A Randomized, Controlled, Masked, Multi-center Study Evaluating the Efficacy, Safety, and Tolerability of Two Doses of AGTC-501 Compared to an Untreated Control Group in Male Participants With X-linked Retinitis Pigmentosa

This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec )) to an untreated control group in male participants with X-linked retinitis pigmentosa caused by RPGR mutations.

开始日期2024-03-14

申办/合作机构

Beacon Therapeutics (USA), Inc.

NCT06275620

/ Enrolling by invitation临床2期

A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa

This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.

开始日期2023-11-14

申办/合作机构-

NCT06333249

/ Active, not recruiting临床2期

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.

开始日期2021-04-13

申办/合作机构

Beacon Therapeutics (USA), Inc.

100 项与 Laruparetigene zosaparvovec 相关的临床结果

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100 项与 Laruparetigene zosaparvovec 相关的转化医学

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100 项与 Laruparetigene zosaparvovec 相关的专利（医药）

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项与 Laruparetigene zosaparvovec 相关的文献（医药）

2025-03-01·American Journal of Ophthalmology

Subretinal Gene Therapy Drug AGTC-501 for XLRP Phase 1/2 Multicenter Study (HORIZON): 24-Month Safety and Efficacy Results

Article

作者: Iannaccone, Alessandro ; Scaria, Abraham ; Sisk, Robert ; Curtiss, Darin ; Yaghy, Antonio ; Jung, JungAh ; Lauer, Andreas ; Yang, Paul ; Birch, David ; Waheed, Nadia K ; Pennesi, Mark E ; Anand, Rajiv

PURPOSETo evaluate the safety and efficacy of subretinal gene therapy using AGTC-501 (rAAV2tYF-GRK1-RPGR) in male participants with X-linked retinitis pigmentosa (XLRP).DESIGNPhase 1/2, open-label, dose-escalation study.METHODSSetting: Four centers in the United States. Patient or Study Population: Twenty-nine males with XLRP and confirmed pathogenic RPGR variants. Mean age was 31.6 years (range 15-55).INTERVENTIONSubretinal injection of AGTC-501 at doses ranging from 2.48 × 10¹⁰ to 1.99 × 10¹² vg/eye administered in one eye per participant. Subretinal injection sites initially targeted the peripheral retina and then transitioned to the macula with successive cohorts.MAIN OUTCOME MEASURESTreatment-emergent adverse events (TEAEs), serious adverse events (SAEs), laboratory parameters, and immunological responses. Efficacy was evaluated by mesopic microperimetry mean sensitivity.RESULTSAll 29 participants experienced ≥1 TEAE. Eleven (38%) experienced ≥1 grade 3 TEAE. Six (21%) experienced ≥1 ocular SAE related to AGTC-501, including retinal detachment (n=4), subcapsular cataract (n=1), and glaucoma (n=1). Two (6.9%) experienced non-ocular treatment-emergent SAEs. Immunological findings did not indicate safety concerns. Three of 4 participants at the highest dose exhibited concerning retinal pigment epithelial changes. Half the participants at the highest tolerated dose (6.8 × 10¹¹vg/eye) maintained ≥7 dB improvement in ≥5 loci at 24 months.CONCLUSIONSSubretinal AGTC-501 was generally well-tolerated. Despite all participants experiencing at least one TEAE, most of these events were mild in nature, exhibited complete resolution, and were associated with the subretinal injection procedure itself rather than the study agent. The highest dose exhibited an unfavorable risk-benefit profile due to the development of RPE changes. Although this group had the highest improvement in retinal sensitivity, our team has decided not to continue this dose in future clinical trials. Preliminary efficacy was observed at the maximum tolerated dose. Further studies are warranted to assess long-term safety and efficacy of AGTC-501 for XLRP treatment.

