A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study

The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.

Start Date04 Jun 2018

Sponsor / Collaborator

NightstaRx Ltd.

NCT03496012 / CompletedPhase 3

A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).

Start Date11 Dec 2017

Sponsor / Collaborator

Biogen, Inc.

NCT03507686 / CompletedPhase 2

An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia With Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).

Start Date29 Nov 2017

Sponsor / Collaborator

Biogen, Inc.

100 Clinical Results associated with Timrepigene emparvovec

100 Translational Medicine associated with Timrepigene emparvovec

100 Patents (Medical) associated with Timrepigene emparvovec

Literatures (Medical) associated with Timrepigene emparvovec

01 Aug 2024·Human Gene Therapy

An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study

Article

Author: Pennesi, Mark E ; Huckfeldt, Rachel M ; Gow, James A ; MacLaren, Robert E ; Zhu, Kan ; Tsang, So-Fai ; Audo, Isabelle ; Fischer, M Dominik ; Li, Jiang ; Sisk, Robert ; Lam, Byron L

Choroideremia, an incurable, progressive retinal degeneration primarily affecting young men, leads to sight loss. GEMINI was a multicenter, open-label, prospective, two-period, interventional Phase II study assessing the safety of bilateral sequential administration of timrepigene emparvovec, a gene therapy, in adult males with genetically confirmed choroideremia (NCT03507686, ClinicalTrials.gov). Timrepigene emparvovec is an adeno-associated virus serotype 2 vector encoding the cDNA of Rab escort protein 1, augmented by a downstream woodchuck hepatitis virus post-transcriptional regulatory element. Up to 0.1 mL of timrepigene emparvovec, containing 1 × 10¹¹ vector genomes, was administered by subretinal injection following vitrectomy and retinal detachment. The second eye was treated after an intrasurgery window of <6, 6-12, or >12 months. Each eye was followed at up to nine visits over 12 months. Overall, 66 participants received timrepigene emparvovec, and 53 completed the study. Visual acuity (VA) was generally maintained in both eyes, independent of intrasurgery window duration, even after bilateral retinal detachment and subretinal injection. Bilateral treatment was well tolerated, with predominantly mild or moderate treatment-emergent adverse events (TEAEs) and a low rate of serious surgical complications (7.6%). Retinal inflammation TEAEs were reported in 45.5% of participants, with similar rates in both eyes; post hoc analyses found that these were not associated with clinically significant vision loss at month 12 versus baseline. Two participants (3.0%) reported serious noninfective retinitis. Prior timrepigene emparvovec exposure did not increase the risk of serious TEAEs or serious ocular TEAEs upon injection of the second eye; furthermore, no systemic immune reaction or inoculation effect was observed. Presence of antivector neutralizing antibodies at baseline was potentially associated with a higher percentage of TEAEs related to ocular inflammation or reduced VA after injection of the first eye. The GEMINI study results may inform decisions regarding bilateral sequential administration of other gene therapies for retinal diseases.

01 Oct 2023·Nature medicineQ1 · MEDICINE

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial.

Q1 · MEDICINE

Article

Author: Panda, Sushil ; Pennesi, Mark E ; Fischer, M Dominik ; Girach, Aniz ; Zhao, Guolin ; Sankila, Eeva-Marja K ; MacLaren, Robert E ; Gow, James A ; Yoon, Dan ; Lam, Byron L

Choroideremia is a rare, X-linked retinal degeneration resulting in progressive vision loss. A randomized, masked, phase 3 clinical trial evaluated the safety and efficacy over 12 months of follow-up in adult males with choroideremia randomized to receive a high-dose (1.0 × 10¹¹ vector genomes (vg); n = 69) or low-dose (1.0 × 10¹⁰ vg; n = 34) subretinal injection of the AAV2-vector-based gene therapy timrepigene emparvovec versus non-treated control (n = 66). Most treatment-emergent adverse events were mild or moderate. The trial did not meet its primary endpoint of best-corrected visual acuity (BCVA) improvement. In the primary endpoint analysis, three of 65 participants (5%) in the high-dose group, one of 34 (3%) participants in the low-dose group and zero of 62 (0%) participants in the control group had ≥15-letter Early Treatment Diabetic Retinopathy Study (ETDRS) improvement from baseline BCVA at 12 months (high dose, P = 0.245 versus control; low dose, P = 0.354 versus control). As the primary endpoint was not met, key secondary endpoints were not tested for significance. In a key secondary endpoint, nine of 65 (14%), six of 35 (18%) and one of 62 (2%) participants in the high-dose, low-dose and control groups, respectively, experienced ≥10-letter ETDRS improvement from baseline BCVA at 12 months. Potential opportunities to enhance future gene therapy studies for choroideremia include optimization of entry criteria (more preserved retinal area), surgical techniques and clinical endpoints. EudraCT registration: 2015-003958-41 .

