XTalks

MARCH 25, 2021

Xtalks is proud to announce the launch of the Xtalks Life Science podcast. Subscribe to the Xtalks Life Science Podcast to never miss a new episode. Fresh Conversations About Life Science Topics. She focuses on news relating to the food industry and writes blogs on recruitment and HR in the life sciences.

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XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. Duvyzat received Priority Review, Orphan Drug and Rare Pediatric Disease designations from the FDA.

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XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. These companies are at various stages of research and clinical trials.

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XTalks

FEBRUARY 19, 2025

It occurs when a genetic error causes the overproduction of a protein called CSF1, which attracts inflammatory cells to the joint, often causing pain, swelling and reduced mobility. In 2024, Deciphera was acquired by ONO Pharmaceutical for $2.4 TGCT is a rare, non-cancerous tumor that develops inside or near joints.

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XTalks

NOVEMBER 26, 2024

BridgeBio has been highly active in the genetic diseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.

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XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. Here is a look at some life science trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.

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XTalks

MARCH 19, 2025

Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. Elevidys aims to address the underlying genetic cause by introducing a functional version of the dystrophin gene, which is essential for muscle function.

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XTalks

JUNE 20, 2022

Amvuttra’s FDA approval is based on positive data from the global, randomized, open-label, multicenter HELIOS-A Phase III study from a period of nine months. The condition has very limited treatment options. Amvuttra is administered via subcutaneous injection four times a year or once every three months (quarterly).

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XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. a biotech company specializing in the development and commercialization of genetic medicines for rare diseases, announced FDA approval for Vyjuvek for the treatment of DEB.

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XTalks

OCTOBER 16, 2024

Hympavzi’s approval, with its innovative approach to balancing clotting, could offer a modern solution that adds to the global effort to reduce the risk of thrombosis. This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year.

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XTalks

APRIL 3, 2024

Related: Novartis’ Fabhalta Gets FDA Approval for Rare Complement Blood Disorder The FDA’s green light for Voydeya comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator in the world to back the therapy. AstraZeneca is also venturing into cell therapy and genetic medicine.

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XTalks

FEBRUARY 4, 2025

Current Share Price: The announcement follows a $115 million Series D financing in December 2024, co-led by Frazier Life Sciences and Deep Track Capital, with participation from Janus Henderson Investors and Logos Capital. MZE829 aims to mimic a protective genetic variant of APOL1. The offering, which includes 8.75

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XTalks

JANUARY 26, 2023

This is the fourth US FDA approval that BeiGene has received for Brukinsa since 2019. Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations.

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XTalks

SEPTEMBER 12, 2022

After approvals in Japan and Europe, Sanofi’s Xenpozyme has secured one from the US Food and Drug Administration (FDA) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Patients in the trial received Xenpozyme or placebo for 52 weeks.

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XTalks

JUNE 23, 2022

In this episode, Ayesha and the team talked about the FDA approval of Eli Lilly’s JAK inhibitor Olumiant for the treatment of alopecia areata, also commonly just known as alopecia. The drug has become the first approved systemic treatment for the autoimmune disorder that causes patchy hair loss.

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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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Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. SK Life Science is a subsidiary of SK, a South Korean holding company.

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XTalks

DECEMBER 9, 2024

Programs like the Rare Disease Endpoint Advancement Pilot encourage collaboration with the FDA to establish novel surrogate endpoints. This initiative is particularly impactful for rare diseases and genetic disorders, where traditional endpoints may be infeasible.

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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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XTalks

FEBRUARY 21, 2025

Current Share Price: PWS is a complex, genetically determined neurodevelopmental disorder that affects multiple organ systems. And hyperphagia is common not only to PWS but also to hypothalamic obesity, binge-eating disorder and certain rare genetic disorders like Bardet-Biedl syndrome.

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XTalks

MARCH 21, 2025

With a 30-month shelf life and rapid absorption, Neffy offers an on-the-go alternative to auto-injectors. Following Neffys FDA approval , ARS Pharma reported $2.3 Iovance Biotherapeutics Iovances Amtagvi (lifileucel) is FDA-approved as the first tumor-infiltrating lymphocyte (TIL)-based therapy for solid tumors.

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XTalks

MAY 31, 2022

The precise cause of psoriasis is unknown, but risk factors include genetics and a family history of the condition, heavy alcohol consumption, stress, smoking and dry or cold weather. It has been estimated that around eight million people in the US and 125 million people in the world have been affected by psoriasis.

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XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). Hyftor became the first FDA-approved topical treatment for facial angiofibroma associated with tuberous sclerosis.

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XTalks

DECEMBER 2, 2024

The US Food and Drug Administration (FDA) has approved AOP Health’s landiolol injection, marketed as Rapiblyk, for the treatment of atrial fibrillation (Afib) and atrial flutter in critical care settings.

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XTalks

MARCH 21, 2023

It is estimated that Rett syndrome affects 6,000 to 9,000 patients in the US, with a diagnosed population of approximately 4,500 patients, according to a press release from Acadia announcing Daybue’s approval. However, in animal studies, trofinetide has been shown to increase dendritic branching and synaptic plasticity signals.

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XTalks

NOVEMBER 10, 2023

Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.” In 2022, the FDA approved 20 orphan drugs out of the 37 novel drugs approved by the FDA’s Center for Drug Evaluation and Research (CDER).

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XTalks

MAY 17, 2023

In 2022, the FDA approved four new gene therapies, and more than a handful are looking to get approval this year for diseases including Duchenne muscular dystrophy (DMD), sickle cell disease (SCD) and hemophilia A. Of the more than 10,000 rare diseases, more than 80 percent are caused by genetic defects.

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XTalks

JANUARY 20, 2025

From Bristol-Myers Squibbs monumental acquisition of Celgene to Pfizers recent purchase of Seagen, these deals illustrate the strategies and ambitions driving innovation in life sciences. This strategic acquisition significantly strengthened Gileads oncology portfolio and provided access to Immunomedics ADC technology.

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Pharmaceutical Technology

NOVEMBER 29, 2022

PharmaTher also has an agreement in place with Case Western Reserve University to develop ketamine as a treatment for Rett syndrome, a rare genetic neurological disorder. In 1970, the FDA approved ketamine as an anesthetic. The company now plans to move this approach into a Phase III trial.

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XTalks

SEPTEMBER 18, 2024

The US Food and Drug Administration (FDA) approved Kisunla (donanemab-azbt) , a treatment for patients with mild cognitive impairment or mild dementia caused by Alzheimer’s. To mitigate this, genetic testing for APOE4 is recommended before treatment.

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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.

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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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XTalks

JUNE 5, 2023

The genetic material of RSV (respiratory syncytial virus) encodes 11 proteins. Today’s FDA approval of Abrysvo recognizes significant scientific progress, and importantly helps provide older adults potential protection against RSV and an opportunity to improve community health by helping prevent the disease,” said Edward E.

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XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. It affects around one in 10,000 girls born each year.

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XTalks

JUNE 29, 2022

The new FDA approval gives Breyanzi (lisocabtagene maraleucel) the broadest patient eligibility of any CAR T-cell therapy in relapsed or refractory LBCL. Treatment with Breyanzi is a tailored method of treatment which involves the genetic reprogramming of T cells. Product logo and product image courtesy of Bristol Myers Squibb.

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