Category: Gene Therapy

Over the years, cell and gene therapies (CGT) and other advanced therapy medicinal products (ATMPs) have managed to capture the interest of both drug developers and healthcare investors. Their popularity can be attributed to various factors, such as minimal side effects, clinical efficacy and their personalized nature. However, there are several complexities associated with the

In order to manipulate the cells, genetic material / nucleic acid is artificially introduced into the cells by using specific carriers / vectors. This process is known as transfection. It does not use viral components for transfection and are aimed at circumventing the challenges, such as inflammation, immunogenicity and non-specific transduction, associated with viral vectors.

Given the potential of gene therapies in targeting the underlying cause of a disease at cellular level,  the demand for such therapies has increased considerably over the past few years. Currently, more than 285 gene therapies are being evaluated in different phases of clinical development. Further, various gene therapy developers have raised more than USD 10 billion

Recent advances in multi-omics approaches, including genomics, transcriptomics, proteomics, metabolomics, cytometry and imaging, in combination with bioinformatics and biostatistics, have been translated into several popular bioanalytical platforms, such as next-generation sequencing (NGS), single-cell analysis, flow cytometry and mass spectrometry. These platforms, although widely adopted, require tissue fixation and are therefore not capable of providing fundamental

With close to half-million infections and the economy showing signs of the 2008 financial crisis, COVID-19 has impacted several industries. Government institutes and industry players have announced various steps to deal with the ongoing situation. In this article, we will highlight the developments in the cell and gene therapy manufacturing industry amid the ongoing COVID-19

In a recent press release, bluebird bio, one of the leading gene therapy developers, announced that COVID-19 has delayed the EU launch and US filing of the company’s gene therapy candidate Zynteglo (LentiGlobin). The company now expects to dose the first commercial patient with Zynteglo in Germany in the second half of 2020. Earlier, the company had

Last week, during our weekly industry update sessions, our prime focus was to understand the recent innovations in the field of cell therapy manufacturing. We were also looking out for players that are driving these innovations. To start with, the first news that caught our attention was Novartis’ decision to sidestep Europe travel ban to

Roots Analysis has actively tracked the developments in the field of cell therapy and gene therapies. The team has focused on identifying the key challenges that are hampering the growth of these revolutionary therapies. To identify the challenges, the team has interviewed several stakeholders involved in cell therapy development and manufacturing. In addition, a recent

Have you had your genomics sequenced? If you asked this question a few years ago, people would have thought you were crazy. However, today, genetic tests are up for sale in supermarkets! Fifteen years ago, the first ever Human Genome Project was carried out, which took more than 13 years to complete. This (Human Genome

There appear to be no Monday blues for Catalent as the company announces the acquisition of MaSTherCell Global, a technology-focused cell and gene therapy contract development and manufacturing organization. Following its last year’s acquisition of Paragon Bioservices (a leading viral vector developer), Catalent’s recent acquisition has extended the company’s leadership in this already crowded space.

As we enter 2020, one drug class that is set to take-off is gene therapy. With three approvals (Zolgensma® (US), Zynteglo™ (Europe) and Beperminogene Perplasmid (Japan), 2019 was a break out year for gene therapy developers. The $16 billion-plus investment by various investors finally seems to be paying off as we enter 2020 with a

At the American Society of Hematology (ASH) annual meeting in Orlando, the team, led by the Abramson Cancer Center of the University of Pennsylvania, will discuss two multiple myeloma patients and one sarcoma patient who is participating in a trial, which is also supported by Tmunity Therapeutics (a company that recently raised $75 million in

The rising demand for genome editing services has resulted in the rise of several players with a wide range of capabilities. Currently, there are close to 80 players that claim to offer CRISPR based genome editing services. To get more insights about the market, check out the detailed report market report – Genome Editing Services.

