Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of EDIT-101 in Adult and Pediatric Participants With Leber Congenital Amaurosis Type 10 (LCA10), With Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in Intron 26 (IVS26) of the CEP290 Gene ("LCA10-IVS26")

The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene ("LCA10-IVS26").

Start Date26 Sep 2019

Sponsor / Collaborator

Editas Medicine, Inc.

100 Clinical Results associated with Brinretigene vesgedparvovec

100 Translational Medicine associated with Brinretigene vesgedparvovec

100 Patents (Medical) associated with Brinretigene vesgedparvovec

Literatures (Medical) associated with Brinretigene vesgedparvovec

01 Mar 2021·Human gene therapyQ2 · MEDICINE

Genome-Editing Strategies for Treating Human Retinal Degenerations

Q2 · MEDICINE

Review

Author: Xue, Kanmin ; Kantor, Ariel ; McClements, Michelle E. ; Cehajic-Kapetanovic, Jasmina ; MacLaren, Robert E. ; Quinn, Joel ; Musa, Ayesha

Inherited retinal degenerations (IRDs) are a leading cause of blindness. Although gene-supplementation therapies have been developed, they are only available for a small proportion of recessive IRD mutations. In contrast, genome editing using clustered-regularly interspaced short palindromic repeats (CRISPR) CRISPR-associated (Cas) systems could provide alternative therapeutic avenues for treating a wide range of genetic retinal diseases through targeted knockdown or correction of mutant alleles. Progress in this rapidly evolving field has been highlighted by recent Food and Drug Administration clinical trial approval for EDIT-101 (Editas Medicine, Inc., Cambridge, MA), which has demonstrated efficacious genome editing in a mouse model of CEP290-associated Leber congenital amaurosis and safety in nonhuman primates. Nonetheless, there remains a significant number of challenges to developing clinically viable retinal genome-editing therapies. In particular, IRD-causing mutations occur in more than 200 known genes, with considerable heterogeneity in mutation type and position within each gene. Additionally, there are remaining safety concerns over long-term expression of Cas9 in vivo. This review highlights (i) the technological advances in gene-editing technology, (ii) major safety concerns associated with retinal genome editing, and (iii) potential strategies for overcoming these challenges to develop clinical therapies.

01 Feb 2019·Nature medicineQ1 · MEDICINE

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

Q1 · MEDICINE

Article

Author: Gloskowski, Sebastian W ; Wang, Tongyao ; Tabbaa, Diana G ; Ta, Terence ; Skor, Maxwell N ; Marco, Eugenio ; Samuelsson, Steven J ; Maeder, Morgan L ; Hopkins, Bei ; Jayaram, Hariharan ; Jiang, Haiyan ; Baral, Reshica ; Myer, Vic E ; Reyon, Deepak ; Bounoutas, George S ; Glucksmann, Alexandra ; Wilson, Christopher J ; Albright, Charles F ; DaSilva, Jennifer A ; Yudkoff, Clifford ; Fennell, Tim J ; Dhanapal, Vidya ; Giannoukos, Georgia ; Shen, Shen ; Gotta, Gregory M ; Chao, Hoson ; Haskett, Scott J ; Joshi, Shivangi ; Dass, Abhishek ; Mepani, Rina ; Ciulla, Dawn M ; Horng, Joy E ; Stefanidakis, Michael ; Bumcrot, David ; Friedland, Ari E ; Stetkiewicz, Pam ; Barrera, Luis Alberto

Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the CEP290 gene^1,2. We developed EDIT-101, a candidate genome-editing therapeutic, to remove the aberrant splice donor created by the IVS26 mutation in the CEP290 gene and restore normal CEP290 expression. Key to this therapeutic, we identified a pair of Staphylococcus aureus Cas9 guide RNAs that were highly active and specific to the human CEP290 target sequence. In vitro experiments in human cells and retinal explants demonstrated the molecular mechanism of action and nuclease specificity. Subretinal delivery of EDIT-101 in humanized CEP290 mice showed rapid and sustained CEP290 gene editing. A comparable surrogate non-human primate (NHP) vector also achieved productive editing of the NHP CEP290 gene at levels that met the target therapeutic threshold, and demonstrated the ability of CRISPR/Cas9 to edit somatic primate cells in vivo. These results support further development of EDIT-101 for LCA10 and additional CRISPR-based medicines for other inherited retinal disorders.

