The phase 1/2 MARVEL-1 clinical trial (NCT04040049) of Freeline Therapeutics’ FLT190, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Fabry Disease, has dosed the first patient in its second cohort.

FLT190 utilizes an AAVS3 capsid and is intended to deliver a functional copy of GLA, the disease-targeted gene, into the liver cells, with the goal of producing greater than normal levels of alpha galactosidase (α-Gal A), the enzyme which is insufficient in patients with 

. Early data from the trial’s first cohort, which was announced in February 2022, indicated that FLT190 showed a potential dose-dependent increase in α-Gal A levels in the plasma, with 1 of the 2 treated patients having durable α-Gal A levels sustained for up to 2 years (though this patient restarted enzyme replacement therapy 6 weeks after dosing) and the other patient continuing to remain off enzyme replacement therapy for over 26 weeks after treatment (data cut-off: December 22, 2021).

In terms of safety, FLT190 was well-tolerated with it being noted that 1 of the 2 patients treated in the first cohort at the time of the data cut-off had experienced a case of transient transaminitis.

 A novel prophylactic immune management regimen introduced after this event may have prevented vector-related transaminitis in the other patient. In both of the patients, cases of myocarditis were reported at about 6 to 8 weeks following treatment. The cases were mild and transient and, on cardiac MRI, were not associated with enduring clinical sequalae.

“Freeline’s proprietary AAVS3 capsid has the potential to deliver transformative treatments for patients with challenging diseases including Fabry disease...,” Pamela Foulds, MD, chief medical officer, Freeline Therapeutics, said in a statement in February.

 “It is highly encouraging to observe early signals of durability and efficacy in MARVEL-1 in Fabry disease, with sustained expression up to 2 years as of our last data reading. Advancing the study will allow further exploration of the potential of FLT190 to provide a functional cure for Fabry disease, which may eliminate the need for enzyme replacement therapy and improve patient outcomes.”

The multicenter, adaptive dose-escalation and dose-expansion clinical trial is recruiting up to 15 male patients aged 18 years or older with confirmed diagnoses classic Fabry disease.

 Participants are required to have decreased plasma α-Gal activity and an estimated glomerular filtration rate (eGFR) of at least 60mL/min/1.73m2 at the time of screening; at least one of the characteristic features of classic Fabry disease; and less than 500 mg/g Urine Protein to Creatinine Ratio (UPCR) in a spot urine sample or less than 1g/24-hours of urinary protein in a 24-hour urine analysis. Patients’ platelet counts must beat least 100x10

. Participants in part 2 of the study must additionally have elevated plasma globotriaosylsphingosine (lyso-Gb3) levels at screening. Patients who have non-classical Fabry disease or presence of AAV neutralizing antibodies or antibodies to α-GLA, Replagal, or Fabrazyme will be excluded from the study. Additional exclusion criteria relate to patient health status and treatment history.

Part 1 of the study is a dose escalation phase for patients previously treated with enzyme replacement therapy or chaperone therapy, while part 2 is a dose expansion phase for patients who have not received previous treatment.

 Participants are treated with a single dose of FLT190 via slow intravenous infusion. Patients in the first cohort of the dose escalation phase received a dose of 7.5x10

 vg/kg, and patients in the second cohort, which is a mid-dose cohort, will receive 1.5x10

 vg/kg. The trial may ultimately include up to 4 dose-level cohorts in total. 

The study’s primary end point is the frequency of treatment-emergent adverse events. The trial will also evaluate efficacy via measures of α-Gal A activity levels and measures of clearance of globotriaosylceramide (Gb3) and lyso-Gb3 from plasma and urine. While participants in the first cohort were recruited from Germany, Italy, Norway, and the United Kingdom, the FDA recently cleared 

 to begin treating patients in the United States.

“We believe FLT190 has the potential to be a life-changing therapy for people with Fabry disease by providing durable enzyme activity above the normal range with a one-time treatment, and we are excited to have initiated this cohort in MARVEL-1,” Foulds said in a statement regarding the initiation of dosing in the second cohort.

 “We have multiple trial sites now open across five countries with more expected to open by year-end. We look forward to reporting updated safety and efficacy data in the first half of next year.”

