Cell Therapy
Gene Therapy
Regulatory & Standards

Clinical News Round-Up: What’s New and ‘Approved’ in Clinical Cell and Gene Therapy?

Georgi Makin
1 June 2021

For this new monthly column, Georgi Makin summarises some of her top picks from clinical news in cell and gene therapy.

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For the first of my clinical columns, I highlight two new gene therapy clinical trials underway, as well as a few recent regulatory approvals and positive recommendations. 

Phase I Gene Therapy Trial for Fatal Genetic Condition Commences in New Brunswick

The Children’s Hospital at Saint Peter’s University Hospital has announced its participation in a global Phase I/II clinical trial program – Imagine-1 – for PBGM01, an AAV-delivery gene therapy under development by Passage Bio.  

PBGM01 is being studied as a potential therapeutic for infantile GM1 gangliosidosis (GM1), a fatal and rare monogenic lysosomal storage disease.

“This clinical study requires exquisite coordination,” said Dr Debra-Lynn Day-Salvatore, Chair of the Department of Medical Genetics and Genomic Medicine, and Principal Investigator for the Imagine-1 trial.

“Everyone plays a pivotal role, and I am extremely proud of the teamwork, precision, and seamless work flow that resulted in the successful dosing of the first patient in the clinical trial program for PBGM01. I am also grateful to the patient’s family for entrusting us with the care of their precious child. We are pleased to celebrate this milestone with the patients, families, and researchers that helped to bring this effort to fruition and share their hope, dreams, and resolve for the future,” Dr Day-Salvatore continued.

Find out more with the full press release >>

Gene Therapy to be Tested as Treatment of Sickle Cell Disease

The Cleveland Clinic has begun enrolling patients for a clinical trial to assess the safety and effectiveness of a single dose of EDIT-301.  

The Clinic is looking to recruit 40 adult patients (from 18–50 years old) with severe sickle cell disease, to be monitored for up to 2 years following treatment.  

“Gene therapy is an incredible technology that works by replacing or inactivating disease-causing genes,” explained Rabi Hanna, Director of the paediatric blood and bone marrow transplant program at Cleveland Clinic and Principal Investigator of the trial.

“In this study, the gene therapy will introduce healthy genes into the body with the goal of correcting genetic abnormalities of red blood cells. By enabling the cells to produce more foetal haemoglobin, this treatment has the potential to cure sickle cell disease in a precise way.” 

“New treatments are critical for people who have sickle cell disease. Despite the fact that it has been more than 100 years since its discovery there have been only a few medications approved in the last 50 years for sickle cell disease. Until now, we didn’t have the technology to work with to create a possible cure for this devastating disease,” Hanna concluded. 

Find out more with the full press release >>

Collaboration Calls for Advanced Therapies Exemption from EU Genetically Modified Organism Legislation

The Alliance for Regenerative Medicine (ARM), the European Federation of Pharmaceutical Industries and Associations (EFPIA), and the European Association of Bioindustries (EuropaBio) have called for gene therapies to be exempt from EU Genetically Modified Organism (GMO) legislation, which, they claim, restricts patient access to transformative medicines.

As Paige Bischoff, Senior Vice President of Global Public Affairs at ARM explained:

“The European Commission recognised that time was of the essence when lifting GMO requirements for COVID-19 vaccines and treatments. Time is also very much of the essence for people with cancer, inherited disorders and other life-threatening conditions. We call on the European Commission to take the same measures for advanced therapies and remove the unnecessary and unintended burden of GMO legislation so patients have timely access to transformative, potentially curative medicines.”

Find out more with the full press release >>

New and ‘Approved’

» AAV Gene Therapy for Limb-Girdle Muscular Dystrophy Cleared IND Application by FDA 

 AskBio has announced FDA IND application clearance for a novel investigational AAV gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 – LION-101 – to proceed in a Phase I/II clinical study.  

The study – with initial dosing planned for early 2022 – will evaluate a single intravenous infusion in adults and adolescents with genotypically confirmed limb-girdle muscular dystrophy type 2I/R9. | Find out more >>

» FDA Grants BCMA CAR-T Therapy Priority Review for the Treatment for Multiple Myeloma 

Legend Biotech Corporation has announced that the FDA has accepted a Biologics License Application (BLA) submitted by Janssen Biotech, Inc. for ciltacabtagene autoleucel (cilta-cel).  

Cilta-cel is an investigational B-cell maturation antigen (BCMA)-directed CAR-T therapy for the treatment of relapsed/refractory multiple myeloma.  

The PDUFA date is currently set for 29 November 2021. | Find out more >> 

» IND Application Clearance Granted for Allogeneic T Cell Therapy for Haematological Malignancies 

GammaDelta Therapeutics has announced FDA clearance of IND application for GDX012, a novel allogeneic variable delta 1 gamma-delta T cell cancer therapy for haematological malignancies.  

The FDA has also reportedly granted orphan drug designation for GDX012 for the treatment of acute myeloid leukaemia, Phase I trials for which are expected to commence later in 2021. | Find out more >>

» Positive Recommendation Announced to Advance Phase I/II Clinical Trial for the Treatment of Huntington’s Disease 

uniQure has announced that the independent Data Safety Monitoring Board (DSMB) has recommended proceeding with another, higher-dose cohort for a gene therapy, AMT-130, for the treatment of Huntingdon’s disease.  

The DSMB has been overseeing a Phase I/II clinical trial of AMT-130, including two patients over nine months. The next step will be to enrol 26 patients over 12 months with a follow-up over 5 years in the US, and 15 patients in Europe. | Find out more >>

» Investigational Gene Therapy for Treatment of Duchenne Muscular Dystrophy Demonstrates Consistent Safety Profile 

Sarepta Therapeutics has announced that its’ investigational gene therapy – SRP-9001 – for the treatment of Duchenne muscular dystrophy, has demonstrated ‘robust expression’ and a ‘consistent safety profile’.  

Study SRP-9001-103 – an open-label study known as ENDEAVOR, conducted in partnership with Roche – used commercially representative material, SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin), demonstrating robust expression of micro-dystrophin and no new safety signals from prior studies. | Find out more >>

» Positive CHMP Opinion Announced for Gene Therapy for Treatment of Early Cerebral Adrenoleukodystrophy 

bluebird bio, Inc. has announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA adopted a positive opinion recommending marketing authorization for SKYSONA™ (elivaldogene autotemcel, Lenti-D™). 

SKYSONA is a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy – a rare, X-linked metabolic disorder that primarily affects males – in patients under 18 years of age with an ABCD1 genetic mutation. | Find out more >>

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2022, Miami, Florida.
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