Around the Helix: Cell and Gene Therapy Company Updates – August 30, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Investing in Long-Term Studies May Provide a Better Picture of Cell Therapy Efficacy
Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed what the company learned from relitigating results of legacy trials for a mesenchymal stem cell therapy in Crohn disease.
X-Linked Retinoschisis Gene Therapy Trial Doses First Patient
ATSN-201 utilizes Atsena’s novel spreading capsid AAV.SPR, which is expected to spread laterally and allow transduction of the gene therapy in the central retina.
FDA Approves Bristol Myers Squibb’s Reblozyl for Expanded Indication in Myelodysplastic Syndromes
The FDA’s decision was based on data from the randomized, open-label phase 3 COMMANDS clinical trial.
BlueRock Therapeutics' Parkinson Disease Cell Therapy Bemdaneprocel Shows Initial Efficacy in Phase 1 Trial
In light of the promising results, BlueRock is preparing for a phase 2 trial.
Allogeneic Natural Killer Cell Trial in Solid Tumors Doses First Patient
NKGen Biotech’s trial for SNK02 was initiated following the clearance of an investigational new drug application by the FDA in October 2022.
Taking a Second Look at Placental-Derived Mesenchymal-Like Adherent Stromal Cell Therapy for Crohn Disease
Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed a new analysis of gene and protein signatures from patients treated in the company’s legacy clinical trials.
BCMA-Directed CAR-T NXC-201 Gets FDA Orphan Drug Designation in Multiple Myeloma
Because of the lack of neurotoxicity seen in patients treated thus far, Nexcella believes NXC-201 may have potential to serve as an outpatient treatment.
Neurophth Therapeutics’ ND1-LHON Gene Therapy Trial Doses First Patient
The single-arm, open-label clinical trial will seek to enroll approximately 18 patients aged 18 to 75 years.
Around the Helix: Cell and Gene Therapy Company Updates – August 23, 2023
Niemann-Pick Disease Type C Gene Therapy Gets Rare Pediatric Disease Designation
Earlier this year Bloomsbury Genetic Therapies met with the UK’s MHRA for a scientific advice meeting to discuss BGT-NPC.
Wugen’s NK Cell Therapy Trial Doses First Patient With AML
The company also reported that WU-NK-101 has received orphan drug designation for treating acute myeloid leukemia from the FDA.
Ornithine Transcarbamylase Deficiency Gene Therapy Gets FDA Orphan Drug Designation
BGT-OTCD is being developed in a collaboration between Bloomsbury Genetic Therapies and University College London.
Artiva Biotherapeutics’ NK Cell Therapy AlloNK Cleared for Trial in Systemic Lupus Erythematosus
AlloNK will be evaluated as part of a combination therapy with anti-CD20 monoclonal antibody rituximab.
Neurogene’s Clinical Trial Seeks to Assess CLN5 Batten Disease Gene Therapy NGN-101
NGN-101 will be delivered to each participant via both intracerebroventricular and intravitreal routes on the same day in the phase 1/2 trial, with the therapy assessed for safety and efficacy.
ImmPACT Bio’s Systemic Lupus Erythematosus CAR-T Cleared for US Trial
IMPT-514 uses the same CAR-T construct as ImmPACT Bio’s IMPT-314, which is already under clinical evaluation for non-Hodgkin lymphoma.
Around the Helix: Cell and Gene Therapy Company Updates – August 16, 2023
Genprex Garners Orphan Drug Designation for Lung Cancer Immunogene Therapy Reqorsa
The new orphan drug designation follows a recently received fast track designation for Reqorsa in combination with Tecentriq for an extensive-stage small cell lung cancer indication.
FDA Lifts Partial Clinical Hold on Arcellx’s Trial for Multiple Myeloma CAR-T CART-ddBCMA
The partial clinical hold was originally placed in June 2023 in relation to the death of a patient participating in the trial.
FDA Places Formal Clinical Hold On 2seventy bio's Trial for Acute Myeloid Leukemia CAR-T In Relation to Patient Death
The formal hold comes approximately 2 months after Seattle Children’s paused the trial in accordance with the clinical study protocol stopping rules.
Rett Syndrome Gene Therapy TSHA-102 Demonstrates Improvements in First Patient Dosed
In terms of safety, Taysha stated that TSHA-102 was well-tolerated and that at 6 weeks after dosing no treatment-emergent serious adverse events had occurred.
mRNA-engineered iPSC-derived Cell Therapies Hold Potential for Tackling Solid Tumors
Kyle Garland, PhD, a senior scientist at Eterna Therapeutics, discussed the company’s preclinical work with partner Factor Bioscience that was presented at ASGCT’s 2023 conference.
Parkinson Disease iPSC-derived Therapy ANPD001 Cleared for Clinical Trial in the US
Aspen Neuroscience noted that the study will be the US’s first multicenter phase 1/2a clinical trial for an autologous iPSC-derived therapy.
HaemaLogiX and Peter MacCallum Cancer Centre to Collaborate on Kappa-Type Multiple Myeloma CAR-T Trial
KMA.CAR-T is directed at Kappa Myeloma Antigen, a receptor exclusively appearing on myeloma cells in kappa-type MM.
AskBio’s Limb-Girdle Muscular Dystrophy Gene Therapy Trial Doses First Patient
LION-CS101, the study evaluating gene therapy AB-1003, is currently recruiting patients aged 18 years through 65 years with LGMD2I/R9 who have a confirmed mutation in FKRP.
Stem Cell Prime Editing May Provide Novel Treatment Approach for p47phox Chronic Granulomatous Disease
Jennifer Gori, PhD, vice president of research at Prime Medicine, discussed what distinguishes prime editing from other forms of gene editing.
Around the Helix: Cell and Gene Therapy Company Updates – August 2, 2023
Trial for Wilson Disease Gene Therapy UX701 Starts Dosing Second Cohort
The initiation of dosing the second cohort in Ultragenyx’s study was recommended by the Data Safety Monitoring Board after a review of safety results from the trial’s first cohort.
Rett Syndrome Gene Therapy Trial Recommended to Dose Second Patient
Taysha Gene Therapies previously reported the dosing of the first patient with TSHA-102 in June 2023.
AAV Immuno-Gene Therapy May Hold Untapped Potential in Oncology Indications
Nicole Paulk, PhD, the CEO, founder, and president of Siren Biotechnology, discussed research she presented at ASGCT’s 2023 conference.
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