An Open-Label Phase 1/2 Study to Evaluate the Safety, Engraftment, and Efficacy of VC-01™Combination Product in Subjects With Type 1 Diabetes Mellitus [T1DM]

VC01-103 will evaluate an experimental combination product, cell replacement therapy intended to provide a functional cure to subjects with Type 1 Diabetes.

Start Date25 Jun 2019

Sponsor / Collaborator

ViaCyte, Inc.

NCT02939118 / CompletedNot Applicable

A One-Year, Multicenter, Observational, Follow-up Safety Study in Subjects Previously Implanted With VC-01™ Combination Product

The purpose of this trial is to evaluate the long-term safety in subjects previously implanted with VC-01™ combination product.

Start Date07 Nov 2016

Sponsor / Collaborator

ViaCyte, Inc.

NCT02239354 / TerminatedPhase 1/2

A Prospective, Multicenter, Open-Label, First-in-Human Phase 1/2 Study With Two Cohorts to Evaluate the Safety, Tolerability, and Efficacy of Various Doses of VC-01™ Combination Product in Subjects With Type 1 Diabetes Mellitus

The purpose of this trial is to test if VC-01™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and maintained safely for two years. It will also test if VC-01 is an effective treatment for subjects with Type 1 Diabetes.

Start Date01 Sep 2014

Sponsor / Collaborator

ViaCyte, Inc.

[+1]

100 Clinical Results associated with PEC Encap

100 Translational Medicine associated with PEC Encap

100 Patents (Medical) associated with PEC Encap

Literatures (Medical) associated with PEC Encap

01 Jan 2023·Journal of environmental science and health. Part. B, Pesticides, food contaminants, and agricultural wastes

Adsorption-desorption characteristics of atrazine on soil and vermicompost prepared with different ratios of raw materials.

Article

Author: Zhang, Lei ; Zhang, Yan ; Xu, Lixin ; Zhang, Luwen ; Chen, Yuxiang

In this work, vermicompost was prepared with maize stover and cattle dung in ratios of 60:40 (VC₁), 50:50 (VC₂) and 40:60 (VC₃), and the physicochemical properties of the vermicompost were related to the ratio of the raw materials used. The effect of the vermicomposts on the adsorption kinetics, adsorption isotherms and desorption of atrazine were investigated in unamended soil (S) and soil amended with 4% (w/w) of VC₁(S-VC₁), VC₂(S-VC₂) and VC₃(S-VC₃). The total organic carbon (TOC) content of VC₁, VC₂ and VC₃ was 38.46, 37.33 and 34.47%, the HA content was 43.50, 42.22 and 39.28 g/kg, and the HA/FA ratios was 1.47, 0.44 and 0.83, respectively. The adsorption of atrazine on the soil, on the vermicompost and on soils amended with vermicompost followed a pseudo-second-order kinetic model. The Freundlich equation better fitted the adsorption isotherm of atrazine. The vermicomposts enhanced atrazine adsorption and decreased atrazine desorption. Correlation analysis showed that the TOC and HA were significantly positively correlated with K_f, which indicated that TOC and HA of the vermicomposts contributed significantly to the adsorption and desorption of atrazine. This study demonstrated that vermicomposts have great potential in the bioremediation of atrazine pollution and that their role is related to the raw materials used to prepare them.

01 Oct 2018·Letters in Applied MicrobiologyQ4 · BIOLOGY

Comparative genomics ofVibrio choleraeO1 isolated from cholera patients in Bangladesh

Q4 · BIOLOGY

Article

Author: Sultana, R. ; Begum, A. ; Hendriksen, R.S. ; Leekitcharoenphon, P. ; Dalsgaard, A. ; Jensen, P.K.M. ; Hossain, Z.Z.

