Explore

Rapid Read Rapid Read
Affinia Therapeutics to Present Gene Therapy Data for Cardiomyopathy at ASGCT Meeting

Affinia Therapeutics to Present Gene Therapy Data for Cardiomyopathy at ASGCT Meeting

Affinia Therapeutics is set to present new preclinical data on its lead program, AFTX-201, at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting. AFTX-201 is an investigational genetic medicine targeting BAG3-associated dilated cardiomyopathy (DCM). The company will highlight the design of the Phase 1/2 UPBEAT clinical trial, which evaluates the safety and efficacy of AFTX-201. The U.S. FDA has granted Fast Track designation for AFTX-201, and the European Medicines Agency has given it Orphan Drug designation.
Trendline Trendline
AIRNA to Present New Preclinical Data on RNA-Editing Therapeutic Pipeline at ASGCT Annual Meeting

AIRNA to Present New Preclinical Data on RNA-Editing Therapeutic Pipeline at ASGCT Annual Meeting

AIRNA to Present New Preclinical Data on RNA-Editing Therapeutic Pipeline at ASGCT Annual Meeting
Rapid Read Rapid Read
FDA Approves First Gene Therapy for Genetic Deafness, Offering Free Access in the U.S.

FDA Approves First Gene Therapy for Genetic Deafness, Offering Free Access in the U.S.

The U.S. Food and Drug Administration (FDA) has approved Regeneron Pharmaceuticals' Otarmeni (lunsotogene parvec-cwha), marking the first gene therapy for a form of genetic hearing loss. This therapy targets otoferlin-related hearing loss, a rare condition caused by mutations in the OTOF gene. The approval is based on the CHORD trial, where 80% of participants showed significant hearing improvement, with 42% achieving normal hearing. Regeneron has announced that the therapy will be available at no cost to U.S. patients, a move aimed at maximizing its reach and impact. The therapy involves a dual hybrid AAV system to deliver the OTOF gene, addressing a previously unmet medical need.
Trendline Trendline
Intellia Therapeutics Submits Biologics License Application for CRISPR-Based Treatment for Hereditary Angioedema

Intellia Therapeutics Submits Biologics License Application for CRISPR-Based Treatment for Hereditary Angioedema

Intellia Therapeutics Submits Biologics License Application for CRISPR-Based Treatment for Hereditary Angioedema
Trendline Trendline
Intellia Therapeutics Begins FDA Submission for CRISPR-Based Hereditary Angioedema Treatment

Intellia Therapeutics Begins FDA Submission for CRISPR-Based Hereditary Angioedema Treatment

Intellia Therapeutics Begins FDA Submission for CRISPR-Based Hereditary Angioedema Treatment
Trendline Trendline
FDA Approves Taiho Oncology's ADC ARC-02 for Phase 1 Clinical Trials in Non-Hodgkin Lymphoma

FDA Approves Taiho Oncology's ADC ARC-02 for Phase 1 Clinical Trials in Non-Hodgkin Lymphoma

FDA Approves Taiho Oncology's ADC ARC-02 for Phase 1 Clinical Trials in Non-Hodgkin Lymphoma
Trendline Trendline
Encoded Therapeutics to Present ETX101 Data at ASGCT Symposium, Highlighting Advances in Dravet Syndrome Treatment

Encoded Therapeutics to Present ETX101 Data at ASGCT Symposium, Highlighting Advances in Dravet Syndrome Treatment

Encoded Therapeutics to Present ETX101 Data at ASGCT Symposium, Highlighting Advances in Dravet Syndrome Treatment
Rapid Read Rapid Read
Taiho Oncology Initiates Phase 1 Clinical Trial for ADC ARC-02 Targeting Non-Hodgkin Lymphoma

Taiho Oncology Initiates Phase 1 Clinical Trial for ADC ARC-02 Targeting Non-Hodgkin Lymphoma

Taiho Oncology, Inc., along with Taiho Pharmaceutical Co., Ltd. and Araris Biotech AG, has announced the initiation of a Phase 1 clinical trial for ARC-02, an antibody-drug conjugate (ADC) designed to treat non-Hodgkin lymphoma. This follows the completion of the U.S. Food and Drug Administration's (FDA) Investigational New Drug (IND) review period. ARC-02 is based on Araris' proprietary AraLinQ™ technology, which allows for the attachment of multiple cancer-fighting payloads to a single antibody, enhancing the targeted delivery and efficacy of the treatment. The trial marks a significant step in Taiho's expansion into ADC development for oncology, leveraging Araris' innovative technology to potentially improve treatment outcomes for patients with B-cell malignancies.
Trendline Trendline
Veradermics Announces Conference Call to Discuss Phase 2/3 Study Results for Hair Loss Treatment

Veradermics Announces Conference Call to Discuss Phase 2/3 Study Results for Hair Loss Treatment

Veradermics Announces Conference Call to Discuss Phase 2/3 Study Results for Hair Loss Treatment
Trendline Trendline
Ascletis Completes Enrollment in U.S. Phase II Study of ASC30 for Diabetes Treatment

Ascletis Completes Enrollment in U.S. Phase II Study of ASC30 for Diabetes Treatment

Ascletis Completes Enrollment in U.S. Phase II Study of ASC30 for Diabetes Treatment
Rapid Read Rapid Read
FDA Approves AstraZeneca's Saphnelo Pen for Self-Administration in Lupus Treatment

FDA Approves AstraZeneca's Saphnelo Pen for Self-Administration in Lupus Treatment

AstraZeneca's Saphnelo (anifrolumab) has received approval from the U.S. Food and Drug Administration (FDA) for self-administration as a once-weekly autoinjector, known as the Saphnelo Pen, for adults with systemic lupus erythematosus (SLE). This decision follows the successful Phase III TULIP-SC trial, which showed that subcutaneous administration of Saphnelo significantly reduced disease activity in patients with moderate to severe SLE. The trial's findings, published in Arthritis & Rheumatology, confirmed the safety and efficacy of the autoinjector, aligning with the known profile of Saphnelo when given intravenously. This approval is expected to enhance treatment accessibility and convenience for lupus patients.
Trendline Trendline
Tr1X to Present Data on Tr1 Treg Cell Therapies at ASGCT 2026

Tr1X to Present Data on Tr1 Treg Cell Therapies at ASGCT 2026

Tr1X to Present Data on Tr1 Treg Cell Therapies at ASGCT 2026