Drugs and Gene Therapy - 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.

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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

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.

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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.

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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.

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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.

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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

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

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.

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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

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.

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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

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.

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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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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.

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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.

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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.

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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.

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Gene Therapy Gene Drugs

Sarepta scraps a Duchenne drug as gene therapy sales rise

Bio Pharma Dive

NOVEMBER 7, 2024

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

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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. You can also subscribe here to receive email notifications when a new issue is available.

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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.

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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.

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Forge Biologics joins BGTC for new AAV gene therapies development

Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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.

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Gene Therapy Gene FDA Approval Manufacturing

Capsida Biotherapeutics and Prevail to develop CNS gene therapies

Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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.

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Cell One and CBM partner for cell and gene therapies

Pharmaceutical Technology

MARCH 10, 2023

Cell One Partners has announced a strategic collaboration with the Center for Breakthrough Medicines (CBM) to expedite the development and commercialisation of cell and gene therapies. Cell One Partners offers virtual company creation and management to its clients for complete aspects of the development and commercialisation of drugs.

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Gene Therapy Gene Manufacturing Development

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

ASRS conference reveals hopeful outcomes for gene therapy RGX-314 in retinal diseases

Pharmaceutical Technology

JULY 22, 2022

The spotlight was placed on many up-and-coming pharmacotherapies for retinal diseases, one of which was AbbVie’s/Regenxbio’s gene therapy RGX-314. The drug is of paramount interest due to addressing vital unmet needs for anti-vascular endothelial growth factor (VEGF) treated retinal diseases, including DME.

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Gene Therapy Gene Trials Compliance

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.

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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.

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Research consortium picks eight rare diseases to advance gene therapies

Pharmaceutical Technology

MAY 16, 2023

The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.

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Gene Therapy Gene Research Clinical Trials

Sarepta asks FDA to approve first gene therapy for Duchenne muscular dystrophy

Bio Pharma Dive

SEPTEMBER 30, 2022

The application comes a year earlier than previously had been expected, as company says drug reviewers are open to accelerated review based on data from early-stage trials.

Gene Therapy

Gene Therapy Gene Trials Drugs

String of gene therapy deals spurs cautious optimism on Wall Street

Bio Pharma Dive

NOVEMBER 1, 2022

To some analysts, the recent acquisitions of Akouos and AGTC, as well as Astellas’ equity investment in Taysha, are a positive signal for an area of drug research that’s been “in investors' penalty box for much of 2022.”

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Gene Therapy Gene Drugs Research

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

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4DMT’s CF gene therapy posts early cues for improving patient outcomes

Pharmaceutical Technology

JUNE 8, 2023

The use of 4D Molecular Therapeutics’ (4DMT) aerosolised gene therapy 4D-710 has improved the quality-of-life and spirometry-measured outcomes in three cystic fibrosis patients , based on early results from a Phase I/II study presented at this year’s annual meeting of the European Cystic Fibrosis Society (ECFS).

Gene Therapy

Gene Therapy Gene Development Trials

Editas continues to trend efficacy signals with sickle cell disease gene therapy

Pharmaceutical Technology

MAY 12, 2023

Editas Medicine will release new efficacy and safety results of its gene therapy EDIT-301 in severe sickle disease as part of an oral presentation at the European Hematology Association’s (EHA) Hybrid Congress. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Regeneron, in search of an eye gene therapy, turns to a young biotech

Bio Pharma Dive

APRIL 6, 2022

Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.

Gene Therapy

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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

FDA advisers offer unanimous support to second Bluebird gene therapy

Bio Pharma Dive

JUNE 10, 2022

By a 13-0 vote, the panel agreed the benefits of Bluebird’s beta thalassemia treatment outweigh its risks, one day after reaching a similar conclusion for another of the company’s drugs.

Gene Therapy

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Gene therapy approval won, Bluebird takes on next challenge: selling it

Bio Pharma Dive

DECEMBER 12, 2022

The biotech is still waiting to treat its first commercial patient with Zynteglo, but says it has not seen insurers deny coverage for the beta thalassemia drug.

Gene Therapy

Gene Therapy Gene Drugs

UniQure moves Huntington's gene therapy to next phase of key trial

Bio Pharma Dive

MAY 27, 2021

Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the genetic disease.

Gene Therapy

Gene Therapy Genetic Disease Gene Genetics

Skyline wins orphan drug designation for retinitis pigmentosa gene therapy

Pharmaceutical Technology

SEPTEMBER 3, 2024

There are currently no approved treatments that can halt or reverse photoreceptor degeneration in patients with retinitis pigmentosa.

Gene Therapy

Gene Therapy Gene Drugs

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

Pfizer hemophilia gene therapy arrives in US to uncertain future

Webinars

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

Pfizer receives FDA approval for haemophilia B gene therapy

Gene Therapy and Pharmacokinetics

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

Forge Biologics and Labcorp partner for gene therapies development

Astellas to support development of Taysha’s gene therapy programmes

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

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

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

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

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

FDA grants approval for CSL’s haemophilia B gene therapy

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

Sarepta scraps a Duchenne drug as gene therapy sales rise

Magazine: CRISPR gene therapies cut through in 2023

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

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

Forge Biologics joins BGTC for new AAV gene therapies development

Krystal Biotech receives FDA approval for DEB topical gene therapy

Capsida Biotherapeutics and Prevail to develop CNS gene therapies

CSL doses first patient with haemophilia B gene therapy Hemgenix

Cell One and CBM partner for cell and gene therapies

Ovid turns to gene therapy startup to restock drug pipeline

ASRS conference reveals hopeful outcomes for gene therapy RGX-314 in retinal diseases

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

Research consortium picks eight rare diseases to advance gene therapies

Sarepta asks FDA to approve first gene therapy for Duchenne muscular dystrophy

String of gene therapy deals spurs cautious optimism on Wall Street

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

4DMT’s CF gene therapy posts early cues for improving patient outcomes

Editas continues to trend efficacy signals with sickle cell disease gene therapy

Regeneron, in search of an eye gene therapy, turns to a young biotech

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

FDA advisers offer unanimous support to second Bluebird gene therapy

Gene therapy approval won, Bluebird takes on next challenge: selling it

UniQure moves Huntington's gene therapy to next phase of key trial

Skyline wins orphan drug designation for retinitis pigmentosa gene therapy

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