XTalks

DECEMBER 20, 2023

In 2023, there were significant advancements and notable trends in the life sciences. Xtalks compiled a list of its top life science news and trends of 2023, which provided readers with the latest developments, information and expert insights across life science industries, including pharma, biotech and medical device.

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Life Science Clinical Trials Clinical Trials FDA Approval 59

XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

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Life Science Gene Therapy Gene Manufacturing 52

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

AUGUST 31, 2022

Hear more about the approval, including clinical trial data showing the treatment can take effect in as little as one week, and how it is the first antidepressant approved in over 60 years that has a new mechanism of action. Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia. Bluebird’s $2.8M

Gene Therapy 52

Gene Therapy FDA Approval Life Science Gene 52

XTalks

FEBRUARY 28, 2024

Also hear about some of the latest trends in rare disease research, including innovative trial designs, patient-centric approaches, the use of cutting-edge technologies like AI and international collaborations. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

Life Science 52

Life Science Research Genetics Drugs 52

XTalks

APRIL 14, 2021

In this episode of the Xtalks Life Science Podcast, Sarah revisits two Parkinson’s biotech companies — Prevail Therapeutics and Voyager Therapeutics — to see what progress they’ve made in their gene therapy clinical development programs since April 2019. Parkinson’s and Melanoma Share an Amyloid Link, Says New Research.

Life Science 98

Life Science Gene Therapy Development Drugs 98

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. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

JUNE 8, 2023

Over the past few years, there has been a significant expansion in the cell and gene therapy landscape, with an increasing number of therapies entering clinical trials and receiving regulatory approvals. “I think if we all go into these relationships with this mindset, we’ll achieve great things together.”

Gene Therapy 98

Gene Therapy Gene Production Development 98

Drug Discovery World

NOVEMBER 24, 2022

With European life sciences R&D investment at a 20-year low, biopharmaceutical associations and MEPs are calling on the EU to modernise its policies. Fortunately, the EFPIA report also offers a detailed list of recommendations and the hope that with a proactive approach to nurturing life sciences, this trend can be reversed. .

Gene Therapy 98

Gene Therapy Gene Clinical Trials Clinical Trials 98

pharmaphorum

JUNE 3, 2021

The arrival of G7 health ministers to Oxford this week (Thursday 3 June) will shine a spotlight on Oxfordshire’s considerable achievements in the life sciences sector – and, according to the county’s Local Enterprise Partnership, there couldn’t be a better location globally for this summit to be held.

Life Science 52

Life Science Vaccination Vaccine Development 52

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). The FDA approval came after several delays and a narrow advisory committee vote of eight to six in favor of the gene therapy’s risk-benefit profile.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

XTalks

JANUARY 17, 2024

Given this hotbed of activity, innovation in the space to drive faster decisions and more efficient trials is intense. They discussed the top new trends in oncology trial design that sponsors are either incorporating into clinical trials or need to start planning to include.

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Clinical Trials Clinical Trials Trials Gene Therapy 124

XTalks

OCTOBER 11, 2023

The company was recently granted clinical trial authorization from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its investigational biologic CAR T-Cell engager ALETA-001. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

Life Science 52

Life Science Clinical Trials Clinical Trials Scientist 52

XTalks

OCTOBER 27, 2021

Ayesha also talked about a new gene silencing treatment for porphyria called Givlaari that received recommendation from England’s NICE after having been initially rejected by the health watchdog last year. Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data.

Gene Silencing 52

Gene Silencing Life Science FDA Approval Gene 52

XTalks

JULY 27, 2022

Toleikis spoke about the latest clinical trial data for the medical device, and also about the company’s new partnership with Evotec to integrate its insulin-producing cells in the Cell Pouch System. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

Life Science 52

Life Science Insulin Clinical Trials Clinical Trials 52

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). As a topical treatment, it is also the first readily redosable gene therapy.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

pharmaphorum

OCTOBER 22, 2020

Sarepta chief commercial officer Bo Cumbo has left to head up gene therapy venture – AavantiBio – with $107 million in backing from his former employer and three high-profile life sciences investors. Unlike a one-shot gene therapy, omaveloxolone would require continuous dosing to maintain its effects.