Whole genome sequencing was utilized to investigate the genomic profile of Vibrio cholerae O1 strains, isolated from symptomatic patients in a low-income urban area of Dhaka, Bangladesh. Comparative genomics using bioinformatics tools were applied to identify major virulence factors, biotype and antimicrobial resistance genes in three V. cholerae O1 strains (VC-1, 2 and 3) isolated from two case patients. A phylogenetic SNP (single nucleotide polymorphism)-based analysis was conducted to infer the relatedness to V. cholerae O1 strains isolated elsewhere. The V. cholerae strains were the El Tor variant carrying ctxB1 (standard classical genotype). SNP-based global phylogeny revealed that the three isolates were strictly clonal and the closest neighbouring genomes were epidemic clones of V. cholerae O1 isolated in 2010 from cholera patients in Pakistan. All strains harboured the integrase gene of the SXT element (int_SXT ), antimicrobial resistance genes for aminoglycosides, phenicol, sulphonamide and trimethoprim except VC-1 that lacked sulphonamide resistance genes. The multilocus sequence typing (MLST) revealed that the strains belonged to sequence type, ST69. The study provides knowledge on current genetic traits of clinical V. cholerae O1 circulating in urban household clusters of Bangladesh which may help in predicting emergence of new pandemic strains in Bangladesh.SIGNIFICANCE AND IMPACT OF THE STUDYVibrio cholerae has frequently experienced genetic changes with rapid evolution of pandemic clones in the Ganges Delta region. Whole genome sequencing can reveal genetic information of current pathogenic V. cholerae in Bangladesh which includes cefotaxime genotypes, virulence factors, altered antimicrobial resistance pattern as well as mobile genetic element compared to global pandemic strains. This study data could be used in planning future surveillance strategies in Ganges Delta region by informing new epidemiology of current outbreak strains.

01 Oct 2015·Stem Cells Translational MedicineQ2 · MEDICINE

Insulin-Producing Endocrine Cells Differentiated In Vitro From Human Embryonic Stem Cells Function in Macroencapsulation Devices In Vivo

Q2 · MEDICINE

ArticleOA

Author: Srijemac, Robert ; Agulnick, Alan D. ; Cesario, Rosemary M. ; Wilson, Alistair Z. ; Ambruzs, Dana M. ; Bhoumik, Anindita ; Kroon, Evert J. ; Kerr, Justin ; Kelly, Jonathan R. ; D'Amour, Kevin A. ; Payne, Janice K. ; Haakmeester, Carl ; Frazier, Mauro A. ; Moorman, Mark A.

AbstractThe PEC-01 cell population, differentiated from human embryonic stem cells (hESCs), contains pancreatic progenitors (PPs) that, when loaded into macroencapsulation devices (to produce the VC-01 candidate product) and transplanted into mice, can mature into glucose-responsive insulin-secreting cells and other pancreatic endocrine cells involved in glucose metabolism. We modified the protocol for making PEC-01 cells such that 73%–80% of the cell population consisted of PDX1-positive (PDX1+) and NKX6.1+ PPs. The PPs were further differentiated to islet-like cells (ICs) that reproducibly contained 73%–89% endocrine cells, of which approximately 40%–50% expressed insulin. A large fraction of these insulin-positive cells were single hormone-positive and expressed the transcription factors PDX1 and NKX6.1. To preclude a significant contribution of progenitors to the in vivo function of ICs, we used a simple enrichment process to remove remaining PPs, yielding aggregates that contained 93%–98% endocrine cells and 1%–3% progenitors. Enriched ICs, when encapsulated and implanted into mice, functioned similarly to the VC-01 candidate product, demonstrating conclusively that in vitro-produced hESC-derived insulin-producing cells can mature and function in vivo in devices. A scaled version of our suspension culture was used, and the endocrine aggregates could be cryopreserved and retain functionality. Although ICs expressed multiple important β cell genes, the cells contained relatively low levels of several maturity-associated markers. Correlating with this, the time to function of ICs was similar to PEC-01 cells, indicating that ICs required cell-autonomous maturation after delivery in vivo, which would occur concurrently with graft integration into the host.SignificanceType 1 diabetes (T1D) affects approximately 1.25 million people in the U.S. alone and is deadly if not managed with insulin injections. This paper describes the production of insulin-producing cells in vitro and a new protocol for producing the cells, representing another potential cell source for a diabetes cell therapy. These cells can be loaded into a protective device that is implanted under the skin. The device is designed to protect the cells from immune rejection by the implant recipient. The implant can engraft and respond to glucose by secreting insulin, thus potentially replacing the β cells lost in patients with T1D.