Orchard Therapeutics, a commercial-stage biopharmaceutical company, announced initial results from a clinical trial (NCT03392987) with a cryopreserved formulation of OTL-200, gene therapy in development for the treatment of metachromatic leukodystrophy (MLD). Get the details of the results here. What is OTL-200: OTL-200 is autologous CD34+ cells transduced with a lentiviral vector containing human arylsulfatase A (ARSA) complementary deoxyribonucleic

Adverum Biotechnologies, a clinical-stage gene therapy company developing drugs to teated rare ocular diseases, announced that the first patient was dosed in the third cohort (n=9) of the ongoing OPTIC phase 1 clinical trial (NCT03748784) for ADVM-022 for the treatment of neovascular or wet age-related macular degeneration (wet AMD). What is ADVM-022: ADVM-022 utilizes a propriety

AVROBIO, a US-based company, developing lentiviral-based gene therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Company’s investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease. What is AVR-RD-02: AVR-RD-02 consists of the patient’s own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient

In a recent press announcement, The U.S. National Institutes of Health (NIH) has agreed to invest in the development of Gene Therapy in an attempt to cure HIV and sickle cell diseases. The program, for which NIH will partner with The Bill & Melinda Gates Foundation, will involve funding of $100 million each over the next

ReNeuron, the UK based, clinical-stage stem cell therapeutics company, announced that new data relating to its CTX stem cell platform will be presented today at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Dr. Steve Pells, Principal Investigator at ReNeuron, will present new data showing the phenotypic stability and scalability

Applied Genetic Technologies Corporation (AGTC), a clinical-stage biotechnology company, developing transformational genetic therapies for patients suffering from rare and debilitating diseases, announced new data from non-clinical studies evaluating the effect of pre-existing anti-AAV antibodies on the transduction and expression efficiency of AAV vectors. According to the company’s CSO, Preexisting immunity to AAV remains a challenge for

Rocket Pharmaceuticals, a New York-based, clinical-stage company, developing first-in-class gene therapies for rare and devastating inherited diseases announced clearance of Investigational New Drug (IND) by the FDA for RP-L301 gene therapy. RP-L301 is a lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a genetic rare disease. Last month, the company received

Homology Medicines, a company using proprietary gene editing and gene therapy platform announced the presentation of preclinical data supporting its investigational gene therapy programs for the treatment of metachromatic leukodystrophy (MLD) and phenylketonuria (PKU) at the American Society of Human Genetics (ASHG) 2019 Meeting. The presented preclinical data showed the results from studies conducted in

The gene therapy company, Exegenesis Bio has become one of the recent players to receive funding in the therapy space. According to a media statement, the company has completed fundraising of over 70 million yuan ($10 million), led by K2 Venture Partners and joined by Kaitai Capital and Legend Star. The company was founded by

In a recent news update, a team of Irish gene therapy researchers announced a breakthrough in the search for a cure for one of the worst strands of the incredibly painful skin disease, EB (epidermolysis bullosa). According to the researchers, the incredibly painful genetic condition, which affects approximately 300 people in Ireland, is caused by a

From being a thing of science fiction to topping the priority list of every big pharma player, gene therapies have come a long way. With multiple approved products, the field of gene therapies has gained substantial momentum over the last couple of decades. The growing burden of genetic disorders and the rising evidence of gene

Last week, I came across an interesting article by Andrew Dunn titled Sanofi investing in gene therapy as R&D focus turns toward rare disease. The article caught my attention because in June, this year, Sanofi scaled back its four-year-old gene therapy alliance with Voyager Therapeutics, for the second time. If we just look at the news titles,

Cell and Gene Therapy are the new entrants in the pharma Superstar Club. They are selling tickets faster than a Queen’s concert in the 1980s. In fact, as per the Alliance of Regenerative Medicine’s recent findings, there has been more than 75% year on year increment in funding to support the development of various cell and gene therapies.

Over 500 cell and gene therapy candidates are being investigated in different stages of clinical development across various geographies. In fact, in the past five years alone, more than 1,000 clinical trials, that are currently active, were initiated to evaluate the potential of these therapies across multiple therapeutic indications. Due to several development challenges, such

Over the last few decades, various Viral Vectors and non-viral vectors have been developed, optimized and standardized for introduction of therapeutic DNA / gene of interest into a patient’s body / cells. Currently, the most popular viral vectors, on the basis of their use in active clinical trials, are those based on AAV, adenovirus, lentivirus

The first gene therapy, Gendicine®, was only approved in 2003 in China; since then, the domain has evolved significantly. The ‘Gene Therapy Market (2nd Edition), 2018-2030’ report provides an extensive study on the current market landscape of gene therapies, with a prime focus on gene augmentation based therapies and oncolytic viral therapies, featuring an elaborate discussion on the

Scientists around the word are debating the scientific and ethical possibilities of using powerful gene editing tools, such as the CRISPR/Cas9 system, to treat heritable diseases in human embryos. In 1987, the discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) genes in single celled prokaryotic systems revolutionized genetic engineering. Initially thought to be