01 Apr 2023·American Journal of Ophthalmology

AAV2-Mediated Gene Therapy for Choroideremia: 5-Year Results and Alternate Anti-sense Oligonucleotide Therapy

Article

Author: Dimopoulos, Ioannis S ; MacLaren, Robert E ; Radziwon, Alina ; MacDonald, Ian M ; Xu, Manlong ; Zhai, Yi ; Tennant, Matthew T ; Mah, Rachel ; Somani, Rizwan ; Crichton, Paul

PURPOSETo assess the long-term safety and efficacy of AAV2-REP1 in choroideremia (CHM) patients, and to test a potential antisense oligonucleotide therapy for CHM.DESIGNExtended, prospective phase 1/2 clinical trial and laboratory investigation.METHODSFive patients who received a single subfoveal injection of AAV2-REP1 were studied. The long-term safety was evaluated by ophthalmic examination, spectral domain optical coherence tomography, and fundus autofluorescence (FAF) for up to 5 years. Functional and structural changes were determined by different test modalities. Four antisense oligonucleotides (ASOs) were designed to treat the CHM c.1245-521A>G mutation, which was present in 2 patients within this trial.RESULTSSubject P3 experienced a localized intraretinal immune response that resulted in a significant loss of preserved retinal pigment epithelium (RPE). P4 experienced an exacerbation of peripheral retinoschisis. P2 had a constant ≥15-letter best-corrected visual acuity (BCVA) gain in the treated eye, whereas P5 had ≥15-letter BCVA improvement once in the untreated eye. The preserved FAF areas declined more rapidly in the treated eyes compared to the untreated eyes (P = .043). A customized 25-mer ASO recovered 83.2% to 95.0% of the normal RNA and 57.5% of the normal protein in fibroblasts from 2 trial patients.CONCLUSIONSIntraretinal inflammation triggered by AAV2-REP1 subretinal injection stabilized after 2 years but resulted in permanent damage to the retinal structure. Long-term progression of the disease was seen in both treated and untreated eyes, casting doubt as to the effectiveness of this approach in late-stage CHM. Alternative approaches such as ASO may have a therapeutic effect in a subgroup of CHM patients.

