Memorandum
- The table below includes an overview of the competitive landscape for type 1 diabetes cure or prevention therapies. It includes all the companies we are aware of with projects in development for beta cell replacement, immune therapy, or other approaches with the aim of preventing, delaying, slowing, or curing type 1 diabetes. We will continuously update the table as timelines change. Our glucose-responsive insulin and automated insulin delivery competitive landscapes are hosted on separate pages.
Beta Cell Encapsulation
|
Company |
Product |
Type |
Status |
Timeline |
|
ViaCyte |
PEC-Direct/VC-02 |
Direct vascularization, requires immunosuppressive therapy |
Phase 1/2 |
Preliminary data from phase 2 study published December 2021 and shared at ADA Scientific Sessions in June 2021, additional patient data expected in 3Q22; Preliminary phase 1/2 data presented at Cell & Gene Meeting on the Mesa in October 2019, company plans to seek RMAT designation in 2H20; “On track” to have a Regenerative Medicine Advanced Therapy (RMAT) designation from FDA as of JPM 2020; European clinical program initiated January 2019; Phase 1/2 trial initiated August 2017, expected to complete December 2020; Phase 3 was expected to initiate mid-2018 with approval projected for 2020 or beyond; Proof-of-efficacy now expected mid-2019 |
|
ViaCyte |
PEC-Encap/VC-01 |
Macroencapsulation, no direct vascularization |
Phase 1/2 |
Anticipate launching clinical study under a new protocol for VC-01 in 2022; Data in 2021 provided learnings applied to guide optimization of approach, existing study protocol closed; ViaCyte and Gore enter clinical phase agreement for membrane technology in August 2020, clinical evaluation “resumed”; Data from new Gore device expected in coming months from JPM 2020; Phase 1/2 trial initiated in 2014; Clinical development halted due to issues with FBR, but expected to resume in 1H19 |
|
Sernova |
Cell Pouch System (CPS) |
Microencapsulation within vascularized tissue matrix |
Phase 1/2 |
Five out of seven patients enrolled in phase 1/2 trial, full enrollment on track for 1Q21 as of November 2020; Sernova pens license agreement with U. of Miami for conformal cell coating technology in August 2020; Sernova acquires conformal coating technology from Converge Biotech in June 2020; Sernova partners with AgeX Therapeutics to develop immune-protected cells in June 2020; Positive preliminary phase 1/2 results released July 2019; Phase 1/2 trial recruiting with expected primary completion July 2019; IND application cleared by FDA in December 2017; Positive interim phase 1/2 results reported at JPM 2016 from a Canadian trial, but the trial has since been terminated; Announced collaboration with CTI Clinical Trial and Consulting Services in November 2016 |
|
Defymed/Semma Therapeutics |
MAILPAN |
Macroencapsulation, no direct vascularization |
Preclinical |
“Safety and function of a new pre-vascularized bioartificial pancreas in an allogeneic rat model” published in Journal of Tissue Engineering in May 2020; Semma acquired by Vertex for $950 million in September 2019; Positive preclinical data released in July 2019; $114M Series B financing in December 2017; Received funding for preclinical trials from JDRF in July 2016; Partnered with Semma Therapeutics for an unlimited stem cell-derived beta cell source in December 2016 |
|
JDRF/UCSF/Stanford |
Undisclosed |
Cell Replacement Cures Without Immunosuppresion |
Preclinical |
Fall 2020 progress report; Spring 2020 progress report; Collaboration announced |
|
Lilly/Sigilon |
Undisclosed |
Macroencapsulation using FBR-resistant Afibromer encapsulation device |
Preclinical |
Collaboration announced April 2018 |
|
Novo Nordisk/Procyon Technologies |
Undisclosed |
Oxygen-enabled cell encapsulation device |
Preclinical |
Partnership announced December 2020 |
|
Novo Nordisk/UCSF/Cornell |
Undisclosed |
Stem cell derived insulin producing cells |
