Beyond Type 1 Provides Grant to ViaCyte to Support Research and Development
of Cell Replacement Therapies for Type 1 Diabetes
There are now multiple immunotherapies targeting clearance of alpha - synuclein from the brain in early - stage clinical trials, and multiple trials underway or in the works on the next generation
of cell replacement therapies for dopaminergic neurons, including the TRANSEURO trial; the Summit4StemCell initiative, put together by Jeanne Loring — a researcher at The Scripps Research Institute who is exceptionally engaged with turning her research into therapies; a Japanese trial to be run by Jun Takahashi of Kyoto University in Japan (cf. here and here); and a trial centered at Memorial Sloan Kettering Cancer Center headed by cell biologist Lorenz Studer.
Our research goal is to understand the molecular and cellular events required for pancreatic β - cell regeneration, and apply this knowledge towards the development
of cell replacement therapies for diabetes.
For the first time, researchers demonstrate long term success
of a cell replacement therapy using autologous induced pluripotent stem cell derivatives
The initial phase of clinical investigation is focusing on the safety
of the cell replacement therapy combination products in patients.
Not exact matches
«This research has broad impact, because by deepening our understanding
of cell reprogramming we have the potential to improve disease modeling and the generation
of better sources
of patient - specific specialized
cells suitable for
replacement therapy,» said Plath.
Diabetes researchers are considering various
replacements for insulin injections: Transplanting new pancreatic islet
cells that make insulin, coaxing the patient's own islets to regenerate, or treating diabetics early in the disease with immune - suppressing
therapies to prevent their body from destroying the rest
of their pancreatic islets.
These
cells, in turn, instruct factor - VIII — specific immune
cells to become tolerant to the coagulation protein, resulting in suppression
of misdirected antibody responses to the
replacement therapy — all without affecting the rest
of the immune system.
HSCT is effectively used today as a form
of «
replacement»
therapy for patients with hard - to - treat blood cancers, providing healthy
cells from either the patient (autologous transplantation) or from a donor (allogeneic transplantation) to better equip patients to fight the disease on their own.
But for
cell replacement therapy to realize its potential, I anticipate that scientists must solve the developmental code, the set
of rules that govern the formation
of tissues, organs, and beings from the original single
cell — the zygote.
«For the broad application
of stem
cell - derived pituitary
cells in the future,
cell replacement therapy may need to be customized to the specific needs
of a given patient population,» Zimmer says.
An EU - funded research project («HumEn»), in which Lickert and his team are participating, shall provide further insights in the field
of beta -
cell replacement therapy.
With these findings, Helmholtz Zentrum München scientists have discovered key molecular functions
of stem
cell differentiation which could be used for beta
cell replacement therapy in diabetes.
Our long - term goal, therefore, is to explore the potential to genetically disrupt both ccr5 and cxcr4 for
cell replacement therapies in HIV infected individuals, and in the case
of cxcr4 do so in a way that specifically targets CXCR4 on T
cells and not the many other
cell types on which it is expressed.
«The loss
of insulin - producing beta
cells leads to type 1 diabetes, making it an ideal target for
cell replacement therapy,» said James Shapiro, MD, PhD, FRCSC, Director
of the Clinical Islet Transplant Program, University
of Alberta.
Regardless
of the targeted organ for
cell replacement or regeneration, the shared challenges in
cell therapies for diabetes such as transplant procedure and immune rejection will be well - served by the collective expertise within CCTD.
Cell therapy, as envisaged by the teams of I - Stem, is primarily based on the identification of experimental protocols that can specifically guide differentiation of pluripotent cells to a cell fate, which presents a interest for the replacement of the defective cell population from the patient (the striatal neurons for Huntington's disease, the cells of the retinal pigment epithelium for retinitis pigmentosa, keratinocytes for genodermatoses, et
Cell therapy, as envisaged by the teams
of I - Stem, is primarily based on the identification
of experimental protocols that can specifically guide differentiation
of pluripotent
cells to a
cell fate, which presents a interest for the replacement of the defective cell population from the patient (the striatal neurons for Huntington's disease, the cells of the retinal pigment epithelium for retinitis pigmentosa, keratinocytes for genodermatoses, et
cell fate, which presents a interest for the
replacement of the defective
cell population from the patient (the striatal neurons for Huntington's disease, the cells of the retinal pigment epithelium for retinitis pigmentosa, keratinocytes for genodermatoses, et
cell population from the patient (the striatal neurons for Huntington's disease, the
cells of the retinal pigment epithelium for retinitis pigmentosa, keratinocytes for genodermatoses, etc.).
