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March 28, 2022 / eMedClub News / -- SQZ Biotechnologies, a clinical-stage biotechnology company, announced today that it has received a $2 million grant from the National Institutes of Health and the National Institute of General Medical Sciences.
grant to support the company's development of novel cell replacement therapies for Parkinson's disease (PD)
.
The cell replacement therapy aims to directly reprogram PD patients' own immune cells into dopamine-producing neurons, a potential new treatment for PD
.
PD is the misfolding and abnormal accumulation of alpha-synuclein in the central and peripheral nervous systems, resulting in progressively worsening motor and non-motor symptoms throughout the body, mainly manifested as bradykinesia, rigidity, tremor, postural gait Abnormal and other movement disorders, often accompanied by a variety of non-motor symptoms such as hyposmia and sleep disturbance
.
The progressive motor symptoms of PD are largely due to the death of dopamine neurons in the substantia nigra, the part of the midbrain that converts levodopa to dopamine, in one step by the enzyme aromatic L-amino acid decarboxylase (AADC).
) catalyzed
.
Neurons in the substantia nigra then release dopamine into the putamen, where dopamine receptors are located
.
In PD, neurons in the substantia nigra degenerate and AADC is markedly reduced in the putamen, which limits the ability of the brain to convert oral levodopa to dopamine
.
PD is the second most common chronic progressive neurodegenerative disease after Alzheimer's disease, affecting nearly 10 million people worldwide
.
At present, clinical treatment is mainly based on drug therapy, rehabilitation training and other symptomatic treatment methods, and there is no effective means to control the disease
.
SQZ Biotechnologies' Novel Cell Replacement Therapy Reprograms PD Patients' Cells to Replace Lost or Diseased Cells
.
Preclinical data presented by SQZ Biotechnologies at the 2021 International Society for Stem Cell Research Annual Meeting demonstrate that the company's proprietary Cell Squeeze technology can deliver a mRNA transcription factor to induce iPSC-derived neurons
.
Cell Squeeze technology can generate reprogrammed cells by controlling the magnitude and timing of single or multiple cell programming factors
.
Because of the speed of the process, the lack of viral or genetic manipulation, and the use of the company's clinically proven manufacturing methods, the specific cells produced by the process have the potential for translational therapy
.
Recommended reading: Using mRNA to successfully induce iPSCs into neurons, breaking through the limitations of traditional reprogrammingDr.
Jonathan Gilbert, vice president and research leader of SQZ Biotechnologies, said: "By reprogramming the patient's own immune cells to directly generate dopamine Neurons will be a major breakthrough, a new paradigm for Parkinson's disease treatment
.
Unlike other allogeneic cell replacement therapies being developed in the Parkinson's disease field, we use the patient's own cells for treatment, This does not require chronic immunosuppression
.
Furthermore, when cells are engineered using RNA-based cell engineering methods, the genome does not undergo any changes that could pose long-term risks
.
" SQZ: Pioneering Cell Squeeze Technology 2014, to 100 With $10,000 in seed funding, SQZ Biotechnologies emerged from the labs of Robert Langer and Klavs Jensen at the Massachusetts Institute of Technology (MIT)
.
And quickly signed a $500 million immuno-oncology deal with global pharmaceutical giant Roche to jointly develop a cell therapy platform
.
The cell therapy will use the Cell Squeeze technology exclusively licensed from MIT owned by SQZ Biotechnologies, where Squeeze (extrusion) is the source of the company's name
.
The technology physically squeezes cells at high speed through a microfluidic chip, temporarily opening the cell membrane and diffusing specific antigens into the cell before the cell membrane re-closes
.
At present, SQZ Biotechnologies has developed dozens of chips to squeeze cells of different sizes, so that the genetic engineering of cells can be completed without relying on viral vectors
.
The platform technology uses physical methods to break cell membranes, so the safety of cell therapy can be greatly improved, and based on this physical delivery method is not only simple to operate, high efficiency, but also low cost, and most importantly, because it does not itself Covers chemical ingredients and other biological ingredients, so there will be no additional side effects
.