Preclinical |
Collaboration announced May 2018 |
|
Encellin (fka Dr. Tejal Desai lab at UCSF) |
nanotemplating |
Next-generation encapsulation approach |
Preclinical |
Research contributed to UCSF spinout Encellin, “hopes to move into the clinic in 2020” as of August 2019; Presented at the 2016 JDRF Bay Area Annual Meeting and Research Update |
|
Goethe University/DRIVE Consortium |
LSFM4Life |
“Organoid” (insulin-producing cells derived from adult stem cells and embedded in a three-dimensional matrix) |
Preclinical |
“Standardized GMP-compliant scalable production of human pancreas organoids” published in Stem Cell Research & Therapy in March 2020; Project launched in April 2016 with goal of entering clinical trials by end of 2019 |
|
DRIVE Consortium/Boston Scientific |
Undisclosed |
Encapsulation device containing gel-embedded clusters of islet cells; capsule releases immunosuppressant; Boston Scientific to help develop minimally invasive injection procedure |
Preclinical |
Participated in TERMIS EU 2019; Announced in June 2015 |
|
Dr. Shuvo Roy lab at UCSF |
Bioartificial pancreas |
Islets contained in semi-permeable silicon ultrafiltration capsule with active blood flow; Graft rather than subcutaneous implant |
Preclinical |
In the midst of three-year $2.4 million NIH grant and two year $1.1 million CIRM grant as of September 2018. Presented at the 2016 JDRF Bay Area Annual Meeting and Research Update |
|
ViaCyte/CRISPR Therapeutics |
PEC-QT |
Islets are derived from genetically engineered stem cells that are immune-evasive |
Preclinical |
Health Canada approved initiation of a Phase 1 trial of immune-evasive, stem cell-derived therapy for the treatment of T1D in November 2021; “Moving into pre-IND activities” as of May 2020; Preclinical data reported at EASD in 2019 showed gene-edited immune-evasive cells demonstrated in vitro protection against activated T-cells compared to controls; Announced at JPM 2018; Received $1.4 million from CIRM for development of immune-evasive stem cells in December 2017; Subsequently partnered with CRISPR Therapeutics |
|
Betalin Therapeutics |
Undisclosed |
Micro-pancreas |
Preclinical |
“Currently raising capital to further optimize the therapy and complete submission of the New Drug (IND) application to the FDA” as of December 2020 on company website; Betalin announces plans to advance into UK clinical testing by early 2021 in July 2020 |
|
Seraxis Therapeutics |
SR-01 |
Implantable biocompatible device |
Preclinical |
The Maryland-based company completed $40 million series C in February 2021 that included investment from JDRF T1D fund, SR-01 is comprised of lab-grown pancreatic islets from Seraxis’ proprietary stem cell line SR1423 and its SeraGraft “implantable biocompatible device,” which protects the cells from immune recognition. |
Beta Cell Replacement Sources
|
Company |
Product |
Status |
Timeline |
|
Vertex (Semma Therapeutics technology) |
Stem cell-derived insulin producing cells |
Phase 2 |
Preliminary data from phase 2 study released in October 2021 shows extremely promising results; Naked cell phase 1/2 launch and FDA Fast Track Designation received in March 2021; Naked cell program IND approved January 2021; Vertex on track to submit IND filing for islet cell transplant program in “late 2020,” phase 2 to commence in 2021 as of 3Q20 update; Cell therapy to move into clinic by late 2020/early 2021, as of JPM 2020; Acquired by Vertex Pharmaceuticals for $950 million in September 2019; Partnership with AZ, Novartis, and Defymed; Published in Nature in 2019 detailing improved beta cell generation/purification process; Semma Therapeutics founded in April 2015 to translate Dr. Doug Melton’s beta cell generation procedure into therapy |
|
Sanofi/Evotec |
Functional stem cell-derived human beta cells for replacement therapies and for identification of active therapeutic targets |
Preclinical |
