Long­time stem cell play­er Vi­a­Cyte has teamed up with a ma­te­ri­als sci­ence com­pa­ny in an ef­fort to solve im­muno­sup­pres­sion chal­lenges and ad­vance its type 1 di­a­betes treat­ments.

Ex­pand­ing on an ex­ist­ing col­lab­o­ra­tion, Vi­a­Cyte and W.L. Gore have agreed to com­bine the biotech’s PEC-En­cap can­di­date with a Gore-pro­duced mem­brane in what they hope will elim­i­nate the need for im­muno­sup­pres­sive drugs. Such treat­ments have cre­at­ed for­eign body re­spons­es in the past, and stamp­ing these re­ac­tions out is the main goal, Vi­a­Cyte CEO Paul Laikind said.

Such re­spons­es “ba­si­cal­ly can lead to a de­po­si­tion of what’s called for­eign-body gi­ant cells on the sur­face of the de­vice” and in­ter­fere with its process­es, Laikind told End­points News. Vi­a­Cyte and Gore’s fo­cus is cut­ting these re­ac­tions out en­tire­ly.

The prod­uct is de­signed to be im­plant­ed un­der­neath the skin and de­liv­er pan­cre­at­ic prog­en­i­tor cells that can se­crete in­sulin and con­trol blood glu­cose lev­els, in essence be­com­ing func­tion­al tis­sue. Not on­ly do the com­pa­nies aim to pro­vide this treat­ment but al­so pro­tect the cells from the hosts’ im­mune sys­tems, as the mem­brane al­lows for the cre­ation of a vas­cu­lar net­work on the sur­face of the de­vice.

Kevin D’Amour

“Those pan­cre­at­ic cells stay in the de­vice, and we’re es­sen­tial­ly re­plac­ing the nor­mal hu­man cells that are lost or dys­func­tion­al in di­a­betes pa­tients,” said Vi­a­Cyte CSO Kevin D’Amour. “They’re be­hold­en to in­ject­ing ex­oge­nous in­sulin to con­trol their blood sug­ar.”

Even though Laikind said the goal is to, es­sen­tial­ly, cure type 1 di­a­betes by way of these cell and hor­mone re­place­ments, the com­pa­nies would be con­tent by sim­ply re­duc­ing the need for in­jectable in­sulin use as that would still con­sti­tute a sig­nif­i­cant ther­a­peu­tic ad­vance.

Erin Hutchin­son

Erin Hutchin­son, a busi­ness leader at Gore, said the tricky part in cre­at­ing the right kind of mem­brane is get­ting the lev­el of per­me­abil­i­ty ex­act­ly cor­rect.

“You don’t want the cells in­side to es­cape and you don’t want the im­mune sys­tem to get in,” Hutchin­son said. “On the oth­er hand, you do want to al­low for trans­port, for things like oxy­gen, in­sulin, glu­cose, etc. And so that’s kind of the chal­lenge, de­sign­ing a mem­brane as part of an over­all de­vice that can do that.”

Stem cell R&D had been a hype train in ear­ly in­vest­ments but has since turned in­to a long-run­ning af­fair, and Vi­a­Cyte is no dif­fer­ent from oth­er such biotechs in this re­gard. The com­pa­ny had at­tempt­ed pair­ing its stem cell tech­nol­o­gy with a dif­fer­ent mem­brane be­fore, but ran in­to trou­ble when pa­tients de­vel­oped those for­eign body re­spons­es. That led them to col­lab­o­rate with Gore, which first signed on to an agree­ment in 2017 and then chipped in with $10 mil­lion as part of a larg­er Vi­a­Cyte fund­ing round in No­vem­ber 2018.

Phase I/II clin­i­cal tri­als of PEC-En­cap with the new mem­brane are un­der­way af­ter some de­lays re­lat­ed to Covid-19. Ear­ly in­di­ca­tions are promis­ing, Laikind said, but the com­pa­nies still need to go down the path of com­mer­cial­iza­tion.

“The im­mune re­sponse is pret­ty con­sis­tent among pa­tients, cer­tain­ly some will re­act more ag­gres­sive­ly than oth­ers,” Laikind said. “But the de­sign of the de­vice is meant to pro­tect the cells from the im­mune re­sponse of any pa­tient … We re­al­ly be­lieve we’re down to the fi­nal step to­ward a com­mer­cial prod­uct in prov­ing the ef­fec­tive de­liv­ery of graft­ing.”

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.

The Senate last week voted along party lines, 51-50, with Vice President Kamala Harris casting the tie-breaker, to make President Biden appointee Alvaro Bedoya the deciding vote on a split 2-2 Federal Trade Commission.

The addition of Bedoya to the FTC could not only spell more trouble for biopharma M&A activity, as he may align with his Democrat partners to break the FTC ties, but it may also mean that FTC Chair Lina Khan has what she needs to move forward on a study around the pharma middlemen known as pharmacy benefit managers.

The story of Henrietta Lacks’ immortal cell line and her family’s fight for justice caught the attention of national media outlets and Hollywood years ago. Now, the case faces an uncertain fate as a Baltimore federal judge considers tossing the case.

After a hearing on Tuesday, Judge Deborah Boardman is weighing Thermo Fisher’s motion to dismiss the claims against it on the grounds that the statute of limitations has passed, and the continuing harm doctrine does not apply. Boardman is grappling with the “extraordinarily unique facts” of the case, according to Maryland Matters, which first reported the news.