A Phase 3 clinical trial has been completed and demonstrated the safety and efficacy of allogeneic islet transplantation in improving glycemic control in Type 1 diabetic patients using the UIC protocol.The objective in offering expanded access to donislecel (allogeneic islets of Langerhans for transplant; IND BB-11807) for the treatment of brittle T1D is to bridge the gap between completed clinical trials and marketing (i.e. approval by the FDA of a biological license application). Expanded access will allow clinical trial subjects, as well as patients outside a clinical trial, to receive treatment. New patients participating in the expanded access protocol are required to meet exclusion and inclusion criteria.

Start Date-

Sponsor / Collaborator

Celltrans, Inc.

NCT00679042 / Active, not recruitingPhase 3

Islet Transplantation in Type 1 Diabetic Patients Using the UIC Protocol, Phase 3

In an earlier Phase 1/2 clinical trial using the Edmonton Protocol of steroid free immunosuppression, investigators at University of Illinois at Chicago (UIC) demonstrated the safety of islet preparation, iset transplantation, and medical treatment at UIC. Therefore, the primary purpose of the present Phase 3 clinical trial is to demonstrate the safety and efficacy of allogeneic islet transplantation in improving glycemic control in Type 1 diabetic patients using the UIC protocol that was developed and proven effective during the Phase 1/2 clinical trial.

Start Date05 Sep 2007

Sponsor / Collaborator

Celltrans, Inc.

100 Clinical Results associated with Donislecel (CellTrans)

100 Translational Medicine associated with Donislecel (CellTrans)

100 Patents (Medical) associated with Donislecel (CellTrans)

Literatures (Medical) associated with Donislecel (CellTrans)

01 Feb 2024·Irish Journal of Medical Science (1971 -)

Donislecel-the first approved pancreatic islet cell therapy medication for type 1 diabetes: a letter to the editor

Letter

Author: Affan, Muhammad ; Dar, Marrium S

Type 1 diabetes is an autoimmune disorder which is caused due to T-cell mediated destruction of β cells of pancreas leading to insulin deficiency and hyperglycemia. The prevalence has been increasing in many countries especially Europe and America. The exogenous therapy which includes insulin administration is not sufficient in maintaining the glucose levels especially in patients with brittle T1D. Therefore, endogenous production of insulin via islet cell therapy has been a center of research for many years and results have largely supported the idea. The US. FDA approved the first cell therapy drug, Donislecel for brittle T1D which may be a crucial achievement in treatment of the autoimmune disorder.

12 Oct 2023·Cureus

Type 1 Diabetes Mellitus Management and Islet Cell Therapy: A New Chapter in Patient Care

Author: Murtaza, Syeda Fatima ; Zaidi, Syed Asjad Tauheed ; Alam, Sadaf ; Lee, Calvin Yee Fen ; Khan, Salman J

Diabetes mellitus (DM) is a complex endocrine disorder characterized by abnormally high levels of glucose, also called hyperglycemia. DM usually occurs when the body does not produce enough insulin or cannot respond to the insulin in the body. Type 1 diabetes mellitus (T1DM) or insulin-dependent diabetes is an autoimmune disease that affects around 8 million people in the world. Patients with T1DM experience an array of symptoms such as polyuria, polydipsia, and weight loss. These patients are prone to immediate life-threatening complications, including hypoglycemia and diabetic ketoacidosis. These patients are also at increased risk of ischemic heart disease, stroke, chronic kidney disease, vision loss, and even damage to nerve endings resulting in neuropathy. In this article, we will discuss type 1 diabetes mellitus and the different treatment options, focusing primarily on the Food and Drug Administration (FDA)-approved first cellular therapy for T1DM, donislecel.

The Medical Letter on Drugs and Therapeutics

Donislecel (Lantidra) for Type 1 Diabetes

Article

The FDA has approved donislecel-jujn (Lantidra – CellTrans), an allogeneic pancreatic islet cellular therapy, for use in conjunction with immunosuppression for treatment of adults with type 1 diabetes who are unable to approach target HbA1c because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education. Donislecel is the first cell-based treatment to be approved in the US for type 1 diabetes.