Evotec regains global rights to beta cell replacement therapy in April 2020, “exploring partnering options to bring this therapy to patients”; Sanofi discontinues R&D in diabetes in December 2019; Management expressed enthusiasm in January 2019 (interview); Partnership announced in August 2015 |
|
AZ MedImmune/Evotec |
Biologic beta cell regeneration |
Preclinical |
Status unknown as of 2020; Partnership announced in December 2010 |
|
ViaCyte |
Stem cell-derived insulin producing cells |
In 2006, ViaCyte published the first study demonstrating successful in vitro differentiation of human embroyonic stem cells into pancreatic tissue; In 2014 BetaLogics (now part of ViaCyte) published the first paper on using in vitro stem-cell derived beta cells to reverse diabetes in mice; ViaCyte is currently in phase 1/2 clinical studies with its stem cell-derived insulin producing cells |
|
|
UCSF Hebrock Lab |
Stem cell-derived enriched beta clusters (more advanced insulin-producing cells) |
Preclinical |
Published methodology for growing enriched beta clusters (eBCs – more mature, insulin-producing cells) from stem cells |
|
Frequency |
Progenitor Cell Activation (PCA) platform, which induces stem cell-like properties in somatic cells |
Preclinical |
No longer active in diabetes as of 2020; Received JDRF funding in February 2017 to optimize the PCA platform for type 1 diabetes |
|
eGenesis |
Islet cell transplantation |
Preclinical |
Completed $125 million Series C in March 2021; HuCo islet cells are currently in preclinical development; working on islet cell transplantations |
|
Pancryos |
PanINSULA stem cell-derived islet cell replacement |
Preclinical |
In May 2022, Pancryos announced a global exclusive license agreement with Brigham and Women’s Hospital to combine PanINSULA with the Harvard Stem Cell Institute’s convection-enhanced microencapsulation device |
Immune Therapies
|
Organization |
Product |
Type |
Status |
Timeline |
|
Provention (previously TrialNet) |
teplizumab |
Anti-CD3 |
Phase 3 |
Provention received a CRL from the FDA in July 2021; In January 2021 ProventionBio filed the BLA with the FDA for type 1 diabetes delay, under the company’s requested Priority Review, MAA filing in Europe slated for 2021, BLA submission completed to FDA with AdComm expected as of 3Q20 update; Follow-up data showing sustained delay of three years presented at ADA 2020; FDA Breakthrough Therapy Designation in August 2019; First therapy shown to delay type 1 diagnosis (ADA 2019); Phase 3 PROTECT study underway as of April 2019, following acquisition of candidate from MacroGeneics |
|
Immune Tolerance Network |
alefacept |
Anti-CD2 |
Phase 2 |
No new dedicated trials as of December 2020; Long-term effect study for multiple ITN candidates recruiting as of December 2020, estimated to complete in March 2025; Alefecept removed from market for psoriasis due to “business reasons” in 2015; Two year T1DAL phase 2 trial results presented at ADA 2015 |
|
TrialNet |
ATG/GCSF |
Combination approach |
Phase 2 |
Two year phase 2 results presented at ADA 2020, combination did not reach statistical significance on primary endpoint; Phase 2 results presented at ADA 2018; ClinicalTrials.gov lists full phase 2 completion in October 2018 |
|
TrialNet |
CTLA4-lg (abatacept) |
Immunosuppressant |
Phase 2 |
Primary completion pushed to November 2021 as of December 2020; Phase 2 trial expected to complete in November 2020, pushed back from February 2018; Results expected in 2020 |
|
Caladrius Biosciences |
CLBS03 |
Treg |
Phase 2 |
Negative topline phase 2a T-Rex (pediatric) results released in February 2019, two-year follow-up data to determine next steps; Phase 1 results presented at ADA 2014; Phase 2 clinical studies launched in March 2016, full completion is expected in March 2020 |
|
GeNeuro |
GNbAC1 |
Monoclonal antibody that neutralizes pathogenic protein MSRV-Env |
Phase 2a |
