World of DTC Marketing

JANUARY 24, 2022

A group of early-stage life science investors and biotech CEOs joined together with a prominent patient advocate to speak out against H.R.3, The panel was hosted by the life sciences venture capital coalition Incubate and held at the National Press Club. Only about $2.2 3, known as the Lower Drug Costs Now Act.

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XTalks

SEPTEMBER 14, 2022

Subscribe to the Xtalks Life Science Podcast to never miss a new episode. Medtronic’s InPen Gets Backing from Disney Star Jennifer Stone in Diabetes Tech Campaign. The weekly podcast is available for streaming every Wednesday on Spotify , Apple Music and wherever you stream your podcasts.

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Life Science FDA Approval Insulin Drugs 90

XTalks

DECEMBER 4, 2024

CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder. Jupiter joins a wave of biotech IPOs this year, stepping into a competitive space while broadening treatment options for CNS patients.

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Bioavailability Genetic Disease Marketing RNA 111

XTalks

AUGUST 7, 2024

In this episode, Ayesha spoke with Lawreen Asuncion, a patient advocate who works to raise awareness for the rare disease Usher syndrome. Lawreen is a seasoned biotech and life science professional with over 25 years of experience. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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Life Science Scientist Production Research 52

STAT News

SEPTEMBER 12, 2022

In the life sciences industry, where company names derived from “biology” or “genetics” are a dime a dozen, it helps to have a brand that stands out. The company is developing treatments for diseases caused by broken mitochondria — the main energy source for human cells.

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Genetic Disease Life Science Genetics Branding 98

BioPharma Reporter

OCTOBER 17, 2024

Wave Life Sciences reports that it has successfully carried out RNA editing in two patients with alpha-1 antitrypsin deficiency, the first time this kind of clinical RNA editing has been completed in humans according to the company.

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RNA Genetic Disease Genetics Life Science 52

XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). Nobelpharma America, a pharmaceutical and medical device company headquartered in Bethesda, Maryland, has launched an educational website called Face Forward.

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Life Science Genetic Disease FDA Approval Marketing 98

Scienmag

JUNE 7, 2021

Credit: Autobahn Labs Cold Spring Harbor Laboratory (CSHL) is collaborating with Autobahn Labs, a new life sciences incubator, to catalyze the Laboratory’s early-stage discovery programs into spin-out companies that commercialize transformational new therapies. Autobahn will provide […].

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Genetic Disease Life Science Genetics Research 52

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences president and CEO Bo Cumbo said: “This collaboration with Phlox Therapeutics is an exciting opportunity to work with a company that shares our commitment and innovative approach to bringing transformative therapies to patients with rare genetic diseases.

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RNA Gene Therapy Genetics Genetic Disease 130

XTalks

DECEMBER 18, 2024

Neurocrine Biosciences Crenessity (crinecerfont) has been given the green light by the US Food and Drug Administration (FDA) as a new treatment for congenital adrenal hyperplasia (CAH). The approval marks a significant step in addressing a condition that has historically relied on decades-old therapies.

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XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases. HS: Absolutely.

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Genome Genomics Medicine Genetic Disease 98

XTalks

MAY 17, 2023

Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall “The BGTC offers promise for rare disease patients around the world,” said Julie Gerberding, MD, CEO of the FNIH, in the press release from the agency.

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pharmaphorum

OCTOBER 16, 2020

Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise. Over the last five years we have focused on the power of drug repurposing for rare genetic diseases. The post Rare diseases, repurposing and the role of AI appeared first on.

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Trials Clinical Trials Clinical Trials Life Science 105

Pharmaceutical Technology

MAY 8, 2023

ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models. It is a chimeric endonuclease, which consists of a custom-designed ZF DNA-binding domain and the non-specific nuclease domain from the FokI restriction enzyme. They are the smallest type of programmable nuclease.

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XTalks

MAY 4, 2021

Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

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XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

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RNA Genetics Genetic Disease Medicine 105

XTalks

FEBRUARY 25, 2025

CTX is a rare, progressive genetic disorder caused by mutations in the CYP27A1 gene, which disrupts the livers ability to produce chenodeoxycholic acid, a bile acid.