1. Freeline initiates dosing of second cohort in MARVEL-1 trial of FLT190 gene therapy candidate for people with fabry disease. News release. Freeline Therapeutics Holdings plc. October 4, 2022. 

https://www.freeline.life/investors/newsroom/freeline-initiates-dosing-of-second-cohort-in-marvel-1-trial-of-flt190-gene-therapy-candidate-for-people-with-fabry-disease/

2. Freeline presents on its fabry and gaucher disease AAV-based gene therapies at the 18th annual Worldsymposium. News release. Freeline Therapeutics Holdings plc. February 8, 2022. 

https://www.freeline.life/investors/newsroom/freeline-presents-on-its-fabry-and-gaucher-disease-aav-based-gene-therapies-at-the-18th-annual-worldsymposium/

3. Hughes DA, Canaan-Kühl S, Barton S, Colliis R, Sherry N. Safety and efficacy of FLT190 for the treatment of patients with Fabry disease: Results from the MARVEL-1 Phase 1/2 clinical trial. Presented at: 18th Annual WORLDSymposium. February 7-11, 2022; San Diego, California.

Articles

The FDA recently cleared Freeline Therapeutics to begin treating patients in the United States.

Fabry Disease Gene Therapy Clinical Trial Moves Onto Dosing Second Cohort

A phase 1/2 clinical trial (NCT05360238) has dosed its first patient with MB-106 

, a chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas (B-NHL) and chronic lymphocytic leukemia (CLL).

The patient did not experience cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS). The trial is a multicenter, open-label trial that builds on the ongoing trial at Fred Hutchinson Cancer Center under the same investigational new drug application (IND). It is evaluating the safety and efficacy of MB-106, a first-in-class CD20-targeted, autologous CAR T cell therapy.

“The first clinical trial under Mustang’s IND is an important milestone in the ongoing development and evaluation of MB-106. Data presented at several prestigious medical meetings earlier this year from the initial, ongoing Phase 1/2 clinical trial at Fred Hutch show that MB-106 continues to demonstrate high efficacy and a favorable safety profile across patients with a wide range of hematologic malignancies. We look forward to providing updates on our multicenter MB-106 clinical trial as it progresses and anticipate reporting efficacy data in the fourth quarter of this year,” Manuel Litchman, MD, president and chief executive officer, Mustang, said, in a statement.

Recent interim data, as of September 9, 2022, from 28 patients treated in the ongoing, investigator-sponsored study, have continued to support MB-106's benefit. No patients experienced CRS or ICANS greater than grade 3. Overall response rate is 96% and complete response (CR) rate is 75% in patients with follicular lymphoma, CLL, diffuse large B-cell lymphoma, and Waldenstrom macroglobulinemia.

Mazyar Shadman, MD, MPH, on MB-106's Potential in Follicular Lymphoma

Twelve patients had a CR for over 12 months with 10 CRs still ongoing. The longest CR is 33 months, and 4 patients had a CR for over 2 years. Six patients with a partial response (PR) improved to ongoing CRs. Three patients had been previously treated with CD19 CAR T-cell therapy and all responded. Investigators also found that CAR-T persistence resulted in deepening responses after the initial 28-day assessment.

“We are excited to broaden the evaluation of MB-106 with this multicenter clinical trial under Mustang’s IND. To date, the data from the initial, ongoing clinical trial at Fred Hutch continue to demonstrate a high rate of complete and durable responses,” Mazyar Shadman, MD, MPH, study chair, associate professor and physician, Fred Hutch and University of Washington, added to the statement. “In addition, MB-106 has shown potential to treat patients in an outpatient setting and provide another immunotherapy option for patients treated previously with CD19-directed CAR T cell therapy.”

The trial is aiming to enroll 287 patients with CLL and B-NHLs, including mantle cell lymphoma. Patients must have evidence of CD20 expression. Phase 1 of the trial will assess escalating dose levels of MB-106 in a 3+3 design in 3 arms. Each arm will have up to 18 patients, with 6 at the maximum tolerated dose. The Safety Review Committee will assess 28-day data and the recommended phase 2 dose will be established. In the phase 2 portion, a total of 71 patients will participate with up to 20 patients in each arm. Up to an additional 51 patients may be added to each arm after an interim analysis.