2020-02-01·Human Gene Therapy2区 · 医学

Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa

2区 · 医学

Article

作者: Ying, Gui-Shuang ; Dufour, Valérie L. ; Swider, Malgorzata ; Weinstein, Nicole M. ; Robinson, Paulette M. ; Cideciyan, Artur V. ; Aguirre, Gustavo D. ; Durham, Amy C. ; Chulay, Jeffrey D. ; Shearman, Mark S. ; Ye, Guo-jie ; Timmers, Adrian ; Pan, Wei ; Beltran, William A. ; Habecker, Perry L. ; Knop, David R. ; Jacobson, Samuel G. ; Song, Chunjuan

Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated rAAV2tYF-GRK1-hRPGRco, to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The vector contains a codon-optimized human RPGR cDNA (hRPGRco) driven by a photoreceptor-specific promoter (G protein-coupled receptor kinase 1 [GRK1]), and is packaged in an AAV2 capsid variant with three surface tyrosine residues changed to phenylalanine (AAV2tYF). We conducted a toxicity and efficacy study of this vector administered by subretinal injection in the naturally occurring RPGR mutant (X-linked progressive retinal atrophy 2 [XLPRA2]) dog model. Sixteen RPGR mutant dogs divided into four groups of three to five animals each received either a subretinal injection of 0.07 mL of AGTC-501 at low (1.2 × 10¹¹ vector genome [vg]/mL), mid (6 × 10¹¹ vg/mL), or high dose (3 × 10¹² vg/mL), or of vehicle control in the right eye at early-stage disease. The left eye remained untreated. Subretinal injections were well tolerated and were not associated with systemic toxicity. Electroretinography, in vivo retinal imaging, and histological analysis showed rescue of photoreceptor function and structure in the absence of ocular toxicity in the low- and mid-dose treatment groups when compared with the vehicle-treated group. The high-dose group showed evidence of both photoreceptor rescue and posterior segment toxicity. These results support the use of AGTC-501 in clinical studies with patients affected with XLRP caused by RPGR mutations and define the no-observed-adverse-effect level at 6 × 10¹¹ vg/mL.

2018-12-01·Human Gene Therapy Clinical Development

Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice

Article

作者: Robinson, Paulette M. ; Plummer, Cayrn ; Ye, Guo-jie ; Sonnentag, Peter ; Deng, Wen-Tao ; Green, Kari B. ; Zhu, Ping ; Chulay, Jeffrey D. ; Coleman, Kirsten E. ; Hauswirth, William W. ; Sharma, Alok K. ; Van Hoosear, Mailin ; Shearman, Mark S. ; Mandapati, Savitri ; Knop, David R. ; Conlon, Thomas J ; Timmers, Adrian ; Song, Chunjuan

Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The vector contains a codon-optimized human RPGR cDNA (RPGRco) driven by a photoreceptor-specific promoter (G protein-coupled receptor kinase 1, GRK1) and is packaged in an AAV2 capsid with three surface tyrosine residues changed to phenylalanine (AAV2tYF). We conducted a safety and potency study of this vector administered by subretinal a injection in the naturally occurring RPGR-deficient Rd9 mouse model. Sixty Rd9 mice (20 per group) received a subretinal injection in the right eye of vehicle (control) or AAV2tYF-GRK1-RPGRco at one of two dose levels (4 × 10⁸ or 4 × 10⁹ vg/eye) and were followed for 12 weeks after injection. Vector injections were well tolerated, with no systemic toxicity. There was a trend towards reduced electroretinography b-wave amplitudes in the high vector dose group that was not statistically significant. There were no clinically important changes in hematology or clinical chemistry parameters and no vector-related ocular changes in life or by histological examination. Dose-dependent RPGR protein expression, mainly in the inner segment of photoreceptors and the adjacent connecting cilium region, was observed in all vector-treated eyes examined. Sequence integrity of the codon-optimized RPGR was confirmed by sequencing of PCR-amplified DNA, or cDNA reverse transcribed from total RNA extracted from vector-treated retinal tissues, and by sequencing of RPGR protein obtained from transfected HEK 293 cells. These results support the use of rAAV2tYF-GRK1-RPGRco in clinical studies in patients with XLRP caused by RPGR mutations.