Frontiers in genome editing

Prime Editing for Inherited Retinal Diseases

Review

Author: Eulau, Eric ; da Costa, Bruna Lopes ; Levi, Sarah R ; Tsai, Yi-Ting ; Quinn, Peter M J

Inherited retinal diseases (IRDs) are chronic, hereditary disorders that lead to progressive degeneration of the retina. Disease etiology originates from a genetic mutation—inherited or de novo—with a majority of IRDs resulting from point mutations. Given the plethora of IRDs, to date, mutations that cause these dystrophies have been found in approximately 280 genes. However, there is currently only one FDA-approved gene augmentation therapy, Luxturna (voretigene neparvovec-rzyl), available to patients with RPE65-mediated retinitis pigmentosa (RP). Although clinical trials for other genes are underway, these techniques typically involve gene augmentation rather than genome surgery. While gene augmentation therapy delivers a healthy copy of DNA to the cells of the retina, genome surgery uses clustered regularly interspaced short palindromic repeats (CRISPR)-based technology to correct a specific genetic mutation within the endogenous genome sequence. A new technique known as prime editing (PE) applies a CRISPR-based technology that possesses the potential to correct all twelve possible transition and transversion mutations as well as small insertions and deletions. EDIT-101, a CRISPR-based therapy that is currently in clinical trials, uses double-strand breaks and nonhomologous end joining to remove the IVS26 mutation in the CEP290 gene. Preferably, PE does not cause double-strand breaks nor does it require any donor DNA repair template, highlighting its unparalleled efficiency. Instead, PE uses reverse transcriptase and Cas9 nickase to repair mutations in the genome. While this technique is still developing, with several challenges yet to be addressed, it offers promising implications for the future of IRD treatment.