Early clinical evidence with the PEC - Encap product supports the potential
of the
replacement cell therapy approach.
«ViaCyte was the first to differentiate human stem
cells into glucose - responsive, insulin - producing
cells, and now we are running the first and only clinical trials
of stem
cell - derived islet
replacement therapies for type 1 diabetes,» said Paul Laikind, PhD, President and CEO
of ViaCyte.
ViaCyte's PEC - EncapTM (also known as VC - 01TM) product candidate is the first pluripotent stem
cell - derived islet
cell replacement therapy for the treatment
of type 1 diabetes in clinical - stage development.
The
cells generated in the Zeng lab may not only provide a potential unlimited source for
cell replacement therapy for Parkinson's disease, but also offer an unprecedented opportunity to develop screening models for assessing small molecule drugs and to clarify the mechanisms
of disease.
SAN DIEGO and BETHESDA, MD, March 3, 2015 — ViaCyte, Inc., a privately - held regenerative medicine company with the first stem
cell - derived islet
replacement therapy for the treatment
of diabetes in clinical trials, today announced a presentation by Dr. Eugene Brandon, Director
of Strategic Relations and Project Management, at the JDRF TypeOneNation Summit.
SAN DIEGO, April 20, 2016 — ViaCyte, Inc., a privately - held regenerative medicine company with the first pluripotent stem
cell - derived islet
replacement therapy for the treatment
of diabetes in clinical - stage development, today announced four presentations at upcoming healthcare events.
The third and final day
of the 2016 Huntington's Disease Therapeutics Conference brings updates on
cell replacement therapies including stem
cells; and new ways to assess and model the progression
of HD to help understand it and run crucial clinical trials.
Comparisons
of genetically matched human pluripotent stem
cells reveals that somatic
cell nuclear transfer is the ideal means
of generating
cells for
replacement therapy
Basically what was happening when you use a hematopoietic stem
cell to correct an inherited metabolic disease is that through engraftment
of that
cell you are allowing that
cell to become the
replacement source for the missing enzyme or other factor - almost like a cellular form
of gene
therapy or, as I call it, «poor man's gene
therapy».
Scientists publishing in the journal
Cell Stem
Cell have reported the production
of functional human oocytes from discarded genetic material, a process which they suggest could one day assist in fertility treatment or mitochondrial
replacement therapy.
The title
of the paper is «The Ideal Gene Delivery Vector: Chromallocytes,
Cell Repair Nanorobots for Chromosome
Replacement Therapy» and it is currently in press at the peer - reviewed Journal
of Evolution and Technology (and is soon to be available online).
While many important developments impacted the field, two that garnered significant public, political and scientific attention in 2016 were the proliferation
of clinics using unproven stem
cell «
therapies,» and the steps forward in therapeutic modification
of human oocytes (unfertilized eggs) through a process called mitochondrial
replacement therapy (MRT).
«These events offer several venues and platforms to share the exciting progress ViaCyte is making in developing novel
cell replacement therapies for diabetes,» said Paul Laikind, PhD, President and CEO
of ViaCyte.
JDRF and ViaCyte Announce Support for Development
of PEC - Direct
Cell Replacement Therapy for Type 1 Diabetes
SAN DIEGO, September 9, 2016 — ViaCyte, Inc., a privately - held regenerative medicine company with the first pluripotent stem
cell - derived islet
replacement therapy for the treatment
of diabetes in clinical - stage development, today announced participation on a panel at the 2016 BioPharm ™ America — 9th Annual International Partnering Conference.
In addition, we use our expertise to facilitate the development
of functionally mature beta
cells from embryonic stem
cells for
cell replacement therapies of type 1 diabetes.
SAN DIEGO, March 17, 2015 — ViaCyte, Inc., a privately - held regenerative medicine company with the first stem
cell - derived islet
replacement therapy for the treatment
of diabetes in clinical trials, today announced a presentation by Dr. Paul Laikind, President and CEO, at the third annual Regen Med Investor Day co-hosted by the Alliance for Regenerative Medicine (ARM) and top financial firm Piper Jaffray.
