Drugs and Gene - Clinical Research Informer

Bispecific cancer drugs and gene therapy advances: What to watch at next month’s ASH meeting

Bio Pharma Dive

NOVEMBER 4, 2022

Competition is increasing behind newly approved drugs from Roche and J&J, while the long-term benefit of gene therapies for chronic blood disorders is being put to the test.

Gene Therapy

Gene Therapy Gene Drugs

FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

Bio Pharma Dive

SEPTEMBER 7, 2021

On the heels of a two-day FDA meeting in which experts debated the risks of gene therapy, the biotech said its early-stage PKU treatment was associated with cancer in mice.

Gene Therapy

Gene Therapy Gene Drugs

Vertex, Verve team up to develop a gene editing drug for liver disease

Bio Pharma Dive

JULY 20, 2022

The deal, which is worth $60 million upfront, broadens Vertex’s reach into gene editing and expands Verve’s research beyond heart disease.

Gene Editing

Gene Editing Gene Development Drugs

Webinars

Bridging Innovation & Patient Care: The Growing Role of AI

MORE WEBINARS

Pfizer hemophilia gene therapy arrives in US to uncertain future

Bio Pharma Dive

APRIL 26, 2024

The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition that were approved earlier struggle to gain traction.

Gene Therapy

Gene Therapy Gene Drugs

Kebilidi FDA-Approved as First Brain-Delivered Gene Therapy for AADC Deficiency

XTalks

NOVEMBER 14, 2024

PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA requires a confirmatory study to validate these findings, which is ongoing.

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

Moderna, teaming with a startup, wades further into gene editing

Bio Pharma Dive

NOVEMBER 2, 2021

The biotech has identified gene editing as a natural fit for the technology it uses to deliver its vaccines and drugs. A deal with Metagenomi, a startup backed by Bayer, will further those ambitions.

Gene Editing

Gene Editing Gene Vaccine Vaccination

FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug

Bio Pharma Dive

MARCH 2, 2023

The announcement, which follows recent regulatory setbacks for some of Intellia’s peers, is a “big win” for the gene editing field, according to one analyst.

Gene Editing

Gene Editing In-Vivo Gene Drugs

In first, FDA approves KRAS-blocking cancer drug from Amgen

Bio Pharma Dive

MAY 28, 2021

For decades, scientists have tried unsuccessfully to target the KRAS gene, which is often mutated in lung, colon and pancreatic cancers. Lumakras is the first drug proven effective.

FDA Approval

FDA Approval Drugs Gene Scientist

Lilly agrees to acquire gene therapy developer Akouos for $610m

Pharmaceutical Technology

OCTOBER 19, 2022

Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss. It has combined expertise in otology, gene therapy and inner ear drug delivery to meet the requirements of individuals with disabling hearing loss globally.

Gene Therapy

Gene Therapy Gene Development Genetics

Gene Therapy and Pharmacokinetics

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy

Gene Therapy Gene Gene Editing Genetics

Boehringer Ingelheim takes next step in development of cystic fibrosis gene therapy

Bio Pharma Dive

OCTOBER 19, 2021

Drugs from Vertex can effectively treat the lung disease in as much as 90% of patients. Gene therapy could be a solution for the remaining 10%, but the path forward is challenging.

Gene Therapy

Gene Therapy Gene Development Drugs

Pfizer receives FDA approval for haemophilia B gene therapy

Pharmaceutical Technology

APRIL 29, 2024

Pfizer has received approval from the US Food and Drug Administration (FDA) for its one-time gene therapy BEQVEZ for haemophilia B.

Gene Therapy

Gene Therapy Gene FDA Approval Drugs

FDA grants accelerated approval for PTC’s AADC deficiency gene therapy

Pharmaceutical Technology

NOVEMBER 14, 2024

The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain.

Gene Therapy

Gene Therapy Gene Drugs

Forge Biologics and Labcorp partner for gene therapies development

Pharmaceutical Technology

MAY 4, 2023

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.

