4D Molecular Therapeutics’ Wet AMD Gene Therapy 4D-150 Reduces Need for antiVEGF Injections in Interim Phase 2 Trial Data
At the 24-week analysis, patients who received the high dose achieved an 89% decrease in annualized antiVEGF injection rates.
Orchard Therapeutics’ Arsa-cel Restores ARSA Enzyme Activity in Patients With Late Juvenile MLD
The gene-edited cell therapy also showed a safety profile consistent with that previously seen in patients with early-onset MLD.
Orchard Therapeutics’ Stem Cell Gene Therapy OTL-201 Reduces Heparan Sulphate Levels in Patients With MPSIIIA
Among the 5 patients treated in the study, sustained supraphysiological expression of the disease-targeted gene, SGSH, was rapidly achieved in the leukocytes and the plasma.
Orchard Therapeutics’ Arsa-cel Restores ARSA Enzyme Activity in Peripheral Blood of Patients With MLD in US Compassionate Use Study
Hematological recovery was achieved and maintained in all 4 patients in the study.
Around the Helix: Cell and Gene Therapy Company Updates – February 7, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Encoded Therapeutics’ Dravet Syndrome Gene Therapy ETX101 Cleared for Separate Clinical Trials in the US and Australia
ETX101 consists of a transgene encoding a GABAergic regulatory element and an engineered transcription factor that is delivered via a nonreplicating, recombinant AAV9 vector.
Sickle Cell Disease Gene Therapies to Be Part of Initial Focus for Biden-Harris Administration’s CGT Access Model
The new Cell and Gene Therapy Access Model will be led by the Centers for Medicare & Medicaid Services’ Innovation Center.
The Field of Neuromuscular Disease is Entering the Era of Treatment
Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed MDA Care Centers and his excitement for the organization’s the Upcoming Clinical & Scientific Conference.
Akouos Seeks to Tackle Otoferlin Gene-Mediated Hearing Loss With Phase 1/2 Clinical Trial and Associated Natural History Study
AK-OTOF delivers transgenes encoding OTOF, the disease-targeted gene, to the inner hair cells of the cochlea via a single unilateral intracochlear administration.
Epilepsy Cell Therapy NRTX-1001 May Eventually Be Evaluated for Alzheimer Disease
Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed future plans for the expanded applications of the allogeneic regenerative neural cell therapy.
FDA Accepts Adamptimmune’s BLA for Synovial Sarcoma TCR T-cell Therapy Afami-cel With Priority Review
The Prescription Drug User Fee Act (PDUFA) target action date has been set for August 4, 2024.
Neurona Therapeutics NRTX-1001 Has Shown Promising Early Signs of Safety and Efficacy in Drug-Resistant Epilepsy
Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed the ongoing first-in-human clinical trial evaluating the cell therapy for drug-resistant mesial temporal lobe epilepsy.
Around the Helix: Cell and Gene Therapy Company Updates – January 31, 2024
Ahead of Announcing Positive Results Seen in Trial for Gene Therapy FBX-101, Forge Biologics’ CEO Makes Comment Supporting Krabbe Disease’s Addition to RUSP
Efficacy and safety results from the RESKUE clinical trial will be presented at at the WORLDSymposium 2024 on February 9.
Sarepta Therapeutics' SRP-5051 Demonstrates Ability to Increase Dystrophin Expression in Patients With Duchenne Muscular Dystrophy
SRP-5051 is an investigational peptide-conjugated phosphorodiamidate morpholino oligomer.
Sarepta Therapeutics' Phase 3 Study for Limb-Girdle Muscular Dystrophy Gene Therapy SRP-9003 Begins Screening Activities
The multinational, open-label EMERGENE study will seek to enroll 15 patients with LGMD2E/R4 in total.
Cell Therapy May Help to Address Unmet Needs in Epilepsy
Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed NRTX-1001, the company’s allogeneic regenerative neural cell therapy currently being evaluated in MTLE.
Sensorion’s OTOF Hearing Loss Gene Therapy Cleared for Phase 1/2 Trial in France
The trial will recruit patients aged 6 to 31 months with the intent of treating them while auditory system plasticity is optimal.
2023: A Landmark Year for Cell and Gene Therapy Approvals
Robert M Califf, MD, MACC, the commissioner of food and drugs at the FDA, discussed how 2023 was an exemplary year for how far the field has come over the past few decades.
Janssen and Legend Biotech’s Carvykti to be Put in Front of FDA AdComm Regarding Multiple Myeloma Expanded Use BLA
The European Medicines Agency Committee for Advanced Therapies will likewise hold a SAG-O meeting regarding the Type II variation application for the CAR-T in the EU.
Around the Helix: Cell and Gene Therapy Company Updates – January 24, 2024
Aruna Bio’s Exosome AB126 Cleared for US Trial in Acute Ischemic Stroke
Aruna Bio stated that it expects to begin a phase 1b/2a clinical trial within the first half of this year.
Autolus Therapeutics’ Obe-cel Will Be Reviewed by the FDA for the Treatment of R/R B-ALL
The agency does not have plans for an advisory committee meeting to discuss obe-cel, which is an investigational autologous CD19-directed CAR-T therapy.
Gene Therapy Will Continue to be a Major Topic at MDA’s 2024 Conference
Sharon Hesterlee, PhD, the chief research officer of MDA, discussed highlights of 2023’s meeting and exciting new things to look forward to at the upcoming 2024 meeting.
Experts Weigh In on Exa-cel's FDA Approval for Transfusion-Dependent Thalassemia
In light of this major decision by the agency, CGTLive™ decided to reach out to several experts to get their thoughts on how exa-cel could impact the landscape of care for TDT.
Triumvira Immunologics’ TAC T-cell Therapy TAC01-CLDN18.2 to be Evaluated in Phase 1/2 Clinical Trail for Solid Tumors
TAC01-CLDN18.2 integrates TAC, Triumvira’s proprietary chimeric receptor that is intended to activate and direct T-cells against tumor cells.
Exa-cel and Lovo-cel Provide Important New Options for Patients With Sickle Cell Disease, but Expanding Access is Necessary
Tami John, MD, a clinical associate professor at Stanford Medicine, also discussed ongoing trends in sickle cell disease research.
Hemogenyx Sends the FDA a Complete Response Related to the IND Hold on AML CAR-T HEMO-CAR-T
Hemogenyx originally submitted the IND to the FDA in May 2023 with the intention of gaining clearance for a phase 1 clinical trial in acute myeloid leukemia.
Around the Helix: Cell and Gene Therapy Company Updates – January 17, 2024
Passage Bio’s Gene Therapy PBFT02 Positively Impacts Biomarker Levels in Patients With Frontotemporal Dementia
Among 3 patients in the study’s first cohort, a 3.6 to 6.6-fold increase in CSF PGRN over baseline was observed at 30 days posttreatment.