News (Medical) associated with Brinretigene vesgedparvovec

08 May 2024

·www.globenewswire.com

Editas Medicine Announces First Quarter 2024 Results and Business Updates

Completed adult cohort enrollment and enrolled multiple patients in the adolescent cohort of the Phase 1/2/3 RUBY clinical trial of reni-cel for severe sickle cell disease On track to present additional clinical data from the RUBY trial and the EdiTHAL trial of reni-cel for transfusion-dependent beta-thalassemia in mid-2024 and additional updates by year-end 2024 Presenting pre-clinical data at ASGCT on in vivo capabilities to support development of transformative in vivo gene editing medicines Strong financial position with operational runway into 2026 CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today reported financial results for the first quarter 2024 and provided business updates. “We made significant progress in all three pillars of our strategy this quarter, including reni-cel, in vivo, and business development, including intellectual property,” commented Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. “We entered 2024 with great momentum, and I am proud of the Editas team’s significant progress toward becoming a commercial-stage company and on developing clinically differentiated, transformational medicines for people living with serious, previously untreatable diseases.” Recent Achievements and Outlook Ex Vivo Hemoglobinopathies Reni-cel (renizgamglogene autogedtemcel, previously EDIT-301) for Severe Sickle Cell Disease (SCD) The Company has completed enrollment of the adult cohort of the Phase 1/2/3 RUBY trial for SCD and continues to dose patients.The Company has enrolled multiple patients in the adolescent cohort in the RUBY trial.The Company remains on-track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up in the RUBY trial in mid-2024 and further data by year-end 2024. Reni-cel for Transfusion-dependent Beta Thalassemia (TDT) The Company continues to enroll and dose patients in the EdiTHAL trial for TDT.The Company remains on-track to present additional clinical data from the EdiTHAL trial in mid-2024 and further data by year-end 2024. In Vivo Medicines The Company is presenting pre-clinical data later this week to support its development of transformative in vivo gene editing medicines at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting on May 9 and 10. Presentations include: Oral presentation of in vivo pre-clinical data from mouse studies using lipid nanoparticle (LNP)-mediated delivery of an optimized guide RNA (gRNA) and engineered AsCas12a messenger RNA (mRNA).Pre-clinical data demonstrating AsCas12a gRNA modifications that enable high-potency gene editing in multiple cell types, including in the liver, and improve gene editing outcomes in vivo, enabling the development of in vivo gene editing medicines.Research on identifying potent large serine recombinases (LSRs) as a foundation to develop novel in vivo gene editing technologies for whole gene knock-in, expanding potential in vivo gene editing targets for developing medicines. The Company is on track to establish in vivo preclinical proof-of-concept for an undisclosed indication by year-end. Other Corporate Highlights Business Development In March 2024, Editas Medicine and Bristol Myers Squibb (NYSE: BMY) signed a two-year extension to the collaboration under which the parties may research, develop, and commercialize autologous and allogeneic alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. The extension also has options to further extend the collaboration for up to an additional two years. Intellectual Property Oral Arguments held in CRISPR patent interferenceOn May 7, 2024, oral arguments were held before the U.S. Court of Appeals for the Federal Circuit (CAFC) regarding an appeal of the Patent Trial and Appeal Board’s (PTAB’s) previous decision favoring Broad Institute (Broad) in the U.S. patent interference involving specific patents for CRISPR/Cas9 editing in human cells between the University of California, the University of Vienna, and Emmanuelle Charpentier (collectively, CVC) and Broad. A decision on the case is expected in the second half of 2024. Leadership Gregory Whitehead named Executive Vice President and Chief Technical and Quality OfficerMr. Whitehead joins the executive leadership team as Chief Technical and Quality Officer (CTQO), responsible for leading the Technical Development, Technical Operations, and Quality departments as the Company advances its gene editing medicines towards biologics license applications (BLAs). Mr. Whitehead has more than 25 years of experience in the biotech industry and extensive cell and gene therapy clinical and commercial development expertise. Prior to his current role, Mr. Whitehead served as Senior Vice President of Quality at Editas Medicine. Before joining Editas Medicine, Mr. Whitehead served as Chief Quality Officer at Rubius Therapeutics, establishing quality capabilities to support first-in-human clinical trials for genetically engineered red blood cells. Earlier in his career, he served as Vice President, Quality at bluebird bio. During his tenure at bluebird bio, Mr. Whitehead scaled the organization in support of multiple gene therapy clinical trials and commercial launch in the U.S. and Europe. Publications The New England Journal of Medicine published the findings from the Phase 1/2 BRILLIANCE trial of EDIT-101, on May 6. The BRILLIANCE trial was the first in vivo CRISPR gene editing study in humans, and the study demonstrated clinical proof of concept in treatment of a rare inherited blindness, Leber Congenital Amaurosis 10, with a favorable safety profile, and preliminary efficacy signals for patients. First Quarter 2024 Financial ResultsCash, cash equivalents, and marketable securities as of March 31, 2024, were $376.8 million compared to $427.1 million as of December 31, 2023. The Company expects the existing cash, cash equivalents, and marketable securities together with the near-term annual license fees and the contingent upfront payment payable under its license agreement with Vertex Pharmaceuticals, Incorporated, to fund operating expenses and capital expenditures into 2026. First Quarter 2024 For the three months ended March 31, 2024, net loss attributable to common stockholders was $62.0 million, or $0.76 per share, compared to net loss of $49.0 million, or $0.71 per share, for the same period in 2023.Collaboration and other research and development revenues decreased to $1.1 million for the three months ended March 31, 2024, compared to $9.9 million for the same period in 2023. The decrease relates to the one-time sale in January 2023 of the Company's wholly owned oncology assets and related licenses. Research and development expenses increased by $11.0 million to $48.8 million for the three months ended March 31, 2024, compared to $37.8 million for the same period in 2023. The increase is primarily attributable to sublicense and license payments as well as clinical and manufacturing costs related to the continued progression of the Company's reni-cel program. General and administrative expenses decreased by $3.7 million to $19.3 million for the three months ended March 31, 2024, compared to $23.0 million for the same period in 2023. The decrease is primarily attributable to one-time professional service expenses related to 2023 strategic initiatives and business development activities as well as reduced legal and patent costs. Upcoming Events Editas Medicine plans to participate in the following scientific and medical conference: American Society of Gene and Cell Therapy (ASGCT) 27th Annual MeetingMay 7-11, 2024Baltimore, MD Editas Medicine plans to participate in the following investor events: Bank of America Health Care Conference 2024May 14, 2024Las Vegas, NV 2024 RBC Capital Markets Global Healthcare Conference May 15, 2024New York, NY Stifel’s 2nd Annual Genetic Medicines ForumMay 28, 2024Virtual Conference Call The Editas Medicine management team will host a conference call and webcast today at 8:00 a.m. ET to provide and discuss a corporate update and financial results for the first quarter of 2024. To access the call, please dial 1-877-407-0989 (domestic) or 1-201-389-0921 (international) and ask for the Editas Medicine earnings call. A live webcast of the call will also be available on the Investors section of the Editas Medicine website at www.editasmedicine.com, and a replay will be available approximately two hours after its completion. About Editas MedicineAs a clinical-stage gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com. Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, including establishing in vivo preclinical proof-of-concept for an undisclosed indication by year-end 2024, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including presenting additional clinical data from the RUBY and EdiTHAL trials in mid-2024 and additional updates by year-end 2024, potential of, and expectations for, the Company’s product candidates, the timing or likelihood of regulatory filings and approvals, the Company’s expectations regarding commercial readiness, and the Company’s expectations regarding cash runway. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials, including the RUBY and EdiTHAL trials, and clinical development of the Company’s product candidates, including reni-cel; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent the Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements. EDITAS MEDICINE, INC.Consolidated Statement of Operations(amounts in thousands, except share and per share data)(Unaudited) Three Months Ended March 31, 2024 2023 Collaboration and other research and development revenues$1,135 $9,851 Operating expenses: Research and development 48,787 37,804 General and administrative 19,339 23,008 Total operating expenses 68,126 60,812 Operating loss (66,991) (50,961)Other income, net: Other income (expense), net 6 (1,584)Interest income, net 5,035 3,509 Total other income, net 5,041 1,925 Net loss$(61,950) $(49,036)Net loss per share, basic and diluted (0.76) (0.71)Weighted-average common shares outstanding, basic and diluted 81,938,839 68,924,180 EDITAS MEDICINE, INC.Selected Consolidated Balance Sheet Items(amounts in thousands)(Unaudited) March 31, December 31, 2024 2023Cash, cash equivalents, and marketable securities$376,776 $427,135Working capital 239,068 277,612Total assets 440,347 499,153Deferred revenue, net of current portion 54,204 60,667Total stockholders' equity 294,400 349,097