项与 Laruparetigene zosaparvovec 相关的新闻（医药）

2025-01-29

·Pharmaceutical Technology

FDA gives RMAT designation to Beacon’s retinitis pigmentosa treatment

Currently, there are no available treatments for XLRP, which primarily impacts young males. Credit: PeopleImages.com – Yuri A/Shutterstock. The US Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to Beacon Therapeutics’ gene therapy, laru-zova, for treating X-linked retinitis pigmentosa (XLRP), a severe, inherited retinal disease. The decision is based on the initial clinical evidence from the Phase II trials, DAWN and SKYLINE, which are assessing the safety, tolerability and efficacy of the therapy in XLRP individuals, who express retinitis pigmentosa GTPase regulator (RPGR) gene mutations. Beacon Therapeutics CEO Lance Baldo stated: “The FDA’s decision to grant RMAT designation to laru-zova is a significant milestone for the XLRP patient community, and underscores our promising data and the potential for laru-zova to significantly improve the lives of patients who suffer from XLRP. “We look forward to working closely with the FDA on continued development activities to support an expedited pathway for laru-zova.” Laru-zova demonstrated improvements in low luminance visual acuity (LLVA) in the DAWN trial. The therapy also gained fast track status from the US regulator, the UK Medicines and Healthcare Products Regulatory Agency’s innovative licensing and access pathway designation, and the European Medicines Agency’s PRIME designation. Designed to express the full-length RPGR protein, laru-zova addresses the extensive photoreceptor damage in XLRP. There are no currently available treatments for this condition, which primarily impacts young males. With a late-stage candidate for XLRP and two preclinical programmes targeting inherited cone-rod dystrophy and dry age-related macular degeneration, Beacon continues to advance its portfolio. The company’s efforts are supported by funds from Advent Life Sciences, Forbion, Oxford Science Enterprises, Syncona and TCGX. In July 2024, the company secured $170m in Series B funding to further the development of its XLRP treatment, AGTC-501. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