News (Medical) associated with PEC Encap

11 Jul 2022

·medcitynews.com

Vertex builds up pipeline of type 1 diabetes cell therapies with $320M buyout

Vertex Pharmaceuticals is developing a cell therapy for type 1 diabetes that replaces the insulin-producing cells lost to the chronic condition. In paying $320 million to acquire ViaCyte, Vertex is getting its hands on the company that was the first to demonstrate this feat in clinical testing. Boston-based Vertex framed the acquisition agreement announced Monday as complementary to its own research efforts. Privately held ViaCyte brings additional tools, technologies and assets, Vertex CEO Reshma Kewalramani said in a prepared statement. In type 1 diabetes, the immune system destroys the islet cells in the pancreas that produce insulin. Patients require regular doses of insulin, either by injection or infused from an insulin pump, to maintain levels of the glucose-regulating hormone. Vertex’s type 1 diabetes cell therapy candidate, VX-880, is comprised of islet cells that are grown in a lab. These cells are infused into a patient along with immunosuppressive therapies to keep the immune system from rejecting them. During the annual meeting of the American Diabetes Association last month, Vertex presented data from two patients who were given half of the targeted dose of cells. Both patients showed changes in blood glucose. One patient achieved insulin independence 270 days after dosing while the other showed a 30% reduction in infused or injected insulin at day 150. Vertex added VX-880 to its pipeline in 2019, paying $950 million up front to acquire startup Semma Therapeutics. At the time of the deal, Semma was preclinical. The Phase 1/2 study underway has a targeted enrollment of 17 patients. It was cleared to resume last week with the resolution of a clinical hold placed on the study in May; the FDA said at the time that there was insufficient information to support dose escalation. San Diego-based ViaCyte has three programs in development for type 1 diabetes. The most advanced is VC-02, stem cell-derived islet cells that are housed in an implantable pouch. This pouch connects with a patient’s blood vessels, enabling them to interact with the transplanted cells. In June 2021, ViaCyte reported clinical data at nine months indicating that transplanted cells were producing insulin in the first patient treated with this approach. Like the Vertex therapy, VC-02 requires immunosuppressive drugs to prevent rejection. A second ViaCyte program, VC-01, could reduce this risk with a pouch designed to prevent the patient’s immune cells from directly coming in contact with the transplanted islet cells. ViaCyte presented additional data for both programs at the American Diabetes Association’s meeting in June. The results are important for showing the potential of cell therapy in type 1 diabetes, according to William Blair analyst Raju Prasad. “Together, the data demonstrated—for the first time in humans—that implanted pancreatic progenitor cells can functionally differentiate into mature, glucose-responsive, (endogenous) insulin-producing islet cells,” Prasad wrote in a note sent to investors Monday morning. Vertex’s main interest in ViaCyte might be its third program, which could eliminate the need for immunosuppressive therapy. The cells in this experimental therapy, VCTX210, are edited with CRISPR technology to evade detection by the immune system. ViaCyte is developing VCTX210 under a partnership with CRISPR Therapeutics. The Switzerland-based gene-editing company is already a partner to Vertex. The two firms are collaborating on gene-editing treatments for two blood disorders, sickle for sickle cell disease and beta thalassemia. The collaboration has also expanded to two muscular disorders, Duchenne muscular dystrophy and myotonic dystrophy type 1. Prasad sees synergies between the type 1 diabetes approaches of Vertex and ViaCyte due to the similarities in the technology, development and manufacturing of the two cell therapies. Those similarities will benefit both CRISPR Therapeutics and Vertex in the development of a CRISPR-edited therapy with a reduced risk of prompting an immune response, he said. However, the ViaCyte acquisition will still need to pass antitrust muster. The Federal Trade Commission has raised concerns about the anti-competitive effects of some pharmaceutical mergers, discussing the topic most recently during a virtual workshop last month. There are other companies developing innovative new treatments for type 1 diabetes. Beta Bionics has also reached clinical testing with a medical device designed to take on the role of a patient’s pancreas. A drug candidate from Provention Bio is currently under FDA review. The technology that might be closest to ViaCyte’s is a cell pouch in development under Canada-based Sernova. This pouch, designed to house therapeutic cells that could offer an alternative to daily dosing with drugs, is in early clinical development in type 1 diabetes.

