Delving into the Latest Updates on Betibeglogene autotemcel with Synapse

Overview

Basic Info

Drug Type

Gene therapy, Hematopoietic stem cell therapy

Synonyms

2009 EGT-produced vector, Autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiglobin BB305 lentiviral vector encoding the beta-A-T87Q-globin gene, Autologous CD34+ cells encoding βA-T87Q-globin gene

+ [13]

Target

β-globin

Mechanism

β-globin stimulants(Hemoglobin beta chain stimulants), Gene transference

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication

ThalassemiaBeta-ThalassemiaAnemia, Sickle Cell+ [1]

Inactive Indication

Transfusion-dependent Thalassemia

Originator Organization

bluebird bio, Inc.

Active Organization

bluebird bio, Inc.

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

EU (29 May 2019),

Transfusion-dependent Beta Thalassemia

RegulationPriority Review (US), Breakthrough Therapy (US), Fast Track (US), Orphan Drug (US), Rare Pediatric Disease (US), Orphan Drug (EU), PRIME (EU), Regenerative Medicine Advanced Therapy (US), Accelerated assessment (EU)

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This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

Start Date08 Jun 2017

Sponsor / Collaborator

bluebird bio, Inc.

100 Clinical Results associated with Betibeglogene autotemcel

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100 Translational Medicine associated with Betibeglogene autotemcel

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100 Patents (Medical) associated with Betibeglogene autotemcel

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42

Literatures (Medical) associated with Betibeglogene autotemcel

01 Dec 2023·Molecular therapy. Methods & clinical development

Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia

Article

Author: Kral, Kelly ; d'Anjou, Marc ; Whitney, Dustin ; Pierciey, Francis J ; Gayron, Marisa ; Colvin, Richard A ; Fincker, Maeva ; Shestopalov, Ilya

Ex vivo autologous hematopoietic stem cell lentiviral-based gene therapy with betibeglogene autotemcel has been studied in patients with transfusion-dependent β-thalassemia in Phase III clinical trials (HGB-207 and HGB-212), with 90% of patients reaching transfusion independence (TI). Here, we explore manufacturing parameters, drug product quality attributes, and limited patient characteristics that had an impact on clinical efficacy in HGB-207 and HGB-212. Retrospective analysis revealed that the peripheral blood vector copy number (VCN) was related to TI, with a strong correlation between peripheral blood VCN at 6 months and gene therapy-derived therapeutic protein (HbA^T87Q) expression at 6 months (correlation coefficient, 0.8681; p < 0.0001; R² = 0.7536). A peripheral blood VCN threshold of ≥0.75 copies per diploid genome at 6 months post betibeglogene autotemcel infusion provided a stringent surrogate biomarker for TI and was used as the outcome variable for multivariate analysis using a random forest classifier. The top predictive feature of clinical efficacy was found to be the percentage of lentiviral vector-positive cells in the drug product. This retrospective analysis is critical to understanding the key product quality attributes that can predict clinical efficacy in lentiviral vector gene therapy within this clinical trial population.

01 Oct 2023·JPMA. The Journal of the Pakistan Medical Association

Betibeglogene Autotemcel; A New Hope For Transfusion Dependent Beta-Thalassaemia.

Article

Author: Jilani, Elahi Sana ; Zulfi, Muhammad Hassan ; Wally, Hiba Tariq

01 Aug 2023·International journal of experimental pathology

Clinical applications of gene therapy for rare diseases: A review.

Review

Author: Owen, James S. ; Papaioannou, Ioannis ; Yanez-Munoz, Rafael J.

Rare diseases collectively exact a high toll on society due to their sheer number and overall prevalence. Their heterogeneity, diversity, and nature pose daunting clinical challenges for both management and treatment. In this review, we discuss recent advances in clinical applications of gene therapy for rare diseases, focusing on a variety of viral and non-viral strategies. The use of adeno-associated virus (AAV) vectors is discussed in the context of Luxturna, licenced for the treatment of RPE65 deficiency in the retinal epithelium. Imlygic, a herpes virus vector licenced for the treatment of refractory metastatic melanoma, will be an example of oncolytic vectors developed against rare cancers. Yescarta and Kymriah will showcase the use of retrovirus and lentivirus vectors in the autologous ex vivo production of chimeric antigen receptor T cells (CAR-T), licenced for the treatment of refractory leukaemias and lymphomas. Similar retroviral and lentiviral technology can be applied to autologous haematopoietic stem cells, exemplified by Strimvelis and Zynteglo, licenced treatments for adenosine deaminase-severe combined immunodeficiency (ADA-SCID) and β-thalassaemia respectively. Antisense oligonucleotide technologies will be highlighted through Onpattro and Tegsedi, RNA interference drugs licenced for familial transthyretin (TTR) amyloidosis, and Spinraza, a splice-switching treatment for spinal muscular atrophy (SMA). An initial comparison of the effectiveness of AAV and oligonucleotide therapies in SMA is possible with Zolgensma, an AAV serotype 9 vector, and Spinraza. Through these examples of marketed gene therapies and gene cell therapies, we will discuss the expanding applications of such novel technologies to previously intractable rare diseases.

