FDA Law Blog

MARCH 13, 2023

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials.

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

Gene 262

Gene Gene Therapy Genetics DNA 262

pharmaphorum

JANUARY 24, 2023

Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates. The logical extension to this kind of approach is treating individual patients, with their individual genetic makeup.

Genetics 98

Genetics Drugs Genome Project Biobanking 98

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. The company has developed various assets based on lentivirus vectors.

Gene Therapy 246

Gene Therapy DNA Gene Genetics 246

pharmaphorum

NOVEMBER 30, 2020

Cure Rare Disease’s Rich Horgan discusses the importance of preclinical NAbs screening and helping rare disease patients better understand their eligibility for gene therapies. Neutralising antibodies (NAbs) present a unique challenge to researchers looking to treat patients with AAV treatment.

Gene Therapy 105

Gene Therapy Gene Antibody Clinical Trials 105

Worldwide Clinical Trials

MAY 30, 2024

This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

XTalks

APRIL 1, 2025

Qfitlia is also the first antithrombin-lowering therapy approved for routine prophylaxis in hemophilia A or B. Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. Among these are three gene therapies.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Delveinsight

DECEMBER 3, 2020

Roche receives FDA authorization for the quantitative COVID-19 antibody test. Roche has got an authorization from the FDA for a more accurate COVID-19 blood test capable of measuring the levels of specific antibodies, which target the cell-unlocking spike protein of coronavirus. Janssen buys a gene therapy asset.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

XTalks

JANUARY 21, 2025

The liquid portion of the blood (serum or plasma) containing antibodies is separated and tested using the Liason Biotrin parvovirus B19 IgG plus test on a Liason XL analyzer. If parvovirus B19 antibodies are present, they bind to the chemicals and particular particles in the test, producing a light signal.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

Delveinsight

FEBRUARY 18, 2021

Bluebird suspends gene therapy trials after two sickle cell patients develop cancer. Bluebird bio has stopped two clinical trials of its sickle cell disease gene therapy after participants developed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Cortexyme tripped up by FDA hold over liver toxicity.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Delveinsight

DECEMBER 22, 2020

FDA Puts uniQure’s Hemophilia B Gene Therapy on Leash. In a recent, uniQure faced backlash after its hemophilia B gene therapy got hit with clinical hold by the USFDA. . The Hemophilia B market has several therapies, however, no therapy has been able to kill the disease from its root.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

Delveinsight

JANUARY 12, 2021

It is an IgM antibody targeting Death Receptor 5 (DR5), and IGM is hoping to test Birinapant with IGM-8444 for the treatment of solid cancers later this year. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences. million after Birinapant successfully becomes a part of the Phase I trials.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

Delveinsight

FEBRUARY 11, 2021

Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Avrobio gene therapy eradicates toxic substrate in Fabry patient. Enhancing the response to immuno-oncology in pancreatic cancer. Median overall survival in those patients was 5.8

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

pharmaphorum

AUGUST 8, 2022

As we reported this morning, the deal gives Pfizer already-approved SCD therapy Oxbryta (voxelator) – which industry watchers reckon could see a dramatic uptick in sales with Pfizer’s marketing muscle – plus a phase 3 antibody candidate, a phase 1 follow-up to Oxbryta that could offer improved dosing.

Marketing 111

Marketing Sales Gene Editing Antibody 111

Delveinsight

DECEMBER 10, 2020

Mice that were genetically modified to be in a deficit of the protein were secured from Type 1 diabetes, and a small peptide inhibitor of OCA-B also prevented the disease in newly diabetic animals, as per the results published in the Journal of Experimental Medicine. UniQure/CSL haemophilia B gene therapy curbs bleeding in phase 3.

Gene Therapy 52

Gene Therapy Gene Vaccination Vaccine 52

Delveinsight

JULY 28, 2020

AstraZeneca has inked an oncology deal with Daiichi Sankyo to jointly develop and commercialize an antibody-drug conjugate DS-1062 (trastuzumab deruxtecan), worldwide except in Japan. No relief to Solid Biosciences as FDA demands more data to lift the hold on the company’s DMD gene therapy trial.

Gene Therapy 52

Gene Therapy Trials Gene Vaccination 52

XTalks

APRIL 12, 2021

Like other neurodegenerative disorders, such as Alzheimer’s disease and dementias, Parkinson’s disease research has been immensely challenging for biotechs, with a high rate of late-stage attrition in trials leading to a lack of disease-modifying therapies being approved. Axovant Gene Therapies. Prevail Therapeutics.

Gene Therapy 96

Gene Therapy Gene Trials Development 96

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. As per the agreement, Alexion will initiate a cash tender offer through a subsidiary to acquire all outstanding shares of LogicBio for $2.07

Gene Editing 64

Gene Editing Gene Genomics Genome 64

pharmaphorum

AUGUST 22, 2022

” For example, Torrance recently met with an expert from the Broad Institute who has been looking into gene therapies and says gene therapy can completely change how drugs are built. The Fund has owned Genmab , a Danish biotechnology company that creates antibody therapies for cancer, for 12 years.