News (Medical) associated with Timrepigene emparvovec

22 Nov 2022

·www.businesswire.com

AAV Vectors in Gene Therapy - Pipeline Insight, 2022 - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "AAV vectors in gene therapy - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering. AAV vectors in gene therapy: Overview Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs, and harnessing revolutionary biotechnologies have contributed extensively to the growth of the gene therapy field. AAV Vectors in Gene Therapy Emerging Drugs GS010: Gensight Biologics LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. "LUMEVOQ" was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. Valoctocogene roxaparvovec: BioMarin Pharmaceutical Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A. It is currently in preregistration stage of development. In June 2021, BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA). In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase III GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA. DTX401: Ultragenyx Pharmaceutical DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-? under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-? activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. In a Phase I/II clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. The drug is currently being evaluated in Phase III clinical trial to treat patients with Glycogen storage disease type I. AAV5-RPGR: MeiraGTx AAV-RPGR is an investigational gene therapy for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase III clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15. Timrepigene emparvovec: Biogen Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase III STAR study. AMT 061: UniQure AMT-061 (etranacogene dezaparvovec) is an experimental gene therapy that uniQure is developing to treat hemophilia B. AMT-061 uses a modified and harmless adeno-associated virus 5 (AAV5) to deliver a highly functional copy of the F9 gene, called FIX-Padua, to patients' cells. The FIX-Padua gene version was shown to result in FIX clotting activity eight times greater than that associated with the standard F9 gene. As such, the one-time therapy - administrated directly into the bloodstream - is expected to increase FIX levels, helping to prevent and control bleeds. If approved, the therapy will be marketed globally by CSL Behring, which closed a commercialization and licensing agreement with uniQure in May 2021. UniQure and CSL Behring expect to file a regulatory application to the U.S. Food and Drug Administration (FDA) in early 2022 seeking AMT-061's approval for hemophilia B. Research and Development. RGX-314: REGENXBIO RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina. REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye. According to the company's pipeline, the drug is currently in the Phase III stage of development. SPK-8011: Spark Therapeutics Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the US, while the European Commission has granted orphan designation to SPK-8011. NFS-01: Neurophth Investigational NR082, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel ophthalmic injection that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND4 mutations. It is currently being evaluated in Phase II/III clinical trial. GT 005: Gyroscope Therapeutics GT005 is designed as an AAV2-based one-time investigational gene therapy for GA secondary to AMD that is delivered under the retina. GT005 aims to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. Complement overactivation has been strongly correlated with the development and progression of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production could dampen the system's overactivity and reduce inflammation, with the goal of preserving a person's eyesight. Gyroscope is also evaluating GT005 in two Phase II clinical trials. Further product details are provided in the report AAV vectors in gene therapy: Therapeutic Assessment This segment of the report provides insights about the different AAV vectors in gene therapy drugs segregated based on following parameters that define the scope of the report, such as: Major Players in AAV vectors in gene therapy There are approx. 70+ key companies which are developing the therapies for AAV vectors in gene therapy. The companies which have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include, BioMarin Pharmaceutical. Phases The publisher's report covers around 235+ products under different phases of clinical development like Late stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) along with the details of Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of Administration Key Players BioMarin Pharmaceutical Gensight Biologics PTC therapeutics Ultragenyx Pharmaceutical MeiraGTx Pfizer Biogen uniQure Pfizer Ultragenyx Pharmaceutical REGENXBIO Biogen Spark therapeuics (Roche) Sarepta therapeutics Neurophth Therapeutics LYSOGENE Gyroscope Therapeutics Nanoscope Therapeutics Homology medicines Ultragenyx Pharmaceutical Passage Bio Freeline therapeutics Astellas Pharma Aspa therapeutics Adrenas Therapeutics ESTEVE Sio Gene Therapies Amicus therapeutics 4D Molecular therapeutics Taysha Gene Therapies Atsena Therapeutics BioMarin Pharmaceutical Abeona Therapeutics REGENXBIO uniQure Taysha Gene Therapies Asklepios BioPharmaceutical Sarepta Therapeutics Abeona Therapeutics Forge Biologics Sangamo therapeuics Bayer LogicBio therapeutics Solid Biosciences Applied Genetic Technologies Spark therapeuics (Roche) Eli lilly and company Lexeo Therapeutics Spark therapeutics Gensight Biologics Alcyone Lifesciences MeiraGTx Audentes therapeutics Vivet Therapeutics Takeda Astellas Gene Therapies Homology Medicines Adverum Biotechnologies Libella Gene Therapeutics Rocket Pharmaceuticals Brain Neurotherapy Bio, Inc. Ocugen Asklepios BioPharmaceutical Neurogene Jaguar Gene Therapy, LLC Axovia Therapeutics Coave therapeutics Novartis/ Arctos medical Novartis/Vedere Bio REGENXBIO Inc. StrideBio Abeona therapeutics Sanofi/ Sirion therapeutics Capsida Biotherapeutics Kriya therapeutics Neurophth Poseida Therapeutics, BridgeBio Pharma DiNAQOR/ Biomarin ViGeneron AskBio/Selecta Biosciences Prevail therapeutics Intas Pharmaceuticals Tenaya Therapeutics Scout Bio therapeutics Ocugen Oyster Point pharmaceuticals Cyprium Therapeutics Solid Biosciences Amicus Therapeutics Coave therapeutics AvantiBio Voyager Therapeutics/ Pfizer Aruvant Sciences Alcyone Lifesciences Gene Therapy Research Institution Co., Ltd. For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/ylu69s .