279

News (Medical) associated with Betibeglogene autotemcel

23 May 2024

·www.prnewswire.com

Levi & Korsinsky Reminds Shareholders of a Lead Plaintiff Deadline of May 28, 2024 in bluebird bio Lawsuit - BLUE

NEW YORK, May 23, 2024 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in bluebird bio, Inc. ("bluebird bio" or the "Company") (NASDAQ: BLUE) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of bluebird bio investors who were adversely affected by alleged securities fraud between April 24, 2023 and December 8, 2023. Follow the link below to get more information and be contacted by a member of our team: BLUE investors may also contact Joseph E. Levi, Esq. via email at [email protected] or by telephone at (212) 363-7500. CASE DETAILS: According to the complaint, on December 8, 2023, Blue announced that the Food and Drug Administration (FDA) approved its drug Lyfgenia (lovotibeglogene autotemcel), also known as lovo-cel for the treatment of sickle cell disease in patients ages 12 and older who have a history of vaso-occlusive events (VOEs). However, the Lyfgenia approval came with a black box warning for haematological malignancies after two patients developed AML during the clinical trials. Analysts noted that they did not expect the black box warning or the absence of a priority review voucher. Following this news, Blue's stock price fell by $1.95 per share, or approximately 40% to close at $2.86 per share. WHAT'S NEXT? If you suffered a loss in bluebird bio during the relevant time frame, you have until May 28, 2024 to request that the Court appoint you as lead plaintiff. Your ability to share in any recovery doesn't require that you serve as a lead plaintiff. NO COST TO YOU: If you are a class member, you may be entitled to compensation without payment of any out-of-pocket costs or fees. There is no cost or obligation to participate. WHY LEVI & KORSINSKY: Over the past 20 years, the team at Levi & Korsinsky has secured hundreds of millions of dollars for aggrieved shareholders and built a track record of winning high-stakes cases. Our firm has extensive expertise representing investors in complex securities litigation and a team of over 70 employees to serve our clients. For seven years in a row, Levi & Korsinsky has ranked in ISS Securities Class Action Services' Top 50 Report as one of the top securities litigation firms in the United States. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 17th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, LLP

Drug ApprovalPatent Infringement

10 May 2024

·www.biopharmadive.com

iTeos shares jump on TIGIT update; Acelyrin swaps CEOs

BioPharma Dive is testing out a new format rounding up smaller updates from around the industry. Have thoughts on what could make this type of story better? Drop us a line!Today, a brief rundown of news from iTeos Therapeutics, as well as notes on Gilead, Acelyrin and Bluebird bio that you may have missed yesterday:Shares in iTeos Therapeutics jumped by nearly 40% Friday morning after the biotechnology company gave an update on a clinical trial of a cancer drug combination its studying with GSK. Interim data surpassed iTeos pre-defined efficacy criteria for clinically relevant activity, the company said. The study is testing iTeos anti-TIGIT antibody together with GSKs PD-1 inhibitor Jemperli in first-line treatment of non-small cell lung cancer.Ned PagliaruloBluebird bio made national headlines in The New York Times this week with news it had collected the cells of the first patient set to undergo treatment with its sickle cell disease therapy Lyfgenia. In an earnings update Thursday, the company disclosed that, so far this year, 14 patients have started the treatment process for its other two marketed gene therapies 11 for Zynteglo, which treats severe beta thalassemia, and 3 for Skysona, approved for a rare brain disorder. Bluebird expects between 85 and 105 patient starts for its three therapies this year. Ned PagliaruloImmune drug developer Acelyrin said Thursday that founder Shao-Lee Lin has stepped down as CEO and will be replaced by current chief legal and administrative officer Mina Kim. The biotech raised $540 million in an initial public offering last year, but has lost much of its value after hitting a clinical trial setback in September. Ben FidlerGilead Sciences and partner Arcellx on Thursday afternoon disclosed the design of a Phase 3 trial they plan to run testing a CAR-T therapy in people with relapsed or refractory multiple myeloma. The therapy, dubbed anito-cel, targets a protein called BCMA, similar to the approved cell therapies Carvykti, from Johnson & Johnson, and Abecma, from Bristol Myers Squibb.Ned Pagliarulo '

Phase 3Cell TherapyExecutive ChangeImmunotherapyGene Therapy

09 May 2024

·endpts.com

Bluebird bio starts 15 patients on its three multimillion-dollar gene therapies

Bluebird bio’s stock $BLUE jumped over 20% on Thursday following its first-quarter earnings release and the announcement of 15 patients starting on its three approved gene therapies: Zynteglo, Skysona and Lyfgenia. The company wrote in its financial report that 15 patients had completed starts on its expensive gene therapies: 11 on Zynteglo for transfusion-dependent thalassemia, three on Skysona for cerebral adrenoleukodystrophy and one on Lyfgenia for sickle cell. Bluebird also expects 85 to 105 more patient starts across its portfolio this year. You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