快速通道基因疗法临床2期

2024-12-14

·药明康德

多款缓解率超90%的CAR-T疗法；使100%患者近20个月无复发的细胞疗法…… | CGT周报

▎药明康德内容团队编辑近期，全球细胞和基因疗法（CGT）领域迎来系列进展。多款嵌合抗原受体（CAR）T细胞疗法在早期临床研究中表现亮眼，总缓解率（ORR）/客观缓解率超90%。源于人类多能干细胞的再生细胞候选疗法NRTX-1001用于治疗耐药性内侧颞叶癫痫（MTLE）患者，接受低剂量治疗的前两名患者在单次治疗两年后仍报告癫痫发作减少>97%。现货型γδ T细胞疗法INB-100用于接受减低强度预处理（RIC）后进行半相合造血干细胞移植（HSCT）的老年血液系统恶性肿瘤患者，在一项1期临床试验中使所有（100%）急性髓系白血病（AML）患者在中位随访时间为19.7个月时仍处于完全缓解（CR）状态，包括3名具有高风险细胞遗传学特征的AML患者。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。图片来源：123RF ———✦研发进展✦——— ◇ 强生（Johnson & Johnson）公司公布了3期临床试验CARTITUDE-4的最新研究结果。结果显示，在接受过1至3种治疗方案（包括蛋白酶体抑制剂），且对来那度胺耐药的复发或难治性多发性骨髓瘤（RRMM）患者中，单次输注与传奇生物联合开发的B细胞成熟抗原（BCMA）靶向CAR-T细胞疗法Carvykit（cilta-cel），与标准治疗方案相比，显著提高了患者的微量残留病（MRD）阴性率。在中位随访期为34个月时，可评估患者的MRD阴性率在cilta-cel治疗组中是标准治疗组的两倍以上（89%比38%；p<0.0001）。在接受治疗后30个月时，cilta-cel治疗组中持续（12个月或更长时间）达到MRD阴性完全缓解或更好的可评估患者比例是标准治疗组的五倍（52%比10%；p<0.0001）。 ◇ Protara Therapeutics公司公布了开放标签2期临床试验ADVANCED-2的最新结果。该试验评估了其基于一种失活的化脓性链球菌（Streptococcus pyogenes）菌株的在研细胞疗法TARA-002对携带高风险原位癌的非肌层浸润性膀胱癌（NMIBC）患者的疗效，包括对卡介苗（BCG）无应答或为BCG初治患者。结果显示，在所有患者中，接受治疗后6个月时的CR率为72%（13/18），任意时间点的CR率为70%（14/20）。在3位随访时间达到9个月的可评估患者中，有2位在9个月时仍保持CR。此外，在对BCG无应答的患者队列中，接受治疗6个月时的CR率为100%（4/4），任意时间点的CR率为80%（4/5）。 ◇ Beacon Therapeutics公司公布了在研基因疗法laru-zova（AGTC-501），在治疗X连锁视网膜色素变性（XLRP）患者的2期临床试验DAWN中获得的3个月中期数据。数据显示在低亮度视力（LLVA）这一关键视觉功能指标上，接受laru-zova治疗3个月的患者有早期改善的良好迹象。Laru-zova是一款基于腺相关病毒载体（AAV）的基因疗法，表达全长RPGR蛋白，因此有望解决XLRP导致的所有感光细胞的损伤，包括杆状细胞和锥状细胞的丧失，代表了针对XLRP患者渐进性视力丧失的潜在“best-in-class”治疗方案。 ◇ Arcellx公司公布了其BCMA靶向CAR-T细胞疗法anitocabtagene autoleucel（anito-cel）用于治疗RRMM的关键性2期研究iMMagine-1的积极数据。截至2024年10月31日的数据，中位随访时间为9.5个月，86例疗效可评估患者的ORR为97%（83/86），CR/严格CR（sCR）率为62%（53/86），非常好的部分缓解或更佳（≥VGPR）率为81%（70/86）。6个月时的无进展生存率和总生存率分别为93.3%和96.5%，12个月时的无进展生存率和总生存率分别为78.5%和96.5%。在此前的1期研究中，中位随访时间为38.1个月时，RRMM患者的中位无进展生存期为30.2个月。迄今为止，在此前的1期研究和iMMagine-1研究中给药的150多名患者中未观察到anito-cel导致的迟发性神经毒性。 ◇ Poseida Therapeutics公司公布了其BCMA靶向同种异体CAR-T疗法P-BCMA-ALLO1用于治疗RRMM患者的1期试验的中期数据。结果显示，优化淋巴细胞清除组（C组）患者的ORR为91%，既往未接受过BCMA靶向治疗患者的ORR为100%，而既往接受过至少一种BCMA和/或GPRC5D靶向治疗患者的ORR为86%。此外，该研究还取得了显著的安全性成果，无剂量限制性毒性反应（DLT），细胞因子释放综合征（CRS）和免疫效应细胞神经毒性综合征（ICANS）发生率低（均≤2级），无移植物抗宿主病（GVHD）或帕金森病。 ◇ Lyell Immunopharma公司公布了其CD19/CD20双靶向自体CAR-T细胞疗法IMPT-314治疗大B细胞淋巴瘤患者的多中心1/2期研究的初步数据。截至2024年10月22日的数据，17例疗效可评估患者的客观缓解率为94%，CR率为72%，这些患者既往接受过至少2线治疗，但未接受过CAR-T细胞疗法治疗。IMPT-314的安全性可控，无高级别CRS且3级ICANS发生率低，这些不良事件可通过标准治疗方案得到解决。 ◇ Synthekine公司公布了其CD19靶向自体CAR-T细胞疗法SYNCAR-001联用工程化改造的聚乙二醇化IL-2细胞因子STK-009治疗复发或难治性CD19阳性血液系统恶性肿瘤的1期研究的初步结果。截至2024年10月8日的数据，该组合疗法展现良好的安全性，未观察到DLT或IL-2相关毒性，无>2级的CRS或>1级的ICANS。所有（100%）接受该组合疗法治疗的非霍奇金淋巴瘤（NHL）患者均达到了CR并在数据截止时保持CR，最长的CR持续时间超过480天。 ◇ Neurona Therapeutics公司公布了其源于人类多能干细胞的再生细胞候选疗法NRTX-1001用于治疗耐药性内侧颞叶癫痫的1/2期临床试验结果。结果显示，低剂量组患者的中位失能性癫痫发作次数比基线减少92%，80%的患者在用药后7-12个月的主要疗效评估期间报告癫痫发作减少>75%。接受低剂量细胞疗法的前两名患者在单次治疗两年后仍报告癫痫发作减少>97%。高剂量组患者的中位失能性癫痫发作次数比基线减少78%，60%的患者在用药后4-6个月的中期评估期间报告癫痫发作减少>75%。在两个剂量组中，均未发现临床上显著的认知和生活质量损害，部分受试者的测试成绩显著提高。