Mustang Bio announces first patient treated in its multicenter phase 1/2 clinical trial of MB-106, a first-in-class CD20-targeted, autologous CAR T cell therapy to treat B-cell non-hodgkin lymphoma and chronic lymphocytic leukemia. News release. Mustang Bio. October 6, 2022. https://www.globenewswire.com/news-release/2022/10/06/2529538/0/en/Mustang-Bio-Announces-First-Patient-Treated-in-Its-Multicenter-Phase-1-2-Clinical-Trial-of-MB-106-a-First-in-Class-CD20-targeted-Autologous-CAR-T-Cell-Therapy-to-Treat-B-cell-Non-H.html

Interim data from an ongoing study demonstrate continuing efficacy in B-NHL, CLL, and FL. 

New, Multicenter Trial of MB-106 Doses First Patient 

The CardiAMP cell therapy showed clinical improvements in patients with 

, according to 2-year data from a phase 3 trial (NCT02438306).

CardiAMP is an autologous cell therapy developed by BioCardia. It is intended to be a comprehensive therapeutic treatment that includes a proprietary molecular diagnostic to determine eligibility of patients’ bone marrow cells, a point of care processing platform that prepares the cells at the patient’s bedside, and the proprietary Helix biotherapeutic delivery system to administer the therapy.

“The 2-year outcomes for patients with heart failure receiving the investigational CardiAMP Cell Therapy in the roll-in cohort showed clinical improvement with 100% survival over two years. These outcomes surpassed our expectations in terms of patient benefit across prespecified primary and secondary endpoints,” Peter Altman, PhD, president and chief executive officer, BioCardia, said in a statement.

 “While this smaller cohort is not a head-to-head comparison, current state of the art therapies for these patients which have been successful in slowing disease, are assessed by us to have 79.9% survival after 2 years.”

These data were presented at Heart Failure Society of America meeting in Washington, D.C by Peter Johnston, MD, assistant professor of medicine and site principal investigator, Johns Hopkins University. The study has enrolled around 114 patients as of the conference call, however, data reported is from 10 patients in a roll-in cohort with 2 years of follow-up. The study’s total enrollment target is 260. Patient characteristics were typical of patients with New York Heart Association (NYHA) Class 2 and 3 and ischemic heart failure.

Daniel Judge, MD, on Translational Gene Therapy for Cardiac Disorders

“We have had our most recent safety data monitoring committee their recommendations were that the study 

 and that no change or actions were required. They did ask that we provide an updated statistical analysis plan that would include an adaptive design,” co-principal investigator Carl Pepine, MD, professor, cardiovascular medicine, University of Florida, said in a conference call.

Investigators observed no serious adverse events (AEs) related to treatment. All 10 patients completed 24 months of follow-up and 2-year survival was 100%. Patients had minimal changes in guideline-directed medical therapy over the study period. Clinical improvements were demonstrated by 6-minute walk distance test (6MWT) scores at 6 months (6% improvement; 28.5 m; 

 = .01) and 24 months (8% improvement; 31.0 m; 

“The remarkable thing is that in patients with very serious heart disease and very impaired cardiac function, there were no deaths in the first 1 years of follow up and there were only 2 hospitalizations out of the 10 patients in the 2 years... 1 was related to heart failure progression and the other patients had multiple return admissions and acute myocardial infarction,” Pepine added.

Quality of life scores were also positive, with 70% of patients reporting improved or stable scores over 24 months.

 Half of patients improved by at least 1 NYHA class, with 4 at class 1, 20% had an unchanged class and 30% deteriorated from class 2 to 3 at 24 months. Median left ventricular ejection fraction (LVEF) improved at months 6, 12, and 24, compared to baseline. Echocardiography also showed recruitment of previously akineticleft ventricular wall segments at month 24 compared to baseline.

“There’s still a great unmet need. Looking at the more recent studies of the various drugs being used, such as the PARADIGM-HF study, EMPEROR, and DAPA-HF studies... you can still see there is an unmet need with around a 13% death rate still exists...our study will follow patients after 24 months and how these numbers unfold in this patient population will still be determined,” co-principal investigator Amish Raval, MD, professor, cardiovascular medicine, University of Wisconsin, said during the call.

