Drugs / gene therapy
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT00002824 | Feb 1996 → ? | central nervous system neoplasm | Abramson Cancer Center at Penn Medicine | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 27
| Date | Issuer | Release |
|---|---|---|
| 2026-08-29 | Bayer | Results AskBio Presents Baseline Characteristics from Phase 2 Heart Failure Gene Therapy Trial of Umiposgene Parvec (AB-1002) at European Society of Cardiology Congress bayer.com ↗
The baseline characteristics provide an overview of the population enrolled in GenePHIT and demonstrate the successful recruitment of a representative non-ischemic heart failure with reduced ejection fraction (HFrEF) population receiving guideline-directed medical therapy. |
| 2026-08-29 | AskBio Inc | Results AskBio Presents Baseline Characteristics from Phase 2 Heart Failure Gene Therapy Trial of Umiposgene Parvec (AB-1002) at European Society of Cardiology Congress askbio.com ↗
The baseline characteristics provide an overview of the population enrolled in GenePHIT and demonstrate the successful recruitment of a representative non-ischemic heart failure with reduced ejection fraction (HFrEF) population receiving guideline-directed medical therapy. |
| 2025-12-09 | Bayer | Regulatory AskBio’s AB-1005 and AB-1002 Receive Pioneering Regenerative Medical Product Designation in Japan bayer.com ↗
“Having AB-1005 and AB-1002 receive the Pioneering Regenerative Medical Product designation in Japan highlights our dedication to advancing innovative gene therapies for participants facing serious diseases,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. |
| 2025-12-09 | AskBio Inc | Regulatory AskBio’s AB-1005 and AB-1002 Receive Pioneering Regenerative Medical Product Designation in Japan askbio.com ↗
“Having AB-1005 and AB-1002 receive the Pioneering Regenerative Medical Product designation in Japan highlights our dedication to advancing innovative gene therapies for participants facing serious diseases,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. |
| 2025-10-21 | Bayer | Results AskBio Announces Publication in Nature Medicine of 12-month data from Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure bayer.com ↗
The publication, which is available online, confirms that no adverse events were deemed related to AB-1002 in this trial and that clinically meaningful improvements were recorded across several efficacy assessments in participants with non-ischemic CHF. |
| 2025-10-21 | AskBio Inc | Results AskBio Announces Publication in Nature Medicine of 12-month data from Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure askbio.com ↗ |
| 2025-05-19 | Bayer | Results AskBio Presents Complete Results of Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure at European Society of Cardiology Heart Failure Meeting bayer.com ↗
No treatment-emergent or serious adverse events were deemed related to trial treatment. |
| 2025-05-19 | AskBio Inc | Results AskBio Presents Complete Results of Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure at European Society of Cardiology Heart Failure Meeting askbio.com ↗
AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, presented—during a dedicated late-breaking science session at this year’s European Society of Cardiology Heart Failure meeting—the complete dataset from a Phase 1 trial investigating AB-1002 for the treatment of congestive heart failure (CHF). |
| 2024-04-18 | Bayer | Regulatory AskBio receives FDA Fast Track Designation for AB-1002 investigational gene therapy program in congestive heart failure bayer.com ↗
the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program. |
| 2024-04-18 | AskBio Inc | Regulatory AskBio receives FDA Fast Track Designation for AB-1002 investigational gene therapy program in congestive heart failure askbio.com ↗
Berlin, Germany, and Research Triangle Park, NC, USA, April 18, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program. |
| 2023-11-13 | Bayer | Results AskBio Presents Preliminary Data from Phase 1 Trial of Gene Therapy for Congestive Heart Failure (CHF) at the 2023 American Heart Association Scientific Sessions bayer.com ↗ |
| 2023-11-12 | AskBio Inc | Results AskBio Presents Preliminary Data from Phase 1 Trial of Gene Therapy for Congestive Heart Failure (CHF) at the 2023 American Heart Association Scientific Sessions askbio.com ↗
today presented first-in-human data from a Phase 1 trial investigating AB-1002 (also known as NAN-101) for the treatment of patients with congestive heart failure (CHF) |
| 2022-08-23 | AskBio Inc | Regulatory BrainVectis, a subsidiary of AskBio, receives clearance to conduct Phase I/II clinical trial in France for its novel gene therapy for early-stage Huntington’s Disease askbio.com ↗ |
| 2021-06-28 | AskBio Inc | Regulatory AskBio Receives FDA Fast Track Designation for LION-101, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) askbio.com ↗
Asklepios BioPharmaceutical , Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced that the U.S. Food & Drug Administration (FDA) has granted Fast Track Designation for the LION-101 gene therapy program. |