▲ Cell extrusion technology platform (Image source: SQZ Biotechnologies) Relying on its unique cell extrusion technology platform, SQZ Biotechnologies has developed SQZ APCs (antigen presenting cells), SQZ AACs (activated antigen carriers) and SQZ TACs (tolerance) These three platforms are used to treat tumors, infectious diseases and autoimmune diseases
.
SQZ APCs (antigen presenting cells) SQZ APCs use specific antigens to extrude PBMCs (peripheral blood mononuclear cells) extracted from patients to form engineered cells and then transport them back to the patient, thereby activating specific T cells against this antigen cellular response
.
At present, the SQZ APCs platform is mainly used in the treatment of tumors and infectious diseases
.
▲ SQZ APCs platform (Image source: SQZ Biotechnologies) In January this year, SQZ Biotechnologies announced that the US FDA has approved the IND application for the Phase 1/2 clinical trial of its mRNA-based cell therapy SQZ-eAPC-HPV
.
The trial will target patients with human papillomavirus (HPV) 16-positive solid tumors, including head and neck, cervical and anal cancers, whose disease has progressed after standard treatment
.
SQZ-eAPC-HPV is based on the company's SQZ APCs platform technology to simultaneously deliver 5 different mRNAs to the patient's monocytes, T cells, B cells and natural killer (NK) cells in one step.
5 different functions
.
Preclinical results showed that SQZ-eAPCs were able to generate robust CD8-positive T cell responses against multiple antigens, including HPV16 protein, by simultaneously expressing antigen, CD86, membrane-bound IL-2, and IL-12
.
Recommended reading: Heavy! For the treatment of HPV-positive tumors, "extruded" cell therapy has entered the clinicYimai Meng broke the news SQZ AACs (activated antigen carrier) SQZ AACs platform and SQZ APCs platform have similar technical principles, the difference is that SQZ AACs are extruded with antigens.
Produced by pressing red blood cells (RBCs) and activating adjuvants
.
After the engineered red blood cells are infused into the patient, the SQZ AACs are rapidly taken up by professional antigen-presenting cells through a natural process to destroy senescent red blood cells in the body
.
To take advantage of this process, SQZ AACs were designed as a "Trojan horse" to deliver large amounts of antigens and activating factors to endogenous antigen-presenting cells in lymphoid organs to induce a specific T-cell response against this antigen
.
At present, the SQZ AACs platform is mainly used in the treatment of tumors
.
Among them, the main candidate product SQZ-AAC-HPV tumor vaccine, its domestic clinical trial application has been accepted by the China National Medical Products Administration Center for Drug Evaluation (CDE) on January 5, 2022
.
On February 1 last year, the US FDA approved a Phase 1 clinical trial of SQZ-AAC-HPV for the treatment of HPV-positive tumors
.
Recommended reading: Erythrocyte vectored tumor vaccine is applied for clinical application in China for the treatment of HPV-positive tumorsYimai Meng broke the news ▲ SQZ AACs platform (Image source: SQZ Biotechnologies) Sexual antigens squeeze red blood cells to form TACs.
After infusion into the patient, when the body destroys the RBCs, the components in the TACs are presented in a tolerable manner, preventing the immune system from attacking its own red blood cells
.
The SQZ TACs platform is still in the preclinical development stage and is mainly used for the treatment of autoimmune diseases
.
▲ SQZ TACs Platform (Image source: SQZ Biotechnologies) SQZ Biotechnologies has demonstrated the use of SQZ platform technology to deliver multiple materials into multiple primary cell types with minimal impact on cell function and viability
.
And this differentiated functionality enables the engineering of multiple cellular functions and the creation of a broad range of cell therapies for multiple disease treatment areas
.
The company has created a broad pipeline of product candidates for different diseases based on this unique technology
.
▲ Product pipeline (image source: SQZ Biotechnologies) Reference: 1.
https:// of-health-to-develop-a-novel-scalable-cell-replacement-therapy-for-parkinson-s-disease/——List of recent popular activities——▼On March 29, nucleic acid drug industry development analysis and process development Highlights▼On March 31, authoritative interpretation of the characterization and analysis technology of AAV gene therapy drugs▼On March 31, Guangzhou, the theme salon of quality control challenge of virus delivery vector AAV▼July 1-3, Shanghai, 2022 Shanghai International Biotechnology Innovative Drug Industry Expo
grant to support the company's development of novel cell replacement therapies for Parkinson's disease (PD)
.