News (Medical) associated with Donislecel (CellTrans)

09 Sep 2024

·www.pharmaceutical-technology.com

EASD24: PolTREG’s T-cell therapy shows long-term remission in type 1 diabetes

PolTREG plans to start a pivotal Phase II/III trial of PTG-007 for the treatment of type 1 diabetes and is pursuing partnership funding. Image Credit: Anatoliy Cherkas / Shutterstock. PolTREG’s has presented positive long-term data for its cell therapy, PTG-007, showing that the therapy induced clinical remission for type 1 diabetes for up to 12 years. The follow-up data from 54 patients with early-onset type 1 diabetes who took part in Phase I and II trials was presented at the European Association for the Study of Diabetes (EASD) taking place from 9-13 September in Spain. In light of the positive data, the Polish company will start a pivotal Phase II/III trial of PTG-007 for the treatment of type 1 diabetes and is pursuing partnership funding. “Today’s results are extremely encouraging and show that some patients remain in clinical remission for up to 12 years after initial treatment,” said Professor Piotr Trzonkowski, PolTREG’s CEO. “This makes us very eager to launch a pivotal study of PTG-007 as soon as we find a suitable partner.” The 54 patients with early-onset type 1 diabetes were followed for 7-12 years after receiving initial treatment as part of Phase I and Phase II trials. The study showed that a “proportion of patients” remained insulin-independent up to 18 to 24 months after treatment. See Also: LACTIN-V by Osel for Human Papillomavirus Infections: Likelihood of Approval Cadisegliatin by VTv Therapeutics for Type 1 Diabetes (Juvenile Diabetes): Likelihood of Approval Additionally, some of the patients remained in clinical remission for 7-12 years after being treated with PTG-007. The trial defined clinical remission as having a low need for external insulin, while retaining proper metabolic/glycaemia control. The study also found that the “best results” were seen in patients who received the combination therapy of Treg therapy and Roche and Biogen ’s anti-CD20 treatment Rituxan (rituximab). The company plans to release detailed data and publish the results in a peer-reviewed scientific publication. PolTREG plans to initiate a Phase II trial evaluating PTG-007 in patients with presymptomatic type 1 diabetes. Cell therapies as a treatment for type 1 diabetes are gaining popularity. Last year saw the US Food and Drug Administration (FDA) approving the first cell therapy, CellTrans’s Lantidra (donislecel) for the treatment of type 1 diabetes. 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

Cell TherapyClinical ResultPhase 1Phase 2Gene Therapy

09 Sep 2024

·www.pharmaceutical-technology.com

EASD 2024: PolTREG’s T-cell therapy shows long-term remission in type 1 diabetes

PolTREG plans to start a pivotal Phase II/III trial of PTG-007 for the treatment of type 1 diabetes and is pursuing partnership funding. Image Credit: Anatoliy Cherkas / Shutterstock. PolTREG’s has presented positive long-term data for its cell therapy, PTG-007, showing that the therapy induced clinical remission for type 1 diabetes for up to 12 years. The follow-up data from 54 patients with early-onset type 1 diabetes who took part in Phase I and II trials was presented at the European Association for the Study of Diabetes (EASD) taking place from 9-13 September in Spain. In light of the positive data, the Polish company will start a pivotal Phase II/III trial of PTG-007 for the treatment of type 1 diabetes and is pursuing partnership funding. “Today’s results are extremely encouraging and show that some patients remain in clinical remission for up to 12 years after initial treatment,” said Professor Piotr Trzonkowski, PolTREG’s CEO. “This makes us very eager to launch a pivotal study of PTG-007 as soon as we find a suitable partner.” The 54 patients with early-onset type 1 diabetes were followed for 7-12 years after receiving initial treatment as part of Phase I and Phase II trials. The study showed that a “proportion of patients” remained insulin-independent up to 18 to 24 months after treatment. See Also: Bavarian Nordic promises two million mpox vaccines for Africa by end of 2024 IBA venture PanTera gains €134m in funding to scale up actinium-225 production Additionally, some of the patients remained in clinical remission for 7-12 years after being treated with PTG-007. The trial defined clinical remission as having a low need for external insulin, while retaining proper metabolic/glycaemia control. The study also found that the “best results” were seen in patients who received the combination therapy of Treg therapy and Roche and Biogen ’s anti-CD20 treatment Rituxan (rituximab). The company plans to release detailed data and publish the results in a peer-reviewed scientific publication. PolTREG plans to initiate a Phase II trial evaluating PTG-007 in patients with presymptomatic type 1 diabetes. Cell therapies as a treatment for type 1 diabetes are gaining popularity. Last year saw the US Food and Drug Administration (FDA) approving the first cell therapy, CellTrans’s Lantidra (donislecel) for the treatment of type 1 diabetes. 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