Positive 12-month results released in May 2019, “GeNeuro believes these data open the door to further development in early-onset T1D pediatric patient population;” Positive six-month phase 2a data released in September 2018; Phase 2a trial initiated April 2017 and expected to complete March 2019 |
|
ActoBio |
AG019 |
Engineered Lactococcus Lactis bacteria that releases proinsulin + IL-10 into gut |
Phase 1b/2a |
Advancing through phase 2a with teplizumab as of 4Q20, positive topline phase 1b results released in August 2020; Phase 1b/2a in combination with teplizumab expected to complete in June 2020 |
|
Imcyse |
Imotopes |
Auto-antigen derived epitopes + thioreductase motif to reprogram autoimmune T-cells |
Phase 1b |
Joined INNODIA in March 2020; Positive poster presented at EASD 2019; Results expected 3Q19; Moving into phase 2 on back of Series B financing |
|
UCSF (Dr. Jeffrey Bluestone) |
Tregs + IL2 |
Combination of CD4, CD127, and CD25 Tregs with IL2 |
Phase 1 |
Phase 1 TILT study expected to complete July 2021 |
|
City of Hope |
PIpepTolDC
|
Combination of patient’s own dendritic cells, vitamin D3, and fragment of pro-insulin |
Phase 1 |
Patient enrollment for phase 1 trial announced in December 2020; Candidate previously delivered positive phase 1 results in the Netherlands in May 2020 |
|
Dr. Camillo Ricordi and Dr. Jay Skyler |
Undisclosed |
Combination of ATG, GCSF, IL-2, etanercept, and exenatide
|
|
Phase 1 trial to initiate in December 2021 (pushed from December 2018); Highlighted by Dr. Jay Skyler at WCIRDC 2020 and EASD 2018 |
|
Pfizer |
nanoformulations (tolerance molecule + antigen) |
Immune tolerance therapy |
Preclinical |
Licensed from AnTolRx in March 2019 |
|
Provention Bio |
Enteroviral vaccine |
Vaccine targeting Coxsackievirus B infection |
Preclinical |
Proof-of-concept data released in May 2020; Announced July 2017; Funding from J&J Innovation and JDRF T1D Fund |
|
Anokion (fka Kanyos Bio) |
Undisclosed |
Immune tolerance therapy |
Preclinical |
Type 1 diabetes program active on pipeline page as of December 2020; Kanyos Bio acquired by Anokion in September 2019; Kanyos Bio created by partnership between Astellas Pharma and Anokion in July 2015 |
|
Selecta |
SVP-Rapamycin/SVP-insulin |
Synthetic Vaccine Particle (SVP) nanotechnology for immunosuppression |
Preclinical |
Type 1 program not listed on pipeline page as of December 2020; Series E financing in September 2015 |
|
Mesoblast |
Undisclosed |
Mesenchymal precursor cell (MPC) treatment |
Preclinical |
Type 1 not listed on pipeline page as of December 2020; “Ex vivo fucosylation” technology licensed from HMS in March 2016; As of January 2016, Mesoblast was investigating a type 1 diabetes application for its MPC treatment |
|
REGiMMUNE/JDRF/Pfizer |
RGI-3100 |
Type 1 diabetes-specific antigen, to be used with reVax immunomodulatory platform to promote Tregs |
Preclinical |
Diabetes no longer listed on REGiMMUNE pipeline as of December 2020; “A Novel Liposome Formulation Carrying Both an Insulin Peptide and a Ligand for Invariant Natural Killer T Cells Induces Accumulation of Regulatory T Cells to Islets in Nonobese Diabetic Mice” published in Journal of Diabetes Research in October 2019; Collaboration with JDRF and Pfizer announced in April 2015 |
|
GSK |
otelixizumab |
Anti-CD3 |
Discontinued |
No longer listed in GSK pipeline; Negative phase 3 results in 2011; Revived phase 2 program completed September 2018 ahead of schedule |
|
Novo Nordisk |
NN9828 |
Anti-IL21/GLP-1 agonist (liraglutide) combination |
Discontinued |
Discontinued in 3Q20; Positive full phase 2 results presented at ADA 2020; Positive phase 2 topline results shared in 2Q19; Phase 2 trial expected to complete March 2019; FDA orphan drug designation in January 2017 |
|
Janssen |
golimumab (Simponi) |
TNF-alpha |
Discontinued |
Type 1 program discontinued in November 2020; Positive phase 2 results presented at ADA 2020; Phase 1 study in youth (n=30) with ≥2 antibodies expected to complete July 2021 |
