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

pharmaphorum

SEPTEMBER 17, 2020

There are two antibodies already on the market that target PCSK9 – Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but these require subcutaneous injection every two to four weeks.

Drugs 62

Drugs Antibody Sales Branding 62

The Pharma Data

NOVEMBER 29, 2020

Naxitamab is a monoclonal antibody that targets GD2, which is found on neuroblastoma and other related tumors. The antibody is designed to destroy GD2-expressing cancer cells by antibody-dependent cell-mediated cytotoxicity (ADCC) and complement dependent cytotoxicity (CDC). The FDA approved the drug on November 24.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

XTalks

SEPTEMBER 22, 2022

GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it. The novel, selective monoclonal antibody spesolimab behind Spevigo reduces flares in individuals by inhibiting interleukin-36 (IL-36) signalling.

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Dermatology 52

Drug Discovery World

MAY 11, 2023

Last, but certainly not least, Samsara Therapeutics is a biopharma company aiming to discovery first-in-class therapeutics that treat rare genetic and neurodegenerative disease, and extend healthy lifespan.

Life Science 52

Life Science Genetics Vaccination Vaccine 52

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases. Shape focuses on unlocking the next breakthrough in RNA technologies in the gene therapy space across many therapeutic areas, as informed by Francois Vigneault, co-founder, and CEO.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

Delveinsight

AUGUST 17, 2021

The vaccine induces neutralizing antibodies as a response against the natural TBE virus, as the sequence and structure of the virus subtype match those found in nature. “We Food and Drug Administration, which could have entered the ultra-rare genetic disease market. The rarity of disease – 1.36

FDA Approval 98

FDA Approval Medicine Vaccination Vaccine 98

The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. The impact of CNS diseases extends beyond patients—to their families and society as well.” The promise to partners is to “move quickly.

RNA 52

RNA Antibody Life Science Protein 52

Delveinsight

FEBRUARY 11, 2021

Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Enhancing the response to immuno-oncology in pancreatic cancer. However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

NOVEMBER 8, 2020

The results from the CANDOR study confirm the potential for Kyprolis to be used in combination with an anti-CD38 monoclonal antibody.”. Progeria, also called Hutchinson-Gilford Progeria Syndrome (HGPS), is an ultra-rare and fatal genetic disease of accelerated aging in children.

Trials 52

Trials Protein Gene Genetic Disease 52

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105

RNA Genetics Genetic Disease Medicine 105

XTalks

SEPTEMBER 17, 2020

Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males. Patients with this disease have a very high level of LDL-C, which increases their risk of coronary artery disease, including heart attack, stroke and atherosclerosis.

Trials 98

Trials Gene Hormones Clinical Trials 98

XTalks

AUGUST 16, 2021

Immune systems can be weakened in individuals with HIV/AIDS; cancer and transplant patients taking certain immunosuppressive drugs; and those with genetic diseases affecting the immune system like congenital agammaglobulinemia and congenital IgA deficiency. According to the CDC, approximately 2.7

Vaccination 104

Vaccination Vaccine Antibody Immune Response 104

The Pharma Data

JULY 6, 2021

Hemlibra is a bispecific factor IXa- and factor X-directed antibody. 12 Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII, 13 making it difficult, if not impossible, to obtain a level of factor VIII sufficient to control bleeding.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 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 Clinical Trials 52

The Pharma Data

JANUARY 9, 2021

The Company is running a Phase 1/2 Study with the 6e13kg/vg dose of valoctocogene roxaparvovec in approximately 10 participants with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors. About Hemophilia A.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

The Pharma Data

NOVEMBER 25, 2020

The FDA recently approved two antibody treatments for the virus, Eli Lilly ’s bamlanivimab and Regeneron ’s REGN-COV2 , which had previously been used to treat the COVID-19 diagnosis of President Donald Trump. Both of the antibody treatments do have limits for their use.

Vaccination 52

Vaccination Vaccine Genetic Disease Genetics 52

Pharmaceutical Technology

APRIL 24, 2023

In an email to this news service, João Conde, PhD, professor at NOVA Medical School, Universidade NOVA de Lisboa, said advances like the mRNA vaccines serve as a testament to the breakthroughs made by science over decades of research at the junction of genetics and nanomedicine. This design protects antibodies from the innate immune system.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147