Cell TherapyAcquisitionCollaborate

21 Jun 2022

·www.biospace.com

CRISPR Therapeutics Eyes Historic First in Gene Therapy

CRISPR Therapeutics (CRISPR) could be on the cusp of achieving a first in gene therapy, the first company to achieve regulatory approval for a CRISPR-Cas9 program. The company is anticipating the filing of a Biologics License Application for CTX001, a potential cure for transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD). On Tuesday, the Cambridge, MA-based gene editing company hosted an Innovation Day presentation that highlighted its pipeline and the promise that its multiple approaches have in mitigating or even curing different diseases. Chief Executive Officer Samarth Kulkarni said it has been less than 10 years since Jennifer Doudna and Emmanuelle Charpentier first published an article in the journal Science about gene editing technology and less than eight years since CRISPR Therapeutics was founded on that technological promise. Now, the company is on the cusp of vying for potential approval of its CRISPR therapy, he said. CTX001, also known as exa-cel, is a CRISPR-Cas9-based gene editing therapy for both TDT and SCD. Earlier this month, CRISPR and its partner Vertex Pharmaceuticals released positive data for exa-cel in TDT and SCD. In the first space, data showed that 42 of 44 patients with TDT who received exa-cel remained transfusion free for up to 37.2 months. The two patients who were not transfusion free had 75% and 89% reductions in transfusion volume, respectively, the companies said. In SCD, all 31 patients with disease characterized by recurrent vaso-occlusive crises (VOCs) were free of the issues following treatment with the gene therapy. Data showed the patients had a duration of up to 32.3 months, CRISPR and Vertex reported. The two companies expanded their partnership in this space last year. “We are extremely excited about exa-cel,” Kulkarni said. Phuong Khanh (P.K.) Morrow, CRISPR’s new chief medical officer, echoed Kulkarni, saying that she believes exa-cel will be the first CRISPR-Cas9 product approved for both TDT and sickle cell disease. Her prediction comes about a week after an advisory committee with the U.S. Food and Drug Administration recommended approval of a different gene therapy approach for these diseases that were developed by bluebird bio. In immuno-oncology, CRISPR is also blazing a trail with its approach, particularly with CTX130, a donor-derived gene-edited allogeneic CAR T therapy that targets CD70, which is expressed on various solid tumors and hematologic malignancies. The company is developing the asset for solid tumors, such as renal cell carcinoma, as well as T cell and B cell hematologic cancers. Morrow suggested the positive data the company has seen with CRX130 in both solid and hematologic tumors is “something of the Holy Grail that researchers have been searching for." In May, CRISPR presented exciting CTX130 data from two Phase I studies for relapsed or refractory renal cell carcinoma and various subtypes of lymphoma. The company reported positive early signs, including an overall response rate of 71% from patients with T-cell lymphoma. Of those, 29% experienced a complete response, CRISPR said. In renal cell carcinoma, cancer that expresses high levels of CD70, CTX130 is also showing significant promise, even leading to a complete response in one patient. Renal cell carcinoma represents a high unmet need, with less than 20% of patients surviving beyond five years. About 40% of RCC patients have shown poor response rates to current therapies. “There is a high potential opportunity with CTX130 because of the CD70 expression in RCC,” Morrow said. Beyond those two assets, CRISPR is also advancing other therapeutics. The company is also developing VCTX210, a potential treatment for type 1 diabetes that is being co-developed with ViaCyte. VCTX210 is an allogeneic, gene-edited, stem cell-derived therapy designed to generate pancreatic cells that can evade recognition by the immune system, which would otherwise destroy them. Earlier this year, the companies dosed the first patient in a Phase I study. As BioSpace previously reported, the goal is for the cell line to be differentiated into pancreatic endoderm cells, generating glucose-responsive insulin-secreting cells in the patient. It is expected that about 10 patients will be included in the study. Data is anticipated by the end of the year. While those programs for CRISPR Therapeutics stand out, Kulkarni said the company sees significant potential with its total pipeline, with a chance to delve into multiple disease indications and potentially bring new treatment options to patients. “We’re very excited about the potential,” he said.

