Month: October 2019

Femtogenix, a UK based biotechnology company developing the next generation of DNA-interactive Antibody Drug Conjugate (ADC) payloads, announced data demonstrating the potent efficacy and favorable toxicity profile of a reduced potency analog from its Pyridinobenzodiazepine (PDD) ADC payload platform in solid tumor models. The presented data demonstrated that Femtogenix’s reduced potency payload has a favorable

The Compal Group’s Raypal Biomedical and Kaohsiung Medical University Chung-Ho Memorial Hospital on October 22 signed an MoU to jointly set up a GTP (good tissue practice) cell therapy manufacturing laboratory in Kaohsiung City, southern Taiwan. What are the key capabilities of Raypal in cell therapy manufacturing: Founded in 2016, Raypal specializes in R&D of

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

MaxCyte, the global Cell Therapy, and life sciences company, announced the initiation of dosing in the third cohort of patients of MaxCyte’s Phase I clinical trial (NCT03608618) with the next higher cell dose of MCY-M11. The dose-escalation trial is evaluating the safety and tolerability, as well as preliminary efficacy, of MCY-M11 administered intraperitoneally across a series

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

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

As per recent news coverage, United Therapeutics has received a permit for $9.5 million build-out of its cell therapy facility on the second floor of Mayo Clinic’s Discovery and Innovation Building. What is the area of the facility:  The facility will have a total of 21,843-square-foot space. What are the capabilities at the facility:  The facility will house

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

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

In one of the most drastic shifts in fortune in the pharmaceutical industry, Biogen has decided to submit its previously failed Alzheimer’s drug for approval. According to the company, The Phase 3 EMERGE (NCT02484547) study met its primary endpoint showing a significant reduction in clinical decline, and Biogen believes that results from a subset of patients

With its acquisition of FloDesign Sonics earlier this month, Merck/MilliporeSigma has become the first company to introduce acoustic technology in cell therapy manufacturing. The acquisition of FloDesign Sonics is a strategic fit for Merck, strengthening its ability alongside to manufacture cell-based therapies for patients. What is Acoustic Cell Processing:  Acoustic cell processing is a disruptive technology that

In the ongoing shift of biologics manufacturers to single-use systems, Cytovance®, a leading biopharmaceutical CDMO, announced the addition of an integrated single-use platform for the manufacturing of plasmid DNA (pDNA) at their facilities in Oklahoma City, OK. According to the company, two grades are available (Critical Reagent Grade™, CGMP-grade) in lot sizes from 1g to 50g,

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 wrote an article on the key innovators in ADC therapeutics space, Technological highlighting the key winners at the 6th World ADC Awards. One of the winners, that has really impressed several drug developers in this space, is Zymeworks. The company has its proprietary conjugation Technological platform, ZymeLink™, which offers site-specificity and customization

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,

Last week, the eyes of all the key stakeholders in the Antibody Drug Conjugates (ADCs) Therapeutics space were on the developments happening in San Diego, California, at the 6th World ADC Awards.  The awards were meant to recognize the companies that are driving innovation in the field of ADCs Therapeutics. With six approved and marketed

Global Antibody Drug Conjugates ADC market is expanding and is estimated to be around USD 7.72 billion in 2023. The compound annual growth rate of ADC market is estimated to be 9.50% in the coming decade. Roots Analysis has been publishing multiple articles and key insights pack for ADC market growing potential to keep the

Gene therapy companies have emerged as the savior in offering sophisticated products to treat patients with defective genes and chronic diseases. There are two types of gene therapies on the basis of targeted gene location: germline gene therapy and somatic gene therapy. These therapies are implemented to treat several diseases, such as hemophilia, Parkinson’s, Alzheimer’s,

T-Cell immunotherapies have gained a lot of traction in recent years. With a plethora of clinical evidence and billions of dollars in investment, calling T-cell therapies as the next big thing might not be incorrect. As per the recent estimates, this space is likely to grow to USD 26 billion by 2030, at a CAGR

Novel Cancer is known to be one of the leading causes of death worldwide, accounting for more than 600,000 deaths in 2018 in the US alone. As per the recent reports by, the World Health Organization (WHO), the number of new cancer cases globally is expected to rise by 70% in the coming 20 years. Despite

The Super Generics industry has enjoyed a remarkable run over the past two decades. The vast healthcare cost-saving potential of these therapies has accelerated their adoption and proven the immense potential that generics have to reach wider patient segments. The super generic drugs market is estimated to be worth $38 billion in 2019 and is

After the immense success of PARP inhibitors at ESMO 2019, synthetic lethality has become the talk of the town. From industry analysts to healthcare researchers, everyone is now interested in this emerging space. In this article, we will look at the financial backing that this domain has received over the last few years and also

Cell and Gene Therapy CROs 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

After a strong showdown at ESMO 2019, both GSK and AstraZeneca have reignited the fight of small molecule anti-cancer drugs against the industry’s brawny immunotherapy push. Leading this fight is poly(ADP-ribose) polymerase (PARP) inhibitor, one of the leading synthetic lethality targets. These better than expected results at ESMO further validate the potential of synthetic lethality