Drugs, FDA Approval, Gene Therapy and Genetic Disease

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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

SEPTEMBER 30, 2022

After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.

Gene Therapy

Gene Therapy FDA Approval Gene Genetic Disease

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease.

Gene Therapy

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Trending Sources

US FDA approves Krystal Biotech’s Vyjuvek for DEB

Pharmaceutical Technology

MAY 22, 2023

The US Food and Drug Administration (FDA) has granted approval for Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB) in patients aged six months and above. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

FDA Approval

FDA Approval Gene Therapy Genetic Disease Gene

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

Gene Therapy

Gene Therapy Clinical Trials Clinical Trials Gene

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB).

Gene Therapy

Gene Therapy Gene FDA Approval Dermatology

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval

FDA Approval Gene Therapy Gene Clinical Trials

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. David Bumcrot PhD, CSO, CAMP4 Therapeutics.

RNA

RNA Gene Expression Gene Gene Therapy

Amicus’ Pombiliti and Opfolda Is a New Therapy for Pompe Disease

XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

FDA Approval

FDA Approval Clinical Trials Clinical Trials Trials

Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million

XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

Gene Therapy

Gene Therapy Gene Drugs FDA Approval

4 Life Sciences Trends for 2023

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies.

Life Science

Life Science Gene Therapy Gene Manufacturing

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year.

Gene Editing

Gene Editing Gene In-Vivo Clinical Trials

First gene therapy for children with rare disease gets US sign-off

Drug Discovery World

MARCH 19, 2024

Lenmeldy (atidarsagene autotemcel) has become the first Food and Drug Administration (FDA)-approved gene therapy for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

Gene Therapy

Gene Therapy Gene FDA Approval Genetic Disease

Advances in neuroscience drug discovery

Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research.

Drugs

Drugs Gene Clinical Trials Clinical Trials

Covid-19 vaccine success bolsters nanoparticle drug delivery research

Pharmaceutical Technology

APRIL 24, 2023

Researchers may develop nanodrugs due to their smaller surface area to allows drugs to dissolve faster, or nanoparticles may encapsulate drugs, so they last longer in the body. Covid-19 vaccines from Moderna and Pfizer /BioNTech, which use lipid nanoparticles, became the only two FDA-approved vaccines for almost all ages.

Drug Delivery

Drug Delivery Vaccination Vaccine Research

Clinical Research Informer

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

Trending Sources

US FDA approves Krystal Biotech’s Vyjuvek for DEB

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Amicus’ Pombiliti and Opfolda Is a New Therapy for Pompe Disease

Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million

4 Life Sciences Trends for 2023

Vertex, CRISPR prep filing for gene-editing blood disorder therapy

First gene therapy for children with rare disease gets US sign-off

Advances in neuroscience drug discovery

Covid-19 vaccine success bolsters nanoparticle drug delivery research

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