Noah Stansfield
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Articles by Noah Stansfield
Cell Therapy TG-C Misses Primary Endpoints in Phase 3 Knee Osteoarthritis Trial
Kolon TissueGene announced topline results from ACTiVION-II (NCT03291470), the first of two US phase 3 trials evaluating TG-C (TissueGene-C), an allogeneic cell and gene therapy for knee osteoarthritis.¹ The randomized, double-blind, placebo-controlled trial missed both co-primary endpoints, change from baseline in VAS pain and WOMAC total score at month 12, and none of the key secondary endpoints were met.
FDA Approves Tregzi for Matched Donor HSCT in Hematologic Malignancies
The FDA approved allogeneic regulatory T cell-based immunotherapy with hematopoietic stem and progenitor cell (HSPC) and T cells-vldq (Tregzi; Orca Bio) on June 30, 2026, for use in matched donor hematopoietic stem cell transplantation (HSCT) with a myeloablative preparative regimen, for hematopoietic and immunologic reconstitution, and to improve chronic graft-versus-host disease (cGVHD)-free survival in adults with hematological malignancies.1 Tregzi received regenerative medicine advanced...
Around the Helix: Cell and Gene Therapy Company Updates – May 27, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Evaluating Miv-Cel for Stiff Person Syndrome
This interview was originally published on our sister site, NeurologyLive®. Stiff person syndrome (SPS) is a rare, chronic autoimmune neurological condition characterized by progressive rigidity of the axial musculature and limbs, along with episodic, stimulus-triggered muscle spasms that can profoundly compromise a patient's ability to walk and maintain quality of life.
Around the Helix: Cell and Gene Therapy Company Updates – May 20, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Phase 3 HOPE-3 Data Reinforce Skeletal and Cardiac Benefits of Deramiocel in Duchenne Muscular Dystrophy
New phase 3 findings presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting further strengthened the case for deramiocel, an investigational allogeneic cell therapy from Capricor Therapeutics, as a potential treatment for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy and progressive skeletal muscle decline.1 The data come at a pivotal regulatory moment for the therapy, with the FDA currently reviewing the biologics license application (BLA) and a...
Assessing Miv-Cel for Myasthenia Gravis
This interview was originally published on our sister site, NeurologyLive®. CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy has emerged as a potentially transformative approach in antibody-mediated autoimmune neurological diseases, including myasthenia gravis (MG). The underlying mechanistic premise involves achieving deep depletion of CD19-expressing B-cells through a single treatment course, with the aim of producing a lasting immune reset.
ASGCT 2026: GS-100 Gene Therapy Demonstrates Early Activity in NGLY1 Deficiency
Interim findings from the ongoing first-in-human phase 1/2/3 trial of GS-100 (Grace Sciences), an investigational intracerebroventricular (ICV) AAV9 gene therapy for NGLY1 Deficiency, showed early improvements in motor and cognitive function among treated children, alongside a dose-dependent safety profile that informed dose selection for the pivotal portion of the study.¹ Presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting by Becky Schweighardt, PhD, and...
ETX101 Gene Therapy Shows Early Developmental, Seizure Benefits in Dravet Syndrome
Interim findings from the ongoing POLARIS phase 1/2 program suggest that Encoded Therapeutics’ investigational gene therapy ETX101 may produce substantial seizure reductions and early neurodevelopmental improvements in young children with SCN1A-positive Dravet syndrome, supporting continued advancement of the program as it enters pivotal-stage development.1 Presented at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, the updated data included outcomes from 21...
Around the Helix: Cell and Gene Therapy Company Updates – May 13, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Beacon Therapeutics Reports Sustained 12-Month Visual Gains With Laru-Zova in XLRP
This story first appeared on Ophthalmology Times, a sister publication to CGTLive. Beacon Therapeutics has reported positive 12-month safety and efficacy findings from the phase 2 DAWN trial evaluating laruparetigene zovaparvovec (laru-zova) in patients with X-linked retinitis pigmentosa (XLRP). The data were presented during an oral session at the 2026 Association for Research in Vision and Ophthalmology Annual Meeting, held May 3-7 in Denver, Colorado.
Thomas Crawford, MD, on High-Dose Nusinersen and the Future of SMA Treatment
“…There's really no reason not to go forward with the 28 milligrams. It is the same price with 1 exception: the the new protocol starts with a single dose at 50 milligrams, as opposed to the continuous dose at 28… So the first loading dose is more expensive… No reason not to do it.” The FDA approved an updated higher-dose formulation of the antisense oligonucleotide therapy nusinersen (Spinraza; Biogen) for spinal muscular atrophy (SMA) on March 30, 2026.
Around the Helix: Cell and Gene Therapy Company Updates – May 6, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Srikanth Muppidi, MD, on Evaluating Miv-Cel in Myasthenia Gravis
“…I'm optimistic for the field that CAR-T certainly offers a new paradigm of how we would treat refractory antibody-mediated diseases.