Gene Therapy

Gene Therapy Gene Development Manufacturing

AskBio’s AB-1002 gene therapy receives FDA fast-track status

Pharmaceutical Technology

APRIL 19, 2024

The US Food and Drug Administration (FDA) has granted fast-track designation to Bayer subsidiary Asklepios BioPharmaceutical’s (AskBio) investigational gene therapy AB-1002, aimed at treating congestive heart failure (CHF).

Gene Therapy

Gene Therapy Gene Drugs

Sarepta scraps a Duchenne drug as gene therapy sales rise

Bio Pharma Dive

NOVEMBER 7, 2024

Now, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment landscape. Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use.

Sales

Sales Gene Therapy Gene Drugs

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

Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy

Gene Therapy Gene Development Genetics

uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

Gene Therapy

Gene Therapy Licensing Licensing Gene

Biogen, Denali begin late-stage testing of Parkinson’s drug

Bio Pharma Dive

OCTOBER 3, 2022

Part of a $1 billion alliance the companies formed in 2020, the drug will be tested in a large study of patients who test positive for mutations in a gene called LRRK2.

Drugs

Drugs Gene

Roche writes off $3B on lower sales forecasts for gene therapies, cancer drugs

Bio Pharma Dive

FEBRUARY 3, 2023

Among the drugs Roche recorded impairment charges on are four gene therapies acquired in the company's 2019 deal for Spark Therapeutics.

Gene Therapy

Gene Therapy Gene Sales Drugs

Sanofi's hemophilia drug comes into focus as delays hit a key competitor

Bio Pharma Dive

SEPTEMBER 10, 2020

With BioMarin's gene therapy sidelined, attention is turning to other drugs, including one Sanofi acquired when it paid $11.6 billion for Bioverativ.

Gene Therapy

Gene Therapy Drugs Gene

Ovid turns to gene therapy startup to restock drug pipeline

Bio Pharma Dive

AUGUST 24, 2022

The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.

Gene Therapy

Gene Therapy Bacteria Gene Drugs

FDA grants approval for CSL’s haemophilia B gene therapy

Pharmaceutical Technology

NOVEMBER 23, 2022

The US Food and Drug Administration (FDA) has granted approval for CSL Behring’s adeno-associated virus vector-based gene therapy, Hemgenix (etranacogene dezaparvovec), to treat haemophilia B (congenital Factor IX deficiency) in adult patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy

Gene Therapy Gene Clinical Trials Clinical Trials

Avrobio to sell cystinosis gene therapy to Novartis for $87.5m

Pharmaceutical Technology

MAY 23, 2023

Avrobio has announced a deal to sell its investigational haematopoietic stem cell (HSC) gene therapy programme , designed to treat cystinosis, to Novartis in an all-cash deal valued at $87.5m. In 2022, Avrobio’s investigational gene therapy, AVR-RD-04, received a rare paediatric disease designation from the US Food and Drug Administration.

Gene Therapy

Gene Therapy Gene Development Drugs

FDA suggests ways to curb constraints with rare disease gene therapy trials

Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

EC grants approval for BioMarin’s gene therapy to treat Hemophilia A

Pharmaceutical Technology

AUGUST 25, 2022

The European Commission (EC) has granted conditional marketing authorisation (CMA) for BioMarin Pharmaceutical ’s gene therapy, Roctavian (valoctocogene roxaparvovec), to treat adults with severe haemophilia A (congenital Factor VIII deficiency). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy

Gene Therapy Gene Clinical Trials Clinical Trials

Royalty Pharma buys into Ferring’s cancer treatment in first gene therapy deal

Bio Pharma Dive

AUGUST 24, 2023

The deal, worth up to $500 million, is a bet by the drug rights dealmaker on the uncertain commercial prospects of Ferring's gene therapy Adstiladrin.

