ASCO Annual Meeting, held May 30 to June 3, in Chicago, Illinois. Abstract #7508

Videos

Shahzad Raza, MD, on Future Plans for CAR T-Cell Therapy NXC-201 in R/R Light Chain Amyloidosis

Bristol Myers Squibb's GPRC5D-targeted chimeric antigen receptor (CAR) T-cell therapy arlocabtagene autoleucel (arlo-cel) is currently being evaluated for the treatment of multiple myeloma (MM) in a phase 2 clinical trial (QUINTESSENTIAL; NCT06297226) and in a phase 1 clinical trial (NCT04674813). A biomarker analysis of data from the phase 1 trial, comparing it to data from the phase 2 KarMMa clinical trial (NCT03361748) evaluating BCMA-directed CAR-T idecabtagene vicleucel (ide-cel) for the treatment of MM, was presented at 

, Kristina Jordahl, PhD, a principal scientist at Bristol Myers Squibb who presented the data at ASCO, discussed the results and their implications. She noted that the data indicates that the GPRC5D-targeted CAR-T may be better able to spare normal plasma cells while maintaining a similar or better efficacy to ide-cel at destroying MM cells.

 This work builds on evidence that GPRC5D has minimal expression on normal plasma cells, despite strong expression on MM cells. Prior research has also described greater depletion of mature B-cells and normal plasma cells from BCMA-targeted as compared to GPRC5D-targeted T-cell engager therapy.

What are some of the key data points you are presenting?

After arlo-cel treatment, we observed that the uninvolved free light chain (FLC) does not clear to the limit of detection in 33% of patients and, when cleared, arlo-cel had a median time to return of 3.3 months with circulating levels that are significantly higher at months 4 and 5. This possible preservation of normal plasma cell function with similar or better targeting of MM cells suggests that arlo-cel has the potential for lower burden of infections while also reducing use of infection-related interventions.

We characterized an observation that FLC profiles from patients treated with arlo-cel are distinct from those from patients treated with BCMA-targeting CAR-T. The results are presented as biomarker-based evidence of higher levels of normal plasma cells with a GPRC5D target as compared to a BCMA target. If clinical evidence continues to support greater antitumor specificity and preservation of humoral immunity, arlo-cel may have the potential to achieve lower rates of hypogammaglobulinemia and infections compared to BCMA-targeting therapies, with fewer interventions.

We look forward to continuing to monitor the observation that targeting GPRC5D with CAR-T results in preservation of the humoral immune system in ongoing phase 2 and phase 3 clinical trials. Possible next steps also include additional data to support the preservation of the humoral immune system, including direct measurement of the B-cell and normal plasma cell levels after treatment.

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

Arlo-cel is a GPRC5D-targeted CAR T-cell therapy with impressive efficacy and a manageable safety profile. Advantages to targeting the GPRC5D antigen with CAR-T in MM may include lower severity of on-target off-tumor toxicity and sparing of the humoral immune system.

REFERENCE
1. Jordahl K. New biomarker analysis following treatment with BMS investigational novel cell therapy targeting GPRC5D, arlocabtagene autoleucel (arlo-cel), in patients who have received at least 3 lines of prior therapy. Presented at the 2025 ASCO Annual Meeting, held May 30 to June 3, in Chicago, Illinois. Abstract #7530

The principal scientist at Bristol Myers Squibb discussed a biomarker analysis she presented at ASCO's 2025 meeting.

Kristina Jordahl, PhD, on Arlo-Cel's Potential to Destroy Multiple Myeloma Cells While Preserving Immune Function

This is the second part of an interview with Laura Aguilar MD, PhD. For the first part, 

The chief medical officer of Diakonos Oncology discussed the broader implications of a positive data readout from a trial for the company’s autologous dendritic cell immunotherapy.

Laura Aguilar MD, PhD, on the Potential to Apply Immunotherapy DOC1021 to a Broad Array of Tumor Types

“This could be used for any tumor where we can get a tumor sample and a patient can undergo a leukapheresis procedure to give us their peripheral blood cells. There's a huge opportunity here for a platform technology that could really benefit a lot of patients with a lot of different tumor types. Needing to do a separate trial for each tumor indication is expensive. More funding to be able to test this in more tumor indications will really allow it to, I think, reach its full potential.”

2025 American Society of Clinical Oncology (ASCO) Annual Meeting

, held May 30 to June 3, in Chicago, Illinois, Diakonos Oncology is presenting data from a phase 1 clinical trial evaluating dubodencel (also known as DOC1021), an investigational autologous dendritic cell immunotherapy made from patients’ own tumor cells and peripheral blood mononuclear cells, for the treatment of gliobastoma. Notably, the results showed that the immunotherapy was well tolerated across all 4 dose levels tested and an 88% 12-month survival rate was observed for a group of newly diagnosed patients (n = 16), which was notably higher than what would be expected for the patient population with standard of care treatment.

 held a discussion with Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology, 

 In the context of this conversation, Aguilar also went over the broader implications of the new data, and discussed potential future plans to apply the DOC1021 platform to a broader range of tumor types. Aguilar pointed out that a separate phase 1 clinical trial for the immunotherapy is already ongoing at Baylor College of Medicine in pancreatic cancer and pancreatic adenocarcinoma and that Diakonos is getting ready for an additional trial in refractory melanoma. She also touched on how work is currently being done aimed at making the manufacturing process for DOC1021 more efficient.

Subhash Tripathi, PhD

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