1. BioCardia presents positive CardiAMP cell therapy heart failure trial two-year data at HFSA Annual Meeting. News release. BioCardia. October 3, 2022. 

https://finance.yahoo.com/news/biocardia-presents-positive-cardiamp-cell-110000572.html


2. CardiAMP Cell Therapy Trial for Heart Failure: Clinical Leadership and Management Call. Webcast. BioCardia. October 5, 2022. https://event.choruscall.com/mediaframe/webcast.html?webcastid=9NhAJ6lm

The new, 2-year data is from a roll-in cohort of 10 patients presented at the HFSA 2022 meeting. 

CardiAMP Cell Therapy Yields 100% Response Rate in Heart Failure

“We're looking at cell therapies for the treatment of autoimmune disease. Our lead indication is a CAR T so it's very similar to the ones that have been in the clinic for oncology, and we are looking at using them starting with the autologous platform... We are also looking at regulatory T cells which can suppress the immune response.”

, held September 19-22 in Boston, Massachusetts, facilitated conversations between industry, academia, and patient advocacy groups ongoing challenges and work to be done with cell therapies in oncology and other indications and showcased emerging data and technologies in these spaces.

One such company in attendance was Kyverna Therapeutics, which is developing cell therapies for autoimmune indications with candidates that will soon be advanced into the clinic. Its lead technology is a chimeric antigen receptor T cell (CAR T), while asynthetic regulatory T cell (synReg T cell) for B-cell autoimmune disorders is in the discovery phase. Both technologies are developed by use of Kyverna’s SmarTcell genetic engineering platform.

spoke with Karen Walker, chief technology officer, Kyverna, to learn more about the company’s technologies and platforms. She also discussed challenges in the space and how Kyverna is aiming to meet them.

Videos

Karen Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies.  

Treating Autoimmune Diseases With Smarter Cell Therapies 

Atsena Therapeutics’ ATSN-101 (previously referred to as SAR439483), an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Leber Congenital Amaurosis caused by biallelic mutations in 

 (LCA1), has demonstrated promising safety and efficacy in a phase 1/2 clinical trial (NCT03920007), according to data presented at the 

American Academy of Ophthalmology (AAO) annual meeting

 which took place from September 30 – October 1, 2022, in Chicago, Illinois.

ATSN-101 utilizes an AAV5 capsid and is delivered via subretinal injection. It is intended to provide photoreceptors with a functional copy of 

, the disease-targeted gene. Among the patients treated with the highest dose in the trial, significant improvement was reported in full-field stimulus testing (FST) across the 3 colors that were tested with dark adapted FST. Clinically meaningful improvements in multi-luminance mobility testing (MLMT) were reported in 3 of 4 participants tested. In terms of safety, no therapy-related serious adverse events (SAEs) were reported. Two cases of ocular inflammation were reported, both being grade 2 and resolving with administration of steroid treatment.

“[I]mportantly, [the majority of patients with 

] have a normal retinal thickness,” presenter Christine Nichols Kay, MD, Atsena Therapeutics, said during her presentation.

 “We can see preserved photoreceptor structure and this does increase the odds of success with retinal gene therapy.”

The ages of the 15 patients who were treated in the clinical trial ranged from 12 years to 76 years (median, 21). The group included 10 female patients and 5 male patients, and racial demographics included 67% white patients and 20% Asian patients, with the race of the remaining patients unreported. The baseline median best corrected visual acuity (BCVA) (logMAR) in the study eye ranged from 0.72 to 4 (median, 1.32).  Patients were required to have the outer nuclear layer identifiale on central retina optical coherence tomography in order to be included in the study.

The patients were divided across 5 cohorts, each of which contained 3 patients. The first 3 cohorts (20/200 BCVA or worse) were treated in the dose escalation phase, with cohort 1 receiving a dose of 1.0x10

 vg/eye of ATSN-101, cohort 2 receiving a dose of 3.0x10

 vg/eye. All patients in these cohorts were older than 18 years of age. The 1.0x10

 vg/eye dose was selected for the expansion phase (BCVA 20/80 or worse), which treated patients older than 18 years in cohort 4 and patients aged 18 years and younger in cohort 5.

“Interestingly, there was [a] 76-year-old in this trial and this patient actually did quite well with a robust FST improvement signal,” Kay added in the presentation.

The BCVA response among the patients treated was variable. However, all 4 patients tested with Spark Therapeutics' MLMT, all of whom received the highest dose, showed either an improvement to the maximum MLMT score of 6 (n=1) or an improvement of greater than 2 levels (n=3). These improvements were compared to baseline when possible and to the untreated eye when baseline data were unavailable.