此外，迄今为止，高剂量和低剂量的NRTX-1001均耐受性良好。 ◇ AAVantgarde Bio公司公布了1/2期开放标签临床试验LUCE-1中前两名受试者的安全性数据。该研究旨在评估以AAV8为载体的视网膜内基因疗法AAVB-081用于治疗Usher综合征1B型（USH1B）相关的视网膜色素变性患者的安全性和耐受性。AAVB-081使用两个AAV8载体，每个载体都携带目标基因的一半信息，两个基因片段在细胞核内重新组合为一个完整的基因，从而能够生成治疗水平的MYO7A蛋白。此次公布的结果显示，两名接受低剂量治疗的受试者的最佳矫正视力（BCVA）在2周内恢复到治疗前的水平。所有不良事件都是轻微的，无需进一步干预即可解决。迄今为止，尚未观察到DLT。 ◇ CRISPR Therapeutics公司公布了其下一代CD19靶向同种异体CAR-T细胞疗法CTX112用于治疗复发或难治性CD19阳性B细胞恶性肿瘤的1/2期临床试验数据。结果显示，CTX112在所有剂量水平上均具有良好的耐受性，并在所有剂量水平下均产生了缓解。12名接受治疗的患者中有5名患者的缓解持续了超过6个月。1名接受剂量1治疗的患者在初始输注后的一年内仍保持CR。 ▲CTX112的临床疗效结果（图片来源：参考资料[12]） ◇ IN8bio公司公布了其在研现货型γδ T细胞疗法INB-100用于接受减低强度预处理后进行半相合造血干细胞移植的老年血液系统恶性肿瘤患者的1期试验的最新数据。历史数据显示，大约25%接受HSCT的AML患者预计在移植后的100天内会出现复发，其中高达50%的患者会在一年内复发。此次公布的结果显示，中位随访时间为19.7个月时，接受INB-100治疗的所有（100%）AML患者仍处于CR状态，包括3名具有高风险细胞遗传学特征的AML患者，他们在未接受维持治疗的情况下，仍然保持CR超过三年。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ Chroma Medicine公司和Nvelop Therapeutics公司宣布合并，将表观遗传编辑技术和下一代体内递送技术相结合，以成立一家名为nChroma Bio的公司重新定义基因药物的未来。在合并的同时，nChroma公司还完成了7500万美元的超额认购融资，为公司提供额外的资金，以推动其主导项目获得有意义的临床数据，并建立强大的下一代肝脏和肝外靶向治疗管线。该公司的主导项目CRMA-1001是一种肝脏靶向表观遗传编辑疗法，具有潜在“best-in-class”的效力和推动慢性乙型肝炎和丁型肝炎功能性治愈的潜力。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] CARVYKTI®▼ (ciltacabtagene autoleucel; cilta-cel) demonstrated significantly higher rates of minimal residual disease negativity compared to standard therapies in the CARTITUDE-4 study. Retrieved December 11, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994233/0/en/CARVYKTI-ciltacabtagene-autoleucel-cilta-cel-demonstrated-significantly-higher-rates-of-minimal-residual-disease-negativity-compared-to-standard-therapies-in-the-CARTITUDE-4-study.html [2] Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP). Retrieved December 6, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-announces-positive-3-month-data-from-phase-2-dawn-trial-of-laru-zova-agtc-501-in-patients-with-x-linked-retinitis-pigmentosa-xlrp-302324627.html [3] Protara Announces Positive Results from the Ongoing Phase 2 ADVANCED-2 Trial of TARA-002 in Patients with NMIBC. Retrieved December 5, 2024, from https://www.globenewswire.com/news-release/2024/12/05/2992222/0/en/Protara-Announces-Positive-Results-from-the-Ongoing-Phase-2-ADVANCED-2-Trial-of-TARA-002-in-Patients-with-NMIBC.html [4] Arcellx Announces New Positive Data for Its iMMagine-1 Study in Patients With Relapsed or Refractory Multiple Myeloma to be Presented During an Oral Presentation at the 66th ASH Annual Meeting and Exposition. Retrieved December 12, 2024, from https://ir.arcellx.com/news/news-details/2024/Arcellx-Announces-New-Positive-Data-for-Its-iMMagine-1-Study-in-Patients-With-Relapsed-or-Refractory-Multiple-Myeloma-to-be-Presented-During-an-Oral-Presentation-at-the-66th-ASH-Annual-Meeting-and-Exposition/default.aspx [5] Poseida Therapeutics Highlights Positive Interim Phase 1 Results for P-BCMA-ALLO1 and Preclinical Data for