Gene Therapy 98

Gene Therapy Gene Life Science Engineer 98

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

Gene Therapy 64

Gene Therapy Gene In-Vitro Development 64

Drug Discovery World

JUNE 12, 2023

It is the UK’s first specifically designed accessible laboratory for the cell and gene therapy industry. The space also includes a Universal Design Lab which has been designed for scientists with and without disabilities to work side by side.

Gene Therapy 52

Gene Therapy Gene Scientist Life Science 52

XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

Gene Therapy 98

Gene Therapy Gene FDA Approval Antibody 98

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

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Gene Therapy Gene Protein Trials 110

XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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Gene Therapy Gene Genome Genomics 105

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

Drug Discovery World

MAY 23, 2023

DDW’s Megan Thomas caught up with George White , General Manager, Product Management, Cell & Gene Therapy at Cytiva, to learn more about the company’s insight into the CGT market. Key challenges for cell and gene therapies In order for cell and gene (CGT) therapies to succeed, George White identifies several challenges to overcome.

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Gene Therapy Gene Gene Editing Production 52

XTalks

APRIL 5, 2024

Amylyx Pharmaceuticals announced yesterday that it will be pulling its ALS (amyotrophic lateral sclerosis) drug Relyvrio from the market after failing in a confirmatory trial. Last month, Amylyx reported results from the global PHOENIX Phase III clinical trial that were unable to confirm the drug’s effectiveness.

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Trials Drugs Marketing FDA Approval 98

Drug Discovery World

SEPTEMBER 6, 2022

Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. As researchers and developers work to transform the way we treat diseases, cell and gene therapies will continue to be the centre of innovation in the coming years. . billion raised.

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Gene Therapy Gene Gene Editing Manufacturing 52

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. It has been estimated that by 2025 the FDA will be approving 10 to 20 cell and gene therapy products per year 2.

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Gene Therapy Gene Development Production 52

BioSpace

OCTOBER 14, 2021

Read about TFF Pharmaceuticals and Augmenta Bioworks' dry powder COVID-19 antibody formula, the world's first gene editing clinical trial for PKU, Bayer's COVID-19 vaccine and other key developments in life sciences research.

Gene Editing 72

Gene Editing Life Science Clinical Trials Clinical Trials 72

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

MAY 6, 2024

Thanks to a prestigious academic legacy, Europe has long been a dominant region for life sciences. Europe is traditionally a life sciences powerhouse, with a strong tradition in pharmaceutical discovery. The oldest, still active pharmaceutical company worldwide, Merck, started life in 1668 in Darmstadt, Germany.

Drugs 59

Drugs Life Science Gene Editing Gene 59

XTalks

DECEMBER 20, 2022

While clinical trials are becoming more complex, oncology trials have generally had a greater level of complexity than trials in many other disease spaces. Despite this, the number of oncology trials worldwide has increased dramatically over the past two decades. Regulatory Considerations. Study Design Considerations.

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Clinical Trials Clinical Trials Trials Regulation 97

XTalks

DECEMBER 27, 2022

Adstiladrin is an adenoviral vector-based gene therapy that is non-replicating, so it cannot multiply in human cells. Boorjian, MD, Carl Rosen Professor and Chair of the Department of Urology at Mayo Clinic, and lead investigator on the recent clinical trial of Adstiladrin, in the company’s press release.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

Pharmaceutical Technology

APRIL 27, 2023

Co-led by new investor Arix Bioscience and current investors EQT Life Sciences and SymBiosis, the financing round also included new and existing investors such as Amplitude Ventures. EVO101 is currently under assessment in a Phase 2a proof-of-concept trial involving patients diagnosed with atopic dermatitis.

Life Science 130

Life Science Gene Trials Medicine 130

Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 130

Gene Therapy Gene In-Vivo Life Science 130

Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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Gene Therapy Gene Manufacturing Production 52

Pharmaceutical Technology

NOVEMBER 23, 2022

One of the primary focus areas includes the continued commercialisation of cutting-edge cell and gene therapies, which involve the modification of cells from patients or donors to produce highly personalised therapies and regenerative medicines. OmniaBio scales CDMO operations in Hamilton.

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Marketing Gene Therapy Life Science Manufacturing 130