All 3 SAEs reported were related to the surgical procedure and included a macular hole in one patient and endopthalmitis and retinal detachment in another patient. The 2 cases of ocular inflammation mentioned above included a case of subretinal inflammation and a case of vitritis. Kay noted that the 2 patients who experienced these cases of ocular inflammation still showed FST improvements. In total, 56 treatment-emergent AEs were reported, and 52 of these were related to the surgical procedure. No patients dropped out of the study due to AEs.

1. Kay CN, Yang P, Cideciyan A, et al. Safety and Efficacy of SAR439483 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1). Presented at: AAO annual meeting. September 30 – October 1, 2022; Chicago, Illinois.

2. Atsena Therapeutics announces late-breaker presentation at the American Academy of Ophthalmology 2022 Annual Meeting. News release. Atsena Therapeutics. September 21, 2022. https://atsenatx.com/press-release/atsena-therapeutics-announces-late-breaker-presentation-at-the-american-academy-of-ophthalmology-2022-annual-meeting/

Significant improvements were reported in full-field stimulus testing.

Gene Therapy Shows Promise in Leber Congenital Amaurosis 

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. 

Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

1. Allogeneic CAR T Recognized for Cutaneous T-Cell Lymphomas

The FDA has granted regenerative medicine advanced therapy (RMAT) designation to CRISPR Therapeutics' allogeneic chimeric antigen receptor (CAR) T-cell therapy for the treatment of CTX130 cutaneous T-cell lymphomas (CTCL), specifically for the treatment of mycosis fungoides and Sézary syndrome (MF/SS).

2. Cellenkos to Try T Regulatory Cell Approach in ALS

The FDA has cleared Cellenkos’ investigational new drug application (IND) for CK0803, a T regulatory (Treg) cell therapy for the potential treatment of amyotrophic lateral sclerosis (ALS).

3. Sarepta’s DMD Gene Therapy Goes In Front of FDA With BLA Submission

Sarepta Therapeutics has submitted its biologics license application (BLA) for SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy developed in collaboration with Roche, for the potential treatment of ambulant patients with Duchenne muscular dystrophy (DMD).

4. KIR-CAR T-Cell Therapy Recognized for Mesothelioma Treatment

The FDA has granted orphan drug designation to Verismo Therapeutics’ SynKIR-110, a killer immunoglobulin-like receptor - chimeric antigen receptor (KIR-CAR) T-cell therapy, for the treatment of mesothelin-expressing mesothelioma.

5. Lineage Cell Therapeutics Expands Manufacturing, R&D Facilities 

With positive data continuing to roll in from its RG6501 (OpRegen) platform, Lineage Cell Therapeutics is investing in an expansion of its GMP manufacturing facility in Israel and opening a new R&D facility in California to support new cell therapy ventures in blindness and hearing loss. 

6. GPRC5D-Targeted CAR-T Shows Promise in Heavily Pre-treated Multiple Myeloma

MCARH109, a chimeric antigen receptor (CAR) T-cell therapy targeted at G protein–coupled receptor, class C, group 5, member D (GPRC5D), yielded responses and demonstrated that GPRC5D is an active immunotherapeutic target in a phase 1 clinical trial (NCT04555551) for patients with heavily pretreated multiple myeloma (MM) carried out at Memorial Sloan Kettering Cancer Center (MSKCC), according to results published in 

7. Modified Immune Cell Therapy Induces Kidney-Donor-Specific Immunosuppression

MIC-Lx cell therapy (TolerogenixX) induced long-term donor-specific immunosuppression and yielded a significant increase in regulatory B lymphocytes in kidney transplant recipients, according to 3-year follow-up data from a phase 1 study (NCT02560220).

8. BioMarin Resubmits BLA for Hemophilia A Gene Therapy Val-Rox

BioMarin has resubmitted the biologics license application (BLA) to the FDA for valoctocogene roxaparvovec (val-rox; BMN-270), an adeno-associated virus (AAV)-based gene therapy intended to treat hemophilia A.

9. Omidubicel Improves Survival in Hematologic Malignancies With HSCT

The umbilical cord blood (UCB) cell therapy omidubicel (NiCord; Gamida Cell) improved survival in patients with hematological malignancies receiving allogeneic hematopoietic stem cell transplantation (HSCT).