Gene TherapyLicense out/in

09 Jun 2021

·endpts.com

ViaCyte adds to the war chest in what looks like an IPO-bound effort to bring regenerative medicine to diabetes

Stem cell therapies have long held promise if providing curative answers to chronic disease, but the field has taken decades to bear fruit despite some big hopes. A long gestating biotech out of San Diego thinks it has the secret sauce to cure type 1 diabetes, and now it’s raking in piles of cash with the potential for a major IPO in the offing. ViaCyte has closed an additional $45 million tranche for its Series D, bringing its total fundraise for the round to $115 million with the backing of a slate of crossover investors that appear to spell a looming IPO for the long-lived regenerative medicine player, the company said Wednesday. It’s a backstory with few contemporary comparators in the go-go world of biotech: ViaCyte was founded back in 1999 and has worked for more than two decades in relative obscurity to bring its stem-cell platform into type 1 diabetes. It’s been a long wait, but not an uncommon one for the slow-to-gestate stem cell field. The company’s lead candidate, dubbed PEC-Encap, delivers pancreatic islet precursor cells as a replacement therapy and is currently in a Phase II study. ViaCyte plans to use the funding to advance that program through the clinic as well as two others: PEC-Direct, a therapy for patients with hypoglycemia unawareness and/or extreme glycemic events that expects a Phase II readout in the first half of 2022, and PEC-QT, a CRISPR Therapeutics-partnered, gene-edited immune-evasive cell line. The latter drug is expected to enter human trials by the end of the year. ViaCyte is calling its slate of programs “major advances in finding a functional cure for type 1 diabetes and other chronic diseases in the future,” CEO Michael Yang said in a statement. Big-name investors, meanwhile, are buying in. The newest fundraising tranche included participation from existing investors in RA Capital Management, Bain Capital Life Sciences, TPG Capital, Sanderling Ventures and a group of “long-time insiders,” ViaCyte said. New investors jumping on board include Adage Capital Partners, Invus Group, Asymmetry Ventures, and Artis Ventures. ViaCyte’s lead drug is the product of a collaboration with material sciences company W.L. Gore, which the partners re-upped in August. The expanded deal aimed to combine PEC-Encap with a Gore-produced membrane in what they hope will eliminate the need for immunosuppressive drugs. The product is designed to be implanted underneath the skin and deliver pancreatic progenitor cells that can secrete insulin and control blood glucose levels, in essence becoming functional tissue. ViaCyte had attempted to pair its stem cell technology with a different membrane before, but ran into trouble when patients’ immune systems flared up. That led them to collaborate with Gore, which first signed on to an agreement in 2017 and then chipped in with $10 million as part of a larger ViaCyte funding round in November 2018.

Cell TherapyCollaborateIPO

100 Deals associated with PEC Encap

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Diabetes Mellitus, Type 1	Phase 2	US	ViaCyte, Inc.	01 Sep 2014
Diabetes Mellitus, Type 1	Phase 2	CA	ViaCyte, Inc.	01 Sep 2014

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04678557 (VC01-103, CTgov)	Phase 1/2	Diabetes Mellitus, Type 1	31	VC-01 Combination Product (Sentinel Units (Aka Cohort 1))	vfofmcmzgl(oatkeduhvp) = gmcjxhtxpp pmwfwnmkpn (ctdnhtigll, elclkntooq - ulapsrcfqj) View more	-	18 Oct 2023
				VC-01 Combination Product (Dose-finding Units (Aka Cohort 2))	qcdmyluwza(nmkqzkslpo) = ayhzzljhkp tnkqctjaul (dnxdpvwqha, peschsabxd - pinkgpcwlk) View more
NCT02239354 (CTgov)	Phase 1/2	Diabetes Mellitus, Type 1	19	VC-01™ Combination Product (Cohort 1)	luteldzqmu(yxothuxwef) = opkjfznkmv jsaqfyfksa (xmogdprnnr, zzmcbawyqs - kirysdupwy) View more	-	24 Mar 2022
				VC-01™ Combination Product (Cohort 2)	qwrzygkptl(qjlbqzxufw) = ypaosdmvpc bxahrtzpcq (kprwoixdgl, cpymdegrxe - lpvbvgppkb) View more

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