The Pharma Data

JANUARY 17, 2021

But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion. Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

Pharmaceutical Technology

OCTOBER 5, 2022

In 2010, two patients with end-stage refractory chronic lymphocytic leukaemia (CLL) were administered Novartis’ autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy, Kymriah (tisagenlecleucel), as part of a Phase I trial.

In-Vitro 279

In-Vitro In-Vivo Sales Clinical Trials 279

Drug Discovery World

MAY 26, 2023

AnaMar has announced positive results from its Phase I study, and the completion of Phase II enabling pre-clinical studies, for AM1476’s initial orphan indication in systemic sclerosis (SSc) characterised by lung and skin fibrosis. There are currently no treatments on the market that effectively reverse scarring.

In-Vivo 52

In-Vivo Trials Marketing Drugs 52

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. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

XTalks

APRIL 24, 2023

Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

Gene Editing 144

Gene Editing Gene Gene Therapy In-Vivo 144

The Pharma Data

JANUARY 12, 2021

INCHEON, South Korea–( BUSINESS WIRE )– Celltrion Group today announced top-line results from its randomised, double-blind, and placebo controlled global Phase II/III clinical trial of CT-P59, an anti-COVID-19 monoclonal antibody treatment candidate. Head of Medical and Marketing Division at Celltrion Healthcare. “We

Clinical Trials 40

Clinical Trials Clinical Trials Trials In-Vivo 40

Drug Discovery World

APRIL 4, 2023

The designation is the first step to gaining a priority review voucher (PRV), which can accelerate market access for therapeutics. The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. Here’s a summary. The drug was granted orphan drug designation by the FDA in May 2021.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

pharmaphorum

FEBRUARY 13, 2022

Quris’ BioAI safety prediction platform – which is based on human tissue samples on chips, nanosensors and machine learning – will be compared to traditional in vitro and in vivo laboratory techniques for spotting toxicity. The post Merck will assess Quris’ AI ‘patient-on-a-chip’ drug safety appeared first on.

Drugs 98

Drugs In-Vivo In-Vitro Pharma Companies 98

pharmaphorum

JULY 22, 2021

We have completed phase 2 trial of a multi-antibody drug for influenza, CT-P27, and since 2015 we have been developing a therapeutic antibody to treat the Middle East Respiratory Syndrome (MERS), CT-P38. In just over a year, Celltrion Healthcare says it has identified and delivered a potential treatment for COVID-19.

Antibody 105

Antibody Clinical Trials Clinical Trials Trials 105

Camargo

NOVEMBER 11, 2020

When the FDA requires a product’s labeling to include a boxed warning (also called a “black box warning” because the text is surrounded by black border), the potential market value of the drug often drops severely. The “skinny label” has been used many times previously to enable ANDA applicants to reach the market in similar circumstances.

Development 242

Development Bioequivalency Generic Drugs Production 242

Drug Discovery World

AUGUST 22, 2023

In September 2021, the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) announced the initiation of a pilot programme 1 to provide parallel scientific advice to pharma companies applying for marketing authorisation for generic versions of hybrid products and complex generic drug products.

Production 52

Production Bioequivalency In-Vivo In-Vitro 52

pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications.

Gene Editing 59

Gene Editing Gene Gene Therapy Drugs 59

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials In-Vivo 52

pharmaphorum

JANUARY 25, 2023

Digital therapeutics company Woebot Health has announced enrolment of the first patient in a pivotal clinical trial to evaluate the safety and efficacy of WB001, an investigational therapeutic for postpartum depression (PPD), granted Breakthrough Device designation by the US Food and Drug Administration (FDA) in 2021.

In-Vivo 98

In-Vivo Clinical Trials Clinical Trials Trials 98

Drug Discovery World

JULY 22, 2022

For example, in randomised clinical trials of patients with non-small cell lung cancer (NSCLC), pembrolizumab has been shown to be effective in slowing down disease progression and improving survival rates 1. demonstrated the ability to model clinical responses to ACT in autologous HIS mice 4. Jespersen et al.

In-Vivo 52

In-Vivo Engineer In-Vitro Antibody 52

Drug Discovery World

FEBRUARY 6, 2023

MT: Can you provide me with an overview of what Infinimmune does currently and the potential it sees in the antibody-based therapeutics market? This is part of why patients in trials and the clinic can experience severe toxicity and adverse events, reject their therapy with an immune response, or completely fail to respond to treatment.

Antibody 52

Antibody Immune Response Genome Genomics 52

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

The Pharma Data

NOVEMBER 22, 2020

I am looking forward to working with the outstanding team already in place to progress this treatment, which has potential against a broad range of tumors both as first line and as combination therapy, further through clinical trials and towards market.”. About Genenta Science. 31 stefania.mazzoleni@genenta.com.

Gene Therapy 40

Gene Therapy Gene In-Vivo Clinical Trials 40

pharmaphorum

JANUARY 25, 2022

As the area of bringing a digital therapeutic to market means passing the same level of regulatory scrutiny as a traditional, pharmaceutical therapy, this means “prescription digital therapeutics must undergo extensive clinical trials to demonstrate their safety and effectiveness before they ever reach patients,” said McCann.