Around the Helix: Cell and Gene Therapy Company Updates – April 29, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Amanda Piquet, MD, FAAN, on Assessing Miv-Cel in Stiff Person Syndrome
“Miv-cell demonstrated efficacy and safety in SPS… We showed videos… of patients using a walker at baseline, and essentially putting that walker aside and being able to walk quickly and normally down the hallway.
Amanda Piquet, MD, FAAN, on Assessing Miv-Cel in Stiff Person Syndrome
“Miv-cell demonstrated efficacy and safety in SPS… We showed videos… of patients using a walker at baseline, and essentially putting that walker aside and being able to walk quickly and normally down the hallway.
Lonvo-z Advances Toward Approval as First In Vivo CRISPR Therapy for Hereditary Angioedema
Intellia Therapeutics has initiated a rolling biologics license application (BLA) submission to the FDA for lonvoguran ziclumeran (lonvo-z), an investigational one-time CRISPR-based gene editing therapy for hereditary angioedema (HAE), following phase 3 data showing substantial reductions in attack rates and treatment burden.¹ The application is supported by topline results from the global phase 3 HAELO trial, which met its primary and all key secondary endpoints, marking what could become...
Regeneron’s Nets Accelerated Approval From FDA for Otoferlin-Related Hearing Loss Gene Therapy Lunsotogene Parvec
The FDA has granted accelerated approval to Otarmeni (lunsotogene parvec-cwha), an adeno-associated virus (AAV) vector–based gene therapy, for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss associated with biallelic pathogenic variants in the OTOF gene.1 Eligible patients must have preserved outer hair cell function and no prior cochlear implant in the affected ear.
Around the Helix: Cell and Gene Therapy Company Updates – April 22, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Reimagining SMA Treatment: Salanersen and the Push for Durable, Low-Frequency Therapies
This article originally appeared on NeurologyLive, a sister publication of CGTLive. Spinal muscular atrophy (SMA) care has undergone a major transformation over the past decade, driven by therapies that restore survival motor neuron (SMN) protein and significantly alter disease trajectories.
Around the Helix: Cell and Gene Therapy Company Updates – April 15, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Around the Helix: Cell and Gene Therapy Company Updates – April 1, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more. Nearly 10 years after its original approval, the FDA has approved a new higher dose strength for nusinersen (Spinraza; Biogen) as a treatment for patients with spinal muscular atrophy (SMA).
Around the Helix: Cell and Gene Therapy Company Updates – March 25, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Angela Lek, PhD, on the Scientific Priorities that Shaped the 2026 MDA Conference
“We're always covering the cutting edge topic—what’s important to have conversations around—not just the readouts for clinical trials, but the really exciting technologies that are emerging around the corner.” The Muscular Dystrophy Association (MDA), an organization dedicated to serving the community of patients with neuromuscular disorders, including muscular dystrophies and other types of neuromuscular disease, holds its Clinical & Scientific Conference each year to bring together...
Around the Helix: Cell and Gene Therapy Company Updates – March 18, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more. At the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 8-11 in Orlando, Florida, CGTLive® sat down with Jerel A.
John F. Brandsema, MD, on the Real-World Use of Givinostat in DMD
"We’re all enthusiastic in the Duchenne community that we have more and more options to consider for treatment of the disease.
Jerel A. Banks, MD, PhD, on Evaluating Gene Therapy BB-301 for OPMD-Related Dysphagia
"We've seen benign safety and clinically meaningful improvements in swallowing function.
Around the Helix: Cell and Gene Therapy Company Updates – March 11, 2026
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
John F. Crowley, JD, MBA, on the Future of Neuromuscular Care
Increasingly, we will be developing therapies tailored to the individual based on their own genetic sequence. That is where medicine is going. Precision medicine will become more and more central to how we care for patients. Over the past few decades, progress in biotechnology, genomics, and translational science has significantly altered the therapeutic landscape for individuals with rare and neuromuscular disorders.
FDA Peels Back CRL on Capricor's DMD Cardiomyopathy Cell Therapy Deramiocel
This article was originally published on our sister site, NeurologyLive®.
Phase 2 FORWARD-53 Trial Reports Sustained Dystrophin Expression With WVE-N531 in Duchenne Muscular Dystrophy
The investigational exon-skipping therapy WVE-N531 (Wave Life Sciences) produced sustained increases in dystrophin levels and early indications of functional improvement in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping, according to 48-week findings from the phase 2 FORWARD-53 study.1 The data were presented at the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 8-11 in Orlando, Florida.
Around the Helix: Cell and Gene Therapy Company Updates – March 4, 2026 | CGTlive®
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Practical Considerations for Implementing SCD Gene Therapy Into Clinical Use
Sickle cell disease (SCD) gene therapy products, namely Vertex Pharmaceuticals' and CRISPR Therapeutics’ gene editing therapy exagamglogene autotemcel (exa-cel; marketed as Casgevy) and bluebird bio’s gene addition therapy lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia), have been commercially approved in the United States since late 2023.
