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LATEST NEWS

Improving Cell and Gene Therapy with Innovative Nonviral Delivery Vectors
By Eda Holl, PhD

November 12th 2024

An overview of novel drug-delivery vehicles and their potential role in
improving gene and cell therapies.

Eureka Therapeutics Tackling Advanced Hepatocellular Carcinoma With T-Cell
Therapy ECT204 in Phase 1/2 ARYA-3 Clinical Trial
By Noah Stansfield

November 11th 2024

Following up on Liver Cancer Awareness Month, which is observed annually in
October by the patient and clinician communities, CGTLive has decided to take a
closer look at this ongoing study.

Gastric Cancer Awareness Month 2024: Looking Back at Progress for Cell Therapy
By CGTLive Staff

November 11th 2024

In observance of Gastric Cancer Awareness Month, held annually in November, we
took a look back at the past few months' news in cell therapy for gastric cancer
indications.

Patient With Sickle Cell Disease Dies From Complications Related to
Busulfan-Conditioning Following Treatment With Beam’s Base-Editing HSC Therapy
BEAM-101
By Noah Stansfield

November 10th 2024

The patient died of respiratory failure 4 months after receiving BEAM-101.

FDA Approves Obe-Cel for Adults With R/R B-Cell Precursor Acute Lymphoblastic
Leukemia
By Matt Hoffman

November 8th 2024

The CAR-T therapy was approved based on the pivotal phase 1b/2 FELIX clinical
trial (NCT04404660), which showed good rates of overall complete remission and
median duration of remission.

Video Interviews
Caroline Diorio, MD, FRCPC, FAAP, on Noninflammatory Neurotoxicity Events in
Patients Receiving CAR-T

R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, on Gene Therapy
for APOE4 Homozygous Alzheimer Disease

Sharon Hesterlee, PhD, on MDA 2025 as a Mixer for Strategies and Collaboration

Daniela van Eickels, MD, PhD, MPH, on Bristol Myers Squibb’s Development of Cell
Therapy for Autoimmune Disease

Paul Melmeyer, MPP, on the Importance of Gene Therapy for MDA’s Upcoming 2025
Conference

Daniela van Eickels, MD, PhD, MPH, on Addressing Unmet Needs in Autoimmune
Disease With Cell Therapy

Arun Upadhyay, PhD, on Results from IRD Gene Therapy OCU400’s Phase 1/2 Trial

Arun Upadhyay, PhD, on the Potential of Gene Therapy in Inherited Retinal
Disease

John Brandsema, MD, on Potential Upcoming Treatments for Duchenne Muscular
Dystrophy

John Brandsema, MD, on Navigating the New Landscape of Care for DMD

Barry J Byrne, MD, PhD, on Making Decisions in the Face of Rapidly Expanding
Treatment Options

John Brandsema, MD, on Balancing Risks and Rewards With Muscular Dystrophy Gene
Therapy

Crystal Proud, MD, on Improving Newborn Screening for Spinal Muscular Atrophy

Chun-Yu Chen, PhD, on Addressing Hemophilia A With CRISPR/Cas9 mRNA LNP Gene
Editing

William Chou, MD, on Targeting Progranulin With Gene Therapy for Frontotemporal
Dementia

Alexandra Collin de l’Hortet, PhD, on Evaluating Epigenetic Gene Therapy for the
Treatment of FSHD



CONFERENCE COVERAGE

View All
Gene Therapy for APOE4 Homozygous Alzheimer Disease
By Kim G. Johnson, MD
R. Nolan Townsend
 * Sandi See Tai, MD
 * Isabella Ciccone, MPH
 * Noah Stansfield

November 6th 2024

R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo
Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and
biomarker effects in patients with early-stage Alzheimer disease.

Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in
Preclinical Parkinson Disease Research
By Marco Meglio

November 5th 2024

The noninvasive gene therapy exceeded the 30% efficacy threshold for normalizing
GCase activity across all doses.

Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients
With Alzheimer Disease
By Marco Meglio

November 4th 2024

LX1001's safety profile was characterized as well-tolerated with no
amyloid-related imaging abnormalities reported.

