Premier 21 Gene Therapy Firms For Immunodeficiency Syndromes
Premier 21 Gene Therapy Firms For Immunodeficiency Syndromes
In the biotechnology and healthcare industry, an emergent body of companies are tailoring their work towards gene therapy for immunodeficiency syndromes. These companies utilize advanced genetic research and cutting-edge techniques to develop treatments for genetic disorders that result in weakened immune systems. Pioneers such as CRISPR Therapeutics and Stoke Therapeutics are utilizing CRISPR/Cas9 gene-editing technology and RNA-based medicines to treat diseases at a genetic level. Nonprofits like Jeffrey Modell Foundation are raising awareness on immunodeficiency disorders and funding innovative research. The future of this industry sees greater promise on the horizon as gene-based therapies continue breaking new ground, fostering advanced treatment options for numerous Immunodeficiency Syndromes.
Top 21 Immunodeficiency Syndromes Gene Therapy Companies
1. CRISPR Therapeutics
- Website: crisprtx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2013
- Headcount: 201-500
- Latest funding type: Grant
CRISPR Therapeutics is a company that specializes in gene editing and cell therapies. They use the CRISPR/Cas9 therapeutic approach to treat genetically-defined diseases by editing cells inside the body.
2. Stoke Therapeutics
- Website: stoketherapeutics.com
- Headquarters: Bedford, Massachusetts, United States
- Founded: 2017
- Headcount: 51-200
- Latest funding type: Post Ipo Equity
Stoke Therapeutics is a biotechnology company that is focused on addressing severe diseases by upregulating protein expression with RNA-based medicines. Their goal is to develop the first disease-modifying therapy for Dravet syndrome, a severe and progressive genetic epilepsy. With their proprietary research platform called TANGO, Stoke aims to restore missing proteins by increasing protein output from healthy genes.
3. Jeffrey Modell Foundation
- Website: info4pi.org
- Headquarters: New York, New York, United States
- Founded: 1987
- Headcount: 1-10
The Jeffrey Modell Foundation (JMF) is a global nonprofit dedicated to early diagnosis, meaningful treatments, and ultimately, cures for Primary Immunodeficiency (PI) through awareness, advocacy, and research. Vicki and Fred Modell established JMF in 1987 in memory of their son, Jeffrey, who died at the age of 15 due to complications from Primary Immunodeficiency (PI). PIs are a group of more than 450 genetic disorders of the immune system that are chronic, serious, and often fatal. PIs affect an estimated 10 million people worldwide. To date, JMF has established a global centers network including over 900 physicians at 402 medical institutions in 87 countries, funded millions of dollars in innovative research for treatments and cures, implemented newborn screening programs in the United States and abroad, and spearheaded a genetic sequencing initiative called Jeffrey's Insights with a goal of 10,000 patients sequenced. Additionally, JMF's awareness-raising activities have reached millions around the world, encouraging early diagnosis and improved quality of life. Join our mission to #CurePI worldwide. Learn more on our website at https://www.info4pi.org.
4. London School of Hygiene and Tropical Medicine, U. of London
- Website: lshtm.ac.uk
- Headquarters: London, Greater London, United Kingdom
- Founded: 1899
- Headcount: 1001-5000
The London School of Hygiene & Tropical Medicine (LSHTM) is a renowned academic institution focused on global health research, education, and policy. It offers a wide range of programs, expertise, and resources to address key healthcare challenges worldwide.
5. Plethora Health
- Website: plethora.health
- Headquarters: Barueri, São Paulo, Brazil
- Founded: 2019
- Headcount: 11-50
Plethora Health is a digital HealthTech platform that integrates the entire healthcare industry in Brazil. It collects and stores data, providing insights and information previously unattainable in the market. With a focus on data analysis, AI, and machine learning, Plethora Health aims to facilitate healthcare and provide the best user experience.
6. Immunodeficiency UK
- Website: immunodeficiencyuk.org
- Headquarters: London, United Kingdom
- Headcount: 1-10
Immunodeficiency UK, launched on the 1st April 2021, is a charity supporting individuals and families affected by a primary and secondary immunodeficiency in the UK. The charity’s day-to-day operations are funded entirely by donations and grants. Our aims are to help ensure that those affected by a primary and secondary immunodeficiency have the knowledge needed to manage their condition effectively and to ensure that their health needs are understood and addressed by those involved in policy and delivery of healthcare. Immunodeficiency UK's registered charity number is 1193166. Our history For the period from 2013 to the 1st April 2021 we were known as the organisation Primary Immunodeficiency UK and managed as part of the charity Genetic Disorders UK.
7. Jeffrey Modell Foundation INC
- Website: jmfworld.com
- Headquarters: New York, New York, United States
- Headcount: 1-10
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8. The XLP Research Trust
- Website: xlpresearchtrust.org
- Headquarters: Romsey, Hampshire, United Kingdom
- Founded: 1995
- Headcount: 1-10
X-linked lymphoproliferative syndrome (XLP), which is also known as Duncan’s syndrome, is a rare always fatal disease that affects only boys. To date only about 100 families and 400+ boys have been diagnosed worldwide. It is likely, however, that there are many more cases where the correct diagnosis has not been made. Founded in 2005, the XLP Research Trust exists to 1. Promote and fund research into the cause, management, symptoms and cure for XLP 2. Provide a point of contact and support for those families affected by XLP 3. Raise the awareness of XLP amongst the medical profession and the general public
9. Hyper IgM Foundation
- Website: hyperigm.org
- Headquarters: New York, New York, United States
- Founded: 2015
- Headcount: 1-10
Hyper IgM Foundation, a non-profit organization, was founded in 2015 by dedicated parents, healthcare professionals, and friends. Hyper IgM Syndrome is a rare disease affecting only 1 in 1,000,000 individuals, which is caused by a genetic mutation that severely compromises the immune system. Those living with Hyper IgM face a significantly shortened life span, multiple hospitalizations, and a high risk of secondary cancers. The only known cure is a bone marrow transplant. Due to its rarity, treatment and diagnosis of this disease is ad hoc, at best. Our mission is to improve the treatment, quality of life and long term outlook for children and adults living with Hyper IgM through research, support, education, and advocacy to families and patients.