Gene Therapy

Gene Therapy Gene Drugs

Avidity drug for muscular dystrophy shows promise; Syntis takes new approach to weight loss

Bio Pharma Dive

JUNE 12, 2024

Study results indicated Avidity’s medicine muted target genes. Elsewhere, Syntis revealed obesity drug plans and Nodthera chalked up more positive data for its NLRP3 inhibitor.

Drugs

Drugs Gene Medicine

Cell and gene therapies: why advanced medicines call for specialised logistics

Pharmaceutical Technology

JULY 7, 2022

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research. Securing the supply chain.

Gene Therapy

Gene Therapy Gene Medicine Genetics

Lilly gene-targeted drug shows potential for use across cancers

Bio Pharma Dive

APRIL 11, 2021

Study results presented at AACR showed Retevmo shrank tumors in nine different cancers, building a case for the type of tissue-agnostic label that only three other medicines have secured.

Gene

Gene Medicine Drugs

FDA sets decision dates for Vertex, CRISPR gene editing drug

Bio Pharma Dive

JUNE 9, 2023

The regulator will issue separate verdicts on the pioneering treatment’s use in sickle cell disease and beta thalassemia, with the first expected late this year.

Gene Editing

Gene Editing Gene Regulation Drugs

Krystal Biotech receives FDA approval for DEB topical gene therapy

Pharmaceutical Technology

MAY 26, 2023

Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy

Gene Therapy Gene FDA Approval Manufacturing

8 key clinical trials to watch for the rest of 2021

Bio Pharma Dive

JUNE 21, 2021

The next six months could feature clinical milestones for CRISPR gene editing, the treatment of COVID-19, microbiome drugs and gene-targeted cancer therapy.

Gene Editing

Gene Editing Clinical Trials Clinical Trials Gene

Magazine: CRISPR gene therapies cut through in 2023

Pharmaceutical Technology

FEBRUARY 2, 2023

If things go as per plan, in a few months, the US Food and Drug Administration (FDA) will deliberate on the first-of-its-kind CRISPR-based gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The post Magazine: CRISPR gene therapies cut through in 2023 appeared first on Pharmaceutical Technology.

Gene Therapy

Gene Therapy Gene Pharma Companies Clinical Trials

Roche licenses Sangamo’s technology for another shot at Alzheimer’s drugs

Bio Pharma Dive

AUGUST 6, 2024

Through a new deal, Roche has exclusive rights to Sangamo molecules designed to repress the gene that makes “tau,” a protein many scientists view as a main driver of Alzheimer’s.

Licensing

Licensing Licensing Protein Gene

Drugmakers flock to Vineti's fix for a cell, gene therapy problem

Bio Pharma Dive

MARCH 24, 2021

A group of drug companies, including Novartis, Takeda and Roche's Genentech, have agreed to collaborate with Vineti to usher in new identification standards for complex medicines.

Gene Therapy

Gene Therapy Gene Medicine Drugs

With $2.8M gene therapy, Bluebird sets new bar for US drug pricing

Bio Pharma Dive

AUGUST 18, 2022

Its market launch is likely to be watched carefully by other gene therapy developers. Approved Wednesday for severe beta thalassemia, Zynteglo will test insurers’ willingness to pay for expensive one-time treatments.

Gene Therapy

Gene Therapy Gene Drugs Marketing

European regulators back hemophilia gene therapy

Bio Pharma Dive

DECEMBER 16, 2022

The EMA’s drug review committee recommended five new drugs be approved in Europe during its December meeting, including CSL’s Hemgenix, AstraZeneca’s Imjudo and Amicus’ Pombiliti.

Gene Therapy

Gene Therapy Regulation Gene Drugs

Takeda wins FDA approval for targeted lung cancer drug

Bio Pharma Dive

SEPTEMBER 16, 2021

Exkivity follows Johnson & Johnson's Rybrevant on the market for treating lung tumors harboring specific mutations in the EGFR gene. Unlike J&J's drug, however, Takeda's is given orally.