| 2019-11-06 | Orchard Therapeutics | Results Orchard Therapeutics Reports Third Quarter 2019 Financial Results and Achievement of Key 2019 Milestones for Neurometabolic and Primary Immune Deficiencies Franchises orchard-tx.com ↗
The initial data showed that cellular engraftment using a cryopreserved formulation was similar to that observed using a fresh formulation, with the longest patient having 12 months of follow-up since treatment. |
| 2019-09-04 | Orchard Therapeutics | Results Orchard Therapeutics Announces Encouraging Update from Proof-of-Concept Study of OTL-203 for the Treatment of Mucopolysaccharidosis Type I (MPS-I) orchard-tx.com ↗
today announced data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, a gene therapy for the treatment of mucopolysaccharidosis type I (MPS-I) developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan. |
| 2019-07-01 | AskBio Inc | Results Statement: Pfizer Phase 1b Clinical Trial Results askbio.com ↗
More importantly, Pfizer presented data that for the first time demonstrated striking improvement in North Star scores for patients of this age range that typically show steady functional decline. |
| 2019-04-29 | Orchard Therapeutics | Results Orchard Therapeutics Announces Clinical Proof-of-Concept Data for Gene Therapy OTL-300 Demonstrating Efficacy in Transfusion-Dependent Beta-Thalassemia orchard-tx.com ↗
At 12 Months Following Treatment, Eight of Nine Patients Had Reduced or Eliminated Need for Transfusions, with Four Pediatric Patients Achieving Transfusion Independence |
| 2019-02-25 | Genethon | Results Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD genethon.com ↗
Six Patients Continue to Show Sustained Levels of Functioning Neutrophils After 12 Months and No Longer Receive Treatment with CGD-related Prophylactic Antibiotics. |
| 2019-02-25 | Orchard Therapeutics | Results Orchard Therapeutics Presents Clinical Proof-of-Concept Data for OTL-102 for the Treatment of X-CGD orchard-tx.com ↗
Six of seven eligible patients showed greater than 10% (ranging from 16%-46%) functioning, oxidase-positive neutrophils in circulation at 12 months, which is the minimum threshold of oxidase-positive neutrophils necessary to demonstrate potential clinical benefit |
| 2018-12-05 | Orchard Therapeutics | Results Donald B. Kohn, MD, Orchard Therapeutics Scientific Advisory Board Member, Presents Clinical Proof-of-Concept Data for OTL-102 Gene Therapy for the Treatment of Patients with X-CGD at the Presidential Symposium at the 2018 ASH Annual Meeting orchard-tx.com ↗
These clinical proof-of-concept data demonstrate, for the first time, 12 months or more of restoration of immunity in X-CGD patients treated with ex-vivo autologous gene therapy, |
| 2018-05-03 | Orchard Therapeutics | Regulatory Orchard Therapeutics’ OTL-200 Receives Rare Pediatric Disease Designation from FDA for Treatment of Metachromatic Leukodystrophy orchard-tx.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation to the company’s gene therapy candidate OTL-200, for the treatment of patients with metachromatic leukodystrophy (MLD). |
| 2017-09-26 | AskBio Inc | Results NanoCor Announces New Data on Congestive Heart Failure Gene Therapy askbio.com ↗
In the study, heart function improved by up to 25 percent using a single treatment of the gene therapy in a large pre-clinical animal (porcine) model. |
| 2016-05-27 | GlaxoSmithKline | Regulatory StrimvelisTM receives European marketing authorisation to treat very rare disease, ADA-SCID gsk.com ↗
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) today announced that the European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency). |
| 2016-05-25 | GlaxoSmithKline | Results GSK, Fondazione Telethon and Ospedale San Raffaele announce publication of pivotal safety and efficacy of gene therapy for children with ADA-SCID gsk.com ↗
All 18 patients were alive after a median follow-up of 6.9 years (ranging from 2.3 to 13.4 years) at the data cut on 8th May 2014. |
| 2016-04-01 | GlaxoSmithKline | Regulatory GSK receives positive CHMP opinion in Europe for Strimvelis™, the first gene therapy to treat very rare disease, ADA-SCID gsk.com ↗
today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), in conjunction with the Committee for Advanced Therapies (CAT), has issued a positive opinion recommending marketing authorisation for Strimvelis |
| 2015-05-05 | GlaxoSmithKline | Regulatory GSK, Fondazione Telethon and Ospedale San Raffaele announce EU regulatory submission for gene therapy to treat rare disease ADA-SCID gsk.com ↗
I am very pleased that we are now at a stage to file for approval of this gene therapy for ADA-SCID – a devastating rare disease which drastically limits and shortens patients’ lives. |
All press releases naming this drug 27 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | gene therapy | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00002824 ↗ |
| Route | Other | “stereotactically injected H5.010RSVTK” NCT00002824 ↗ |