Dual CAR-T P-CD19CD20-ALLO1 at the 66th American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2024, from https://www.prnewswire.com/news-releases/poseida-therapeutics-highlights-positive-interim-phase-1-results-for-p-bcma-allo1-and-preclinical-data-for-dual-car-t-p-cd19cd20-allo1-at-the-66th-american-society-of-hematology-ash-annual-meeting-302325606.html [6] Lyell Presents Positive Initial Clinical Data from the Phase 1-2 Clinical Trial of IMPT-314 for the Treatment of B-cell Lymphoma at the 2024 ASH Annual Meeting. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994158/0/en/Lyell-Presents-Positive-Initial-Clinical-Data-from-the-Phase-1-2-Clinical-Trial-of-IMPT-314-for-the-Treatment-of-B-cell-Lymphoma-at-the-2024-ASH-Annual-Meeting.html [7] Synthekine Presents Positive Initial Results from Phase 1 Clinical Trial of CD19 CAR-T (SYNCAR-001) and Orthogonal IL-2 (STK-009) Combination Therapy for Treatment of Hematologic Malignancies at ASH 2024 Annual Meeting. Retrieved December 12, 2024, from https://www.synthekine.com/news/synthekine-presents-positive-initial-results-from-phase-1-clinical-trial-of-cd19-car-t-syncar-001-and-orthogonal-il-2-stk-009-combination-therapy-for-treatment-of-hematologic-malignancies-at-ash-2/ [8] AAVantgarde presents safety data of the LUCE-1 study of AAVB-081 for RP related to Usher 1B. Retrieved December 12, 2024, from https://www.aavantgarde.com/en/news/aavantgarde-presents-safety-data-of-the-luce-1-study-of-aavb-081-for-rp-related-to-usher-1b/ [9] Neurona Therapeutics Presents Positive Clinical Update: New Data from NRTX-1001 Cell Therapy Trial in Drug-resistant Epilepsy Announced at 2024 Annual Meeting of the American Epilepsy Society. Retrieved December 12, 2024, from https://www.neuronatherapeutics.com/news/press-releases/120624/ [10] IN8bio Reports Continued Progression-Free Survival in Phase 1 Investigator-Sponsored Trial of INB-100 Allogeneic Gamma-Delta T Cells for Leukemias at the 2024 American Society of Hematology Annual Meeting. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994365/0/en/IN8bio-Reports-Continued-Progression-Free-Survival-in-Phase-1-Investigator-Sponsored-Trial-of-INB-100-Allogeneic-Gamma-Delta-T-Cells-for-Leukemias-at-the-2024-American-Society-of-H.html [11] CRISPR Therapeutics Presents Data at the 2024 American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2993974/0/en/CRISPR-Therapeutics-Presents-Data-at-the-2024-American-Society-of-Hematology-ASH-Annual-Meeting.html [12] Chroma Medicine and Nvelop Therapeutics Unite to Form nChroma Bio, Securing $75 Million to Accelerate Genetic Medicines. Retrieved December 12, 2024, from https://chromamedicine.com/2024/12/10/chroma-medicine-and-nvelop-therapeutics-unite-to-form-nchroma-bio-securing-75-million-to-accelerate-genetic-medicines/ 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