Other
|
Organization |
Name |
Type |
Status |
Timeline |
|
TrialNet |
Oral insulin |
Oral insulin |
Phase 3 |
“Slowed Metabolic Decline After 1 Year of Oral Insulin Treatment Among Individuals at High Risk for Type 1 Diabetes in the Diabetes Prevention Trial–Type 1 (DPT-1) and TrialNet Oral Insulin Prevention Trials” published in Diabetes in August 2020; Negative results in relatives of patients with type 1 released in November 2017; Phase 3 trial fully enrolled; Results presented at ADA 2017; Phase 2 dose-ranging trial expected to complete in December 2017 |
|
Evolve Systems |
EVC001 |
B. infantis |
Not applicable |
March 2021 announced that the primary prevention SINT1A trial of 1,444 participants will be treated with the EVC001 candidate as a potential preventive treatment for type 1 diabetes in infants at-risk of developing the condition; Closed $55 million Series D in February 2021 |
|
Global Platform for the Prevention of Autoimmune Diabetes |
Oral insulin |
Oral insulin (high-dose in infants) |
Phase 2b |
Primary Oral Insulin Trial (POInT) currently screening in Europe; Primary completion slated for January 2025 as of December 2020 |
|
Kamada |
Undisclosed |
Alpha-1 Antitrypsin (AAT) |
Phase 2 |
Type 1 program active on company website as of December 2020; Topline phase 2 data released November 2017; Early signs of efficacy in people age 12-18 with new-onset type 1 diabetes |
|
Massachusetts General Hospital (Dr. Denise Faustman) |
BCG vaccine |
Bacillus Calmette- Guérin (BCG)
|
Phase 2 |
Phase 2 trial slated to complete in July 2020; Boundless Investing webinar highlights BCG vaccine in July 2020; Phase 1 results released in July 2018 controversial; Primary completion expected in July 2020 with full completion expected in July 2023 |
|
vTv Therapeutics |
TTP399 |
Liver-selective glucokinase activator |
Phase 2 |
Positive results from mechanistic study announced in October 2021; In April 2021 vTv received an FDA Breakthrough Designation following positive results from phase 2 trial |
|
Novo Nordisk |
NN-1845 |
Glucose Sensitive Insulin |
Phase 1 |
Phase 1 trial of glucose sensitive insulin competed 3Q21 –established proof of concept, shown to be well-tolerated |
|
IM Therapeutics |
IMT-002 |
human leukocyte antigen (HLA) |
Phase 1 |
In July 2021, announced positive results from a phase 1b trial assessing the safety, tolerability, pharmacokinetics, and mechanistic action with small drug molecule IMT-002. IMT-002 works by blocking HLA-DQ8, which inhibits the activation of T-cells, effectively “starving the autoimmune process” and ultimately preserving beta-cell function. IM Therapeutics, a Boston area based company, closed a $10 million Series A funding round in 2019 with investment from the JDRF T1D Fund. |
|
Sonoma Biotherapeutics |
SBT-11-5301 |
Teff debulking |
Preclinical |
Closed $265 million oversubscribed Series B in August 2021 that included funding from JDRF T1D Fund; Technology selectively depletes T effector cells |
|
GentiBio |
Undisclosed |
Regulatory T cells |
Preclinical |
GentiBio closes $157 million Series A in August 2021;GentiBio technology converts the more abundant T effector cell from a patient into potent, durable engineered Treg cells that can be injected back into the patient’s body |
|
Calibr |
Neratinib |
Mammalian sterile 20–like kinase-1 (MST1) inhibitor
|
Preclinical |
“Neratinib protects pancreatic beta cells in diabetes” published in Nature Communications in November 2019; Discussed at 2016 JDRF Mission Summit |
|
Kriya Therapeutics |
KT-A112 |
AAV |
Preclinical |
Closed $100 million Series B with help from JDRF T1D Fund in July 2021; Adeno-associated viruses (AAVs) are non-pathogenic viruses engineered to deliver DNA to drive the endogenous production of insulin |
-- by Claire Holleman, Hanna Gutow, Abigail Dove, Helen Gao, Zhazira Irgebay, Sahaj Shah, Rhea Teng, and Kelly Close