10. Jasper Therapeutics
- Website: jaspertherapeutics.com
- Headquarters: Redwood City, California, United States
- Founded: 2018
- Headcount: 11-50
- Latest funding type: Post Ipo Equity
Jasper Therapeutics is a biotechnology company specializing in stem cell grafts and mRNA therapies. They focus on developing innovative treatments for various diseases and conditions. Through their cutting-edge research and scientific expertise, Jasper Therapeutics aims to revolutionize the field of regenerative medicine.
11. Fortress Biotech
- Website: fortressbiotech.com
- Headquarters: Bay Harbor Islands, Florida, United States
- Founded: 2006
- Headcount: 11-50
- Latest funding type: Post Ipo Equity
Fortress Biotech is a commercial-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for various diseases. They specialize in the treatment of skin cancer, amyloid light chain (AL) amyloidosis, cytomegalovirus (CMV), acute myeloid leukemia (AML), breast cancer, Menkes disease, X-linked severe combined immunodeficiency (XSCID), multiple myeloma (MM), and other genetically driven cancers. Their portfolio includes both marketed products and clinical-stage product candidates, utilizing gene therapy, immunotherapy, and targeted therapies. They are committed to improving patient outcomes and addressing unmet medical needs.
12. Severe Combined Immune Deficiency
- Website: scid.net
- Headquarters: Lakeland, Florida, United States
- Headcount: 11-50
SCID.net is a website that provides information and resources related to Severe Combined Immunodeficiency (SCID), which is a primary immune deficiency that can cause serious infections and other health issues.
13. BioMarin Pharmaceutical Inc.
- Website: biomarin.com
- Headquarters: San Rafael, California, United States
- Founded: 1997
- Headcount: 1001-5000
- Latest funding type: Post Ipo Debt
BioMarin is a pharmaceutical company specializing in genetic therapies. They develop and provide transformative treatments for various genetic diseases, such as Morquio A Syndrome, PKU, and MPS. With a focus on gene therapy research, BioMarin aims to maximize the impact of genetic innovation and improve patients' lives.
14. Ultragenyx
- Website: ultragenyx.com
- Headquarters: Novato, California, United States
- Founded: 2010
- Headcount: 501-1000
- Latest funding type: Post Ipo Equity
Ultragenyx is a biopharmaceutical company focused on developing and commercializing novel treatments for rare genetic diseases. They have a diverse portfolio of approved therapies and product candidates targeting diseases with high unmet medical need. Their goal is to provide safe and effective therapies to patients with a sense of urgency.
15. Generium
- Website: generium.ru
- Headquarters: Moscow, Moscow City, Russian Federation
- Founded: 2009
- Headcount: 1001-5000
Generium is a biotechnology company specializing in the production of pharmaceutical drugs. They are known for their research and development in the field of biotechnology and chemistry.
16. Genethon
- Website: genethon.fr
- Headquarters: Évry, Île-De-France, France
- Founded: 1990
- Headcount: 51-200
Genethon is a company dedicated to the development of gene therapy drugs for rare diseases. They focus on the preclinical and clinical development of gene therapy drugs for rare diseases. Genethon's pipeline includes therapies for various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and Wiskott-Aldrich syndrome.
17. Neurogene Inc.
- Website: neurogene.com
- Headquarters: New York, New York, United States
- Founded: 2018
- Headcount: 51-200
- Latest funding type: Series A
Neurogene is a biotech company focused on developing gene therapies for rare neurological diseases. They are dedicated to addressing the high unmet need in this field and aim to revolutionize medical treatment through innovation and scientific execution. With a strong sense of purpose, Neurogene aims to develop life-changing genetic medicines for individuals and families affected by devastating neurological conditions.
18. I-Stem
- Website: istem.eu
- Headquarters: Evry Cedex, Ile-De-France, France
- Founded: 2005
- Headcount: 51-200
I-Stem is an institute specializing in stem cell research for the treatment and study of monogenic diseases.
19. Homology Medicines, Inc.
- Website: homologymedicines.com
- Headquarters: Bedford, Massachusetts, United States
- Founded: 2015
- Headcount: 51-200
- Latest funding type: Post Ipo Equity
Homology Medicines is a genetic medicine company dedicated to developing new treatments and cures for rare diseases. With their powerful in vivo genetic medicines platform, they aim to transform the lives of patients by potentially offering them a chance at a cure.
20. Avrobio
- Website: avrobio.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2015
- Headcount: 51-200
- Latest funding type: Ipo
AVROBIO is a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease. They are focused on targeting lysosomal disorders and developing personalized therapies for patients. Their goal is to enable individuals with genetic diseases to live longer and free from disease progression.
21. Encoded Therapeutics Inc.
- Website: encoded.com
- Headquarters: South San Francisco, California, United States
- Founded: 2014
- Headcount: 51-200
- Latest funding type: Series D
Encoded Therapeutics is a company focused on translating next-generation science into transformative gene therapies. They aim to develop innovative treatments using gene therapy to address unmet medical needs.
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