Phase 3Orphan DrugGene TherapyLicense out/inClinical Result

09 Sep 2021

·www.globenewswire.com

Drug Development of Choroideremia Pipeline Report Analysis

Los Angeles, USA, Sept. 08, 2021 (GLOBE NEWSWIRE) -- Drug Development of Choroideremia Pipeline Report Analysis | DelveInsight The need for a new therapeutic option is acute. Clinical research is underway investigating the gene therapies for the effective treatment of Choroideremia. DelveInsight’s “Choroideremia Pipeline Insight” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in the Choroideremia pipeline landscapes. It comprises Choroideremia pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Choroideremia therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Choroideremia pipeline products. Some of the key takeaways from the Choroideremia Pipeline Report Get an overview of pipeline landscape @ Choroideremia Clinical Trials Analysis Choroideremia is a rare genetic disorder of vision that is more common in men. It is an X-linked recessive condition that gradually degrades the choroid, retinal pigment epithelium (RPE), and photoreceptors. Choroideremia Emerging Drugs NightstaRx Ltd initiated the development of BIIB111 for the treatment of Choroideremia. BIIB111 is a gene therapy-based treatment consisting of AAV2 vectors with recombinant human DNA designed to deliver REP1 protein to the retina. Spark Therapeutics is advancing an open-label, dose-escalating Phase I/II trial designed to assess the safety and preliminary efficacy of subretinal administration of investigational SPK-7001. Each of the investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and their collaborators. For further information, refer to the detailed report @ Choroideremia Pipeline Therapeutics Scope of Choroideremia Pipeline Drug Insight · Choroideremia Therapies Late-stage (Phase III) · Choroideremia Therapies Mid-stage (Phase II)· Choroideremia Therapies Early-stage (Phase I) · Choroideremia Preclinical stage and Discovery candidates · Discontinued and Inactive candidates · Gene transference· CHM protein expression stimulants; Gene transference· Gluconeogenesis inhibitors· CHM protein expression stimulants; Gene transference · Peptides· Monoclonal antibodies· Small molecules · Polymer · Gene therapy · Parenteral · Intravitreal· Oral· Subretinal · Monotherapy· Combination· Mono/Combination Key Questions regarding Current Choroideremia Treatment Landscape and Emerging Therapies Answered in the Pipeline Report Table of Contents Get a customized pipeline report @ Choroideremia Drugs Pipeline Report Other Reports Browse Blog Posts About DelveInsightDelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also provides Healthcare Consulting services comprising credible market analysis that will help accelerate the business growth and overcome challenges with a practical approach.