Medicine 110

Medicine In-Vivo In-Vitro Development 110

Drug Discovery World

MARCH 12, 2024

To this end, a grant of $3 million offered in the Bio Studio programme to mature a technology for ‘platform-market-fit’ is a unique opportunity for translational projects. Also, most second- generation antipsychotics have delayed onset, which may influence patient adherence. million deaths annually.

Medicine 59

Medicine Production Drugs In-Vivo 59

FDA Law Blog

JANUARY 2, 2022

The Draft Guidance describes a nine-step framework for evaluating the credibility of CM&S information submitted in pre-market applications. Computational modeling and simulation (CM&S) can sometimes be useful to demonstrate the safety and effectiveness of medical devices or incorporated into devices. Draft Guidance at 4.

In-Vivo 52

In-Vivo Development Engineer Clinical Trials 52

Drug Discovery World

OCTOBER 4, 2022

million from the National Institute on Aging of the National Institutes of Health, the company plans to begin a Phase Ia clinical trial evaluating its lead tau self- association small molecule inhibitor OLX-07010. . OLX-07010 has been well characterised through an extensive program of pre-clinical toxicology and metabolism testing.

Development 52

Development In-Vivo Compound Screening Clinical Development 52

Drug Discovery World

JULY 5, 2023

Global Cancer Segment Market Manager at Agilent Technologies, recently sat down with DDW Editor Reece Armstrong to tell us about the developments in the ‘golden age’ of cancer research. Mark Garner, PhD., RA: Please can you tell us a bit more about your role at Agilent? MG: I am responsible for Agilent’s cancer segment.

Research 98

Research Protein Scientist Immune Response 98

The Pharma Data

NOVEMBER 15, 2020

16, 2020 /PRNewswire/ –: iSTAR Medical , a medtech company developing minimally invasive ophthalmic implants for treatment of patients with glaucoma, today announced positive one-year results from the European STAR-II clinical trial of its micro-invasive glaucoma surgery (MIGS) device, MINIject TM. WAVRE, Belgium , Nov.

Trials 52

Trials In-Vivo Engineer Clinical Trials 52

Drug Discovery World

NOVEMBER 16, 2022

Therefore, while researchers can understand just how a drug molecule impacts a cell, they can’t be sure that it will perform the same when used in-vivo. . One of these benefits is how cells grown in this type of culture are able to interact with their surroundings, similar to how they would perform in-vivo. 2D vs 3D .

Drugs 97

Drugs In-Vivo In-Vitro Scientist 97

Drug Discovery World

SEPTEMBER 28, 2022

In fact, the market research firm, Bekryl, predicts that AI has the potential to offer over $70 billion in savings for the drug discovery process by 2028. . According to Hampleton Patners’ latest Healthtech M&A market report2, digital health companies raised a total of $57.2 The simple answer is no.

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

FDA Law Blog

JANUARY 2, 2022

The Draft Guidance describes a nine-step framework for evaluating the credibility of CM&S information submitted in pre-market applications. Computational modeling and simulation (CM&S) can sometimes be useful to demonstrate the safety and effectiveness of medical devices or incorporated into devices. Draft Guidance at 4.

In-Vivo 40

In-Vivo Regulation Development Engineer 40

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Pharmaceutical Technology

SEPTEMBER 19, 2022

The latest development is based on findings from the single-arm, open-label Phase II/III ALD-102 (Starbeam) and Phase III ALD-104 clinical trials. In June 2019, the European Commission granted conditional marketing authorisation for bluebird bio’s Zynteglo to treat transfusion-dependent ?-thalassemia. thalassemia.

FDA Approval 246

FDA Approval In-Vivo Gene Therapy Clinical Trials 246

Pharmaceutical Technology

AUGUST 18, 2022

The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study. In June 2019, the European Commission (EC) granted conditional marketing authorisation for Zynteglo to treat transfusion-dependent beta-thalassemia.

Gene Therapy 246

Gene Therapy Gene Genotype Genetics 246

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

Drug Discovery World

SEPTEMBER 6, 2022

And whilst clinical trials activity decreased by around 15% compared to 2020, last year still saw a number of regenerative medicines reach the bedside. Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7 billion, compared to $19.9 billion, compared to $19.9 million price point per dose.

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

DECEMBER 10, 2020

.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.

Vaccination 52

Vaccination Vaccine Immune Response Protein 52

Pharmaceutical Technology

JULY 20, 2022

Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes. Roche will also make further payments to Avista during the collaboration’s research phase and clinical, sales-based milestone and royalty payments for resulting products. Avista is entitled to receive $7.5m

Gene Therapy 147

Gene Therapy Gene Development In-Vivo 147

Pharmaceutical Technology

FEBRUARY 1, 2023

Ellipses Pharma stated that the dual FLT3 inhibition and Aurora kinase demonstrated to overcome the acquired resistance to selectively inhibit FLT3 in vitro and in vivo. According to the company, approximately one-third of AML patients are diagnosed with FLT3 mutations related to a higher relapse risk and poor clinical outcomes.

FDA Approval 130

FDA Approval In-Vivo In-Vitro Trials 130