R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, on Gene Therapy
for APOE4 Homozygous Alzheimer Disease
By Kim G. Johnson, MD
R. Nolan Townsend
 * Sandi See Tai, MD
 * Isabella Ciccone, MPH

October 31st 2024

The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that
demonstrated promising safety and biomarker effects in patients with early-stage
Alzheimer disease.

Continuing Medical Education

MultimediaCME,NCPD

Community Oncology Connections™: Overcoming Clinical Challenges and Embracing
New Opportunities in the Management of Multiple Myeloma

View More




VideoCME,NCPD

Exploring the Dynamic Shifts in the MDS Treatment Paradigm: Unveiling Advances
in Disease Modifying Therapies, Spotlight on Cutting-Edge Clinical Trials, and
the Latest Approvals

View More




VideoCME,NCPD

Cases and Conversations™: Applying Guidelines to Practice for the Management of
Paroxysmal Nocturnal Hemoglobinuria

View More




MultimediaCME,NCPD

Community Practice Connections™: 5th Annual Precision Medicine Symposium – An
Illustrated Tumor Board

View More




MultimediaCME

Oncology Briefings™: Selecting Therapy for Patients with Heavily Pretreated
Myeloma Based on Patient and Disease-Specific Factors

View More




MultimediaCME,NCPD

Oncology Briefings™: Case Discussions in Smoldering Myeloma—To Treat or Not to
Treat?

View More




VideoNCPD

Medical Crossfire®: How Has the Nurse’s Role Evolved in the Management of
Pediatric Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas?

View More




MultimediaCME

Community Practice Connections™: How Can We Optimize the Real World Application
of BCMA-Targeted Therapies for Our Patients with R/R Multiple Myeloma?

View More






ALL NEWS



CGTLive®’s Weekly Rewind – November 8, 2024

By CGTLive Staff
November 8th 2024


Review top news and interview highlights from the week ending November 8, 2024.



NTLA-2002 Gene Editing Reduces Angioedema Attacks in Hereditary Angioedema

By Matt Hoffman
November 8th 2024


Intellia’s investigational CRISPR-Cas9 gene-editing therapy NTLA-2002 reduced
monthly attacks by more than 70% in both tested dose arms.



FDA Removes Clinical Holds Previously Placed on 3 of CARsgen's Oncology Cell
Therapies

By Noah Stansfield
November 7th 2024


The clinical holds on the 3 products were originally placed by the FDA in
December 2023 based on findings from an inspection of CARsgen’s manufacturing
facility.



Lung Cancer Awareness Month 2024: Looking Back at Progress for Cell and Gene
Therapy

By CGTLive Staff
November 7th 2024


In observance of Lung Cancer Awareness Month, held annually in November, we took
a look back at the past few months' news in cell and gene therapy for lung
cancer indications.



Around the Helix: Cell and Gene Therapy Company Updates – November 6, 2024

By Noah Stansfield
November 6th 2024


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



HuidaGene Therapeutics’ CRISPR/Cas13 RNA-Editing Therapy HG202 Gets Greenlight
for US Trial in Age-Related Macular Degeneration

By Noah Stansfield
November 6th 2024


According to HuidaGene, it is the first CRISPR/Cas13Y RNA-targeting therapy to
receive clearance for a clinical trial from the FDA or any other regulatory
agency.



Gene Therapy for APOE4 Homozygous Alzheimer Disease

By Kim G. Johnson, MD
R. Nolan Townsend
 * Sandi See Tai, MD
 * Isabella Ciccone, MPH
 * Noah Stansfield

November 6th 2024


R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo
Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and
biomarker effects in patients with early-stage Alzheimer disease.



Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in
Preclinical Parkinson Disease Research

By Marco Meglio
November 5th 2024


The noninvasive gene therapy exceeded the 30% efficacy threshold for normalizing
GCase activity across all doses.



Noninflammatory Neurotoxicity Events in Patients Receiving CAR-T

By Caroline Diorio, MD, FRCPC, FAAP
Noah Stansfield
November 5th 2024


Caroline Diorio, MD, FRCPC, FAAP, an attending physician at the Cancer Center at
Children's Hospital of Philadelphia, discussed a study she coauthored that was
recently published in Blood.



Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves
Visual Acuity in Randomized Controlled Clinical Trial

By Noah Stansfield
November 5th 2024


The randomized, controlled clinical trial treated patients with either a high
dose of MCO-010, a low dose of MCO-010, or a sham–control.



Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients
With Alzheimer Disease

By Marco Meglio
November 4th 2024


LX1001's safety profile was characterized as well-tolerated with no
amyloid-related imaging abnormalities reported.



FDA Activity Recap: October 2024 Features IND Clearances and Company Alignment
on Plans in FDA Meetings

By CGTLive Staff
November 4th 2024


Catch up on any of the key FDA news stories you may have missed last month, with
coverage highlights from the CGTLive® team.



RiboX's Circular RNA Therapy RXRG001 Cleared for Phase 1/2a Trial in
Radiation-Induced Xerostomia and Hyposalivation

By Noah Stansfield
November 3rd 2024


RiboX stated that this is the first IND clearance the FDA has granted for any
circular RNA therapy.



Caroline Diorio, MD, FRCPC, FAAP, on Noninflammatory Neurotoxicity Events in
Patients Receiving CAR-T

By Caroline Diorio, MD, FRCPC, FAAP
Noah Stansfield
November 3rd 2024


The attending physician at the Cancer Center at Children's Hospital of
Philadelphia discussed a study she coauthored that was recently published in
Blood.



CGTLive®’s Weekly Rewind – November 1, 2024

By CGTLive Staff
November 1st 2024


Review top news and interview highlights from the week ending November 1, 2024.



Data Roundup: October 2024 Features Update for TCR-Based Autologous Cell Therapy
in Melanoma, the First Clinical Demonstration of Therapeutic RNA Editing in
Humans, and More

By CGTLive Staff
November 1st 2024


Catch up on any of the key data updates you may have missed last month, with
coverage highlights from the CGTLive™ team.



Abeona Therapeutics Resubmits BLA to FDA for Epidermolysis Bullosa Gene Therapy
Pz-Cel

By Noah Stansfield
October 31st 2024


The company made the resubmission after reaching an accord with the FDA in a
Type A meeting with the agency that took place in August 2024.



R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, on Gene Therapy
for APOE4 Homozygous Alzheimer Disease

By Kim G. Johnson, MD
R. Nolan Townsend
 * Sandi See Tai, MD
 * Isabella Ciccone, MPH

October 31st 2024


The 3 experts discussed Lexeo Therapeutics’ LX1001 gene therapy trial that
demonstrated promising safety and biomarker effects in patients with early-stage
Alzheimer disease.



Targeting Progranulin With Gene Therapy for Frontotemporal Dementia

By William Chou, MD
Noah Stansfield
October 31st 2024


William Chou, MD, president and chief executive officer of Passage Bio,
discussed feedback from a recent Type C meeting with the FDA.



Around the Helix: Cell and Gene Therapy Company Updates – October 30, 2024

By Noah Stansfield
October 30th 2024


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



BridgeBio Pharma’s Gene Therapy BBP-812 Effects Progressive Improvement in Motor
Function in Patients With Canavan Disease

By Noah Stansfield
October 30th 2024


Furthermore, the majority of patients treated with the gene therapy showed
improved myelination as measured by T-2-weighted MRI.



Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy

By Melissa Spencer, PhD
CGTLive Staff
October 29th 2024


Melissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as
a delivery system for gene therapies targeting muscular dystrophies, and the
challenbges associated with them.



Immatics’ TCR-T Therapy Shows Good Phase 1 Safety and Survival Data, Phase 3
Plans Confirmed

By Matt Hoffman
October 29th 2024


Patients with melanoma treated with ACTengine IMA203 achieved high response
rates, with progression-free survival of 6 months and duration of response
beyond 1 year.



MDA 2025 as a Mixer for Strategies and Collaboration

By Sharon Hesterlee, PhD
Noah Stansfield
October 28th 2024


Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy
Association discussed the upcoming MDA Clinical and Scientific Conference, which
will be held March 16-19, in Dallas, Texas.



Intellia’s CRISPR Gene Editing Therapy NTLA-2002 Appears to Eliminate Hereditary
Angioedema Attacks in Some Patients Following One-Time Treatment

By Noah Stansfield
October 27th 2024


Participants in the trial received either a single dose of 25 mg of NTLA-2002, a
single dose of 50 mg of NTLA-2002, or a placebo.


See All News >
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