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PJ Anand, Alcyone Therapeutics CEO

A gene ther­a­py biotech is burst­ing on­to the scene with 12 pro­grams in tow. An ul­tra-rare neu­ro dis­ease is up first

In downtown Lowell, MA, tucked into one of the country’s oldest surviving textile mill complexes is a new biotech toiling away on next-gen CNS therapies for severe neurological disorders. And it’s starting with Rett syndrome — a rare neurodevelopmental condition that almost exclusively affects girls.

Alcyone Therapeutics emerged from stealth mode Wednesday with $23 million from RTW Investments and 12 new gene therapy programs in the works. The company was created last summer, shortly after founder and CEO PJ Anand met Kathrin Meyer, a principal investigator in the gene therapy unit at the Abigail Wexner Research Institute at Nationwide Children’s Hospital.

Stuart Schreiber (Maria Nemchuk, Broad Institute)

Po­laris, Ab­b­Vie, Bay­er back Stu­art Schreiber's hunt to turn can­cer treat­ments in­to cures

What happens to cancer cells that don’t die after powerful treatment? Scientists have long known that in all but a handful of cases, they don’t vanish even if the patients appear cancer-free — instead lurking somewhere, unreachable and undetectable and preparing, like a vanquished movie villain at the start of a sequel, for a return.

Research over the last decade began to suggest there was something different about these survivor cells. It wasn’t just that they might mutate. They reprogrammed themselves, like an operating system sealing itself off after too many incorrect passwords, and entered a protective state that other cells in the body have been known to enter when threatened.

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Henry Colecraft and Scott Kanner (via Columbia Technology Ventures)

Sal­vaging 'ug­ly fruit': Af­ter a se­ries of pro­tein degra­da­tion bets, Ver­sant turns to pro­tein sta­bi­liza­tion plat­form out of Co­lum­bia

As an expert on ion channels, Henry Colecraft was familiar with the damage that an overzealous ubiquitination system can do to the body. While that process, often described as a garbage disposal mechanism, is crucial for getting rid of defective or unnecessary proteins, it can also mistakenly send still functional proteins to destruction.

It is a process involved in a diverse group of diseases known as ion channelopathies, ranging from cystic fibrosis to epilepsy to the heart disease known as long QT syndrome.

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Sarah Cole, Takeda Ventures partner

Al­most a decade af­ter iden­ti­fy­ing a new class of RNA, a group of sci­en­tists sparks in­ter­est from Take­da Ven­tures

Over the last several years, scientists have turned their attention to long noncoding RNAs (lncRNAs) — a mysterious class of naturally occurring molecules that don’t code for proteins, but fulfill important regulatory functions in the cell. Stefano Gustincich and his team identified a new class of them back in 2012, and now the scientists have pulled in seed funding to see if the molecules can go where current therapeutics can’t.

Lara Sullivan, Pyxis Oncology CEO

Al­loy and Pyx­is spin out new 'com­pa­ny' around high-risk I/O, im­munol­o­gy tar­gets

When CEO Lara Sullivan and the team at Pyxis sat down in 2019 to see which of the many targets from Thomas Gajewski’s immuno-oncology lab they would try to drug, there were too many for any startup to pursue at once. They prioritized the targets they thought had the best chance of success, leaving the rest for some future date.

That date came sooner than expected. Last year, the team at Alloy Therapeutics —the conglomerate of biotech services, technologies and spinouts backed by billionaire investor Peter Thiel — got wind of all that Pyxis was leaving on the table. Now, the two are spinning out a new company, Kyma Therapeutics, dedicated to finding antibodies that can hit two of the high-risk targets, potentially opening up paths to treatments in cancer and immunology.

Samir Ounzain, Haya CEO

A new RNA start­up looks to reimag­ine heart treat­ments, with eyes set on En­tresto

When Samir Ounzian left London a decade ago to study a little-understood branch of the genome at a Swiss lab, he wasn’t sure what he would find. Known as long-non-coding RNA, these sections were translated into looping rings of RNA — hence the name — but didn’t actually translate into proteins, and no one was quite sure how many there were or precisely what they were doing.

“We didn’t expect anything at the time,” Ounzian told Endpoints News, “because we didn’t really understand how frequent they were in the genome.”

David Ho (L) and Yaoxing Huang (RenBio)

HIV re­searcher David Ho has a new line of at­tack on SARS-CoV-2, and his fledg­ling start­up is gun­ning for the clin­ic

The famed AIDS researcher David Ho started off the Covid-19 pandemic working to develop an antibody against the SARS-CoV-2 virus, but now he’s behind a new biotech that’s taking a slightly different approach to fight the disease.

Ho is a co-founder of RenBio, which pulled in its first fundraise Thursday morning with a $24 million Series A, the company announced. The biotech is pouring its first efforts into a bispecific antibody targeting two sites on the SARS-CoV-2 spike protein, and is touting activity against several Covid-19 variants of concern including strains originating from the UK, South Africa and Brazil, among others.

Cyrus Mozayeni, Vedere Bio CEO

Months af­ter No­var­tis buy­out, the old Vedere Bio team is back at it with a new pipeline and a fresh $77M

What do you do when Novartis plunks down $150 million to buy your startup before you even hit the clinic? If you’re Cyrus Mozayeni, you start a new one.

On Tuesday, Mozayeni pulled the curtain on a hefty $77 million Series A round to get the ball rolling at Vedere Bio II — an ocular gene therapy company launched by the same leadership and research team from the first Vedere. After handing their lead program to Novartis in 2020, Mozayeni’s team is now regrouping around a different preclinical pipeline which, like the first, is based on tech from the UC Berkeley labs of Ehud Isacoff and John Flannery and UPenn.

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An Or­biMed-backed start­up looks to cut ex­cess cor­ti­cos­teroids with mid-stage tests for its lead drug queued up

OrbiMed has had a busy few months with a slate of startups launching out of its always productive pipeline of business concepts. Now, an OrbiMed-backed player in Oregon is looking to tamp down intracellular steroid toxicity, and it’s got a healthy check to get off the ground.

Portland-based Sparrow Pharmaceuticals launched Tuesday with a $50 million Series A led by OrbiMed and a lead corticosteroid lowering candidate geared up for Phase II trials, the company said.

Jim Wil­son and Tachi Ya­ma­da are at it again — launch­ing a 2nd-gen gene ther­a­py com­pa­ny with an ap­petite for risk, glob­al vi­sion and $200M to gam­ble with

When Tachi Yamada and Jim Wilson partnered on launching Passage Bio $PASG, the game plan was to use their considerable combined scientific knowhow and global business connections to leap out with new programs for a slate of prospects — therapies that they could be relatively sure of getting through pivotal studies and opening fields for rare CNS diseases. Working with Penn’s Gene Therapy Program, now close to 400 strong on staff, they have a product engine and clinical development plan to get drugs through to the marketplace.

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