The cell replacement therapy aims to directly reprogram PD patients' own immune cells into dopamine-producing neurons, a potential new treatment for PD
.
PD is the misfolding and abnormal accumulation of alpha-synuclein in the central and peripheral nervous systems, resulting in progressively worsening motor and non-motor symptoms throughout the body, mainly manifested as bradykinesia, rigidity, tremor, postural gait Abnormal and other movement disorders, often accompanied by a variety of non-motor symptoms such as hyposmia and sleep disturbance
.
The progressive motor symptoms of PD are largely due to the death of dopamine neurons in the substantia nigra, the part of the midbrain that converts levodopa to dopamine, in one step by the enzyme aromatic L-amino acid decarboxylase (AADC).
) catalyzed
.
Neurons in the substantia nigra then release dopamine into the putamen, where dopamine receptors are located
.
In PD, neurons in the substantia nigra degenerate and AADC is markedly reduced in the putamen, which limits the ability of the brain to convert oral levodopa to dopamine
.
PD is the second most common chronic progressive neurodegenerative disease after Alzheimer's disease, affecting nearly 10 million people worldwide
.
At present, clinical treatment is mainly based on drug therapy, rehabilitation training and other symptomatic treatment methods, and there is no effective means to control the disease
.
SQZ Biotechnologies' Novel Cell Replacement Therapy Reprograms PD Patients' Cells to Replace Lost or Diseased Cells
.
Preclinical data presented by SQZ Biotechnologies at the 2021 International Society for Stem Cell Research Annual Meeting demonstrate that the company's proprietary Cell Squeeze technology can deliver a mRNA transcription factor to induce iPSC-derived neurons
.
Cell Squeeze technology can generate reprogrammed cells by controlling the magnitude and timing of single or multiple cell programming factors
.
Because of the speed of the process, the lack of viral or genetic manipulation, and the use of the company's clinically proven manufacturing methods, the specific cells produced by the process have the potential for translational therapy
.
Recommended reading: Using mRNA to successfully induce iPSCs into neurons, breaking through the limitations of traditional reprogrammingDr.
Jonathan Gilbert, vice president and research leader of SQZ Biotechnologies, said: "By reprogramming the patient's own immune cells to directly generate dopamine Neurons will be a major breakthrough, a new paradigm for Parkinson's disease treatment
.
Unlike other allogeneic cell replacement therapies being developed in the Parkinson's disease field, we use the patient's own cells for treatment, This does not require chronic immunosuppression
.
Furthermore, when cells are engineered using RNA-based cell engineering methods, the genome does not undergo any changes that could pose long-term risks
.
" SQZ: Pioneering Cell Squeeze Technology 2014, to 100 With $10,000 in seed funding, SQZ Biotechnologies emerged from the labs of Robert Langer and Klavs Jensen at the Massachusetts Institute of Technology (MIT)
.
And quickly signed a $500 million immuno-oncology deal with global pharmaceutical giant Roche to jointly develop a cell therapy platform
.
The cell therapy will use the Cell Squeeze technology exclusively licensed from MIT owned by SQZ Biotechnologies, where Squeeze (extrusion) is the source of the company's name
.
The technology physically squeezes cells at high speed through a microfluidic chip, temporarily opening the cell membrane and diffusing specific antigens into the cell before the cell membrane re-closes
.
At present, SQZ Biotechnologies has developed dozens of chips to squeeze cells of different sizes, so that the genetic engineering of cells can be completed without relying on viral vectors
.
The platform technology uses physical methods to break cell membranes, so the safety of cell therapy can be greatly improved, and based on this physical delivery method is not only simple to operate, high efficiency, but also low cost, and most importantly, because it does not itself Covers chemical ingredients and other biological ingredients, so there will be no additional side effects
.
▲ Cell extrusion technology platform (Image source: SQZ Biotechnologies) Relying on its unique cell extrusion technology platform, SQZ Biotechnologies has developed SQZ APCs (antigen presenting cells), SQZ AACs (activated antigen carriers) and SQZ TACs (tolerance) These three platforms are used to treat tumors, infectious diseases and autoimmune diseases
.