Gene TherapyFinancial StatementExecutive ChangeLicense out/in

06 May 2024

·www.sciencedaily.com

Participants of pioneering CRISPR gene editing trial see vision improve

About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a new article. About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published today in the New England Journal of Medicine. "This trial shows CRISPR gene editing has exciting potential to treat inherited retinal degeneration," said Mark Pennesi, M.D., Ph.D., a corresponding author on the paper, an ophthalmologist and Oregon Health & Science University's lead scientist for the Phase 1/2 BRILLIANCE trial. "There is nothing more rewarding to a physician than hearing a patient describe how their vision has improved after a treatment. One of our trial participants has shared several examples, including being able to find their phone after misplacing it and knowing that their coffee machine is working by seeing its small lights. "While these types of tasks might seem trivial to those who are normally sighted, such improvements can have a huge impact on quality of life for those with low vision." The BRILLIANCE trial evaluated the safety and effectiveness of EDIT-101, an experimental gene editing treatment developed by Editas Medicine that uses CRISPR technology. The experimental treatment was designed to edit a mutation in the CEP290 gene, which provides instructions to create a protein that is critical for sight. People with this gene mutation have a rare condition that is commonly called Leber Congenital Amaurosis, or LCA, Type 10, for which there is currently no Food and Drug Administration-approved treatment. LCA's various types occur in about 2 or 3 out of 100,000 newborns. The OHSU Casey Eye Institute treated the trial's first participant in early 2020. That procedure also marked the first time that CRISPR had been used to edit genes within the human body, called in vivo gene editing. The new paper describes the study's findings through February 2023 and details how the trial's 14 participants -- 12 adults and two children -- responded to receiving EDIT-101 in one eye. Key results include: Four specific outcomes were used to evaluate the experimental treatment's effectiveness: Further research for a future treatment In November 2022, trial sponsor Editas Medicine announced that it was pausing the trial's enrollment and would seek another partner to continue the experimental therapy's development. Pennesi and colleagues are exploring working with other commercial partners to conduct additional trials, in collaboration with Editas. The researchers hope future studies can examine ideal dosing, whether a treatment effect is more pronounced in certain age groups such as younger patients, and include refined endpoints to measure impacts on activities of daily living. "This research demonstrates that CRISPR gene therapy for inherited vision loss is worth continued pursuit in research and clinical trials," said Mass Eye & Ear ophthalmologist Eric Pierce, M.D., Ph.D., who is also a corresponding author. "While more research is needed to determine who may benefit most, we consider the early results promising. To hear from several participants how thrilled they were that they could finally see the food on their plates -- that is a big deal. These were individuals who could not read any lines on an eye chart and who had no treatment options, which is the unfortunate reality for most people with inherited retinal disorders." "Our patients are the first congenitally blind children to be treated with gene editing, which significantly improved their daytime vision," said the paper's third corresponding author, Tomas S. Aleman, M.D., a pediatric ophthalmologist at the Children's Hospital of Philadelphia and the University of Pennsylvania's Scheie Eye Institute. "Our hope is that the study will pave the road for treatments of younger children with similar conditions and further improvements in vision. This trial represents a landmark in the treatment of genetic disease, in specific genetic blindness, by offering important alternative treatment when traditional forms of therapy, such as gene augmentation, are not an option." "The results from the BRILLIANCE trial provide proof of concept and important learnings for the development of new and innovative medicines for inherited retinal diseases. We've demonstrated that we can safely deliver a CRISPR-based gene editing therapeutic to the retina and have clinically meaningful outcomes," said Editas Medicine Chief Medical Officer Baisong Mei, M.D., Ph.D. The OHSU Casey Eye Institute is one of five clinical sites that recruited participants for the trial. The other sites are: Bascom Palmer Eye Institute in Miami, Florida; Mass Eye and Ear in Boston, Massachusetts; Scheie Eye Institute at the University of Pennsylvania and Children's Hospital of Philadelphia; and Kellogg Eye Center in Ann Arbor, Michigan.