Gene Therapy

100 Deals associated with Betibeglogene autotemcel

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External Link

KEGG	Wiki	ATC	Drug Bank
-	-	B06AX	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Thalassemia	US	bluebird bio, Inc.	17 Aug 2022
Transfusion-dependent Beta Thalassemia	EU	bluebird bio, Inc.	29 May 2019
Transfusion-dependent Beta Thalassemia	IS	bluebird bio, Inc.	29 May 2019
Transfusion-dependent Beta Thalassemia	LI	bluebird bio, Inc.	29 May 2019
Transfusion-dependent Beta Thalassemia	NO	bluebird bio, Inc.	29 May 2019
Beta-Thalassemia	GB	bluebird bio, Inc.	-

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Anemia, Sickle Cell	NDA/BLA	US	bluebird bio, Inc.	25 Apr 2023
Transfusion-dependent Thalassemia	Phase 3	US	bluebird bio, Inc.	08 Aug 2016
Transfusion-dependent Thalassemia	Phase 3	FR	bluebird bio, Inc.	08 Aug 2016
Transfusion-dependent Thalassemia	Phase 3	DE	bluebird bio, Inc.	08 Aug 2016
Transfusion-dependent Thalassemia	Phase 3	IT	bluebird bio, Inc.	08 Aug 2016
Transfusion-dependent Thalassemia	Phase 3	TH	bluebird bio, Inc.	08 Aug 2016
Transfusion-dependent Thalassemia	Phase 3	GB	bluebird bio, Inc.	08 Aug 2016

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04628585 (LTF-307, ASH2023) Manual	Not Applicable	Anemia, Sickle Cell	47	Lovotibeglogene Autotemcel	ewsblfvykd(zsihbsllix) = sokgfkzdvi mmairoehiz (smvffsntdv ) View more	Positive	11 Dec 2023
NCT02633943 (LTF-303, ASH2023) Manual	Not Applicable	Transfusion-dependent Beta Thalassemia	51	Betibeglogene Autotemcel	wmikbqwual(ljfaqdxvyx) = mjudgmnwmt oaolkjqvij (nhmccgaphv, -22.3 to 10.5) View more	Positive	10 Dec 2023
HGB-210 (BusinessWire) Manual	Not Applicable	Anemia, Sickle Cell	47	LYFGENIA	vqmmlxblvm(wobioiveqq) = hscrcxmbth jiudqnqeaa (poxiihzlxc ) View more	Positive	09 Dec 2023
FDA Manual	Phase 1/2	Anemia, Sickle Cell	45	LYFGENIA	dkdzvzfdvy(bwtcuiklid) = qlrhfpnydk ttmcbmpoci (yjsdlyrzxr, 71 - 97) View more	Positive	08 Dec 2023
NCT02140554 (HGB-206, Pubmed) Manual	Phase 1/2	Anemia, Sickle Cell	9	Lovo-cel (Group A)	qyhdveferg(cnfpgufbkf) = zbskrihdbx hnxkybaybh (xpdwjgqmrb ) View more	Positive	25 Sep 2022
NCT02140554 (HGB-206, Pubmed) Manual	Phase 1/2	Anemia, Sickle Cell	9	Lovo-cel (Group B)	qyhdveferg(cnfpgufbkf) = iwfkuocbzt hnxkybaybh (xpdwjgqmrb ) View more	Positive	25 Sep 2022
NCT02140554 (HGB-206, Pubmed \| Br Dent J) Manual	Phase 1/2	Anemia, Sickle Cell	35	LentiGlobin	qphbsnnxgm(xvrhibhzxx) = zubruerfst ktqhglcjbf (ftfciejqjf ) View more	Positive	12 Dec 2021
NCT02906202 (Pubmed \| Br Dent J) Manual	Phase 3	Thalassemia	23	Betibeglogene autotemcel	ddswvnnujd(tvmkidnxyp) = qjedjbstls xyypqvchnb (igbezvyynl ) View more	Positive	11 Dec 2021
NCT02633943 (LTF-303, EHA2021) Manual	Not Applicable	Transfusion-dependent Thalassemia	44	BETIBEGLOGENE AUTOTEMCEL	vljybedaqg(kxwzvznjoj) = gonadotropic insufficiency, ec ~ pic pregnancy, fetal death, gallbladder wall thickening/polyp, bacteremia with neutropenia, and major depression (all n=1) zphtnbgffx (hqkqevugdc ) View more	Positive	09 Jun 2021
HGB-207, HGB-212 (EHA2021)	Phase 3	Transfusion-dependent Beta Thalassemia	27	betibeglogene autotemcel gene therapy	uztxbsgvqs(ytyovfemjp) = thrombocytopenia, tachycardia (2 patients < 12 years of age), abdominal pain (2 patients 12-18 years of age) hlhfeqwntw (mfooawaznl ) View more	-	09 Jun 2021
HGB-207 (Northstar-2), HGB-212 (Northstar-3) (EHA2021)	Phase 3	Transfusion-dependent Beta Thalassemia	41	BETIBEGLOGENE AUTOTEMCEL (Beti-cel)	cxygbugbgh(najicvyxct) = rqdfmugnzu anfkmtelaf (ysyljoqwvy ) View more	-	09 Jun 2021