SQZ APCs (antigen presenting cells) SQZ APCs use specific antigens to extrude PBMCs (peripheral blood mononuclear cells) extracted from patients to form engineered cells and then transport them back to the patient, thereby activating specific T cells against this antigen cellular response
.
At present, the SQZ APCs platform is mainly used in the treatment of tumors and infectious diseases
.
▲ SQZ APCs platform (Image source: SQZ Biotechnologies) In January this year, SQZ Biotechnologies announced that the US FDA has approved the IND application for the Phase 1/2 clinical trial of its mRNA-based cell therapy SQZ-eAPC-HPV
.
The trial will target patients with human papillomavirus (HPV) 16-positive solid tumors, including head and neck, cervical and anal cancers, whose disease has progressed after standard treatment
.
SQZ-eAPC-HPV is based on the company's SQZ APCs platform technology to simultaneously deliver 5 different mRNAs to the patient's monocytes, T cells, B cells and natural killer (NK) cells in one step.
5 different functions
.
Preclinical results showed that SQZ-eAPCs were able to generate robust CD8-positive T cell responses against multiple antigens, including HPV16 protein, by simultaneously expressing antigen, CD86, membrane-bound IL-2, and IL-12
.
Recommended reading: Heavy! For the treatment of HPV-positive tumors, "extruded" cell therapy has entered the clinicYimai Meng broke the news SQZ AACs (activated antigen carrier) SQZ AACs platform and SQZ APCs platform have similar technical principles, the difference is that SQZ AACs are extruded with antigens.
Produced by pressing red blood cells (RBCs) and activating adjuvants
.
After the engineered red blood cells are infused into the patient, the SQZ AACs are rapidly taken up by professional antigen-presenting cells through a natural process to destroy senescent red blood cells in the body
.
To take advantage of this process, SQZ AACs were designed as a "Trojan horse" to deliver large amounts of antigens and activating factors to endogenous antigen-presenting cells in lymphoid organs to induce a specific T-cell response against this antigen
.
At present, the SQZ AACs platform is mainly used in the treatment of tumors
.
Among them, the main candidate product SQZ-AAC-HPV tumor vaccine, its domestic clinical trial application has been accepted by the China National Medical Products Administration Center for Drug Evaluation (CDE) on January 5, 2022
.
On February 1 last year, the US FDA approved a Phase 1 clinical trial of SQZ-AAC-HPV for the treatment of HPV-positive tumors
.
Recommended reading: Erythrocyte vectored tumor vaccine is applied for clinical application in China for the treatment of HPV-positive tumorsYimai Meng broke the news ▲ SQZ AACs platform (Image source: SQZ Biotechnologies) Sexual antigens squeeze red blood cells to form TACs.
After infusion into the patient, when the body destroys the RBCs, the components in the TACs are presented in a tolerable manner, preventing the immune system from attacking its own red blood cells
.
The SQZ TACs platform is still in the preclinical development stage and is mainly used for the treatment of autoimmune diseases
.
▲ SQZ TACs Platform (Image source: SQZ Biotechnologies) SQZ Biotechnologies has demonstrated the use of SQZ platform technology to deliver multiple materials into multiple primary cell types with minimal impact on cell function and viability
.
And this differentiated functionality enables the engineering of multiple cellular functions and the creation of a broad range of cell therapies for multiple disease treatment areas
.
The company has created a broad pipeline of product candidates for different diseases based on this unique technology
.
▲ Product pipeline (image source: SQZ Biotechnologies) Reference: 1.
https:// of-health-to-develop-a-novel-scalable-cell-replacement-therapy-for-parkinson-s-disease/——List of recent popular activities——▼On March 29, nucleic acid drug industry development analysis and process development Highlights▼On March 31, authoritative interpretation of the characterization and analysis technology of AAV gene therapy drugs▼On March 31, Guangzhou, the theme salon of quality control challenge of virus delivery vector AAV▼July 1-3, Shanghai, 2022 Shanghai International Biotechnology Innovative Drug Industry Expo