FDA Approval

FDA Approval Drugs Gene Marketing

Vertex, CRISPR strengthen case for pioneering gene-editing treatment

Bio Pharma Dive

JUNE 11, 2022

Positive data from 75 patients with either sickle cell disease or beta thalassemia keep the companies’ therapy on track to become the first CRISPR-based treatment submitted to drug regulators, possibly by the end of the year.

Gene Editing

Gene Editing Gene Regulation Drugs

Vertex, CRISPR finish US filing for gene editing drug approval

Bio Pharma Dive

APRIL 3, 2023

The approval application is the first in the U.S. for a CRISPR-based medicine and puts the partners ahead of a rival therapy from Bluebird bio.

Gene Editing

Gene Editing Gene Medicine Drugs

Regeneron, UPenn to use gene therapy tools to deliver a COVID-19 antibody drug

Bio Pharma Dive

DECEMBER 1, 2020

Working with University of Pennsylvania researcher Jim Wilson, Regeneron aims to develop a nasal spray version of the coronavirus treatment recently cleared by the Food and Drug Administration.

Gene Therapy

Gene Therapy Antibody Gene Drugs

CSL doses first patient with haemophilia B gene therapy Hemgenix

Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

Gene Therapy

Gene Therapy Gene Protein Sales

Bispecific cancer drugs and gene therapy advances: What to watch at next month’s ASH meeting

FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

Webinars

Trending Sources

Vertex, Verve team up to develop a gene editing drug for liver disease

Webinars

Pfizer hemophilia gene therapy arrives in US to uncertain future

Kebilidi FDA-Approved as First Brain-Delivered Gene Therapy for AADC Deficiency

Moderna, teaming with a startup, wades further into gene editing

FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug

In first, FDA approves KRAS-blocking cancer drug from Amgen

Lilly agrees to acquire gene therapy developer Akouos for $610m

Gene Therapy and Pharmacokinetics

Boehringer Ingelheim takes next step in development of cystic fibrosis gene therapy

Pfizer receives FDA approval for haemophilia B gene therapy

FDA grants accelerated approval for PTC’s AADC deficiency gene therapy

Forge Biologics and Labcorp partner for gene therapies development

AskBio’s AB-1002 gene therapy receives FDA fast-track status

Sarepta scraps a Duchenne drug as gene therapy sales rise

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

Astellas to support development of Taysha’s gene therapy programmes

uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS

Biogen, Denali begin late-stage testing of Parkinson’s drug

Roche writes off $3B on lower sales forecasts for gene therapies, cancer drugs

Sanofi's hemophilia drug comes into focus as delays hit a key competitor

Ovid turns to gene therapy startup to restock drug pipeline

FDA grants approval for CSL’s haemophilia B gene therapy

Avrobio to sell cystinosis gene therapy to Novartis for $87.5m

FDA suggests ways to curb constraints with rare disease gene therapy trials

EC grants approval for BioMarin’s gene therapy to treat Hemophilia A

Royalty Pharma buys into Ferring’s cancer treatment in first gene therapy deal

Avidity drug for muscular dystrophy shows promise; Syntis takes new approach to weight loss

Cell and gene therapies: why advanced medicines call for specialised logistics

Lilly gene-targeted drug shows potential for use across cancers

FDA sets decision dates for Vertex, CRISPR gene editing drug

Krystal Biotech receives FDA approval for DEB topical gene therapy

8 key clinical trials to watch for the rest of 2021

Magazine: CRISPR gene therapies cut through in 2023

Roche licenses Sangamo’s technology for another shot at Alzheimer’s drugs

Drugmakers flock to Vineti's fix for a cell, gene therapy problem

With $2.8M gene therapy, Bluebird sets new bar for US drug pricing

European regulators back hemophilia gene therapy

Takeda wins FDA approval for targeted lung cancer drug

Vertex, CRISPR strengthen case for pioneering gene-editing treatment

Vertex, CRISPR finish US filing for gene editing drug approval

Regeneron, UPenn to use gene therapy tools to deliver a COVID-19 antibody drug

CSL doses first patient with haemophilia B gene therapy Hemgenix

Stay Connected