In addition, the funding will be used to support other operations including the continued development
of the PEC - Encap ™ product candidate, ViaCyte's stem
cell - derived islet
replacement therapy that is being developed as a treatment for all diabetes patients who require insulin to control their disease.
Cell replacement therapies require clinically safe stem
cells that can be used to generate many (possibly all) types
of cells.
The first - in - human STEP ONE * trial is evaluating the safety and efficacy
of ViaCyte's PEC - Encap (a.k.a. VC - 01 ™) product candidate, a stem
cell - derived, encapsulated
cell replacement therapy.
San Diego, January 8, 2015 — ViaCyte, Inc., a privately - held regenerative medicine company with the first stem
cell - derived islet
replacement therapy for the treatment
of diabetes in clinical trials, has received a No Objection Letter from Health Canada providing clearance to proceed with sites in Canada for the Company's Phase 1/2 clinical trial
of its VC - 01TM product candidate.
ViaCyte's VC - 01 ™ product candidate, a first - in - class
cell replacement therapy for the treatment
of type 1 diabetes, is currently being evaluated in a Phase 1/2 trial called STEP ONE, or Safety, Tolerability, and Efficacy
of VC - 01 Combination Product in Type One Diabetes.
San Diego, California, October 6, 2014 — ViaCyte, Inc., a privately - held regenerative medicine company developing a stem
cell - derived islet replacement therapy for the treatment of diabetes, called VC - 01 ™, will give two presentations at the annual Stem Cell Meeting on the Mesa Conference, being held at the Estancia La Jolla Hotel & Spa and The Salk Institute for Biological Studies, as foll
cell - derived islet
replacement therapy for the treatment
of diabetes, called VC - 01 ™, will give two presentations at the annual Stem
Cell Meeting on the Mesa Conference, being held at the Estancia La Jolla Hotel & Spa and The Salk Institute for Biological Studies, as foll
Cell Meeting on the Mesa Conference, being held at the Estancia La Jolla Hotel & Spa and The Salk Institute for Biological Studies, as follows:
«We are grateful to CIRM and the citizens
of California for their continued support
of our potentially transformative stem
cell - derived,
cell replacement therapies for diabetes,» said Paul Laikind, Ph.D., President and CEO
of ViaCyte.
Ultimately, we use all this information to establish innovative culture techniques for generating unlimited neural stem
cell sources for the derivation
of specific types
of clinically relevant neuronal or glial
cells, for their use in disease modelling and drug discovery platforms, and towards refining their efficacy for future
cell replacement - based
therapy.
With the STEP ONE trial underway, and the addition
of W. L. Gore & Associates as collaborators to optimize the Encaptra ®
cell delivery system, ViaCyte felt that the PEC - Direct approach might be the most rapid way to get the invaluable PEC - 01
cell replacement therapy to those who need it most.
The new iPS
cells generated using novel methods may represent biomedically - and clinically - ideal
cells, providing potential platforms for studying human disease mechanisms and achieving the long - term goal
of personalized
cell -
replacement therapy.
SAN DIEGO, March 1, 2016 — ViaCyte, Inc., a privately - held regenerative medicine company with the first pluripotent stem
cell - derived islet
replacement therapy for the treatment
of diabetes in clinical - stage development, today announced presentations at the JDRF TypeOneNation Summit meetings being held in Washington DC and Los Angeles.
Partial clinical hold on high dose SGT - 001 resolved, additional information requested on full clinical hold NIH grant will further development
of replacement beta
cells for type 1 and type 2 diabetes New trial will assess MSCs to improve heart function in heart failure patients ReNeuron wins grant for retinal
cell therapy development
Information on the clinical testing
of ViaCyte's implantable
cell replacement therapies can be found at Clinicaltrials.gov.
«JDRF remains dedicated to accelerating the delivery
of beta
cell replacement therapies to the T1D community, and we commend ViaCyte in its announcement
of the first patients to be implanted with the PEC - Direct islet
cell replacement therapy,» said Derek Rapp, JDRF President and Chief Executive Officer.
The WFIRM research team is working to engineer
replacement tissues and organs and develop healing
cell therapies for more than 30 different areas
of the body.
A number
of treatments exist, including enzyme
replacement therapy and hematopoietic stem
cell transplantation, but efficacy depends upon diagnosing the disease and its specific form as early as possible.