Month: October 2019

ADCs Pipeline Update: Sutro Biopharma Presents Encouraging Data in Patients with Advanced Ovarian Cancer

Sutro Biopharma, a California based, clinical-stage company, announced initial safety data in ovarian cancer patients from its ongoing phase I study (NCT03748186) of STRO-002. The drug is a folate receptor alpha (FolRα)-targeting antibody-drug conjugate (ADC) and potent anti-tumor activity in preclinical endometrial cancer patient-derived xenograft (PDX) models. The data are being presented at the AACR-NCI-EORTC Molecular Targets

ADCs Pipeline Update: Femtogenix Presents Novel Data on ADCs Containing Reduced Potency Payloads

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

Antibody Drug Conjugates Market Update: Ambrx and NovoCodex Collaborate

Ambrx and NovoCodex Biopharmaceuticals announced that they have formed a second collaboration to develop and commercialize Ambrx’s internally developed site-specific antibody drug conjugates (ADCs). What is the focus of the agreement: Under the agreement, Ambrx and NovoCodex will join forces to continue the development of ARX305, an Ambrx enabled ADC for the treatment of CD70

Cell Therapy Manufacturing Market Update: Compal and University to set up GTP lab

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) cellular laboratory in Kaohsiung City, southern Taiwan. What are the key capabilities of Raypal: Founded in 2016, Raypal specializes in R&D of immunocyte and immunotherapy, stem cell and

Cell Therapy Pipeline Update: Talaris Therapeutics Announces Initiation of Phase 3 Clinical Trial

Talaris Therapeutics, a late-clinical stage biotechnology company based in Boston, MA and Louisville, KY, announced the initiation of FREEDOM-1 (NCT03995901), the company’s Phase 3 clinical trial of FCR001 in living donor kidney transplant (LDKT) recipients. What is the objective of the trial: This pivotal trial will evaluate the safety and efficacy of a single dose of FCR001,

Gene Therapy Pipeline Update: Orchard Therapeutics Presents Data on Metachromatic Leukodystrophy

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

Cell Therapy Market Update: MaxCyte Advances Phase I Clinical Trial of Lead mRNA-based Cell Therapy

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

Genome Editing Market Update: Intellia Therapeutics Presents Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy

Intellia Therapeutics, one of the leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology is presenting one oral presentation and four poster presentations at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) meeting taking place October 22-25, 2019, in Barcelona, Spain. Get a complete

Gene Therapy Pipeline Update: Adverum Biotechnologies Doses First Patient for Wet AMD

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

Gene Therapy Pipeline Update: AVROBIO Receives Orphan-Drug Designation

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

Cell Therapy Manufacturing Update: United Therapeutics Receives Permit

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

Gene Therapy Funding Update: With a Promise of $100 Million in Funding, US Government Bets Big on Gene Therapy

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

Cell and Gene Therapy Technology Update: ReNeuron Presents Positive Data

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

Gene Therapy Pipeline Update: AGTC Presents Promising Data of its AAV Vectors for Ocular Gene Therapy

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

Gene Therapy Pipeline Update: Rocket Pharmaceuticals Receives IND Clearance for Gene Therapy for Pyruvate Kinase Deficiency

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

Gene Therapy Pipeline Update: Homology Medicines Presents Data Demonstrating Preclinical Proof-of-Concept

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

Alzheimer’s Pipeline Update: Biogen Takes a U-Turn on Failed Drug; Approval in Sight

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

Cell Therapy Manufacturing Update: Merck Becomes the First to Introduce Acoustic Technology

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 allows

Plasmid DNA Manufacturing Market Update: Cytovance®Introduces Integrated Single-Use Platform

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,

Gene Therapy Market Update: Researchers Deliver a Cure for Epidermolysis Bullosa (EB)

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

Technological Evolution of ADC Conjugation Platforms

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 of

Gene Therapy Market: Big Pharma Bets Big

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,

Super Generics Market: The Untapped Gold Mine

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 problems arrive: While the generics market is highly attractive, the increasing competition in this domain

Growing Cell and Gene Therapy Market: Vector Manufacturers Make Merry

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.

Synthetic Lethality Market: Riding on the ESMO 2019 Success of PARP Inhibitors

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