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XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company.

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Gene Therapy Gene Dermatology FDA Approval 97

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. “They called him ‘The Boy.’ Who wasn’t supposed to sit up. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S.

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XTalks

AUGUST 19, 2020

Cystic fibrosis (CF) patients must keep a six feet distance from others at all times to keep germs away that could exacerbate their disease. CF is a progressive, genetic disease that causes persistent lung infection and could limit a person’s ability to breathe over time.

Genetic Disease 111

Genetic Disease Genetics Life Science Research 111

XTalks

AUGUST 16, 2021

Immune systems can be weakened in individuals with HIV/AIDS; cancer and transplant patients taking certain immunosuppressive drugs; and those with genetic diseases affecting the immune system like congenital agammaglobulinemia and congenital IgA deficiency. According to the CDC, approximately 2.7

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XTalks

AUGUST 29, 2022

The team’s approach to cellular engineering with non-viral CRISPR technology could be used to replace the genes IL2RA and CTLA4 entirely, which are associated with rare genetic diseases. Scientists have previously shown that they could replace small sections of the IL2RA gene where mutations occur in patients.

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Gene Editing DNA Gene Genomics 98

XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes.

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Clinical Trials Clinical Trials Genetics Trials 98

XTalks

SEPTEMBER 27, 2024

In a news release published yesterday, Pfizer announced it will voluntarily withdraw its sickle cell disease (SCD) therapy Oxbryta (voxelotor) from all markets worldwide. The company said it is also discontinuing all active clinical trials for voxelotor and expanded access programs around the world.

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XTalks

OCTOBER 16, 2024

Pfizer’s Hympavzi (marstacimab-hncq) has received approval from the US Food and Drug Administration (FDA) for treating hemophilia A or B in adults and adolescents aged 12 and older with factor VIII deficiency (hemophilia A) or factor IX deficiency (hemophilia B), who do not have inhibitors to these factors.

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pharmaphorum

JANUARY 27, 2023

Developing medicines – for people living with disease Dr Mullen’s current role involves providing medical monitoring support, as well as safety, pharmacovigilance, scientific, and medical advice across a range of client projects, including advanced therapies and orphan drugs. I still feel that it is very important,” Dr Mullen said. “We’ve

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Clinical Trials Clinical Trials Trials Development 111

XTalks

MAY 4, 2021

Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. They found that as cells transitioned from stem cells to differentiated adult cells, which involves significant epigenetic changes, the methylation marks made by CRISPRoff were inherited in a significant fraction of cells.

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The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. The impact of CNS diseases extends beyond patients—to their families and society as well.”

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RNA Antibody Life Science Protein 52

XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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Gene Therapy Gene FDA Approval In-Vivo 98

pharmaphorum

DECEMBER 8, 2021

If approved, the drug will be sold by Neuren Pharma, which licensed North American rights to the drug in 2018 and is also developing another drug for Rett blarcamesine, in collaboration with Anavex Life Sciences. Results from a phase 3 trial of blarcamesine are due next year.

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Marketing Drugs Life Science Trials 59

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).

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FDA Approval Clinical Trials Clinical Trials Trials 104

Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

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Gene Therapy Gene Immune Response In-Vivo 52

XTalks

MAY 12, 2023

What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.

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FDA Approval Genetics Trials Drugs 97

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug.”.

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XTalks

SEPTEMBER 12, 2022

ASMD is a rare genetic lysosomal storage disease caused by mutations in the sphingomyelin phophodiesterase-1 ( SMPD1 ) gene that codes for the acid sphingomyelinase (ASM) enzyme.

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FDA Approval Genetics Trials Gene 98

XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. The approval was granted to BridgeBio Pharma, Inc. Nasdaq: BBIO ) and its affiliate Origin Biosciences, Inc. MoCD Type A usually presents in the first couple of days after birth and causes intractable seizures, brain injury and death.

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FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

SEPTEMBER 22, 2022

GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it. The novel, selective monoclonal antibody spesolimab behind Spevigo reduces flares in individuals by inhibiting interleukin-36 (IL-36) signalling.

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