细胞疗法临床结果基因疗法免疫疗法临床1期

2024-12-08

·药明康德

潜在“best-in-class”基因疗法积极2期临床结果公布，改善致盲疾病患者视力指标

▎药明康德内容团队编辑 Beacon Therapeutics日前公布了在研基因疗法laru-zova（AGTC-501），在治疗X连锁视网膜色素变性（XLRP）患者的2期临床试验DAWN中获得的3个月中期安全性和疗效数据。三个月的数据表明： Laru-zova在所有参与者中均具有良好的耐受性，未报告与研究药物相关的治疗伴发不良事件，包括无眼部炎症不良事件。数据还显示在低亮度视力（LLVA）这一关键视觉功能指标上有早期改善的良好迹象。获益风险评估结果支持继续进行针对由视网膜色素变性GTPase调节剂（RPGR）基因突变引起的XLRP患者的临床开发。 ▲Laru-zova在2期临床试验DAWN中改善患者低亮度视力（图片来源：Beacon Therapeutics官网） XLRP是一种严重的侵袭性遗传性视网膜疾病，主要影响年轻男性，常在患者中年时导致失明，目前尚无可用的治疗方法。Laru-zova是一款基于腺相关病毒载体的基因疗法，表达全长RPGR蛋白，因此有望解决XLRP导致的所有感光细胞的损伤，包括杆状细胞和锥状细胞的丧失，代表了针对XLRP患者渐进性视力丧失的潜在“best-in-class”治疗方案。目前，Beacon Therapeutics公司正在进行关键性2/3期临床试验VISTA，开发laru-zova作为XLRP患者的潜在疗法，患者注册正在进行中。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料： [1] Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP). Retrieved December 6, 2024, from https://www.prnewswire.com/news-releases/beacon-therapeutics-announces-positive-3-month-data-from-phase-2-dawn-trial-of-laru-zova-agtc-501-in-patients-with-x-linked-retinitis-pigmentosa-xlrp-302324627.html [2] Subretinal Gene Therapy laru-zova (AGTC-501) for X-Linked Retinitis Pigmentosa (XLRP) Phase 2 Multicenter Study (DAWN): Preliminary Results. Retrieved December 6, 2024, from https://www.beacontx.com/wp-content/uploads/2024/12/2024-Dec-3-FLORetina-2024-DAWN-3M-data-presentation-FINALw.pdf 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

基因疗法临床结果临床2期引进/卖出

100 项与 Laruparetigene zosaparvovec 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
X连锁视网膜色素变性	临床3期	英国	Beacon Therapeutics Ltd.初创企业	2024-03-14
X连锁视网膜色素变性	临床3期	英国	Beacon Therapeutics (USA), Inc.	2024-03-14
X连锁视网膜色素变性	临床3期	澳大利亚	Beacon Therapeutics (USA), Inc.	2024-03-14
X连锁视网膜色素变性	临床3期	美国	Beacon Therapeutics (USA), Inc.	2024-03-14

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06275620 (VISTA, PipelineReview) 人工标引	临床2期	X连锁视网膜色素变性	-	AGTC-501	(襯製夢衊餘鑰憲壓壓構) = None 糧膚蓋壓憲餘鹹糧積範 (鏇製願蓋鑰簾夢餘淵製 ) 更多	积极	2024-12-06
NCT06333249 (SKYLINE, Company_Website) 人工标引	临床2期	X连锁视网膜色素变性	-	AGTC-501	(積憲遞製衊獵積鹽廠壓) = 壓選積憲夢窪餘窪顧夢鏇鹽鏇顧壓範齋選顧願 (夢憲遞齋範餘鑰鑰遞鹹 )	积极	2024-10-15
EURETINA2024	N/A	X连锁视网膜色素变性	-	AGTC-501	憲衊襯範窪醖鹹襯憲蓋(膚顧範遞艱艱觸齋範窪) = No SUSARs or endophthalmitis were reported out to Month 36, and no participants early terminated due to a TEAE. Seven ocular SAEs were reported in 7 participants (retinal detachment [4], subcapsular cataract [1], glaucoma [1] and reduced visual acuity [1]). None of these were thought to be related to the investigational material. Of note, 3 of the retinal detachments occurred in periperally dosed patients. The TEAEs deemed related to AGTC-501 were all non-serious (mostly Grade 1-2 in severity), except one Grade 3 retinal depigmentation in the highest dose group which was not associated with any visual sequelae. Temporally, 73% of ocular TEAEs occurred within 3 months of AGTC-501 administration, 52% of which resolved within 1 month of onset. 餘夢鑰範鑰鹽糧鑰構醖 (窪鬱範壓壓鑰鹹顧憲廠 )	-	2024-09-19
NCT03316560 (Horizon, EURETINA2023)	临床1/2期	X连锁视网膜色素变性	29	AGTC-501	繭鬱繭鑰鑰糧製願窪網(鹽憲齋獵夢鏇觸築簾夢) = All non-serious (mostly Grade 1-2 in severity), except one Grade 3 retinal depigmentation 齋築簾齋網製獵遞餘繭 (壓範鬱網廠願艱遞遞窪 ) 更多	积极	2023-10-05