10. Allogeneic Anti-CD7 CAR-T Demonstrates Promising Results in Hematological Malignancies

Bioheng Biotech’s RD13-01, an investigational allogeneic CD7-targeted chimeric antigen receptor T-cell (CAR-T) product with genetic modifications to resist fratricide, graft versus host disease (GvHD), and rejection, demonstrated promising safety and efficacy data in a phase 1 clinical trial (NCT04538599) for patients with CD7+ hematological malignancies, according to results published in 

11. Wet AMD Gene Therapy Reduces Anti-VEGF Treatment Burden

RGX-314, REGENXBIO’s investigational gene therapy for wet age-related macular degeneration (AMD), was well-tolerated and reduced treatment burden in participants treated in the phase 2 AAVIATE trial (NCT04514653).

12. Solid Bio Adds Friedreich Ataxia to Pipeline With Aavanti Acquisition

Solid Biosciences has acquired AavantiBio, a company whose lead program, AVB-202, is a gene therapy candidate in preclinical studies for Friedreich’s ataxia.

13. Gene Therapy Yields Improvements in Danon Disease

The gene therapy RP-A501 (Rocket Pharmaceuticals) was well-tolerated and yielded functional improvements in adult and pediatric patients with Danon disease, according to data from a phase 1 trial (NCT03882437) presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022.

14. Gene Therapy Trial Makes Headway in Canavan Disease

A phase 1/2 clinical trial (NCT04833907) of Myrtelle’s rAAV-Olig001-ASPA (MYR-101), an investigational recombinant adeno-associated virus (rAAV) vector-based gene therapy intended to treat Canavan disease (CD), has completed dosing of the first 8 patients.

15. B-NHL Cell Therapy Cleared for Trials

The FDA has granted IND clearance for a phase 1/2 dose escalation study of Oncternal Therapeutics’ chimeric antigen receptor (CAR) T therapy ONCT-808 for treating aggressive B cell non-Hodgkin’s lymphoma (B NHL).

16. Frontera Therapeutics' FT-001 IND Cleared by China's Center for Drug Evaluation

An IND for FT-001, an investigational gene therapy intended to treat inherited retinal degenerations, was previously cleared by the US FDA earlier this year.

17. Krystal Biotech's Dystrophic Epidermolysis Bullosa Gene Therapy Gets Positive Opinion on Pediatric Investigation Plan from the EMA's PDCO

The European Medicines Agency (EMA) Pediatric Committee (PDCO)'s positive opinion clears the way for a European Marketing Authorization Application (MAA) for beremagene geperpavec (B-VEC), which is expected in late 2022.

18. New Bill Could Expand Access to Blood and Blood-derived Biomaterial Storage Services for Americans With Flexible Spending and Healthcare Savings Accounts

The "Access to Future Cures Act" (H.R.8912) bill, which was introduced to Congress in September 2022, intends to give Americans with Health Savings Accounts (HSA), Health Reimbursement Arrangements (HRA), and Flexible Spending Accounts (FSA) additional options in light of the rapidly expanding field of approved cell and gene therapies.

19. Circularis Acquired by Ginkgo Bioworks

Ginkgo Bioworks' plans include the use of Circularis' promoter screening platform in an attempt to identify promoters that modulate expression in tumor micro-environments for the purpose of cell therapy development.

20. BCMA-targeted Cell Therapy CARVYKTI Approved in Japan for R/R Multiple Myeloma

The CARVYKTI (ciltacabtagene autoleucel) approval is restricted to patients who have no history of treatment with CAR-positive T-cell infusion therapy targeting BCMA, who have peviously received therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody, and who are relapsed/refractory (r/r) to their most recent therapy.

21. Real Endpoints Marketplace, Bluebird Bio Partner to Enable Additional Patient Access to Zynteglo

The collaboration will provide options for an outcomes-based agreement for ZYNTEGLO to plans that cover almost 16 million Americans.

22. Pfizer Licenses Next-Generation AAV Capsid from Voyager Therapeutics

Pfizer intends to utilize the capsid in its development of an investigational gene therapy for an undisclosed rare neurologic disease. The licensing is part of an agreement announced late last year.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies. 