Gene TherapyClinical StudyClinical Result

28 Apr 2023

·www.pharmaceutical-technology.com

Editas grabs orphan drug status for sickle cell disease CRISPR therapy

Adam Zamecnik @a_zamecnik Image Credit: Shutterstock / Ezume Images The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022. The company is studying EDT-301 in sickle cell disease in a Phase I/II RUBY study (NCT04853576), and is on track to provide a clinical update by mid-2023. Editas had previously announced positive safety and efficacy data with two patients enrolled in the RUBY study, stating that the therapy was well-tolerated and that both patients were successfully engrafted and free of vaso-occlusive events during the follow-up. The first patient had a total haemoglobin level of 16.4 g/dL and 45.4% fetal haemoglobin after five months, per a December 2022 update. Editas has plans to dose 20 patients on this study by the end of this year. EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. This increases the expression of fetal haemoglobin. Editas’ CRISPR therapies represent a different approach to the gene editing technology than the one used by CRISPR Therapeutics , which relies on the CRISPR/Cas9 platform . Earlier in the year, the company had announced a move towards developing treatments for hemoglobinopathies like sickle cell disease and beta thalassemia and a focus on in vivo discovery. In a January 9 press release, Editas stated it will be laying off roughly 20% of its workforce , alongside the discontinuation of its investments in its therapies for inherited retinal diseases. Specifically, this meant that the company stopped investments into EDIT-101 and its use in Leber Congenital Amaurosis and EDIT-103 for rhodopsin-associated autosomal retinitis pigmentosa. Editas also discontinued investments in its preclinical natural killer (NK) cell program, which included candidates like EDIT-202 for use in solid tumours. On January 19, Editas announced that it entered into a definitive agreement with Shoreline Biosciences for the licensing of its natural killer cell program. Shoreline acquired EDIT-202 as part of this agreement. 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 Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. By Cytiva Thematic By downloading this case study, 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

Gene TherapyLicense out/inOrphan DrugCell TherapyClinical Study

100 Deals associated with Brinretigene vesgedparvovec

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Eye Abnormalities	Phase 2	US	Editas Medicine, Inc.	26 Sep 2019
Hereditary retinal dystrophy	Phase 2	US	Editas Medicine, Inc.	26 Sep 2019
Leber Congenital Amaurosis 10	Phase 2	US	Editas Medicine, Inc.	26 Sep 2019
Retinal Degeneration	Phase 2	US	Editas Medicine, Inc.	26 Sep 2019
Vision Disorders	Phase 2	US	Editas Medicine, Inc.	26 Sep 2019
Leber Congenital Amaurosis	Phase 2	-	Allergan Ltd. (Ireland)	-

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03872479 (BRILLIANCE, Literature) Manual	Phase 1/2	Retinal Degeneration	14	EDIT-101	eztkacikdz(uwueuottmu) = none bzsdhsmffd (zwstnqrpyg ) View more	Positive	06 May 2024
NCT03872479 (Corporate Publications) Manual	Phase 1/2	Leber Congenital Amaurosis 10	14	EDIT 101	yaovovdxwm(xfiqsubvoh) = No ocular serious adverse events were observed tutcmuqqfm (vvhhhtaagr ) View more	Positive	17 Nov 2022

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