Robin E. Miller, MD
The director of the Sickle Cell Disease Program at Nemours Children’s Hospital discussed what clinics need in order to start administering gene therapy effectively.
Around the Helix: Cell and Gene Therapy Company Updates – February 25, 2026 | CGTlive®
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more. CGTLive® spoke with Robin E.
Robin E. Miller, MD, on Integrating Gene Therapy into Clinical Care for Sickle Cell Disease
“It's a very new therapy and the press has been very positive, and so families often don't understand how difficult the process is going to be. As such, we need to explore all the options that the patients have.
MDA’s Upcoming 2026 Clinical & Scientific Conference
The Muscular Dystrophy Association (MDA) is an organization dedicated to both providing state-of-the-art care for patients with neuromuscular diseases and to advancing research related to the development of new therapies for neuromuscular disease indications. Each year, the organization holds the MDA Clinical & Scientific Conference to bring together various experts and stakeholders to share and discuss the latest findings in neuromuscular disease research.
Around the Helix: Cell and Gene Therapy Company Updates – February 18, 2026 | CGTlive®
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Barry J Byrne, MD, PhD, on MDA’s Upcoming 2026 Clinical & Scientific Conference
"There will be some tracks on cell and gene therapy throughout the meeting. Certainly, we've faced a number of safety events that have come to light in these past 6 months. Strategies for mitigating risk and therapeutic strategies to improve outcomes I think will be part of what people will come away with from those sessions.
CGTLive®’s Weekly Rewind – February 13, 2026 | CGTlive®
Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field. FDA objections centered on study population misclassification risk, external control validity, and surrogate end point evidentiary strength. In episode 8 of ImmunoLogic, Julia C.
Around the Helix: Cell and Gene Therapy Company Updates – February 11, 2026 | CGTlive®
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
American College of Physicians Calls for An End of the Term 'Provider' in Policy Paper
This article was originally published on our sister site, OphthalmologyTimes®. The American College of Physicians (ACP) has released a policy paper asserting that the use of the term “provider” to describe physicians carries ethical implications that go beyond semantics and may contribute to the erosion of professional identity, trust, and the physician–patient relationship. In the paper, ACP places the discussion within longstanding concerns about the commercialization of medicine.
FDA Slams REGENXBIO's MPS II Gene Therapy RGX-121 With CRL
This article was originally published on our sister site, NeurologyLive®. The FDA has issued a complete response letter (CRL) to REGENXBIO’s biologics license application (BLA) for clemidsogene lanparvovec (RGX-121), an investigational adeno-associated virus (AAV) vector–based gene therapy being developed for the treatment of mucopolysaccharidosis type 2 (MPS II), also known as Hunter syndrome.¹ REGENXBIO reported that it received the CRL on February 7, 2026.
Ultragenyx Puts BLA for MPS IIIA Gene Therapy UX111 in Front of FDA Again
Ultragenyx has resubmitted a biologics license application (BLA) for UX111 (rebisufligene etisparvovec), an AAV9 vector-based gene therapy intended to treat mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome), to the FDA.1 The new BLA includes “substantial longer-term data on multiple measures of neurologic benefit”, and is aimed at obtaining an accelerated approval for the gene therapy product.1 Notably, a previous BLA submission for UX111 was met with a complete...
Around the Helix: Cell and Gene Therapy Company Updates – February 4, 2026 | CGTlive®
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
Pivotal Trial for Taysha's Rett Syndrome Gene Therapy TSHA-102 Doses First Patient
This article was originally published on our sister site, NeurologyLive®. Taysha Gene Therapies has dosed the first patient in the pivotal phase 3 portion (Part B) of the REVEAL clinical trial (NCT05606614), which is assessing the investigational adeno-associated virus (AAV) vector–based gene therapy TSHA-102 for the treatment of Rett syndrome.¹ The company reported that the initial dosing occurred in the fourth quarter of 2025.
CGTLive®’s Weekly Rewind – January 30, 2026 | CGTlive®
Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field. The therapy provided a +6-letter gain in BCVA-evaluable patients. Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, also discussed open questions that remain for the future of the hemophilia B gene therapy.
Ocugen’s Gene Therapy OCU410ST Continues to Show Efficacy in Phase 1 Stargardt Disease Trial
This article was originally published on our sister site, NeurologyLive®. Ocugen’s investigational adeno-associated virus (AAV)–based gene therapy OCU410ST demonstrated a +6-letter improvement in best-corrected visual acuity (BCVA) among BCVA-evaluable patients without confounders in the phase 1 GARDian1 clinical trial (NCT05956626).¹ These findings, along with additional study results, were published in Eye. The BCVA analysis included 6 of the 9 patients treated in the study.
Around the Helix: Cell and Gene Therapy Company Updates – January 28, 2026 | CGTlive®
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. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
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