What is Gene Therapy?
Gene therapy is a cutting-edge medical approach that aims to treat or prevent genetic diseases by modifying a person's genes. It involves introducing healthy copies of genes or modifying existing genes to correct genetic abnormalities or enhance desired traits. This can be achieved by using viral vectors or other delivery methods to deliver the therapeutic genes into target cells. Gene therapy has shown promise in treating various genetic disorders, such as cystic fibrosis and certain types of cancer, and holds potential for future advancements in personalized medicine.
AAV Gene Therapy
AAV (Adeno-Associated Virus) gene therapy is a type of gene therapy that utilizes adeno-associated viruses as vectors to deliver therapeutic genes into target cells. AAVs are non-pathogenic viruses that have been modified to remove their ability to cause disease and to carry a therapeutic gene payload instead. Once administered to the patient, AAV gene therapy vectors can efficiently enter target cells and deliver the therapeutic genes, allowing for long-term gene expression. AAV gene therapy has shown promise in treating various genetic disorders, such as inherited retinal diseases and certain types of muscular dystrophy, and has been approved for clinical use in some cases. Ongoing research aims to optimize AAV vector design, enhance delivery efficiency, and address potential immune responses to further improve the efficacy of AAV gene therapy.
CRISPR Gene Therapy
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) gene therapy is a revolutionary approach that utilizes the CRISPR-Cas9 system to edit and modify specific genes within an organism's genome. It involves using a guide RNA molecule to target a specific DNA sequence, and the Cas9 enzyme acts as a molecular scissors to cut the DNA at that location. This enables precise modifications such as gene knockout, gene insertion, or gene correction. CRISPR gene therapy holds immense potential for treating genetic diseases, as it offers a highly precise and customizable tool for altering the genome.
The Gene Therapy Market
Gene therapy is one of the fastest-growing markets in the world, it was valued at USD 2.6 billion in 2020 and is expected to grow at an exponential rate of 8.3% per year.
Gene therapy is concerned with the genetic modification of genes inside the body in order to treat or stop diseases. This is done by either replacing a faulty gene or adding a new gene in order to achieve the desired result. Gene therapy holds considerable promise for treating diseases such as cancer, hemophilia, AIDS, and diabetes.
Although Massachusetts is one of the smallest US states, its size benefits hospitals, universities, and biotech companies by bringing them closer together. This leads to more collaboration which promotes innovation. Along with support from the government, this nucleated ecosystem has helped Massachusetts build one of the world’s top biotech hubs with gene therapy companies at the forefront.
Innovative Gene Therapy Companies
AGTC is a clinical-stage biotechnology company founded in 1999 by Richard Jude Samulski, Nicholas Muzyczka, William W. Hauswirth, Terence R. Flotte, and Barry J. Byrne. The company is located in Cambridge and currently has around 122 employees.
This company uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, other ophthalmology, CNS and otology indications. In addition to its product pipeline, AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.
Akouos is a precision genetic medicine company founded in 2017 by Manny Simons and Michael Mckenna. The company is located in Boston and currently has around 83 employees. Akouos is dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide.
They leverage adeno-associated viral (AAV) vector-based gene therapy in order to restore and preserve hearing in genetically-defined patient populations in a range of forms such as a single genetic mutation to ototoxic drug exposure. This enables the healthcare industry to treat sensorineural hearing loss in an efficient manner.
Ally Therapeutics
Ally Therapeutics is a relatively new biotech company founded in 2018 by Jessica Chiang and Tina Liu. The company is based in Cambridge and has around 10 employees. They’re developing a new technology that enables broader use of AAV gene therapies. The technology improves both safety and efficacy of gene therapy, specifically by reducing innate immune responses against the DNA.
Apic Bio is an innovative gene therapy company founded in 2017 by Christian Mueller and, John Reilly. The company is based in Cambridge and has around 40 employees.
Apic Bio is focused on developing first-in-class treatment options for rare, undertreated neurological and liver diseases and is advancing several novel gene therapies to treat Alpha-1 Antitrypsin Deficiency (Alpha-1), genetic Amyotrophic Lateral Sclerosis (ALS), and other CNS diseases.
bluebird bio Inc is a clinical-stage biotechnology company founded by Philippe Leboulch in 1993. The company is located in Cambridge and has around 1,300 employees.
bluebird bio develops gene therapies for rare and severe genetic diseases. Their s gene therapy procedures aim to genetically modify a patient's cells to fundamentally correct or address the genetic basis underlying a disease.
Entrada Therapeutics was founded by Dehua Pei, Leo Qian in 2016. The company is located in Boston and has around 80 employees.
Entrada Threapeutic’s mission is to treat devastating diseases through the intracellular delivery of biologics. Entrada’s technology enables the efficient intracellular delivery of proteins, peptides and nucleic acids, thus allowing for the development of programs across several intracellular target classes. The Company's novel approach addresses current challenges associated with both large and small molecule therapeutics and represents a fundamental advancement in the delivery of molecules into the cytosol.
Nephrogen Inc. is a medical device company founded by Demetri Maxim. The company is based in Boston and has around 10 employees.
Nephrogen develops gene therapy for kidney disease and harnesses recent advances in genome editing (CRISPR-Cas9) and nucleic acid delivery (AAV, nanoparticles) to develop outright cures for genetic kidney disease. They use different therapeutic approaches including ex vivo perfusion and re-transplantation, in vivo local delivery, and in vivo systemic delivery, enabling physicians to cure cystic kidney diseases, including and nephronophthisis.
Orchard Therapeutics is a leading global fully integrated commercial-stage company founded by Andrea Spezzi, Ben Auspitz. The company is based in Boston and has about 300 employees.
Orchard Therapeutics is dedicated to transforming the lives of patients with rare diseases through innovative gene therapies. Orchard’s portfolio of autologous ex vivo gene therapy programs has demonstrated sustained clinical benefits in over 150 patients across five disease areas. These programs include Strimvelis®, the first autologous ex vivo gene therapy approved by the EMA in 2016, 3 programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (Wiskott Aldrich syndrome) and ADA-SCID (adenosine deaminase severe combined immunodeficiency), 2 other clinical programs in X-CGD (X-linked chronic granulomatous disease) and beta-thalassemia, as well as an extensive preclinical pipeline
SalioGen Therapeutics is a new company founded by Joseph Higgins. It is located in Cambridge and has about 20 employees.
SalioGen Therapeutics has made advancements in curative genetic therapies using its Exact DNA Integration Technology (EDIT) platform, a mammal-derived genome engineering technology. It is focused on providing durable, safe, and affordable non-viral gene therapies to more patients with inherited diseases. Looking ahead, SalioGen will also explore the EDIT platform’s capabilities to streamline cell therapy, cell engineering, and biologics manufacturing to help an even broader population of patients in need.
Transgene is an integrated biopharmaceutical company founded by Pierre Chambon. It is located in Boston and has about 200 employees.
Transgene designs and develops cutting-edge immunotherapeutic products to treat cancers and chronic infectious diseases.
Lab Management Software for Modern Gene Therapy Companies: Scispot.io
Scispot has become the primary lab management software of choice for numerous drug discovery startups, including gene therapy companies.
Scispot creates a connected digital replica of these innovative bioscience companies. It centralizes their company-wide data, templatizes routine research and automates non-scientific tasks. It makes other lab software such as electronic lab notebooks (ELNs) and lab information management systems (LIMS) redundant.
Modern biotech companies use Scispot's operating platform to manage their research projects, inventory, samples, and partners all in one spot. Request a demo to learn how you can accelerate your gene therapy research using Scispot.
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