Clinical ResultCell TherapyPhase 1Phase 2Gene Therapy

24 Apr 2024

·www.fiercepharma.com

Vertex jumps to TreeFrog to enhance production of Type 1 diabetes cell therapy candidates

Vertex has acquired access to an advanced cell therapy manufacturing platform developed by TreeFrog which will allow the Boston company to boost production of its three pipeline candidates to treat Type 1 diabetes. Following success in cystic fibrosis and sickle cell disease, Vertex’s next big leap is Type 1 diabetes (T1D). Helping the Boston company develop three pipeline candidates in the indication will be French cell therapy developer and manufacturer TreeFrog Therapeutics. The companies announced their partnership on Tuesday, with Vertex licensing exclusive use of TreeFrog’s cell manufacturing platform, C-Stem, which will optimize production by allowing cells to grow “exponentially in 3D,” Vertex said. “The technology will enhance Vertex’s ability to generate large amounts of fully differentiated cells for its portfolio of cell therapies,” the companies wrote. Terms of the milestone-heavy partnership include a $25 million payment upfront, with $215 million available related to the development of a process to produce differentiated islet cells. TreeFrog can also gain sales royalties and another $540 million when clinical, regulatory and commercial milestones are met on up to two future products. Vertex will apply TreeFrog’s technology to its primary T1D candidate VX-880, which successfully completed a phase 1/2 trial last year. Both VX-880 and VX-264 are stem-cell-derived, fully differentiated insulin-producing islet cell therapies. “Our expectation is that C-Stem will enhance our ability to generate large amounts of fully differentiated cells for our portfolio of T1D cell therapies,” a Vertex spokesperson wrote in an email. “We will be implementing TreeFrog’s technology into our current processes to potentially enhance stem cell production and scale-up.” In addition to developing a pipeline of cell therapy products—including a lead candidate for Parkinson’s disease—six-year-old TreeFrog licenses out its C-Stem technology. Three years ago, the company touted C-Stem as a “leap forward in cell manufacturing,” allowing companies to mass-produce off-the-shelf cell therapies with standardized quality. “This is a fantastic opportunity to apply and further develop our C-Stem technology,” Frédéric Desdouits, Ph.D., CEO of TreeFrog, said in a release. “This collaboration with Vertex advances our business strategy of helping bring best-in-class cell therapies to millions of people—both through our own therapeutic programs and with best-in-class partners like Vertex.” Last year, Vertex struck a manufacturing deal with Lonza in which the companies are co-investing in a new facility in Portsmouth, New Hampshire, which will employ up to 300 people. The plant will support the scale-up and manufacture of Vertex’s two clinical T1D programs. “Our scale up at Lonza is ongoing and we will determine the right time to incorporate TreeFrog’s technology into that process as the collaboration progresses,” the Vertex spokesperson said. Last year, Chicago startup CellTrans scored the first approval for a T1D cell therapy when the FDA signed off on Lantidra (donislecel). Stem cell treatments—like those under development by Vertex—are believed to be more reliable and healthier than those from deceased donors, as is the case with Lantidra.

Cell TherapyDrug ApprovalLicense out/inClinical Study

100 Deals associated with Donislecel (CellTrans)

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Diabetes Mellitus, Type 1	US	Celltrans, Inc.	28 Jun 2023

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


FDA Manual	Not Applicable	Diabetes Mellitus, Type 1	30	LANTIDRA	(pqqqvjcqfu) = edaylymhrn uocsfpyfnb (kdjjpfjtxr ) View more	Positive	28 Jun 2023
FDA Manual	Not Applicable	Diabetes Mellitus, Type 1	30	LANTIDRA (Study 1)	yzvjrhmwdf(zzvyxtkkdq) = situsgupka uahnigeycq (npayfqpaar, 4.2)	Positive	28 Jun 2023
NCT00679042 (CTgov)	Phase 3	Diabetes Mellitus, Type 1	21	Islets of Langerhan+Simulect+Rapamune+Prograf+Byetta+Enbrel	atsmvstpmn(uymapjvlfd) = svtndoaesw sezpjxhccc (wdowsrqovu, daeldyxmaa - eyfufiiaup) View more	-	06 Apr 2021

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