Gene Therapy 102

Gene Therapy Antibody Gene Medicine 102

Pharma Packaging Solutions

JUNE 30, 2023

Whole blood, organ and tissue transplants, breast milk, antibodies for passive immunity, fecal microbiota, human reproductive cells, and stem-cell therapy are all examples of this type of biologic. As biopharma technology progresses, becoming both more prevalent and available, the need for high-quality biopharma packaging increases.

Recombinant DNA Technology 52

Recombinant DNA Technology Packaging Packaging Gene Therapy 52

The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Cloudbyz

MARCH 6, 2024

This allows for the creation of highly specific and targeted therapies that can modulate biological pathways with remarkable precision. Personalized Medicine: Biologics offer the potential for personalized treatment approaches by taking into account individual variations in genetics, immune responses, and disease characteristics.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

XTalks

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease. In November 2023, Hookipa Pharma Inc.,

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

pharmaphorum

FEBRUARY 17, 2022

The drug – which costs $125,000 at US list prices – can be used either alone or in combination with hydroxyurea, a well-established therapy for the disease. The confounder for both GBT and Novartis’ hopes for their drug could be genetic therapies for SCD, which offer a one-shot treatment for the disease.

Drugs 59

Drugs Gene Therapy Gene Editing Gene 59

Scienmag

MAY 17, 2021

Patients’ genetic profile strongly influences size of effect in early-phase trial People with extremely high levels of triglycerides (a type of fat in the blood) and a specific genetic profile saw a substantial reduction in triglycerides after taking the human monoclonal antibody evinacumab compared with those taking a placebo, in a study presented (..)

Genetics 42

Genetics Antibody Trials Gene Therapy 42

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Hemlibra is a bispecific factor IXa- and factor X-directed antibody.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Delveinsight

AUGUST 5, 2021

Sanofi is betting the genetic technology behind the fast development of two highly effective coronavirus shots last year will result in vaccines for other viruses as well as drugs for diseases of the lung and liver, announcing a deal to acquire a research partner Translate Bio for USD 3.2 Sanofi bets USD 3.2

Vaccination 98

Vaccination Vaccine Gene Therapy Development 98

Delveinsight

DECEMBER 8, 2020

As its lead program, KaliVir is developing VET2-L2 leveraging its unique technology platform based on a genetically modified vaccinia virus. Canadian antibody-drug discovery platform AbCellera Biologics is setting the groundwork for a massive Initial public offering worth USD 391 million. million from the Canadian government. .

Licensing 52

Licensing Licensing Gene Therapy Antibody 52

pharmaphorum

JUNE 7, 2022

Alectos’ drug helps to maintain the acid pH level in lysosomes required for their function in the hope of restoring their activity and – hypothetically at least – slow the progression of Parkinson’s in patients with and without known genetic risks. Along with the upfront payment, Biogen has pledged up to $77.5

Licensing 80

Licensing Licensing Gene Therapy Genetics 80

Roots Analysis

MAY 21, 2024

LNPs comprise of a lipid bilayer that surrounds a hydrophobic core, which can be loaded with therapeutic agents ( such as drugs, genetic material and proteins ). Several LNP-based therapies are currently being evaluated in the clinical trials; majority of these are conducted for treating breast cancer, ovarian cancer and lung cancer.

Drug Delivery 40

Drug Delivery Drugs Gene Therapy Bioavailability 40

Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. Adoptive T Cell Therapies Adoptive T cell therapy is another form of cancer treatment leveraging the patient’s immune system.

Vaccination 52

Vaccination Vaccine Protein Antibody 52

Delveinsight

AUGUST 20, 2020

FDA rejects BioMarin’s gene therapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec. The team successfully protected HILOs from the immune system without genetic manipulation. J&J has agreed to pay USD 52.50

Gene Therapy 52

Gene Therapy Research Gene Antibody 52

Delveinsight

SEPTEMBER 23, 2021

The biotech has evolved a T-cell receptor-like antibody to damage a selected immune checkpoint in oncology. Novartis is expanding its optogenetics portfolio with the acquisition of gene therapy company Arctos Medical. Mutations can cause IRDs in more than 100 genes.

RNA 61

RNA Gene Therapy Gene Protein 61

The Pharma Data

JANUARY 7, 2021

To meet that goal, Sanofi is investing in areas like synthetic biology and cell and gene therapy. He also noted innovations are occurring with protein degraders, antibody conjugates, nanobodies and multi-specific antibodies, specifically bi- and tri-specific. billion acquisition of Synthorx.

Protein 52

Protein Antibody Genetics Gene Therapy 52

The Pharma Data

DECEMBER 27, 2020

Vir Biotechnology and GlaxoSmithKline dosed the first patient in a new sub-trial of a Phase III study of monoclonal antibody VIR-7831 for hospitalized adults with COVID-19. The drug is a humanized IgG4 monoclonal antibody that blocks CCR5 and is being developed for COVID-19, HIV and metastatic triple-negative breast cancer.

Trials 52

Trials Antibody Gene Therapy Gene 52

pharmaphorum

OCTOBER 12, 2022

Now, he leads Whitelab Genomics as its artificial intelligence (AI) platform powers the development of genomic therapies – an emerging field in which genetic sequences are injected into cells to target and repair damaged genes. and France to accelerate the development of genomic therapies.

Genomics 105

Genomics Genome Medicine Development 105