Gene TherapyAntibodySmall molecular drug

15 Jun 2021

·www.biospace.com

After Controversial AD Drug Win, Biogen's Retinal Gene Therapy Flops

John Tlumacki/The Boston Globe via Getty Images Pivoting from conflicting and controversial press over its recently approved Alzheimer’s disease treatment, Biogen has announced yesterday that its gene therapy drug timrepigene emparvovec has failed to show a clinically meaningful benefit for a rare inherited eye disease in a Phase III trial. The late-stage STAR study enrolled 169 adult males with choroideremia, an inherited retinal disease characterized by progressive vision loss and ultimate blindness. The investigators evaluated the efficacy and safety of a single subretinal injection of the investigational gene therapy timrepigene emparvovec. This drug was designed to deliver a functional human choroideremia gene into photoreceptor cells and the retinal pigment epithelium to address the condition’s underlying genetic causative mechanisms. In a statement on the STAR findings, Biogen stated the study failed to meet the primary endpoint of the proportion of patients who experienced a ≥15-letter improvement in the best corrected visual acuity (BCVA) at one year in the intervention arm versus the control group. The primary endpoint was assessed using the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart. Also, Biogen reported that the trial failed to show efficacy regarding the key secondary endpoints. However, the safety findings from the study were generally consistent with previous research trials. “We extend our deepest gratitude to all those who contributed to the STAR study, including the participants, investigators, site staff and the broader choroideremia community,” said Katherine Dawson, M.D., Biogen’s Head of the Therapeutics Development Unit. “While we are disappointed by the results of the STAR study, we are hopeful that the clinical insights gleaned from this study may help to shape therapeutic innovation for inherited retinal diseases including choroideremia, so that in the future there may be treatment options for the community affected by these debilitating disorders.” The company noted it would continue to assess the STAR study’s complete data set before confirming its future plans for clinical development of timrepigene emparvovec. These topline results from the STAR trial follow a controversial approval of Biogen’s Alzheimer’s drug Aduhelm (aducanumab). As reported by the company on June 7, the U.S. Food and Drug Administration (FDA) granted accelerated approval to the therapy to address the accumulation of amyloid beta plaques in the brain associated with the neurologic disease. The approval was based on clinical trial data showing the therapy could reduce these plaques, but these studies did not demonstrate an overall clinically meaningful reduction in cognitive decline. Instead, the plaques serve as a biomarker for cognitive dysfunction in patients with Alzheimer’s disease, and both Biogen and some regulators at the FDA surmise the reduction in these plaques could possibly provide a beneficial effect. But as reported in opinion pieces in the New York Times and Bloomberg, among other publications, the approval of the dementia drug is based on “spotty evidence,” which serves to threaten the FDA’s reputation. As such, three-panel members of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee have resigned from their posts in protest over the Aduhelm approval. “My rationale was that the FDA needs to re-evaluate how it solicits and uses the advisory committees … because I didn’t think that the firm recommendations from the committee in this case … were appropriately integrated into the decision-making process,” said one of the resigning members, Aaron Kesselheim, a professor of Medicine at Harvard Medical School, in an interview with Reuters.

Gene TherapyAccelerated Approval

100 Deals associated with Timrepigene emparvovec

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
X-linked retinitis pigmentosa	Phase 3	FR	NightstaRx Ltd.	04 Jun 2018
X-linked retinitis pigmentosa	Phase 3	GB	NightstaRx Ltd.	04 Jun 2018
X-linked retinitis pigmentosa	Phase 3	DE	NightstaRx Ltd.	04 Jun 2018
X-linked retinitis pigmentosa	Phase 3	FI	NightstaRx Ltd.	04 Jun 2018
X-linked retinitis pigmentosa	Phase 3	DK	NightstaRx Ltd.	04 Jun 2018
X-linked retinitis pigmentosa	Phase 3	CA	NightstaRx Ltd.	04 Jun 2018
X-linked retinitis pigmentosa	Phase 3	US	NightstaRx Ltd.	04 Jun 2018
Retinal Dysplasia	Phase 3	-	University of Oxford	-
Retinal Dysplasia	Phase 3	-	Biogen, Inc.	-
Choroideremia	Discovery	US	-	21 Sep 2017

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03507686 (GEMINI, CTgov)	Phase 2	Choroideremia	66	BIIB111 (Treatment Period 1: BIIB111)	lseebcfdwj(rpihmlwtkv) = ckkpfxjceo fjchrskgcz (bitqqtsgbn, owfpoaegzx - qdyoqdcfbv) View more	-	18 Sep 2023
NCT03507686 (GEMINI, CTgov)	Phase 2	Choroideremia	66	BIIB111 (Treatment Period 2: BIIB111)	lseebcfdwj(rpihmlwtkv) = qtpwslysgw fjchrskgcz (bitqqtsgbn, mviwgzgykd - rmdnyvhadt) View more	-	18 Sep 2023
NCT02553135 (CTgov)	Phase 2	Choroideremia	6	Injection of AAV2-REP1 (10e11 vg)	xnyukyptot(sbcpaugzzn) = kpsdukxdhy vfmhgdymwi (pvotrwxqxs, squkcwggry - jzilnxtaxn) View more	-	29 Jul 2019
NCT02671539 \| NCT01461213 \| NCT02077361 \| NCT02553135 (NSR-REP1, AAO2018)	Phase 1/2	Choroideremia AAV2 vector encoding REP1	32	NSR-REP1	(jkynsbgkmh) = fkswarypvt ydjewyqret (vcoftqgpbn, -0.1 to 5.7)	Positive	29 Oct 2018

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